Genetic Targeting of the Albumin Locus to Treat Hemophilia

N Engl J Med. 2016 Mar 31;374(13):1288-90. doi: 10.1056/NEJMcibr1600347.
No abstract available

MeSH terms

  • Animals
  • Dependovirus / genetics
  • Disease Models, Animal
  • Factor IX / genetics*
  • Gene Targeting*
  • Gene Transfer Techniques*
  • Genetic Vectors
  • Hemophilia B / genetics
  • Hemophilia B / therapy*
  • Humans
  • Liver
  • Mice

Substances

  • Factor IX