Abstract
The initial results of the first clinical trial of gene therapy in patients with hemophilia B have been reported 1 Kay M.A. et al. Evidence for gene transfer and expression of factor IX in hemophilia B patients treated with an AAV vector. Nat. Genet. 2000; 24: 257-261 Crossref PubMed Scopus (859) Google Scholar . This on-going trial is assessing the intra-muscular injection of an adeno-associated viral (AAV) vector expressing factor IX (F.IX) and, like most of the Phase I trials of gene therapy, the primary goal of the trial is safety.
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