Progress and challenges in CRISPR-mediated therapeutic genome editing for monogenic diseases

• 作者: Colin T.Konishi Chengzu Long
• 来源期刊: 生物医学研究杂志（英文版）
• 年，卷（期）: 2021,（2）
• 所属期刊栏目: REVIEW ARTICLES
• 页码范围: 148-162
• 页数: 15页
• 分类号: Q789
• 语种: 英文

摘要

There are an estimated 10 000 monogenic diseases affecting tens of millions of individuals worldwide.The application of CRISPR/Cas genome editing tools to treat monogenic diseases is an emerging strategy with the potential to generate personalized treatment approaches for these patients.CRISPR/Cas-based systems are programmable and sequence-specific genome editing tools with the capacity to generate base pair resolution manipulations to DNA or RNA.The complexity of genomic insults resulting in heritable disease requires patientspecific genome editing strategies with consideration of DNA repair pathways,and CRISPR/Cas systems of different types,species,and those with additional enzymatic capacity and/or delivery methods.In this review we aim to discuss broad and multifaceted therapeutic applications of CRISPR/Cas gene editing systems including in harnessing of homology directed repair,non-homologous end joining,microhomology-mediated end joining,and base editing to permanently correct diverse monogenic diseases.