New insights into applications of base editor in hereditary disorders

Le résumé fourni par la source

Abstract Hereditary disorders are a group of diseases caused by genetic mutations or chromosomal variations. Although the incidence of each genetic disorder is relatively low, patients affected by the disease generally experience a range of severe symptoms, including blindness, disability, and even premature death. In addition, the available treatments for hereditary disorders are limited. Fortunately, the emergence of advanced gene‐editing tools such as Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and base editors (BEs) has provided promising prospects for gene editing therapies in treating hereditary disorders. In this review, we mainly summarize the recent progress on BEs and their applications in hereditary disorders. Additionally, we discuss the prospects and challenges associated with the use of BEs, aiming to provide new insights into the area of gene therapy.

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.