Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

ABSTRACT:

Background

In patients with severe hemophilia B, gene therapy that is mediated by a novel self-complementary adeno-associated virus serotype 8 (AAV8) vector has been shown to raise factor IX levels for periods of up to 16 months. We wanted to determine the durability of transgene expression, the vector dose-response relationship, and the level of persistent or late toxicity.

Methods

We evaluated the stability of transgene expression and long-term safety in 10 patients with severe hemophilia B: 6 patients who had been enrolled in an initial phase 1 dose-escalation trial, with 2 patients each receiving a low, intermediate, or high dose, and 4 additional patients who received the high dose (2×10(12) vector genomes per kilogram of body weight). The patients subsequently underwen

SUBMITTER: Nathwani AC 

PROVIDER: S-EPMC4278802 | biostudies-literature | 2014 Nov

REPOSITORIES: biostudies-literature