Gene therapy finds its niche

Nat Biotechnol. 2011 Feb;29(2):121-8. doi: 10.1038/nbt.1769.


Gene therapy is finally poised to make a contribution to the treatment of debilitating, highly penetrant genetic diseases that have proved intractable to other regimens.

MeSH terms

  • Adult
  • Child
  • Clinical Trials as Topic
  • Genetic Therapy*
  • Genetic Vectors
  • Humans