1.
Transl Res
; 161(4): 307-12, 2013 Apr.
Artigo
em Inglês
| MEDLINE
| ID: mdl-23352600
RESUMO
Genetic correction of hemophilia A and B was long considered amenable to the available gene transfer technologies. This assumption has come to fruition with the recent results of a phase I/II trial for hemophilia B. Here we review the clinical application of gene therapy for the hemophilia's as a paradigm of the evolution of gene transfer science and technology. This review is not intended as comprehensive but rather to highlight current clinical developments of gene therapy for the hemophilias.