Gene Therapy for Hemoglobinopathies: Tremendous Successes and Remaining Caveats

Mol Ther. 2016 Apr;24(4):668-70. doi: 10.1038/mt.2016.57.

MeSH terms

  • Animals
  • Clinical Trials as Topic
  • Disease Models, Animal
  • Genetic Therapy / methods*
  • Genetic Vectors / genetics
  • Hemoglobinopathies / therapy*
  • Humans
  • Lentivirus / genetics