Detalhe da pesquisa
1.
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A.
N Engl J Med
; 385(21): 1961-1973, 2021 11 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-34788507
2.
THE JEREMIAH METZGER LECTURE: TURNING GENES INTO MEDICINES: HIGHLIGHTS AND HURDLES IN THE DEVELOPMENT OF GENE THERAPY FOR GENETIC DISEASE.
Trans Am Clin Climatol Assoc
; 133: 204-233, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-37701622
3.
Entering the Modern Era of Gene Therapy.
Annu Rev Med
; 70: 273-288, 2019 01 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-30477394
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.
The Effect of CpG Sequences on Capsid-Specific CD8+ T Cell Responses to AAV Vector Gene Transfer.
Mol Ther
; 28(3): 771-783, 2020 03 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-31839483
6.
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
7.
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
N Engl J Med
; 377(23): 2215-2227, 2017 12 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-29211678
8.
Gene and cell therapy in 2023: Rich pipeline, slimming resources?
Mol Ther
; 32(1): 3-4, 2024 Jan 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-38118443
9.
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
10.
Adeno-associated viral vectors for the treatment of hemophilia.
Hum Mol Genet
; 25(R1): R36-41, 2016 Apr 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-26614390
11.
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
12.
Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII.
Blood
; 127(5): 565-71, 2016 Feb 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-26702064
13.
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
14.
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
15.
Gene Therapy.
N Engl J Med
; 381(5): 455-464, 2019 08 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31365802
16.
Long-term safety and efficacy of factor IX gene therapy in hemophilia B.
N Engl J Med
; 371(21): 1994-2004, 2014 Nov 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-25409372
17.
In vivo genome editing of the albumin locus as a platform for protein replacement therapy.
Blood
; 126(15): 1777-84, 2015 Oct 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-26297739
18.
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.
Nat Rev Genet
; 12(5): 341-55, 2011 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-21499295
19.
In vivo genome editing restores haemostasis in a mouse model of haemophilia.
Nature
; 475(7355): 217-21, 2011 Jun 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-21706032
20.
Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid.
Mol Ther
; 24(6): 1042-1049, 2016 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-27019999