Long-term safety and efficacy of factor IX gene therapy in hemophilia B Academic Article uri icon

Overview

MeSH Major

  • Factor IX
  • Genetic Therapy
  • Genetic Vectors
  • Hemophilia B

abstract

  • In 10 patients with severe hemophilia B, the infusion of a single dose of AAV8 vector resulted in long-term therapeutic factor IX expression associated with clinical improvement. With a follow-up period of up to 3 years, no late toxic effects from the therapy were reported. (Funded by the National Heart, Lung, and Blood Institute and others; ClinicalTrials.gov number, NCT00979238.).

publication date

  • January 2014

Research

keywords

  • Academic Article

Identity

Language

  • eng

PubMed Central ID

  • PMC4278802

Digital Object Identifier (DOI)

  • 10.1056/NEJMoa1407309

PubMed ID

  • 25409372

Additional Document Info

start page

  • 1994

end page

  • 2004

volume

  • 371

number

  • 21