Detalhe da pesquisa
1.
Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model.
Gene Ther
; 29(9): 520-535, 2022 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-35105949
2.
Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital Amaurosis.
Mol Ther
; 26(1): 256-268, 2018 01 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-29033008
3.
AAV-ID: A Rapid and Robust Assay for Batch-to-Batch Consistency Evaluation of AAV Preparations.
Mol Ther
; 25(6): 1375-1386, 2017 06 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-28427840
4.
AAV-mediated Gene Therapy Halts Retinal Degeneration in PDE6ß-deficient Dogs.
Mol Ther
; 24(5): 867-76, 2016 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-26857842
5.
Successful gene therapy in the RPGRIP1-deficient dog: a large model of cone-rod dystrophy.
Mol Ther
; 22(2): 265-277, 2014 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-24091916
6.
Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates.
Mol Ther
; 22(9): 1605-13, 2014 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-24954473
7.
Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.
Mol Ther
; 22(11): 1923-35, 2014 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-25200009
8.
Humoral and cellular capsid-specific immune responses to adeno-associated virus type 1 in randomized healthy donors.
J Immunol
; 188(12): 6418-24, 2012 Jun 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-22593612
9.
Mannose-coupled AAV2: A second-generation AAV vector for increased retinal gene therapy efficiency.
Mol Ther Methods Clin Dev
; 32(1): 101187, 2024 Mar 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-38327809
10.
Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6.
J Virol
; 86(12): 6620-31, 2012 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-22496229
11.
PCR-based detection of gene transfer vectors: application to gene doping surveillance.
Anal Bioanal Chem
; 405(30): 9641-53, 2013 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-23912835
12.
Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver.
Mol Ther
; 20(6): 1177-86, 2012 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-22453768
13.
Restoration of vision in the pde6ß-deficient dog, a large animal model of rod-cone dystrophy.
Mol Ther
; 20(11): 2019-30, 2012 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-22828504
14.
Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy.
Mol Ther Methods Clin Dev
; 30: 30-47, 2023 Sep 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-37746247
15.
Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate.
Hum Mol Genet
; 19(1): 147-58, 2010 Jan 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-19837699
16.
A 10 patient case report on the impact of plasmapheresis upon neutralizing factors against adeno-associated virus (AAV) types 1, 2, 6, and 8.
Mol Ther
; 19(11): 2084-91, 2011 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-21629225
17.
Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes.
Mol Ther
; 19(2): 251-9, 2011 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-21139569
18.
Cellular and Structural Characterization of VP1 and VP2 Knockout Mutants of AAV3B Serotype and Implications for AAV Manufacturing.
Hum Gene Ther
; 33(21-22): 1142-1156, 2022 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-36082996
19.
Neonatal systemic delivery of scAAV9 in rodents and large animals results in gene transfer to RPE cells in the retina.
Exp Eye Res
; 93(4): 491-502, 2011 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-21723863
20.
Regulation of retinal function but nonrescue of vision in RPE65-deficient dogs treated with doxycycline-regulatable AAV vectors.
Mol Ther
; 18(6): 1085-93, 2010 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-20354505