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CRISPR/Cas9: Implications for Modeling and Therapy of Neurodegenerative Diseases
Weili Yang1, Zhuchi Tu1, Qiang Sun1
1Institute of Genetics and Developmental Biology, Chinese Academy of Sciences Beijing, China.
Abstract:
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 disease (HD), and to treat these disorders that are caused by genetic mutations.
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