CRISPR/Cas9: implication for modeling and therapy of amyotrophic lateral sclerosis

Frontiers in Neuroscience

Neuroscience

Yan Zhao¹  Likui Lu¹  Dongyi Yu²  Qinqin Gao³  Miao Sun³  Yajun Shi³ 

[1] Institute for Fetology, the First Affiliated Hospital of Soochow University, Suzhou, Jiangsu, China;Key Laboratory of Birth Defect Prevention and Genetic Medicine of Shandong Health Commission, Key Laboratory of Birth Regulation and Control Technology of National Health Commission of China, Center for Medical Genetics and Prenatal Diagnosis, Shandong Provincial Maternal and Child Health Care Hospital Affiliated to Qingdao University, Jinan, Shandong, China;Key Laboratory of Birth Defect Prevention and Genetic Medicine of Shandong Health Commission, Key Laboratory of Birth Regulation and Control Technology of National Health Commission of China, Center for Medical Genetics and Prenatal Diagnosis, Shandong Provincial Maternal and Child Health Care Hospital Affiliated to Qingdao University, Jinan, Shandong, China;Institute for Fetology, the First Affiliated Hospital of Soochow University, Suzhou, Jiangsu, China;

关键词: amyotrophic lateral sclerosis;    gene editing;    CRISPR/Cas9;    ALS models;    gene therapeutic;    clinical trials;   

DOI  :  10.3389/fnins.2023.1223777

 received in 2023-05-16, accepted in 2023-06-21,  发布年份 2023

来源: Frontiers

【 摘 要 】

Amyotrophic lateral sclerosis (ALS) is a deadly neurological disease with a complicated and variable pathophysiology yet to be fully understood. There is currently no effective treatment available to either slow or terminate it. However, recent advances in ALS genomics have linked genes to phenotypes, encouraging the creation of novel therapeutic approaches and giving researchers more tools to create efficient animal models. Genetically engineered rodent models replicating ALS disease pathology have a high predictive value for translational research. This review addresses the history of the evolution of gene editing tools, the most recent ALS disease models, and the application of CRISPR/Cas9 against ALS disease.

【 授权许可】

Unknown   
Copyright © 2023 Shi, Zhao, Lu, Gao, Yu and Sun.