Gene Therapy Uses HIV to Rescue Kids Born Without Immune System


Sumary of Gene Therapy Uses HIV to Rescue Kids Born Without Immune System:

  • The life-saving therapy utilizes a harmless, modified form of the AIDS-causing human immunodeficiency virus (HIV) to work its magic..
  • Cora is one of 48 out of 50 young children who were essentially cured of their severe combined immunodeficiency (SCID) using this new procedure, researchers reported May 11 in the New England Journal of Medicine..
  • The children all suffered from a form of immune deficiency called ADA-SCID, in which a faulty gene causes a buildup in the bloodstream of a naturally occurring biochemical called adenosine..
  • “Lymphocytes are particularly sensitive to adenosine, so you basically poison off the lymphocytes, and that’s what results in SCID,”.
  • Kids with this form of SCID are kept alive by receiving regular injections of the enzyme they cannot make that breaks down adenosine, along with a battery of other medications that take the place of their missing immune system….

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