-
1
-
-
84879867061
-
Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome
-
Aiuti, A. et al. Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science 341, 1233151 (2013).
-
(2013)
Science
, vol.341
, pp. 1233151
-
-
Aiuti, A.1
-
2
-
-
51349090473
-
Insertional oncogenesis in 4 patients after retrovirusmediated gene therapy of SCID-X1
-
Hacein-Bey-Abina, S. et al. Insertional oncogenesis in 4 patients after retrovirusmediated gene therapy of SCID-X1. J. Clin. Invest. 118, 3132-3142 (2008).
-
(2008)
J. Clin. Invest.
, vol.118
, pp. 3132-3142
-
-
Hacein-Bey-Abina, S.1
-
3
-
-
84879264708
-
ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering
-
Gaj, T., Gersbach, C. A. & Barbas, C. F. III. ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering. Trends Biotechnol. 31, 397-405 (2013).
-
(2013)
Trends Biotechnol.
, vol.31
, pp. 397-405
-
-
Gaj, T.1
Gersbach, C.A.2
Barbas, C.F.3
-
4
-
-
84898046908
-
Quantifying genome-editing outcomes at endogenous loci with SMRT sequencing
-
Hendel, A. et al. Quantifying genome-editing outcomes at endogenous loci with SMRT sequencing. Cell Rep. 7, 293-305 (2014).
-
(2014)
Cell Rep.
, vol.7
, pp. 293-305
-
-
Hendel, A.1
-
5
-
-
84891710947
-
Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases
-
Cho, S. W. et al. Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases. Genome Res. 24, 132-141 (2014).
-
(2014)
Genome Res.
, vol.24
, pp. 132-141
-
-
Cho, S.W.1
-
6
-
-
84880570576
-
High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells
-
Fu, Y. et al. High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells. Nature Biotechnol. 31, 822-826 (2013).
-
(2013)
Nature Biotechnol.
, vol.31
, pp. 822-826
-
-
Fu, Y.1
-
7
-
-
34547610960
-
AAV vector integration sites inmouse hepatocellular carcinoma
-
Donsante, A. et al. AAV vector integration sites inmouse hepatocellular carcinoma. Science 317, 477 (2007).
-
(2007)
Science
, vol.317
, pp. 477
-
-
Donsante, A.1
-
8
-
-
84866973619
-
Ribosomal DNA integrating rAAV-rDNA vectors allow for stable transgene expression
-
Lisowski, L. et al. Ribosomal DNA integrating rAAV-rDNA vectors allow for stable transgene expression. Mol. Ther. 20, 1912-1923 (2012).
-
(2012)
Mol. Ther.
, vol.20
, pp. 1912-1923
-
-
Lisowski, L.1
-
9
-
-
33747046320
-
Gene targeting in vivo by adeno-associated virus vectors
-
Miller, D. G. et al. Gene targeting in vivo by adeno-associated virus vectors. Nature Biotechnol. 24, 1022-1026 (2006).
-
(2006)
Nature Biotechnol.
, vol.24
, pp. 1022-1026
-
-
Miller, D.G.1
-
10
-
-
79960424171
-
Vivo genome editing restores haemostasis in a mouse model of haemophilia
-
Li, H. et al. In vivo genome editing restores haemostasis in a mouse model of haemophilia. Nature 475, 217-221 (2011).
-
(2011)
Nature
, vol.475
, pp. 217-221
-
-
Li, H.1
-
11
-
-
84885599195
-
Role of antigen-specific regulatory CD41CD251 T cells in tolerance induction after neonatal IP administration of AAV-hF.IX
-
Shi, Y., Falahati, R., Zhang, J., Flebbe-Rehwaldt, L. & Gaensler, K. M. Role of antigen-specific regulatory CD41CD251 T cells in tolerance induction after neonatal IP administration of AAV-hF.IX. Gene Ther. 20, 987-996 (2013).
-
(2013)
Gene Ther.
, vol.20
, pp. 987-996
-
-
Shi, Y.1
Falahati, R.2
Zhang, J.3
Flebbe-Rehwaldt, L.4
Gaensler, K.M.5
-
12
-
-
84864360337
-
AAV-mediated gene targeting is significantly enhanced by transient inhibition of nonhomologous end joining or the proteasome in vivo
-
Paulk, N. K., Loza, L. M., Finegold, M. J. & Grompe, M. AAV-mediated gene targeting is significantly enhanced by transient inhibition of nonhomologous end joining or the proteasome in vivo. Hum. Gene Ther. 23, 658-665 (2012).
-
(2012)
Hum. Gene Ther.
, vol.23
, pp. 658-665
-
-
Paulk, N.K.1
Loza, L.M.2
Finegold, M.J.3
Grompe, M.4
-
13
-
-
84887017005
-
Albumin: Pathophysiologic basis of its role in the treatment of cirrhosis and its complications
-
Garcia-Martinez, R. et al. Albumin: pathophysiologic basis of its role in the treatment of cirrhosis and its complications. Hepatology 58, 1836-1846 (2013).
-
(2013)
Hepatology
, vol.58
, pp. 1836-1846
-
-
Garcia-Martinez, R.1
-
14
-
-
44949131860
-
Vitro and in vivo gene therapy vector evolution via multispecies interbreedingandretargeting of adeno-associated viruses
-
Grimm, D. et al. In vitro and in vivo gene therapy vector evolution via multispecies interbreedingandretargeting of adeno-associated viruses. J. Virol. 82, 5887-5911 (2008).
-
(2008)
J. Virol.
, vol.82
, pp. 5887-5911
-
-
Grimm, D.1
-
15
-
-
84894242894
-
Selection and evaluation of clinically relevant AAV variants in a xenograft liver model
-
Lisowski, L. et al. Selection and evaluation of clinically relevant AAV variants in a xenograft liver model. Nature 506, 382-386 (2014).
-
(2014)
Nature
, vol.506
, pp. 382-386
-
-
Lisowski, L.1
-
16
-
-
84864390047
-
Singleamino acidmodificationofadeno-associatedvirus capsidchanges transduction and humoral immune profiles
-
Li, C. et al.Singleamino acidmodificationofadeno-associatedvirus capsidchanges transduction and humoral immune profiles. J. Virol. 86, 7752-7759 (2012).
-
(2012)
J. Virol.
, vol.86
, pp. 7752-7759
-
-
Li, C.1
-
17
-
-
84880540708
-
Vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous
-
Dalkara, D. et al. In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous. Sci. Transl Med. 5, 189ra176 (2013).
-
(2013)
Sci. Transl Med.
, vol.5
, Issue.189
, pp. 176
-
-
Dalkara, D.1
-
18
-
-
84855161388
-
Adenovirus-associated virus vector-mediated gene transfer in hemophilia
-
Nathwani, A. C. et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N. Engl. J. Med. 365, 2357-2365 (2011).
-
(2011)
B. N. Engl. J. Med.
, vol.365
, pp. 2357-2365
-
-
Nathwani, A.C.1
-
19
-
-
84897051037
-
Retinal gene therapy in patients with choroideremia: Initial findings from a phase 1/2 clinical trial
-
MacLaren, R. E. et al. Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial. Lancet 383, 1129-1137 (2014).
-
(2014)
Lancet
, vol.383
, pp. 1129-1137
-
-
MacLaren, R.E.1
-
20
-
-
77950610272
-
Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo
-
Paulk, N. K. et al. Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo. Hepatology 51, 1200-1208 (2010).
-
(2010)
Hepatology
, vol.51
, pp. 1200-1208
-
-
Paulk, N.K.1
-
21
-
-
84874307076
-
Mechanisms of retroviral integration and mutagenesis
-
Cavazza, A., Moiani, A. & Mavilio, F. Mechanisms of retroviral integration and mutagenesis. Hum. Gene Ther. 24, 119-131 (2013).
-
(2013)
Hum. Gene Ther.
, vol.24
, pp. 119-131
-
-
Cavazza, A.1
Moiani, A.2
Mavilio, F.3
-
22
-
-
79955707349
-
High cleavage efficiency of a 2A peptide derived from porcine teschovirus-1 in human cell lines zebrafish and mice
-
Kim, J. H. et al. High cleavage efficiency of a 2A peptide derived from porcine teschovirus-1 in human cell lines, zebrafish and mice. PLoS ONE 6, e18556 (2011).
-
(2011)
PLoS ONE
, vol.6
, pp. 18556
-
-
Kim, J.H.1
-
23
-
-
70149114880
-
Gene therapy withhumanandmouse T-cell receptorsmediates cancer regression and targets normal tissues expressing cognate antigen
-
Johnson, L. A. et al.Gene therapy withhumanandmouse T-cell receptorsmediates cancer regression and targets normal tissues expressing cognate antigen. Blood 114, 535-546 (2009).
-
(2009)
Blood
, vol.114
, pp. 535-546
-
-
Johnson, L.A.1
-
24
-
-
10644289281
-
Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice
-
Nakai, H. et al. Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice. J. Virol. 79, 214-224 (2005).
-
(2005)
J. Virol.
, vol.79
, pp. 214-224
-
-
Nakai, H.1
-
25
-
-
76249102739
-
Organogenesisand development of the liver
-
Si-Tayeb, K., Lemaigre, F. P.&Duncan, S. A. Organogenesisand development of the liver. Dev. Cell 18, 175-189 (2010).
-
(2010)
Dev. Cell
, vol.18
, pp. 175-189
-
-
Si-Tayeb, K.1
Lemaigre, F.P.2
Duncan, S.A.3
-
26
-
-
0034960344
-
Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo
-
Nakai, H. et al. Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo. J. Virol. 75, 6969-6976 (2001).
-
(2001)
J. Virol.
, vol.75
, pp. 6969-6976
-
-
Nakai, H.1
-
28
-
-
84923180631
-
ZFN mediated targeting of albumin safe harbor results in therapeutic levels of human factor viii in a mouse model of hemophilia
-
Anguela, X. M. et al. ZFN mediated targeting of albumin "safe harbor" results in therapeutic levels of human factor viii in a mouse model of hemophilia A. Blood 122, 720 (2013).
-
(2013)
A. Blood
, vol.122
, pp. 720
-
-
Anguela, X.M.1
-
29
-
-
84892768755
-
Gene Therapy for Lysosomal Storage Disorders
-
suppl. 1
-
Yew, N. S. & Cheng, S. H. Gene therapy for lysosomal storage disorders. Pediatr. Endocrinol. Rev. 11 (suppl. 1), 99-109 (2013).
-
(2013)
Pediatr. Endocrinol. Rev.
, vol.11
, pp. 99-109
-
-
Yew, N.S.1
Cheng, S.H.2
-
30
-
-
84855466746
-
Antibody-based protection against HIV infection by vectored immunoprophylaxis
-
Balazs, A. B. et al. Antibody-based protection against HIV infection by vectored immunoprophylaxis. Nature 481, 81-84 (2011).
-
(2011)
Nature
, vol.481
, pp. 81-84
-
-
Balazs, A.B.1
-
31
-
-
33645992763
-
Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype
-
Grimm, D., Pandey, K., Nakai, H., Storm, T. A. & Kay, M. A. Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype. J. Virol. 80, 426-439 (2006).
-
(2006)
J. Virol.
, vol.80
, pp. 426-439
-
-
Grimm, D.1
Pandey, K.2
Nakai, H.3
Storm, T.A.4
Kay, M.A.5
-
32
-
-
84877015804
-
A mini-intronic plasmid (MIP): A novel robust transgene expression vector in vivo and in vitro
-
Lu, J., Zhang, F. & Kay, M. A. A mini-intronic plasmid (MIP): a novel robust transgene expression vector in vivo and in vitro. Mol. Ther. 21, 954-963 (2013).
-
(2013)
Mol. Ther.
, vol.21
, pp. 954-963
-
-
Lu, J.1
Zhang, F.2
Kay, M.A.3
-
33
-
-
50249150340
-
A reproducible and well-tolerated method for 2/3 partial hepatectomy in mice
-
Mitchell, C. & Willenbring, H. A reproducible and well-tolerated method for 2/3 partial hepatectomy in mice. Nature Protocols 3, 1167-1170 (2008).
-
(2008)
Nature Protocols
, vol.3
, pp. 1167-1170
-
-
Mitchell, C.1
Willenbring, H.2
-
34
-
-
84975628037
-
Optimal immunofluorescent staining for human factor ix and infiltrating T cells following gene therapy for hemophilia
-
Rogers, G. L. & Hoffman, B. E. Optimal immunofluorescent staining for human factor ix and infiltrating T cells following gene therapy for hemophilia B. J. Genet. Syndr. Gene Ther. Suppl. 1, 012 (2012).
-
(2012)
B. J. Genet. Syndr. Gene Ther. Suppl.
, vol.1
, pp. 12
-
-
Rogers, G.L.1
Hoffman, B.E.2
-
35
-
-
33745299723
-
Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways
-
Grimm, D. et al. Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature 441, 537-541 (2006).
-
(2006)
Nature
, vol.441
, pp. 537-541
-
-
Grimm, D.1
|