A 37-year-old member asked:
help please! is gene therapy a permanent cure for sickle cell anemia?
1 doctor answer
Dr. Craig MacArthur answered
33 years experience Pediatric Hematology and Oncology
In theory yes: As sickle cell anemia is a genetic disease, correction of the gene defect by stem cell transplantation or gene therapy would theoretically correct the genetic defect. Gene therapy is experimental at this time. Stem cell transplantation is more common, but is limited by the availability of suitable tissue matched donors. In either case, correction of the genetic defect will not fix prior strokes.
Answered on May 28, 2016
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