Detalhe da pesquisa
1.
Pompe disease gene therapy.
Hum Mol Genet
; 20(R1): R61-8, 2011 Apr 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-21518733
2.
Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation.
Mol Ther Methods Clin Dev
; 30: 303-314, 2023 Sep 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-37601414
3.
A simplified purification protocol for recombinant adeno-associated virus vectors.
Mol Ther Methods Clin Dev
; 1: 14034, 2014.
Artigo
em Inglês
| MEDLINE | ID: mdl-26015974
4.
Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes.
Hum Gene Ther
; 24(6): 630-40, 2013 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-23570273