A 37-year-old member asked:
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help please! is gene therapy a permanent cure for sickle cell anemia?

1 doctor answer
Dr. Craig MacArthur
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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Related questions:

A 40-year-old member asked:
Dr. Aniket Saha
13 years experience Pediatric Hematology and Oncology
Not as far I know: This may be the future. But as of right now, I am not aware of gene therapy for sickle cell disease. The only cure for it at the moment is bone marr ... Read More
A 46-year-old member asked:
Dr. Peter Francis
28 years experience Retinal Surgery
Replace gene: Gene therapy could be used to transfer a new replacement normal gene to cells producing red blood cells. That is how gene therapy would work however t ... Read More
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A 21-year-old female asked:
Dr. Michael Gabor
32 years experience Diagnostic Radiology
Gene mutations: are random. The sickle gene confers some resistance to malaria due to the abnormal configuration of hemoglobin. Sickle trait/sickle cell anemia is ... Read More
A 46-year-old member asked:
Dr. Michael Dugan
Specializes in Hematology
It should not: Be a problem.

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