Detalhe da pesquisa
1.
Gene Editing for CEP290-Associated Retinal Degeneration.
N Engl J Med
; 2024 May 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-38709228
2.
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
3.
Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy.
Mol Ther
; 29(2): 442-463, 2021 02 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33278565
4.
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
5.
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
6.
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
7.
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
8.
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
9.
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
10.
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
11.
Evaluation of Ocular Gene Therapy in an Italian Patient Affected by Congenital Leber Amaurosis Type 2 Treated in Both Eyes.
Adv Exp Med Biol
; 854: 533-9, 2016.
Artigo
em Inglês
| MEDLINE | ID: mdl-26427456
12.
Adeno-associated virus 8-mediated gene therapy for choroideremia: preclinical studies in in vitro and in vivo models.
J Gene Med
; 16(5-6): 122-30, 2014.
Artigo
em Inglês
| MEDLINE | ID: mdl-24962736
13.
Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2.
Ophthalmology
; 120(6): 1283-91, 2013 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-23474247
14.
Lessons Learned from the Development of the First FDA-Approved Gene Therapy Drug, Voretigene Neparvovec-rzyl.
Cold Spring Harb Perspect Med
; 13(5)2023 05 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-36167727
15.
Motion-selective areas V5/MT and MST appear resistant to deterioration in choroideremia.
Neuroimage Clin
; 38: 103384, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-37023490
16.
Vitreoretinal lymphoma presenting as frosted branch angiitis in a patient with diffuse large B-cell lymphoma.
Am J Ophthalmol Case Rep
; 30: 101838, 2023 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-37131529
17.
Optimization and Validation of a Virtual Reality Orientation and Mobility Test for Inherited Retinal Degenerations.
Transl Vis Sci Technol
; 12(1): 28, 2023 01 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-36716040
18.
Proliferative retinopathy and retinal detachment in pediatric atypical hemolytic uremic syndrome.
J AAPOS
; 26(1): 31-34, 2022 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-34785364
19.
Efficacy of Maribavir in Valganciclovir-Resistant Cytomegalovirus Retinitis.
Retin Cases Brief Rep
; 2022 Nov 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-36730596
20.
Short-term Assessment of Subfoveal Injection of Adeno-Associated Virus-Mediated hCHM Gene Augmentation in Choroideremia Using Adaptive Optics Ophthalmoscopy.
JAMA Ophthalmol
; 140(4): 411-420, 2022 04 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-35266957