ISSN : 2663-2187

Gene Therapy Treatment for Sickle Cell Disease: Casgevy

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Mr. Harshkumar Brahmbhatt,Gadhavi Shrushti,Mitali Dalwadi,,Mr. Mahavir Sharma,Dr. Ashim kumar Sen
ยป doi: 10.48047/AFJBS.6.5.2024.10371-10379

Abstract

Sickle cell disease (SCD) presents a notable burden on global health, necessitating innovative approaches beyond traditional biomedical paradigms. While existing treatments aim to alleviate symptoms, recent advancements in gene therapy, exemplified by CASGEVY (Exagamglogene Autotemcel), offer transformative potential. CASGEVY, Introducing the maiden FDA-approved gene therapy employing CRISPR/Cas9 technology, targets recurrent vaso-occlusive crises by enhancing fetal hemoglobin (HbF) production. This ex vivo therapy involves precise genome editing of hematopoietic stem cells, promising enduring benefits by reactivating HbF expression. Despite its groundbreaking nature, CASGEVY warrants thorough consideration of administration protocols and potential side effects, emphasizing the need for comprehensive patient evaluation and monitoring.

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