Gene therapy returns to centre stage

Nature. 2015 Oct 15;526(7573):351-60. doi: 10.1038/nature15818.


Recent clinical trials of gene therapy have shown remarkable therapeutic benefits and an excellent safety record. They provide evidence for the long-sought promise of gene therapy to deliver 'cures' for some otherwise terminal or severely disabling conditions. Behind these advances lie improved vector designs that enable the safe delivery of therapeutic genes to specific cells. Technologies for editing genes and correcting inherited mutations, the engagement of stem cells to regenerate tissues and the effective exploitation of powerful immune responses to fight cancer are also contributing to the revitalization of gene therapy.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Clinical Trials as Topic
  • Gene Targeting
  • Genetic Therapy / methods*
  • Genetic Therapy / trends*
  • Hematopoietic Stem Cells / metabolism
  • Humans
  • Immunotherapy
  • Liver / metabolism
  • Neoplasms / immunology
  • Neoplasms / pathology
  • Neoplasms / therapy
  • Organ Specificity
  • T-Lymphocytes / immunology
  • T-Lymphocytes / transplantation