Detalhe da pesquisa
1.
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
2.
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
3.
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
4.
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
5.
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
6.
Safety and efficacy of gene transfer for Leber's congenital amaurosis.
N Engl J Med
; 358(21): 2240-8, 2008 May 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-18441370
7.
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.
Lancet
; 374(9701): 1597-605, 2009 Nov 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-19854499
8.
Plasticity of the human visual system after retinal gene therapy in patients with Leber's congenital amaurosis.
Sci Transl Med
; 7(296): 296ra110, 2015 Jul 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-26180100
9.
AAV2 gene therapy readministration in three adults with congenital blindness.
Sci Transl Med
; 4(120): 120ra15, 2012 Feb 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-22323828
10.
The human visual cortex responds to gene therapy-mediated recovery of retinal function.
J Clin Invest
; 121(6): 2160-8, 2011 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-21606598