-
1
-
-
77956928344
-
Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia
-
Cavazzana-Calvo, M. et al. Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia. Nature 467, 318-322 (2010).
-
(2010)
Nature
, vol.467
, pp. 318-322
-
-
Cavazzana-Calvo, M.1
-
2
-
-
70449427834
-
Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy
-
Cartier, N. et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 326, 818-823 (2009).
-
(2009)
Science
, vol.326
, pp. 818-823
-
-
Cartier, N.1
-
3
-
-
84879873039
-
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy
-
Biffi, A. et al. Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 341, 1233158 (2013).
-
(2013)
Science
, vol.341
, pp. 1233158
-
-
Biffi, A.1
-
4
-
-
77954833516
-
Efficacy of gene therapy for X-linked severe combined immunodeficiency
-
Hacein-Bey-Abina, S. et al. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 363, 355-364 (2010).
-
(2010)
N Engl J Med
, vol.363
, pp. 355-364
-
-
Hacein-Bey-Abina, S.1
-
5
-
-
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
-
6
-
-
77955608799
-
RNA-based gene therapy for HIV with lentiviral vector-modified CD34(+ ) cells in patients undergoing transplantation for AIDS-related lymphoma
-
DiGiusto, D. L. et al. RNA-based gene therapy for HIV with lentiviral vector-modified CD34(+ ) cells in patients undergoing transplantation for AIDS-related lymphoma. Sci Transl Med 2, 36ra43 (2010).
-
(2010)
Sci Transl Med
, vol.2
, pp. 36ra43
-
-
DiGiusto, D.L.1
-
7
-
-
84879264708
-
3rd ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering
-
Gaj, T., Gersbach, C. A. & Barbas, C. F. 3rd 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
-
8
-
-
84927175081
-
High-throughput screening identifies compounds that enhance lentiviral transduction
-
Johnston, J. M. et al. High-throughput screening identifies compounds that enhance lentiviral transduction. Gene Ther 21, 1008-1020 (2014).
-
(2014)
Gene Ther
, vol.21
, pp. 1008-1020
-
-
Johnston, J.M.1
-
9
-
-
84926339367
-
Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of beta-thalassemia and sickle cell disease
-
Negre, O. et al. Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of beta-thalassemia and sickle cell disease. Curr Gene Ther 15, 64-81 (2015).
-
(2015)
Curr Gene Ther
, vol.15
, pp. 64-81
-
-
Negre, O.1
-
10
-
-
79952688973
-
Large-scale manufacture and characterization of a lentiviral vector produced for clinical ex vivo gene therapy application
-
Merten, O. W. et al. Large-scale manufacture and characterization of a lentiviral vector produced for clinical ex vivo gene therapy application. Hum Gene Ther 22, 343-356 (2011).
-
(2011)
Hum Gene Ther
, vol.22
, pp. 343-356
-
-
Merten, O.W.1
-
11
-
-
84883743436
-
Integrase-defective lentiviral vectors as a delivery platform for targeted modification of adenosine deaminase locus
-
Joglekar, A. V. et al. Integrase-defective lentiviral vectors as a delivery platform for targeted modification of adenosine deaminase locus. Mol Ther 21, 1705-1717 (2013).
-
(2013)
Mol Ther
, vol.21
, pp. 1705-1717
-
-
Joglekar, A.V.1
-
12
-
-
84878528467
-
Genomic editing of the HIV-1 coreceptor CCR5 in adult hematopoietic stem and progenitor cells using zinc finger nucleases
-
Li, L. et al. Genomic editing of the HIV-1 coreceptor CCR5 in adult hematopoietic stem and progenitor cells using zinc finger nucleases. Mol Ther 21, 1259-1269 (2013).
-
(2013)
Mol Ther
, vol.21
, pp. 1259-1269
-
-
Li, L.1
-
13
-
-
84897550064
-
Gene therapy for Wiskott-Aldrich syndrome-long-term efficacy and genotoxicity
-
Braun, C. J. et al. Gene therapy for Wiskott-Aldrich syndrome-long-term efficacy and genotoxicity. Sci Transl Med 6, 227ra233 (2014).
-
(2014)
Sci Transl Med
, vol.6
, pp. 227ra233
-
-
Braun, C.J.1
-
14
-
-
0040417435
-
Adeno-associated virus 2-mediated high efficiency gene transfer into immature and mature subsets of hematopoietic progenitor cells in human umbilical cord blood
-
Zhou, S. Z. et al. Adeno-associated virus 2-mediated high efficiency gene transfer into immature and mature subsets of hematopoietic progenitor cells in human umbilical cord blood. J Exp Med 179, 1867-1875 (1994).
-
(1994)
J Exp Med
, vol.179
, pp. 1867-1875
-
-
Zhou, S.Z.1
-
15
-
-
0030879933
-
Adeno-associated virus type 2-mediated transduction in primary human bone marrow-derived CD34+ hematopoietic progenitor cells: Donor variation and correlation of transgene expression with cellular differentiation
-
Ponnazhagan, S. et al. Adeno-associated virus type 2-mediated transduction in primary human bone marrow-derived CD34+ hematopoietic progenitor cells: donor variation and correlation of transgene expression with cellular differentiation. J Virol 71, 8262-8267 (1997).
-
(1997)
J Virol
, vol.71
, pp. 8262-8267
-
-
Ponnazhagan, S.1
-
16
-
-
0033559749
-
Transduction of primitive human marrow and cord blood-derived hematopoietic progenitor cells with adenoassociated virus vectors
-
Chatterjee, S. et al. Transduction of primitive human marrow and cord blood-derived hematopoietic progenitor cells with adenoassociated virus vectors. Blood 93, 1882-1894 (1999).
-
(1999)
Blood
, vol.93
, pp. 1882-1894
-
-
Chatterjee, S.1
-
17
-
-
45549090635
-
Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses
-
Zhong, L. et al. Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses. Proc Natl Acad Sci USA 105, 7827-7832 (2008).
-
(2008)
Proc Natl Acad Sci USA
, vol.105
, pp. 7827-7832
-
-
Zhong, L.1
-
18
-
-
78650898983
-
High-efficiency transduction and correction of murine hemophilia B using AAV2 vectors devoid of multiple surface-exposed tyrosines
-
Markusic, D. M. et al. High-efficiency transduction and correction of murine hemophilia B using AAV2 vectors devoid of multiple surface-exposed tyrosines. Mol Ther 18, 2048-2056 (2010).
-
(2010)
Mol Ther
, vol.18
, pp. 2048-2056
-
-
Markusic, D.M.1
-
19
-
-
77957838569
-
Enhanced long-term transduction and multilineage engraftment of human hematopoietic stem cells transduced with tyrosine-modified recombinant adeno-associated virus serotype 2
-
Kauss, M. A. et al. Enhanced long-term transduction and multilineage engraftment of human hematopoietic stem cells transduced with tyrosine-modified recombinant adeno-associated virus serotype 2. Hum Gene Ther 21, 1129-1136 (2010).
-
(2010)
Hum Gene Ther
, vol.21
, pp. 1129-1136
-
-
Kauss, M.A.1
-
20
-
-
84892967754
-
Optimizing the transduction efficiency of capsid-modified AAV6 serotype vectors in primary human hematopoietic stem cells in vitro and in a xenograft mouse model in vivo
-
Song, L. et al. Optimizing the transduction efficiency of capsid-modified AAV6 serotype vectors in primary human hematopoietic stem cells in vitro and in a xenograft mouse model in vivo. Cytotherapy 15, 986-998 (2013).
-
(2013)
Cytotherapy
, vol.15
, pp. 986-998
-
-
Song, L.1
-
21
-
-
84875040149
-
High-efficiency transduction of primary human hematopoietic stem cells and erythroid lineage-restricted expression by optimized AAV6 serotype vectors in vitro and in a murine xenograft model in vivo
-
Song, L. et al. High-efficiency transduction of primary human hematopoietic stem cells and erythroid lineage-restricted expression by optimized AAV6 serotype vectors in vitro and in a murine xenograft model in vivo. PLoS One 8, e58757 (2013).
-
(2013)
PLoS One
, vol.8
, pp. e58757
-
-
Song, L.1
-
22
-
-
84955240212
-
Adeno-associated virus: The naturally occurring virus versus the recombinant vector
-
Srivastava, A. Adeno-Associated Virus: The Naturally Occurring Virus Versus the Recombinant Vector. Hum Gene Ther 27, 1-6 (2016).
-
(2016)
Hum Gene Ther
, vol.27
, pp. 1-6
-
-
Srivastava, A.1
-
23
-
-
84897931068
-
Pristimerin enhances recombinant adeno-associated virus vector-mediated transgene expression in human cell lines in vitro and murine hepatocytes in vivo
-
Wang, L. N. et al. Pristimerin enhances recombinant adeno-associated virus vector-mediated transgene expression in human cell lines in vitro and murine hepatocytes in vivo. J Integr Med 12, 20-34 (2014).
-
(2014)
J Integr Med
, vol.12
, pp. 20-34
-
-
Wang, L.N.1
-
24
-
-
84964313851
-
Gene transfer properties and structural modeling of human stem cell-derived AAV
-
Smith, L. J. et al. Gene transfer properties and structural modeling of human stem cell-derived AAV. Mol Ther 22, 1625-1634 (2014).
-
(2014)
Mol Ther
, vol.22
, pp. 1625-1634
-
-
Smith, L.J.1
-
25
-
-
84918780947
-
Cytotoxic genes from traditional Chinese medicine inhibit tumor growth both in vitro and in vivo
-
Zhang, Y. H. et al. Cytotoxic genes from traditional Chinese medicine inhibit tumor growth both in vitro and in vivo. J Integr Med 12, 483-494 (2014).
-
(2014)
J Integr Med
, vol.12
, pp. 483-494
-
-
Zhang, Y.H.1
-
26
-
-
84875092627
-
Optimization of the capsid of recombinant adeno-associated virus 2 (AAV2) vectors: The final threshold?
-
Aslanidi, G. V. et al. Optimization of the capsid of recombinant adeno-associated virus 2 (AAV2) vectors: the final threshold? PLoS One 8, e59142 (2013).
-
(2013)
PLoS One
, vol.8
, pp. e59142
-
-
Aslanidi, G.V.1
-
27
-
-
0033010884
-
Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2
-
Qing, K. et al. Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2. Nat Med 5, 71-77 (1999).
-
(1999)
Nat Med
, vol.5
, pp. 71-77
-
-
Qing, K.1
-
28
-
-
77953229919
-
Epidermal growth factor receptor is a co-receptor for adeno-associated virus serotype 6
-
Weller, M. L. et al. Epidermal growth factor receptor is a co-receptor for adeno-associated virus serotype 6. Nat Med 16, 662-664 (2010).
-
(2010)
Nat Med
, vol.16
, pp. 662-664
-
-
Weller, M.L.1
-
29
-
-
84937905397
-
Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells
-
Hendel, A. et al. Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells. Nat Biotechnol (2015).
-
(2015)
Nat Biotechnol
-
-
Hendel, A.1
-
30
-
-
84922720418
-
CRISPR genome engineering and viral gene delivery: A case of mutual attraction
-
Schmidt, F. & Grimm, D. CRISPR genome engineering and viral gene delivery: a case of mutual attraction. Biotechnol J 10, 258-272 (2015).
-
(2015)
Biotechnol J
, vol.10
, pp. 258-272
-
-
Schmidt, F.1
Grimm, D.2
-
31
-
-
84922032690
-
Gene therapy: Editorial control
-
Gammon, K. Gene therapy: editorial control. Nature 515, S11-S13 (2014).
-
(2014)
Nature
, vol.515
, pp. S11-S13
-
-
Gammon, K.1
-
32
-
-
84942921684
-
Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template
-
Sather, B. D. et al. Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template. Sci Transl Med 7, 307ra156 (2015).
-
(2015)
Sci Transl Med
, vol.7
, pp. 307ra156
-
-
Sather, B.D.1
-
33
-
-
84949814888
-
Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors
-
Wang, J. et al. Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors. Nat Biotechnol 33, 1256-1263 (2015).
-
(2015)
Nat Biotechnol
, vol.33
, pp. 1256-1263
-
-
Wang, J.1
-
34
-
-
84963516618
-
Targeted gene addition in human CD34 hematopoietic cells for correction of X-linked chronic granulomatous disease
-
De Ravin, S. S. et al. Targeted gene addition in human CD34 hematopoietic cells for correction of X-linked chronic granulomatous disease. Nat Biotechnol 34, 424-431 (2016).
-
(2016)
Nat Biotechnol
, vol.34
, pp. 424-431
-
-
De Ravin, S.S.1
-
35
-
-
79955376402
-
A simple method to increase the transduction efficiency of single-stranded adeno-associated virus vectors in vitro and in vivo
-
Ma, W. et al. A simple method to increase the transduction efficiency of single-stranded adeno-associated virus vectors in vitro and in vivo. Hum Gene Ther 22, 633-640 (2011).
-
(2011)
Hum Gene Ther
, vol.22
, pp. 633-640
-
-
Ma, W.1
-
36
-
-
84880550933
-
A concept of eliminating nonhomologous recombination for scalable and safe AAV vector generation for human gene therapy
-
Dong, B. et al. A concept of eliminating nonhomologous recombination for scalable and safe AAV vector generation for human gene therapy. Nucleic Acids Res 41, 6609-6617 (2013).
-
(2013)
Nucleic Acids Res
, vol.41
, pp. 6609-6617
-
-
Dong, B.1
-
37
-
-
84906667355
-
Efficient production of dual recombinant adeno-associated viral vectors for factor VIII delivery
-
Wang, Q. et al. Efficient production of dual recombinant adeno-associated viral vectors for factor VIII delivery. Hum Gene Ther Methods 25, 261-268 (2014).
-
(2014)
Hum Gene Ther Methods
, vol.25
, pp. 261-268
-
-
Wang, Q.1
|