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1
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0035958048
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Foreign DNA integration. Genome wide perturbations of methylation and transcription in the recipient genomes
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Even nonrandom integration may have deleterious effects on the host genome
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Müller K., Heller H., Doerfler W. Foreign DNA integration. Genome wide perturbations of methylation and transcription in the recipient genomes. J Biol Chem. 276:2001;14271-14278. Even nonrandom integration may have deleterious effects on the host genome.
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Müller, K.1
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Aberrant patterns of DNA methylation, chromatin formation and gene expression in cancer
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Baylin S.B., Esteller M., Rountree M.R., Bachman K.E., Schuebel K., Herman J.G. Aberrant patterns of DNA methylation, chromatin formation and gene expression in cancer. Hum Mol Genet. 10:2001;687-692.
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Yáñez, R.J.1
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4
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0035902445
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Manipulating the mammalian genome by homologous recombination
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A nice review of homologous recombination, including TFOs
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Vasquez K.M., Marburger K., Intody Z., Wilson J.H. Manipulating the mammalian genome by homologous recombination. Proc Natl Acad Sci USA. 98:2001;8403-8410. A nice review of homologous recombination, including TFOs.
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Proc Natl Acad Sci USA
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Vasquez, K.M.1
Marburger, K.2
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0028048780
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A role for RNA synthesis in homologous pairing events
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Kotani H., Kmiec E.B. A role for RNA synthesis in homologous pairing events. Mol Cell Biol. 14:1994;6097-6106.
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Mol Cell Biol
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Kotani, H.1
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0011675801
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Targeted gene repair in mammalian cells using chimeric RNA/DNA oligonucleotides
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T. Friedman. Cold Spring Harbor Laboratory Press
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Kmiec E.B., Kren B.T., Steer C.J. Targeted gene repair in mammalian cells using chimeric RNA/DNA oligonucleotides. Friedman T. Development of Human Gene Therapy. 1999;643-670 Cold Spring Harbor Laboratory Press.
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Development of Human Gene Therapy
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Kmiec, E.B.1
Kren, B.T.2
Steer, C.J.3
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7
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0039594943
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A plausible mechanism for gene correction by chimeric oligonucleotides
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Outstanding biochemical dissection of the putative mechanisms and factors required for chimeraplasty
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Gamper H.B. Jr, Cole-Strauss A., Metz R., Parekh H., Kumar R., Kmiec E.B. A plausible mechanism for gene correction by chimeric oligonucleotides. Biochemistry. 39:2000;5808-5816. Outstanding biochemical dissection of the putative mechanisms and factors required for chimeraplasty.
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Biochemistry
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, pp. 5808-5816
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Gamper H.B., Jr.1
Cole-Strauss, A.2
Metz, R.3
Parekh, H.4
Kumar, R.5
Kmiec, E.B.6
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8
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0029817766
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Correction of the mutation responsible for sickle cell anemia by an RNA-DNA oligonucleotide
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Cole-Strauss A., Yoon K., Xiang Y., Byrne B.C., Rice M.C., Gryn J., Holloman W.K., Kmiec E.B. Correction of the mutation responsible for sickle cell anemia by an RNA-DNA oligonucleotide. Science. 273:1996;1386-1389.
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Science
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, pp. 1386-1389
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Cole-Strauss, A.1
Yoon, K.2
Xiang, Y.3
Byrne, B.C.4
Rice, M.C.5
Gryn, J.6
Holloman, W.K.7
Kmiec, E.B.8
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9
-
-
0031953132
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In vivo site-directed mutagenesis of the factor IX gene by chimeric RNA/DNA oligonucleotides
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Kren B.T., Bandyopadhyay P., Steer C.J. In vivo site-directed mutagenesis of the factor IX gene by chimeric RNA/DNA oligonucleotides. Nat Med. 4:1998;285-290.
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Nat Med
, vol.4
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Kren, B.T.1
Bandyopadhyay, P.2
Steer, C.J.3
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10
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0033621041
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Correction of the UDP-glucuronosyltransferase gene defect in the Gunn rat model of Crigler-Najjar syndrome type I with a chimeric oligonucleotide
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Demonstration that chimeraplast treatment in vivo in an accurate animal model of a human disease can provide sufficient genomic modification for therapeutic benefit
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Kren B.T., Parashar B., Bandyopadhyay P., Chowdhury N.R., Chowdhury J.R., Steer C.J. Correction of the UDP-glucuronosyltransferase gene defect in the Gunn rat model of Crigler-Najjar syndrome type I with a chimeric oligonucleotide. Proc Natl Acad Sci USA. 96:1999;10349-10354. Demonstration that chimeraplast treatment in vivo in an accurate animal model of a human disease can provide sufficient genomic modification for therapeutic benefit.
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Proc Natl Acad Sci USA
, vol.96
, pp. 10349-10354
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Kren, B.T.1
Parashar, B.2
Bandyopadhyay, P.3
Chowdhury, N.R.4
Chowdhury, J.R.5
Steer, C.J.6
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11
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0033537836
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Nucleotide exchange in genomic DNA of rat hepatocytes using RNA/DNA oligonucleotides. Targeted delivery of liposomes and polyethyleneimine to the asialoglycoprotein receptor
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Bandyopadhyay P., Ma X., Linehan-Stieers C., Kren B.T., Steer C.J. Nucleotide exchange in genomic DNA of rat hepatocytes using RNA/DNA oligonucleotides. Targeted delivery of liposomes and polyethyleneimine to the asialoglycoprotein receptor. J Biol Chem. 274:1999;10163-10172.
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J Biol Chem
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Bandyopadhyay, P.1
Ma, X.2
Linehan-Stieers, C.3
Kren, B.T.4
Steer, C.J.5
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12
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0001937705
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Liver-directed gene therapy
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I.M. Arias, J.L. Boyer, F.V. Chisari, N. Fausto, D. Schachter, & D.A. Shafritz. Lippincott Williams & Wilkins
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Steer C.J., Kren B.T., Roy Chowdhury N., Roy Chowdhury J. Liver-directed gene therapy. Arias I.M., Boyer J.L., Chisari F.V., Fausto N., Schachter D., Shafritz D.A. The Liver: Biology and Pathobiology. 4th edn :2001;941-962 Lippincott Williams & Wilkins.
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The Liver: Biology and Pathobiology 4th edn
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Steer, C.J.1
Kren, B.T.2
Roy Chowdhury, N.3
Roy Chowdhury, J.4
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13
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0036129569
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1-antitrypsin deficiency
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This report suggests that the structure/function relationships and factors/pathways involved in chimeraplasty are conserved between bacteria and mammals
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1-antitrypsin deficiency. Chest. 1221:2002;91S-97S. This report suggests that the structure/function relationships and factors/pathways involved in chimeraplasty are conserved between bacteria and mammals.
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(2002)
Chest
, vol.1221
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Metz, R.1
DiCola, M.2
Kurihara, T.3
Bailey, A.4
Frank, B.5
Roecklein, B.6
Blaese, M.7
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14
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0035886376
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In vivo gene repair of point and frameshift mutations directed by chimeric RNA/DNA oligonucleotides and modified single-stranded oligonucleotides
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Lui L., Rice M.C., Kmiec E.B. In vivo gene repair of point and frameshift mutations directed by chimeric RNA/DNA oligonucleotides and modified single-stranded oligonucleotides. Nucleic Acids Res. 29:2001;4238-4250.
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(2001)
Nucleic Acids Res
, vol.29
, pp. 4238-4250
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Lui, L.1
Rice, M.C.2
Kmiec, E.B.3
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15
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0034995488
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In vitro and in vivo nucleotide exchange directed by chimeric RNA/DNA oligonucleotides in Saccharomyces cerevisae
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This study identifies some of the factors involved in chimeraplasty, including ones that suppress conversion
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Rice M.C., Bruner M., Czymmek K., Kmiec E.B. In vitro and in vivo nucleotide exchange directed by chimeric RNA/DNA oligonucleotides in Saccharomyces cerevisae. Mol Microbiol. 40:2001;857-868. This study identifies some of the factors involved in chimeraplasty, including ones that suppress conversion.
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(2001)
Mol Microbiol
, vol.40
, pp. 857-868
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Rice, M.C.1
Bruner, M.2
Czymmek, K.3
Kmiec, E.B.4
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16
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0033989521
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Localized in vivo genotypic and phenotypic correction of the albino mutation in skin by RNA-DNA oligonucleotide
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The second demonstration that in vivo correction of a single point-mutation led to a phenotypic alteration
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Alexeev V., Igoucheva O., Domashenko A., Cotsarelis G., Yoon K. Localized in vivo genotypic and phenotypic correction of the albino mutation in skin by RNA-DNA oligonucleotide. Nat Biotechnol. 18:2000;43-47. The second demonstration that in vivo correction of a single point-mutation led to a phenotypic alteration.
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(2000)
Nat Biotechnol
, vol.18
, pp. 43-47
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Alexeev, V.1
Igoucheva, O.2
Domashenko, A.3
Cotsarelis, G.4
Yoon, K.5
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17
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0032931129
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Homologous recombination based gene therapy
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Lai L.-W., Lein Y.-H.H. Homologous recombination based gene therapy. Exp Nephrol. 7:1999;11-14.
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(1999)
Exp Nephrol
, vol.7
, pp. 11-14
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Lai, L.-W.1
Lein, Y.-H.H.2
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18
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85031353730
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Conversion of a single base pair mutation in newborn carbonic anhydrase (CA) II deficient mice by chimeric RNA/DNA oligonucleotides
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Lai L, Doty D, Khan R, Omera M, Lien Y-H: Conversion of a single base pair mutation in newborn carbonic anhydrase (CA) II deficient mice by chimeric RNA/DNA oligonucleotides. Mol Therapy 2001, S295. Chimeraplasty can repair multiple tissue types in vivo. Moreover, it demonstrates that the route of administration profoundly influences efficiency.
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(2001)
Mol Therapy
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Lai, L.1
Doty, D.2
Khan, R.3
Omera, M.4
Lien, Y.-H.5
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19
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0036211418
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Dystrophin gene repair in mdx muscle precursor cells in vitro and in vivo mediated by RNA-DNA chimeric oligonucleotides
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Thought provoking ex vivo gene therapy approach for muscular dystrophy
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Bertoni C., Rando T.A. Dystrophin gene repair in mdx muscle precursor cells in vitro and in vivo mediated by RNA-DNA chimeric oligonucleotides. Hum Gene Ther. 13:2002;707-718. Thought provoking ex vivo gene therapy approach for muscular dystrophy.
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(2002)
Hum Gene Ther
, vol.13
, pp. 707-718
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Bertoni, C.1
Rando, T.A.2
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20
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0034624974
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Rescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotides
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Rando T.A., Disatnik M.H., Zhou L.Z. Rescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotides. Proc Natl Acad Sci USA. 97:2000;5363-5368.
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(2000)
Proc Natl Acad Sci USA
, vol.97
, pp. 5363-5368
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Rando, T.A.1
Disatnik, M.H.2
Zhou, L.Z.3
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21
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0034086776
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In vivo targeted repair of a point mutation in the canine dystrophin gene by a chimeric RNA/DNA oligonucleotide
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Gene repair by chimeraplasty works in a large animal model of muscular dystrophy
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Bartlett R.J., Stockinger S., Denis M.M., Bartlett W.T., Inverardi L., Le T.T., thi Man N., Morris G.E., Bogan D.J., Metcalf-Bogan J., et al. In vivo targeted repair of a point mutation in the canine dystrophin gene by a chimeric RNA/DNA oligonucleotide. Nat Biotechnol. 18:2000;615-622. Gene repair by chimeraplasty works in a large animal model of muscular dystrophy.
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(2000)
Nat Biotechnol
, vol.18
, pp. 615-622
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-
Bartlett, R.J.1
Stockinger, S.2
Denis, M.M.3
Bartlett, W.T.4
Inverardi, L.5
Le, T.T.6
Thi Man, N.7
Morris, G.E.8
Bogan, D.J.9
Metcalf-Bogan, J.10
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22
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0033587679
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A tool for functional plant genomics: Chimeric RNA/DNA oligonucleotides cause in vivo gene-specific mutations
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Beetham P.R., Kipp R.B., Sawycky X.L., Arntzen C.J., May G.D. A tool for functional plant genomics: Chimeric RNA/DNA oligonucleotides cause in vivo gene-specific mutations. Proc Natl Acad Sci USA. 96:1999;8774-8778.
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(1999)
Proc Natl Acad Sci USA
, vol.96
, pp. 8774-8778
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Beetham, P.R.1
Kipp, R.B.2
Sawycky, X.L.3
Arntzen, C.J.4
May, G.D.5
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23
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0033587695
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Targeted manipulation of maize genes in vivo using chimeric RNA/DNA oligonucleotides
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Zhu T., Peterson D.J., Tagliani L., St Claire G., Baszczynski C.L., Bowen B. Targeted manipulation of maize genes in vivo using chimeric RNA/DNA oligonucleotides. Proc Natl Acad Sci USA. 96:1999;8768-8773.
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(1999)
Proc Natl Acad Sci USA
, vol.96
, pp. 8768-8773
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Zhu, T.1
Peterson, D.J.2
Tagliani, L.3
St Claire, G.4
Baszczynski, C.L.5
Bowen, B.6
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24
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0034966380
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Targeted correction of the β-thalassemia and targeted mutagenesis of the nucleotide associated with HPFH by RNA/DNA oligonucleotides: Potential for β-thalassemia gene therapy
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Li Z.-H., Liu D.-P., Yin W.-X., Guo Z.-C., Liang C.-C. Targeted correction of the β-thalassemia and targeted mutagenesis of the nucleotide associated with HPFH by RNA/DNA oligonucleotides: potential for β-thalassemia gene therapy. Blood Cells Mol Dis. 27:2001;530-538.
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(2001)
Blood Cells Mol Dis
, vol.27
, pp. 530-538
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Li, Z.-H.1
Liu, D.-P.2
Yin, W.-X.3
Guo, Z.-C.4
Liang, C.-C.5
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25
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85047696977
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- cells
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This study suggests that ex vivo gene therapy using chimeraplasts may be feasible for certain hemoglobinopathies
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- cells. Gene Ther. 9:2002;118-126. This study suggests that ex vivo gene therapy using chimeraplasts may be feasible for certain hemoglobinopathies.
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(2002)
Gene Ther
, vol.9
, pp. 118-126
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Lui, H.1
Agawal, S.2
Kmiec, E.3
Davis, B.R.4
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26
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0035918314
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Gene correction of the apolipoprotein (apo) E2 phenotype to wild-type apoE3 by in situ chimeraplasty
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Confirmation that high-efficieny gene repair using chimeraplasts can be achieved both in vitro and in vivo
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Tagalakis A.D., Graham I.R., Riddell D.R., Dickson J.G., Owen J.S. Gene correction of the apolipoprotein (apo) E2 phenotype to wild-type apoE3 by in situ chimeraplasty. J Biol Chem. 276:2001;13226-13230. Confirmation that high-efficieny gene repair using chimeraplasts can be achieved both in vitro and in vivo.
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(2001)
J Biol Chem
, vol.276
, pp. 13226-13230
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Tagalakis, A.D.1
Graham, I.R.2
Riddell, D.R.3
Dickson, J.G.4
Owen, J.S.5
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27
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0035013301
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Gene repair validation
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Excellent discussion of the variability and complexities associated with targeted gene repair using chimeraplasts
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Graham I.R., Manzano A., Tagalakis A.D., Sperber G., Hill V., Beattie S., Schepelmann S., Dickson G., Owen J.S. Gene repair validation. Nat Biotechnol. 19:2001;507-508. Excellent discussion of the variability and complexities associated with targeted gene repair using chimeraplasts.
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(2001)
Nat Biotechnol
, vol.19
, pp. 507-508
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-
Graham, I.R.1
Manzano, A.2
Tagalakis, A.D.3
Sperber, G.4
Hill, V.5
Beattie, S.6
Schepelmann, S.7
Dickson, G.8
Owen, J.S.9
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28
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85031358178
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Chimeraplast-mediated exon skipping as an approach to gene therapy for frame-shift deletions of the dystrophin gene
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A novel approach using chimeraplasty and possible strategy for deleting deleterious coding sequences, such as the expanded repeats associated with neurodegenerative disorders
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Bertoni C., Lau C., Rando T.A. Chimeraplast-mediated exon skipping as an approach to gene therapy for frame-shift deletions of the dystrophin gene. Mol Ther. 5:2002;S361. A novel approach using chimeraplasty and possible strategy for deleting deleterious coding sequences, such as the expanded repeats associated with neurodegenerative disorders.
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(2002)
Mol Ther
, vol.5
, pp. 361
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Bertoni, C.1
Lau, C.2
Rando, T.A.3
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29
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0011626169
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Effect of ultraviolet B radiation on chimeric RNA-DNA oligonucleotide induced gene correction
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Increasing DNA repair activity can improve chimeraplast-mediated gene conversion
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Lai L.-W., Whitehair O., Campbell P.D., Lein Y.-H.H. Effect of ultraviolet B radiation on chimeric RNA-DNA oligonucleotide induced gene correction. Mol Ther. 5:2002;S346. Increasing DNA repair activity can improve chimeraplast-mediated gene conversion.
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(2002)
Mol Ther
, vol.5
, pp. 346
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Lai, L.-W.1
Whitehair, O.2
Campbell, P.D.3
Lein, Y.-H.H.4
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30
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0034816091
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Mitochondria isolated from liver contain the essential factors required for RNA/DNA oligonucleotide-targeted gene repair
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This article suggests the potential to modify the mitochondrial genome using chimeraplasts
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Chen Z., Felsheim R., Wong P., Augustin L.B., Metz R., Kren B.T., Steer C.J. Mitochondria isolated from liver contain the essential factors required for RNA/DNA oligonucleotide-targeted gene repair. Biochem Biophys Res Commun. 285:2001;188-194. This article suggests the potential to modify the mitochondrial genome using chimeraplasts.
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(2001)
Biochem Biophys Res Commun
, vol.285
, pp. 188-194
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-
Chen, Z.1
Felsheim, R.2
Wong, P.3
Augustin, L.B.4
Metz, R.5
Kren, B.T.6
Steer, C.J.7
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31
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0036512266
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Functional correction of episomal mutations with short DNA fragments and RNA - DNA oligonucleotides
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Thorpe P.H., Stevenson B.J., Porteous D.J. Functional correction of episomal mutations with short DNA fragments and RNA - DNA oligonucleotides. J Gene Med. 4:2002;195-204.
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(2002)
J Gene Med
, vol.4
, pp. 195-204
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Thorpe, P.H.1
Stevenson, B.J.2
Porteous, D.J.3
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32
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0035066680
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Persistent failures in gene repair
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van der Steege G., Schuilenga-Hut P.H., Buys C.H., Scheffer H., Pas H.H., Jonkman M.F. Persistent failures in gene repair. Nat Biotechnol. 19:2001;305-306.
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(2001)
Nat Biotechnol
, vol.19
, pp. 305-306
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-
Van der Steege, G.1
Schuilenga-Hut, P.H.2
Buys, C.H.3
Scheffer, H.4
Pas, H.H.5
Jonkman, M.F.6
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33
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0037075199
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Targeted gene repair and its application to neurodegenerative disorders
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Parekh-Olmedo H., Kranic D., Kmiec E.B. Targeted gene repair and its application to neurodegenerative disorders. Neuron. 33:2002;495-498.
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(2002)
Neuron
, vol.33
, pp. 495-498
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Parekh-Olmedo, H.1
Kranic, D.2
Kmiec, E.B.3
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34
-
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0035069295
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The potential of nucleic acid repair in functional genomics
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Rice M.C., Czymmek K., Kmiec E.B. The potential of nucleic acid repair in functional genomics. Nat Biotechnol. 19:2001;321-326.
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(2001)
Nat Biotechnol
, vol.19
, pp. 321-326
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Rice, M.C.1
Czymmek, K.2
Kmiec, E.B.3
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35
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0035853251
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Targeted gene repair in mammalian cells using chimeric RNA/DNA oligonucleotides and modified single-stranded vectors
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A well-written technical article on using chimeraplasts and SSONs
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Parekh-Olmedo H., Czymmek K., Kmiec E.B. Targeted gene repair in mammalian cells using chimeric RNA/DNA oligonucleotides and modified single-stranded vectors. Sci STKE. 73:2001;PL1-12. A well-written technical article on using chimeraplasts and SSONs.
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(2001)
Sci STKE
, vol.73
, pp. 1-12
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Parekh-Olmedo, H.1
Czymmek, K.2
Kmiec, E.B.3
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36
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0032457288
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Different frequency of gene targeting events by the RNA-DNA oligonucleotide among epithelial cells
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Santana E., Peritz A.E., Iyer S., Uitto J., Yoon K. Different frequency of gene targeting events by the RNA-DNA oligonucleotide among epithelial cells. J Invest Dermatol. 111:1998;1172-1177.
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(1998)
J Invest Dermatol
, vol.111
, pp. 1172-1177
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Santana, E.1
Peritz, A.E.2
Iyer, S.3
Uitto, J.4
Yoon, K.5
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37
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0034327613
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The DNA strand of chimeric RNA/DNA oligonucleotides can direct gene repair/conversion activity in mammalian and plant cell-free extracts
-
Seminal article demonstrating that site-directed alteration of DNA can be achieved with SSONs
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Gamper H.B., Parekh H., Rice M.C., Bruner M., Youkey H., Kmiec E.B. The DNA strand of chimeric RNA/DNA oligonucleotides can direct gene repair/conversion activity in mammalian and plant cell-free extracts. Nucleic Acids Res. 28:2000;4332-4339. Seminal article demonstrating that site-directed alteration of DNA can be achieved with SSONs.
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(2000)
Nucleic Acids Res
, vol.28
, pp. 4332-4339
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Gamper, H.B.1
Parekh, H.2
Rice, M.C.3
Bruner, M.4
Youkey, H.5
Kmiec, E.B.6
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38
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0036640365
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Rad51p and Rad54p, but not Rad52p, elevate gene repair in Saccharomyces cerevisiae directed by modified single-stranded oligonucleotide vectors
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An excellent study defining various factors that may be involved in mediating SSON genomic alteration
-
Lui L., Cheng S., van Brabant A.J., Kmiec E.B. Rad51p and Rad54p, but not Rad52p, elevate gene repair in Saccharomyces cerevisiae directed by modified single-stranded oligonucleotide vectors. Nucleic Acids Res. 30:2002;2742-2750. An excellent study defining various factors that may be involved in mediating SSON genomic alteration.
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(2002)
Nucleic Acids Res
, vol.30
, pp. 2742-2750
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-
Lui, L.1
Cheng, S.2
Van Brabant, A.J.3
Kmiec, E.B.4
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39
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0035077792
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Targeted gene correction by small single-stranded oligonucleotides in mammalian cells
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A detailed comparison of targeted gene repair using SSONs with cell-free extracts, together with episomal and chromosomal reporter genes in cultured cells
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Igoucheva O., Alexeev V., Yoon K. Targeted gene correction by small single-stranded oligonucleotides in mammalian cells. Gene Ther. 8:2001;391-399. A detailed comparison of targeted gene repair using SSONs with cell-free extracts, together with episomal and chromosomal reporter genes in cultured cells.
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(2001)
Gene Ther
, vol.8
, pp. 391-399
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-
Igoucheva, O.1
Alexeev, V.2
Yoon, K.3
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40
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0036097061
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Strand bias in targeted gene repair is influenced by transcriptional activity
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An important and thought-provoking article that underscores the importance of understanding the mechanisms and factors involved in DNA repair
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Lui L., Rice M.C., Dury M., Cheng S., Gamper H., Kmiec E.B. Strand bias in targeted gene repair is influenced by transcriptional activity. Mol Cell Biol. 22:2002;3852-3863. An important and thought-provoking article that underscores the importance of understanding the mechanisms and factors involved in DNA repair.
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(2002)
Mol Cell Biol
, vol.22
, pp. 3852-3863
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-
Lui, L.1
Rice, M.C.2
Dury, M.3
Cheng, S.4
Gamper, H.5
Kmiec, E.B.6
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41
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0026704096
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Strand-specificity in the transformation of yeast with synthetic oligonucleotides
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A seminal paper on gene alteration using SSONs in vivo. A reminder that most 'new technology' is simply an improvement of previously described strategies
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Yamamoto T., Moerschell R.P., Wakem L.P., Komar-Panicucci S., Sherman F. Strand-specificity in the transformation of yeast with synthetic oligonucleotides. Genetics. 131:1992;811-819. A seminal paper on gene alteration using SSONs in vivo. A reminder that most 'new technology' is simply an improvement of previously described strategies.
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Genetics
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Yamamoto, T.1
Moerschell, R.P.2
Wakem, L.P.3
Komar-Panicucci, S.4
Sherman, F.5
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42
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Fujioka K.-i, Aratani Y., Kusano K., Koyama H. Targeted recombination with single-stranded DNA vectors in mammalian cells. Nucleic Acids Res. 3:1993;407-412.
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Nucleic Acids Res
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Fujioka K.-i1
Aratani, Y.2
Kusano, K.3
Koyama, H.4
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43
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Bertling, W.3
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44
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0033987325
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Site-directed alteration of genomic DNA by small-fragment homologous replacement
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An excellent how-to-do methods article on small fragment homologous replacement
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Goncz K.K., Gruenert D.C. Site-directed alteration of genomic DNA by small-fragment homologous replacement. Methods Mol Biol. 133:1999;85-99. An excellent how-to-do methods article on small fragment homologous replacement.
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Goncz, K.K.1
Gruenert, D.C.2
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45
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0034743705
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Targeted correction of a defective selectable marker gene in human epithelial cells by small DNA fragments
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Colosimo A., Goncz K.K., Novelli G., Dallapiccola B., Gruenert D.C. Targeted correction of a defective selectable marker gene in human epithelial cells by small DNA fragments. Mol Ther. 3:2001;178-185.
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Mol Ther
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Colosimo, A.1
Goncz, K.K.2
Novelli, G.3
Dallapiccola, B.4
Gruenert, D.C.5
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46
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0029959582
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Gene targeting of CFTR DNA in CF epithelial cells
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Kunzelmann K., Legendre J.Y., Knoell D.L., Escobar L.C., Xu Z., Gruenert D.C. Gene targeting of CFTR DNA in CF epithelial cells. Gene Ther. 3:1996;859-867.
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Gene Ther
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Kunzelmann, K.1
Legendre, J.Y.2
Knoell, D.L.3
Escobar, L.C.4
Xu, Z.5
Gruenert, D.C.6
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47
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0031796816
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Targeted replacement of normal and mutant CFTR sequences in human airway epithelial cells using DNA fragments
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Goncz K.K., Kunzelmann K., Xu Z., Gruenert D.C. Targeted replacement of normal and mutant CFTR sequences in human airway epithelial cells using DNA fragments. Hum Mol Genet. 7:1998;1913-1919.
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Hum Mol Genet
, vol.7
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Goncz, K.K.1
Kunzelmann, K.2
Xu, Z.3
Gruenert, D.C.4
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48
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0036272055
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Isolation of cell lines corrected at ΔF508-CFTR locus by SFHR-mediated targeting
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The latest report from the group that has advanced SFHR as an effective method of gene conversion for the three-base pair ΔF508 CFTR mutation
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Bruscia E., Sangiuolo F., Sinibaldi P., Goncz K.K., Gruenert D.C. Isolation of cell lines corrected at ΔF508-CFTR locus by SFHR-mediated targeting. Gene Ther. 9:2002;683-685. The latest report from the group that has advanced SFHR as an effective method of gene conversion for the three-base pair ΔF508 CFTR mutation.
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(2002)
Gene Ther
, vol.9
, pp. 683-685
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Bruscia, E.1
Sangiuolo, F.2
Sinibaldi, P.3
Goncz, K.K.4
Gruenert, D.C.5
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49
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0036268053
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Application of SFHR to gene therapy of monogenic disorders
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Reinforces the notion that nuclear delivery of the molecules is essential for gene conversion. Would cells that are refractory to SFHR be amenable to chimeraplasty for gene correction, or vice versa?
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Goncz K.K., Prokopishyn N.L., Chow B.L., Davis B.R., Gruenert D.C. Application of SFHR to gene therapy of monogenic disorders. Gene Ther. 9:2002;691-694. Reinforces the notion that nuclear delivery of the molecules is essential for gene conversion. Would cells that are refractory to SFHR be amenable to chimeraplasty for gene correction, or vice versa?
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(2002)
Gene Ther
, vol.9
, pp. 691-694
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Goncz, K.K.1
Prokopishyn, N.L.2
Chow, B.L.3
Davis, B.R.4
Gruenert, D.C.5
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50
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0035831649
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In vivo and in vitro correction of the mdx dystrophin gene nonsense mutation by short-fragment homologous replacement
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This report indicates that SFHR can correct the mdx mutation at the genomic level, both in vitro and in vivo
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Kapsa R., Quigley A., Lynch G.S., Steeper K., Kornberg A.J., Gregorevic P., Austin L., Byrne E. In vivo and in vitro correction of the mdx dystrophin gene nonsense mutation by short-fragment homologous replacement. Hum Gene Ther. 12:2001;629-642. This report indicates that SFHR can correct the mdx mutation at the genomic level, both in vitro and in vivo.
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(2001)
Hum Gene Ther
, vol.12
, pp. 629-642
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-
Kapsa, R.1
Quigley, A.2
Lynch, G.S.3
Steeper, K.4
Kornberg, A.J.5
Gregorevic, P.6
Austin, L.7
Byrne, E.8
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51
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0034743499
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Expression of ΔF508 CFTR in normal mouse lung after site-specific modification of CFTR sequences by SFHR
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The first in vivo generation of a three-base pair deletion in lung. This proof-of-principle article underscores the difficulty of transferring work from cell culture to animals and the importance of delivery
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Goncz K.K., Colosimo A., Dallapiccola B., Gagne L., Hong K., Novelli G., Papahadjopoulos D., Sawa T., Schreier H., Wiener-Kronish J., et al. Expression of ΔF508 CFTR in normal mouse lung after site-specific modification of CFTR sequences by SFHR. Gene Ther. 8:2001;961-965. The first in vivo generation of a three-base pair deletion in lung. This proof-of-principle article underscores the difficulty of transferring work from cell culture to animals and the importance of delivery.
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(2001)
Gene Ther
, vol.8
, pp. 961-965
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-
Goncz, K.K.1
Colosimo, A.2
Dallapiccola, B.3
Gagne, L.4
Hong, K.5
Novelli, G.6
Papahadjopoulos, D.7
Sawa, T.8
Schreier, H.9
Wiener-Kronish, J.10
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52
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0033997905
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Design and packaging of adeno associated virus gene targeting vectors
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An excellent article describing the technical aspects of an approach to use viral vectors for gene augmentation and/or gene repair
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Hirata R.K., Russell D.W. Design and packaging of adeno associated virus gene targeting vectors. J Virol. 74:2000;4612-4620. An excellent article describing the technical aspects of an approach to use viral vectors for gene augmentation and/or gene repair.
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(2000)
J Virol
, vol.74
, pp. 4612-4620
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Hirata, R.K.1
Russell, D.W.2
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53
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0034991469
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Introduction of single base substitutions at homologous chromosomal sequences by adeno-associated virus vectors
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Inoue N., Dong R., Hirata R.K., Russell D.W. Introduction of single base substitutions at homologous chromosomal sequences by adeno-associated virus vectors. Mol Ther. 3:2001;526-530.
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(2001)
Mol Ther
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, pp. 526-530
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Inoue, N.1
Dong, R.2
Hirata, R.K.3
Russell, D.W.4
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54
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0032874343
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High-fidelity correction of mutations at multiple chromosomal positions by adeno-associated virus vectors
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A novel approach showing that a viral vector could be effective at both gene augmentation as well as gene repair
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Inoue N., Hirata R.K., Russell D.W. High-fidelity correction of mutations at multiple chromosomal positions by adeno-associated virus vectors. J Virol. 73:1999;7376-7380. A novel approach showing that a viral vector could be effective at both gene augmentation as well as gene repair.
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(1999)
J Virol
, vol.73
, pp. 7376-7380
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Inoue, N.1
Hirata, R.K.2
Russell, D.W.3
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55
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0035475787
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Triplex forming oligonucleotides: Sequence specific tools for gene targeting
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An outstanding review of the triplex-forming oligonucleotide field and the strategies exploiting their ability to induce endogenous DNA alteration pathways
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Knauert M.P., Glazer P.M. Triplex forming oligonucleotides: sequence specific tools for gene targeting. Hum Mol Genet. 10:2001;2243-2251. An outstanding review of the triplex-forming oligonucleotide field and the strategies exploiting their ability to induce endogenous DNA alteration pathways.
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(2001)
Hum Mol Genet
, vol.10
, pp. 2243-2251
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Knauert, M.P.1
Glazer, P.M.2
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56
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0033486098
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A search for base analogs to enhance third-strand binding to 'inverted' target base pairs of triplexes in the pyrimidine/parallel motif
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Amosova O.A., Fresco J.R. A search for base analogs to enhance third-strand binding to 'inverted' target base pairs of triplexes in the pyrimidine/parallel motif. Nucleic Acids Res. 27:1999;4632-4635.
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(1999)
Nucleic Acids Res
, vol.27
, pp. 4632-4635
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Amosova, O.A.1
Fresco, J.R.2
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57
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0035914386
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Chromosome targeting at short polypurine sites by cationic triplex-forming oligonucleotides
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Vasquez K.M., Dagel J.M., Weeks D.L., Glazer P.M. Chromosome targeting at short polypurine sites by cationic triplex-forming oligonucleotides. J Biol Chem. 276:2001;38536-38541.
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J Biol Chem
, vol.276
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Vasquez, K.M.1
Dagel, J.M.2
Weeks, D.L.3
Glazer, P.M.4
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58
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0033557144
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Chromosomal mutations induced by triplex-forming oligonucleotides in mammalian cells
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Vasquez K.M., Wang G., Havre P.A., Glazer P.M. Chromosomal mutations induced by triplex-forming oligonucleotides in mammalian cells. Nucleic Acids Res. 27:1999;1176-1181.
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(1999)
Nucleic Acids Res
, vol.27
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Vasquez, K.M.1
Wang, G.2
Havre, P.A.3
Glazer, P.M.4
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59
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0033597251
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Targeted correction of an episomal gene in mammalian cells by a short DNA fragment tethered to a triplex-forming oligonucleotide
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One of the first reports on the use of this strategy for site-specific genomic alteration
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Chan P.P., Lin M., Faruqi A.F., Powell J., Seidman M.M., Glazer P.M. Targeted correction of an episomal gene in mammalian cells by a short DNA fragment tethered to a triplex-forming oligonucleotide. J Biol Chem. 274:1999;11541-11548. One of the first reports on the use of this strategy for site-specific genomic alteration.
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(1999)
J Biol Chem
, vol.274
, pp. 11541-11548
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Chan, P.P.1
Lin, M.2
Faruqi, A.F.3
Powell, J.4
Seidman, M.M.5
Glazer, P.M.6
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60
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0032874955
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Correction of chromosomal point mutations in human cells with bifunctional oligonucleotides
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A bifunctional TFO can induce genomic modification at a level sufficient to have potential therapeutic application
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Culver K.W., Hsieh W.-T., Huyen Y., Chen V., Liu J., Khripine Y., Khorlin A. Correction of chromosomal point mutations in human cells with bifunctional oligonucleotides. Nat Biotechnol. 17:1999;989-993. A bifunctional TFO can induce genomic modification at a level sufficient to have potential therapeutic application.
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(1999)
Nat Biotechnol
, vol.17
, pp. 989-993
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Culver, K.W.1
Hsieh, W.-T.2
Huyen, Y.3
Chen, V.4
Liu, J.5
Khripine, Y.6
Khorlin, A.7
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61
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0035947657
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Triplex-induced recombination in human cell-free extracts. Dependence on XPA and HsRad51
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Datta H.J., Chan P.P., Vasquez K.M., Gupta R.C., Glazer P.M. Triplex-induced recombination in human cell-free extracts. Dependence on XPA and HsRad51. J Biol Chem. 276:2001;18018-18023.
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J Biol Chem
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, pp. 18018-18023
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Datta, H.J.1
Chan, P.P.2
Vasquez, K.M.3
Gupta, R.C.4
Glazer, P.M.5
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62
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0037197844
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Human XPA and RPA DNA repair proteins participate in specific recognition of triplex-induced helical distortions
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Vasquez K.M., Christensen J., Li L., Finch R.A., Glazer P.M. Human XPA and RPA DNA repair proteins participate in specific recognition of triplex-induced helical distortions. Proc Natl Acad Sci USA. 99:2002;5848-5853.
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(2002)
Proc Natl Acad Sci USA
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, pp. 5848-5853
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Vasquez, K.M.1
Christensen, J.2
Li, L.3
Finch, R.A.4
Glazer, P.M.5
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63
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0034255524
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High-frequency intrachromosomal gene conversion induced by triplex-forming oligonucleotides microinjected into mouse cells
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This report suggests that harnessing the endogenous HR activity induced by TFOs has potential as a useful tool to modify the genome
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Luo Z., Macris M.A., Faruqi A.F., Glazer P.M. High-frequency intrachromosomal gene conversion induced by triplex-forming oligonucleotides microinjected into mouse cells. Proc Natl Acad Sci USA. 97:2000;9003-9008. This report suggests that harnessing the endogenous HR activity induced by TFOs has potential as a useful tool to modify the genome.
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(2000)
Proc Natl Acad Sci USA
, vol.97
, pp. 9003-9008
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Luo, Z.1
Macris, M.A.2
Faruqi, A.F.3
Glazer, P.M.4
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64
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0033766239
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Specific mutations induced by triplex-forming oligonucleotides in mice
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Vasquez K.M., Narayanan L., Glazer P.M. Specific mutations induced by triplex-forming oligonucleotides in mice. Science. 290:2000;530-533.
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Science
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Vasquez, K.M.1
Narayanan, L.2
Glazer, P.M.3
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65
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0035945236
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Intracellular generation of single-stranded DNA for chromosomal triplex formation and induced recombination
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This vector system has the potential to generate in vivo single-stranded repair molecules for different strategies described in this article for site-directed genomic alteration
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Datta H.J., Glazer P.M. Intracellular generation of single-stranded DNA for chromosomal triplex formation and induced recombination. Nucleic Acids Res. 29:2001;5140-5147. This vector system has the potential to generate in vivo single-stranded repair molecules for different strategies described in this article for site-directed genomic alteration.
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(2001)
Nucleic Acids Res
, vol.29
, pp. 5140-5147
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Datta, H.J.1
Glazer, P.M.2
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66
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0035852796
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Stimulation of Rec-A-mediated D-loop formation by oligonucleotide-directed triple-helix formation: Guided homologous recombination (GOREC)
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Biet E., Maurisse R., Dutreix M., Sun J.-S. Stimulation of Rec-A-mediated D-loop formation by oligonucleotide-directed triple-helix formation: guided homologous recombination (GOREC). Biochemistry. 40:2001;1779-1786.
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(2001)
Biochemistry
, vol.40
, pp. 1779-1786
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Biet, E.1
Maurisse, R.2
Dutreix, M.3
Sun, J.-S.4
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67
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0036268483
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A new method (GOREC) for directed mutagenesis and gene repair by homologous recombination
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If the high rates of gene conversion observed in this system can be replicated in vivo, this gene repair technology could have great potential
-
Maurisse R., Feugeas J.-P., Biet E., Kuzniak I., Leboulch P., Dutreix M., Sun J.-S. A new method (GOREC) for directed mutagenesis and gene repair by homologous recombination. Gene Ther. 9:2002;703-707. If the high rates of gene conversion observed in this system can be replicated in vivo, this gene repair technology could have great potential.
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(2002)
Gene Ther
, vol.9
, pp. 703-707
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Maurisse, R.1
Feugeas, J.-P.2
Biet, E.3
Kuzniak, I.4
Leboulch, P.5
Dutreix, M.6
Sun, J.-S.7
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68
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0034913116
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Nonviral vectors in the new millennium: Delivery barriers in gene transfer
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An excellent review on the current status of the nonviral delivery for gene therapy, emphasizing the barriers that have made this process less efficient than viral delivery methods
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Nishikawa M., Huang L. Nonviral vectors in the new millennium: delivery barriers in gene transfer. Hum Gene Ther. 12:2001;861-870. An excellent review on the current status of the nonviral delivery for gene therapy, emphasizing the barriers that have made this process less efficient than viral delivery methods.
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(2001)
Hum Gene Ther
, vol.12
, pp. 861-870
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Nishikawa, M.1
Huang, L.2
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69
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0035941087
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Gene therapy: Safer and virus free?
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An insightful look at the current status of gene therapy
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Ferber D. Gene therapy: safer and virus free? Science. 294:2001;1638-1642. An insightful look at the current status of gene therapy.
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(2001)
Science
, vol.294
, pp. 1638-1642
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Ferber, D.1
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70
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85031357251
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Simultaneous targeted alteration of the tyrosinase and c-kit genes by single-stranded oligonucleotides
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in press
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Alexeev V, Igoucheva O, Yoon K: Simultaneous targeted alteration of the tyrosinase and c-kit genes by single-stranded oligonucleotides. Gene Ther: in press. The first report to demonstrate the feasibility of simultaneously targeting two genes in a single cell.
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Gene Ther
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Alexeev, V.1
Igoucheva, O.2
Yoon, K.3
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71
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85031362566
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Oligonucleotide-directed single-base DNA alterations in mouse embryonic stem cells
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in press
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Pierce EA, Liu Q, Igoucheva O, Omarrudin R, Ma HC, Diamond S, Yoon K: Oligonucleotide-directed single-base DNA alterations in mouse embryonic stem cells. Gene Ther: in press. A seminal report demonstrating that SSONs can produce ES cells with precise mutations in any gene. The next step is to test whether SSONs can create accurate mouse models of inherited diseases by this relatively simple method.
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Gene Ther
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Pierce, E.A.1
Liu, Q.2
Igoucheva, O.3
Omarrudin, R.4
Ma, H.C.5
Diamond, S.6
Yoon, K.7
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