Gene Editing and Sickle Cell Disease

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Sickle cell disease is an inherited condition that predominantly affects people of African descent. The disease results in chronic pain and early death, and is caused by a misfolding of oxygen-toting hemoglobin, a central protein in red blood cells.  Vertex Pharmaceuticals and the CRISPR Therapeutics companies have combined to test a potential therapy.  A patients stem cells are gene edited using CRISPR/Cas9 so that they stop producing adult mutant hemoglobin, and produce a fetal version instead.  The engineered stem cells are returned to the patient, who then manufactures fetal hemoglobin in their own blood cells, potentially curing the disease. Clinical trials have just begun.  Dr. Brenda Eustace, Director of Discovery Research, takes us through the problem, its effects and the Vertex solution that could bring needed relief to millions worldwide.
Website:  VRTX.com
Twitter:  @VertexPharma

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The Talking Biotech podcast is distinct from Dr. Kevin Folta's teaching and research roles at the University of Florida. The views expressed on the show are those of Dr. Folta and his guests, and do not reflect the opinions of the university or Colabra.