CRISPR/Cas9: implications for modeling and therapy of neurodegenerative diseases

Frontiers in Molecular Neuroscience

Xiao-Jiang eLi¹  Zhuchi eTu²  Qiang eSun²  Weili eYang² 

[1] Emory University School of Medicine;Institute of Genetics and Developmental Biology;

关键词: Gene Therapy;    Neurodegenerative Diseases;    Animal Models;    CRISPR/Cas9;    Genetic mutations;   

DOI  :  10.3389/fnmol.2016.00030

来源: DOAJ

【 摘 要 】

CRISPR/Cas9 is now used widely to genetically modify the genomes of various species. The ability of CRISPR/Cas9 to delete DNA sequences and correct DNA mutations opens up a new avenue to treat genetic diseases that are caused by DNA mutations. In this review, we describe the advantages of using CRISPR/Cas9 to engineer genomic DNAs in animal embryos, as well as in specific regions or cell types in the brain. We also discuss how to apply CRISPR/Cas9 to establish animal models of neurodegenerative diseases, such as Parkinson’s and Huntington's diseases, and to treat these disorders that are caused by genetic mutations.

【 授权许可】

Unknown