Saturday, January 31, 2009

Gene Therapy Works and Bubble Boy Lives

  • Gene Therapy works for one type of Severe Combined Immune Deficiency, made popular by the movie "Bubble Boy"
  • The promise of gene therapy exists for other more common diseases such as Sickle Cell Anemia
  • It is much harder to use gene therapy than we thought when the first articles extolling its promise were written about forty years ago.
  • It will involve a lot more work and money to take this promising technology to the point of using it for more common diseases
NEJM January 29, 2009: Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency and Editorial Gene Therapy Fulfilling Its Promise