Some Useful Articles on Gene Therapy

It is difficult to avoid the promise of gene therapy with article appearing the popular press on almost a daily basis.  Today I read article on gene therapy in Time magazine on the bus on the way to the gym.  While in vivo application of the technology of gene therapy including gene editing is a heady prospect, the problem of accurate delivery (off-target effects).  It is far more likely that engineered stem cells will be the first to market.  An article in Nature Biotechnology (1)describes the use of hematopoietic stem cells in severe combined immunodeficiency (SCID) while a very short (and available) article by Alain Fischer (2) in Comptes Rendue Biologies describes the stem cell engineering process.  Both articles are highly recommended for those who wish to keep up with the rapidly moving field of gene therapy.

  1.  Scott, C.T. and DeFranscesco, L., Gene therapy’s out-of-body experience, Nature Biotechnology 34, 600-607, 2016
  2. Fischer, A., Gene therapy: Myth or reality?, Comp.Rend.Biol. in press, 2016