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Volumn 13, Issue 5, 2002, Pages 473-481

The application of DNA repair vectors to gene therapy

Author keywords

[No Author keywords available]

Indexed keywords

DNA; DNA FRAGMENT; GENOMIC DNA; SINGLE STRANDED DNA;

EID: 0036811533     PISSN: 09581669     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0958-1669(02)00370-1     Document Type: Review
Times cited : (13)

References (71)
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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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    • 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?
    • 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?
    • (2002) Gene Ther , vol.9 , pp. 691-694
    • Goncz, K.K.1    Prokopishyn, N.L.2    Chow, B.L.3    Davis, B.R.4    Gruenert, D.C.5
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    • In vivo and in vitro correction of the mdx dystrophin gene nonsense mutation by short-fragment homologous replacement
    • This report indicates that SFHR can correct the mdx mutation at the genomic level, both in vitro and in vivo
    • 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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    • 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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    • Expression of ΔF508 CFTR in normal mouse lung after site-specific modification of CFTR sequences by SFHR
    • 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
    • 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.
    • (2001) Gene Ther , vol.8 , pp. 961-965
    • 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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    • Design and packaging of adeno associated virus gene targeting vectors
    • An excellent article describing the technical aspects of an approach to use viral vectors for gene augmentation and/or gene repair
    • 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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    • High-fidelity correction of mutations at multiple chromosomal positions by adeno-associated virus vectors
    • A novel approach showing that a viral vector could be effective at both gene augmentation as well as gene repair
    • 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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    • Triplex forming oligonucleotides: Sequence specific tools for gene targeting
    • An outstanding review of the triplex-forming oligonucleotide field and the strategies exploiting their ability to induce endogenous DNA alteration pathways
    • 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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    • Knauert, M.P.1    Glazer, P.M.2
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    • Targeted correction of an episomal gene in mammalian cells by a short DNA fragment tethered to a triplex-forming oligonucleotide
    • One of the first reports on the use of this strategy for site-specific genomic alteration
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    • A bifunctional TFO can induce genomic modification at a level sufficient to have potential therapeutic application
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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.1    Macris, M.A.2    Faruqi, A.F.3    Glazer, P.M.4
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    • Intracellular generation of single-stranded DNA for chromosomal triplex formation and induced recombination
    • 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
    • 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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    • Datta, H.J.1    Glazer, P.M.2
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    • A new method (GOREC) for directed mutagenesis and gene repair by homologous recombination
    • 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.
    • (2002) Gene Ther , vol.9 , pp. 703-707
    • 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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    • Nonviral vectors in the new millennium: Delivery barriers in gene transfer
    • 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
    • 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.
    • (2001) Hum Gene Ther , vol.12 , pp. 861-870
    • Nishikawa, M.1    Huang, L.2
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    • Gene therapy: Safer and virus free?
    • An insightful look at the current status of gene therapy
    • 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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    • Simultaneous targeted alteration of the tyrosinase and c-kit genes by single-stranded oligonucleotides
    • in press
    • 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.
    • Gene Ther
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    • Oligonucleotide-directed single-base DNA alterations in mouse embryonic stem cells
    • in press
    • 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.
    • Gene Ther
    • Pierce, E.A.1    Liu, Q.2    Igoucheva, O.3    Omarrudin, R.4    Ma, H.C.5    Diamond, S.6    Yoon, K.7


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.