Detalhe da pesquisa
1.
Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B.
Mol Ther
; 28(9): 2073-2082, 2020 09 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-32559433
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.
Genetic Medicine for Hearing Loss: OTOF as Exemplar.
J Am Acad Audiol
; 32(10): 646-653, 2021 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-35609591
5.
The Natural History of Inherited Retinal Dystrophy Due to Biallelic Mutations in the RPE65 Gene.
Am J Ophthalmol
; 199: 58-70, 2019 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-30268864