ASH 2023 | Correcting the function of mutated hemoglobin with gene therapy for SCD
Akshat Jain
• 8 Dec 2023
Akshat Jain, MD, MPH, FRCP, Loma Linda University, San Bernadino, CA, shares insights into the recently FDA-approved gene therapies for the treatment of sickle cell disease (SCD). Utilizing CRIPSR/Cas9 technology or lentiviral-based vectors, these therapies edit the genome to correct dysfunctional hemoglobin, preventing the sickling of red blood cells. This mitigates anemia and reduces pain ratings reported by patients. This interview took place at the 65th ASH Annual Meeting and Exposition, held in San Diego, CA.
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