Detalhe da pesquisa
1.
ß-secretase 1 overexpression by AAV-mediated gene delivery prevents retina degeneration in a mouse model of age-related macular degeneration.
Mol Ther
; 31(7): 2042-2055, 2023 07 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-37016576
2.
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
3.
Optimization of Capillary-Based Western Blotting for MYO7A.
Adv Exp Med Biol
; 1415: 125-130, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-37440024
4.
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
5.
Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis.
Mol Ther
; 29(8): 2456-2468, 2021 08 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-33781914
6.
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
7.
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
8.
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
9.
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
10.
Complement C3-Targeted Gene Therapy Restricts Onset and Progression of Neurodegeneration in Chronic Mouse Glaucoma.
Mol Ther
; 26(10): 2379-2396, 2018 10 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-30217731
11.
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
12.
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
13.
Overexpression of Type 3 Iodothyronine Deiodinase Reduces Cone Death in the Leber Congenital Amaurosis Model Mice.
Adv Exp Med Biol
; 1074: 125-131, 2018.
Artigo
em Inglês
| MEDLINE | ID: mdl-29721936
14.
Consequences of zygote injection and germline transfer of mutant human mitochondrial DNA in mice.
Proc Natl Acad Sci U S A
; 112(42): E5689-98, 2015 Oct 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-26438859
15.
Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease.
Proc Natl Acad Sci U S A
; 112(43): E5844-53, 2015 Oct 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-26460017
16.
Viral-mediated vision rescue of a novel AIPL1 cone-rod dystrophy model.
Hum Mol Genet
; 24(3): 670-84, 2015 Feb 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-25274777
17.
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
18.
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
19.
Long-term photoreceptor rescue in two rodent models of retinitis pigmentosa by adeno-associated virus delivery of Stanniocalcin-1.
Exp Eye Res
; 165: 175-181, 2017 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-28974356
20.
Targeting iodothyronine deiodinases locally in the retina is a therapeutic strategy for retinal degeneration.
FASEB J
; 30(12): 4313-4325, 2016 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-27623928