Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.

ABSTRACT:

Background

Hemophilia B, an X-linked disorder, is ideally suited for gene therapy. We investigated the use of a new gene therapy in patients with the disorder.

Methods

We infused a single dose of a serotype-8-pseudotyped, self-complementary adenovirus-associated virus (AAV) vector expressing a codon-optimized human factor IX (FIX) transgene (scAAV2/8-LP1-hFIXco) in a peripheral vein in six patients with severe hemophilia B (FIX activity, <1% of normal values). Study participants were enrolled sequentially in one of three cohorts (given a high, intermediate, or low dose of vector), with two participants in each group. Vector was administered without immunosuppressive therapy, and participants were followed for 6 to 16 months.

Results

AAV-mediated expression of FIX at 2

SUBMITTER: Nathwani AC 

PROVIDER: S-EPMC3265081 | biostudies-literature | 2011 Dec

REPOSITORIES: biostudies-literature