Anita wang

Biotechnology Research Center, Cuixi Academy of Biotechnology, Zhuji, 311800, Zhejiang, China
Author    Correspondence author
International Journal of Molecular Medical Science, 2024, Vol. 14, No. 3   
Received: 18 May, 2024    Accepted: 20 Jun., 2024    Published: 30 Jun., 2024

This is an open access article published under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

Sickle cell anemia (SCA) is a severe monogenic disorder characterized by the presence of abnormal hemoglobin, leading to various clinical complications. Recent advancements in gene editing technologies, particularly CRISPR/Cas9, have shown promising potential in treating SCA by targeting and modifying specific genetic loci to upregulate fetal hemoglobin (HbF) or correct the sickle mutation. This study explores the prospects of gene editing technologies in the treatment of SCA, focusing on the efficacy, safety, and durability of these approaches. Studies have demonstrated that CRISPR/Cas9 can efficiently edit hematopoietic stem and progenitor cells (HSPCs) to recreate genetic variants associated with elevated HbF, resulting in significant therapeutic benefits. Additionally, various delivery methods for CRISPR/Cas9, including ribonucleoprotein complexes and lentiviral vectors, have been optimized to balance efficiency and cytotoxicity. Clinical trials and preclinical studies have shown that gene-edited cells can engraft and persist in vivo, maintaining therapeutic benefits over extended periods. Despite these advancements, challenges such as off-target effects and the need for improved targeting methods remain. This study provides a comprehensive overview of the current state of gene editing technologies in SCA treatment, highlighting key findings and future directions.

Sickle cell anemia; CRISPR/Cas9; Fetal hemoglobin; Gene editing; Hematopoietic stem cells