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Life Sci ; 259: 118165, 2020 Oct 15.
Artigo em Inglês | MEDLINE | ID: mdl-32735884

RESUMO

CRISPR (clustered regularly interspaced short palindromic Repeats)/Cas9 is a new genetic editing technology that can be a beneficial method to advance gene therapy. CRISPR technology is a defense system of some bacteria against invading viruses. Genome editing based on the CRISPR/Cas9 system is an efficient and potential technology that can be a viable alternative to traditional methods. This system is a compound of a short guide RNAs (gRNAs) for identifying the target DNA sequence and Cas9 protein as nuclease for breaking and cutting of DNA. In this review, recent advances in the CRISPR/Cas9-mediated genome editing tools are presented as well as their use in gene therapy strategies for the treatment of neurological disorders including Parkinson's disease, Alzheimer's disease, and Huntington's disease.


Assuntos
Sistemas CRISPR-Cas , Terapia Genética/métodos , Doenças Neurodegenerativas/terapia , Animais , Humanos
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