Gene Therapy with Lentivectors Today

Lentiviral vectors are now used in several gene therapy clinical trials worldwide.

They have proven safe and efficient for the following diseases:

  • X-linked adrenoleukodystrophy (article)
  • Metachromatic leukodystrophy (article)
  • Wiskott-Aldrich syndrome (article)

A recent review of Luigi Naldini describes the current research and use of vectors in general and lentivectors in particular in human gene and cell therapies (article). On campuses where you have both the expertise and a vision, lentivectors are now used to treat human diseases such as X-Linked Severe Combined Immunodeficiency (SCID). (link)

Lentivectors are also now widely used for the generation of CAR T-cells. (link)