What is the role of genetic manipulation (CRISPR) in the treatment of sickle cell disease (SCD)?

Updated: Sep 30, 2020
  • Author: Mark Ventocilla, OD, FAAO; Chief Editor: Hampton Roy, Sr, MD  more...
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Answer

Answer

Recent advances in genetic manipulation tools such as CRISPR raise the possibility that a systemic cure for SCD may be attainable. One such study involves CRISPR Therapeutics and Vertex Pharmaceuticals, who seek to enroll approximately 45 people aged 18-35 years in a joint study to determine if genetically modifying blood cells with CRISPR permanently remedies faulty sickle cells. This and other similar studies may offer great hope for individuals with SCD.

Results presented in June 2020 at the Annual European Hematology Association Congress indicate that the first patient treated through the Vertex study for SCD was free from painful crisis at 9 months after treatment. Some side effects were reported but were believed to be caused by chemotherapy rather than genome editing. [14]


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