Detalhe da pesquisa
1.
Design of experiments as a decision tool for cell therapy manufacturing.
Cytotherapy
; 24(6): 590-596, 2022 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-35227602
2.
Safety Assessment of AAV2-hGDNF Administered Via Intracerebral Injection in Rats for Treatment of Parkinson's Disease.
Int J Toxicol
; 40(1): 4-14, 2021.
Artigo
em Inglês
| MEDLINE | ID: mdl-33131343
3.
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
4.
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
5.
Re-administration of AAV vectors by masking with host albumin: A Goldilocks hypothesis.
Mol Ther
; 31(7): 1870-1873, 2023 07 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-37369207
6.
Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery.
Mol Ther
; 26(9): 2282-2294, 2018 09 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-30196853
7.
Progress and challenges in viral vector manufacturing.
Hum Mol Genet
; 25(R1): R42-52, 2016 Apr 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-26519140
8.
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
9.
AAV vector manufacturing process design and scalability - Bending the trajectory to address vector-associated immunotoxicities.
Mol Ther
; 30(6): 2119-2121, 2022 06 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-35594866
10.
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
11.
Chimeric antigen receptor-modified T cells for acute lymphoid leukemia.
N Engl J Med
; 368(16): 1509-1518, 2013 Apr 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-23527958
12.
Quantification of CpG Motifs in rAAV Genomes: Avoiding the Toll.
Mol Ther
; 28(8): 1756-1758, 2020 08 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-32710825
13.
Codon Modification and PAMPs in Clinical AAV Vectors: The Tortoise or the Hare?
Mol Ther
; 28(3): 701-703, 2020 03 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-32035026
14.
AAV-mediated expression of an ADAMTS13 variant prevents shigatoxin-induced thrombotic thrombocytopenic purpura.
Blood
; 121(19): 3825-9, S1-3, 2013 May 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-23515928
15.
Cerebral infusion of AAV9 vector-encoding non-self proteins can elicit cell-mediated immune responses.
Mol Ther
; 21(1): 158-66, 2013 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-22929660
16.
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
17.
Assessing the potential for AAV vector genotoxicity in a murine model.
Blood
; 117(12): 3311-9, 2011 Mar 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-21106988
18.
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.
Nat Med
; 12(3): 342-7, 2006 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-16474400
19.
Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B.
Mol Ther
; 20(7): 1410-6, 2012 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-22565846
20.
Safe, long-term hepatic expression of anti-HCV shRNA in a nonhuman primate model.
Mol Ther
; 20(9): 1737-49, 2012 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-22735378