Detalhe da pesquisa
1.
Central visual pathways affected by degenerative retinal disease before and after gene therapy.
Brain
; 2024 Mar 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-38538211
2.
AAV2 vector optimization for retinal ganglion cell-targeted delivery of therapeutic genes.
Gene Ther
; 31(3-4): 175-186, 2024 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-38200264
3.
Adeno-Associated Virus Serotype 2-hCHM Subretinal Delivery to the Macula in Choroideremia: Two-Year Interim Results of an Ongoing Phase I/II Gene Therapy Trial.
Ophthalmology
; 129(10): 1177-1191, 2022 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-35714735
4.
Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy.
Mol Ther
; 29(2): 442-463, 2021 02 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33278565
5.
Rescue of retinal ganglion cells in optic nerve injury using cell-selective AAV mediated delivery of SIRT1.
Gene Ther
; 28(5): 256-264, 2021 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-33589779
6.
Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease: Phase 3 Results at 3 and 4 Years.
Ophthalmology
; 128(10): 1460-1468, 2021 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-33798654
7.
AKT3 Gene Transfer Promotes Anabolic Reprogramming and Photoreceptor Neuroprotection in a Pre-clinical Model of Retinitis Pigmentosa.
Mol Ther
; 27(7): 1313-1326, 2019 07 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-31043342
8.
Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials.
Ophthalmology
; 126(9): 1273-1285, 2019 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-31443789
9.
Amelioration of Neurosensory Structure and Function in Animal and Cellular Models of a Congenital Blindness.
Mol Ther
; 26(6): 1581-1593, 2018 06 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-29673930
10.
CHOROIDEREMIA: Retinal Degeneration With an Unmet Need.
Retina
; 39(11): 2059-2069, 2019 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-31021898
11.
In vitro and in vivo rescue of aberrant splicing in CEP290-associated LCA by antisense oligonucleotide delivery.
Hum Mol Genet
; 25(12): 2552-2563, 2016 06 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-27106101
12.
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.
Lancet
; 390(10097): 849-860, 2017 Aug 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-28712537
13.
Federal Response to the Opioid Crisis.
Curr HIV/AIDS Rep
; 15(4): 293-301, 2018 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-29968173
14.
Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward.
Mol Ther
; 25(5): 1076-1094, 2017 05 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-28391961
15.
Novel mobility test to assess functional vision in patients with inherited retinal dystrophies.
Clin Exp Ophthalmol
; 46(3): 247-259, 2018 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-28697537
16.
Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.
Lancet
; 388(10045): 661-72, 2016 Aug 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-27375040
17.
The Role of the Human Visual Cortex in Assessment of the Long-Term Durability of Retinal Gene Therapy in Follow-on RPE65 Clinical Trial Patients.
Ophthalmology
; 124(6): 873-883, 2017 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-28237426
18.
Natural History of the Central Structural Abnormalities in Choroideremia: A Prospective Cross-Sectional Study.
Ophthalmology
; 124(3): 359-373, 2017 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-27986385
19.
Gene Therapy and Stem Cell Transplantation in Retinal Disease: The New Frontier.
Ophthalmology
; 123(10S): S98-S106, 2016 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-27664291
20.
Broader Implications of Progressive Liver Dysfunction and Lethal Sepsis in Two Boys following Systemic High-Dose AAV.
Mol Ther
; 28(8): 1753-1755, 2020 08 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-32710826