Detalhe da pesquisa
1.
Effect of gene therapy on visual function in Leber's congenital amaurosis.
N Engl J Med
; 358(21): 2231-9, 2008 May 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-18441371
2.
Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi.
Proc Natl Acad Sci U S A
; 105(15): 5868-73, 2008 Apr 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-18398004
3.
Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice.
Mol Ther
; 17(6): 1053-63, 2009 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-19240687
4.
Charting a clear path: the ASGCT Standardized Pathways Conference.
Mol Ther
; 22(7): 1235-1238, 2014 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-24981438
5.
Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial.
Hum Gene Ther
; 18(8): 726-32, 2007 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-17685853
6.
Suppressing Nonsense for Gene Therapy.
Hum Gene Ther
; 33(11-12): 577-578, 2022 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-35537480
7.
AAV Infection: Protection from Cancer.
Hum Gene Ther
; 28(4): 323-327, 2017 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-27832705
8.
Correlation between DNA transfer and cystic fibrosis airway epithelial cell correction after recombinant adeno-associated virus serotype 2 gene therapy.
Hum Gene Ther
; 16(8): 921-8, 2005 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-16076250
9.
My Pathway to Adeno-Associated Virus and Adeno-Associated Virus Gene Therapy: A Personal Perspective.
Hum Gene Ther
; 31(9-10): 494-498, 2020 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-32275185
10.
Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study.
Hum Gene Ther
; 14(11): 1079-88, 2003 Jul 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-12885347
11.
A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies.
Hum Gene Ther
; 13(11): 1349-59, 2002 Jul 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-12162817
12.
Authors' Response to Jesse D. Riordan, Hum Gene Ther 2017;28:375-376; DOI: 10.1089/hum.2017.045.
Hum Gene Ther
; 28(5): 376-377, 2017 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-28498777
13.
Adeno-associated virus vectors in clinical trials.
Hum Gene Ther
; 16(5): 541-50, 2005 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-15916479
14.
Redox modifier genes and pathways in amyotrophic lateral sclerosis.
Antioxid Redox Signal
; 11(7): 1569-86, 2009 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-19187001
15.
Adeno-associated viral vectors for hereditary emphysema; progressing along the clinical development highway.
Hum Gene Ther
; 22(10): 1175-7, 2011 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-22044094
16.
AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice.
Mol Ther
; 9(2): 231-40, 2004 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-14759807
17.
Adeno-associated virus and the development of adeno-associated virus vectors: a historical perspective.
Mol Ther
; 10(6): 981-9, 2004 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-15564130
18.
Nonclinical toxicology in support of licensure of gene therapies. Arlington, VA, USA, March 13-14, 2003.
Mol Ther
; 8(1): 8-10, 2003 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-12872763
19.
Workshop on long-term follow-up of participants in human gene transfer research.
Mol Ther
; 10(6): 976-80, 2004 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-16156012