Gene therapy. Factor IX Padua: them that have, give

Blood. 2012 Nov 29;120(23):4452-3. doi: 10.1182/blood-2012-09-452821.

Abstract

In this issue of Blood, Finn et al have taken a factor IX variant with increased specific activity associated with thrombophilia and used it to improve gene therapy of hemophilia B in dogs, and Cantore et al have shown similar results in mice.

Publication types

  • Comment

MeSH terms

  • Animals
  • Factor IX / genetics*
  • Genetic Therapy / methods*
  • Hemophilia B / therapy*
  • Humans
  • Male
  • Mutation*

Substances

  • Factor IX