Progress and challenges in CRISPR-mediated therapeutic genome editing for monogenic diseases
Colin T.Konishi1
Chengzu Long2
1.Leon H.Charney Division of Cardiology, New York University Grossman School of Medicine, New York, NY 10016, USA2.Leon H.Charney Division of Cardiology, New York University Grossman School of Medicine, New York, NY 10016, USA;Helen and Martin Kimmel Center for Stem Cell Biology, New York University Grossman School of Medicine, New York, NY 10016, USA;Department of Neurology, New York University Grossman School of Medicine, New York, NY 10016, USA;Department of Neuroscience and Physiology, New York University Grossman School of Medicine, New York, NY 10016, USA
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.
机标关键词：
分类号：Q789(基因工程（遗传工程）)
论文发表日期：2021-03-30
在线出版日期：2025-08-15（本平台首次上网日期，不代表文献的发表时间）
页数：15( 148-162 )
英文信息