Detalhe da pesquisa
1.
Intravitreal injection of a rationally designed AAV capsid library in non-human primate identifies variants with enhanced retinal transduction and neutralizing antibody evasion.
Mol Ther
; 31(12): 3441-3456, 2023 Dec 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-37814449
2.
Optimization of Capillary-Based Western Blotting for MYO7A.
Adv Exp Med Biol
; 1415: 125-130, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-37440024
3.
Effects of Altering HSPG Binding and Capsid Hydrophilicity on Retinal Transduction by AAV.
J Virol
; 95(10)2021 04 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-33658343
4.
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Mol Ther
; 29(2): 464-488, 2021 02 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33309881
5.
Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse.
Gene Ther
; 28(7-8): 447-455, 2021 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-33244179
6.
Novel AAV44.9-Based Vectors Display Exceptional Characteristics for Retinal Gene Therapy.
Mol Ther
; 28(6): 1464-1478, 2020 06 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-32304666
7.
Identifying Treatments for Taste and Smell Disorders: Gaps and Opportunities.
Chem Senses
; 45(7): 493-502, 2020 10 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-32556127
8.
A Drug-Tunable Gene Therapy for Broad-Spectrum Protection against Retinal Degeneration.
Mol Ther
; 26(10): 2407-2417, 2018 10 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-30078764
9.
Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse.
Gene Ther
; 30(9): 736, 2023 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-37495717
10.
Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations.
Mol Ther
; 25(8): 1866-1880, 2017 08 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-28566226
11.
Intravitreal delivery of a novel AAV vector targets ON bipolar cells and restores visual function in a mouse model of complete congenital stationary night blindness.
Hum Mol Genet
; 24(21): 6229-39, 2015 Nov 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-26310623
12.
Impact of Heparan Sulfate Binding on Transduction of Retina by Recombinant Adeno-Associated Virus Vectors.
J Virol
; 90(8): 4215-4231, 2016 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-26865709
13.
Functional study of two biochemically unusual mutations in GUCY2D Leber congenital amaurosis expressed via adenoassociated virus vector in mouse retinas.
Mol Vis
; 22: 1342-1351, 2016.
Artigo
em Inglês
| MEDLINE | ID: mdl-27881908
14.
Targeted gene delivery to the enteric nervous system using AAV: a comparison across serotypes and capsid mutants.
Mol Ther
; 23(3): 488-500, 2015 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-25592336
15.
Gene therapy with the caspase activation and recruitment domain reduces the ocular inflammatory response.
Mol Ther
; 23(5): 875-884, 2015 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-25698151
16.
A Mini-review: Animal Models of GUCY2D Leber Congenital Amaurosis (LCA1).
Adv Exp Med Biol
; 854: 253-8, 2016.
Artigo
em Inglês
| MEDLINE | ID: mdl-26427419
17.
Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber's hereditary optic neuropathy in a mouse model.
Proc Natl Acad Sci U S A
; 109(20): E1238-47, 2012 May 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-22523243
18.
A comprehensive review of retinal gene therapy.
Mol Ther
; 21(3): 509-19, 2013 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-23358189
19.
Cone specific promoter for use in gene therapy of retinal degenerative diseases.
Adv Exp Med Biol
; 801: 695-701, 2014.
Artigo
em Inglês
| MEDLINE | ID: mdl-24664760
20.
Long-term RNA interference gene therapy in a dominant retinitis pigmentosa mouse model.
Proc Natl Acad Sci U S A
; 108(45): 18476-81, 2011 Nov 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-22042849