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Gene Therapy: An Experimental Technique to Treat Genetic Diseases

key information

source: Sickle Cell Anemia News

year: N/A

summary/abstract:

Gene therapy is an experimental technique that aims to treat genetic diseases by altering a disease-causing gene or introducing a healthy copy of a mutated gene to the body. The U.S. Food and Drug Administration approved the first gene therapy for an inherited disease.

Gene Therapy for Sickle Cell Anemia

Sickle cell anemia is caused by a mutation in the HBB gene which provides the instructions to make part of hemoglobin, the protein in red blood cells that carries oxygen.

Researchers are working on two different strategies to treat sickle cell anemia with gene therapy. Both of these strategies involve genetically altering the patient’s own hematopoietic stem cells. These are cells in the bone marrow that divide and specialize to produce different types of blood cells, including the red blood cells.

 

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