Detalhe da pesquisa
1.
5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency.
Mol Ther
; 25(6): 1387-1394, 2017 06 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-28408179
2.
Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy.
Ophthalmology
; 123(7): 1606-20, 2016 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-27102010
3.
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy.
Proc Natl Acad Sci U S A
; 106(38): 16363-8, 2009 Sep 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-19706466
4.
Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy.
Hum Gene Ther
; 29(12): 1428-1437, 2018 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-29869534
5.
Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults.
Hum Gene Ther
; 17(12): 1177-86, 2006 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-17115945
6.
Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults.
Hum Gene Ther
; 15(1): 93-128, 2004 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-14965381
7.
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
8.
Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression.
J Clin Invest
; 123(12): 5310-8, 2013 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-24231351
9.
Preclinical evaluation of a recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin made using a recombinant herpes simplex virus production method.
Hum Gene Ther
; 22(2): 155-65, 2011 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-20812844
10.
Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results.
Hum Gene Ther
; 22(10): 1239-47, 2011 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-21609134
11.
Recent developments in the protection of pediatric research subjects.
J Pediatr
; 149(3): 285-286, 2006 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-16939731