CRISPR/Cas9 novel therapeutic road for the treatment of neurodegenerative diseases

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 e...

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Veröffentlicht in:Life sciences (1973) 2020-10, Vol.259, p.118165-118165, Article 118165
Hauptverfasser: Karimian, Ansar, Gorjizadeh, Negar, Alemi, Forough, Asemi, Zatollah, Azizian, Khalil, Soleimanpour, Jafar, Malakouti, Faezeh, Targhazeh, Niloufar, Majidinia, Maryam, Yousefi, Bahman
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Sprache:eng
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Zusammenfassung: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. [Display omitted]
ISSN:0024-3205
1879-0631
DOI:10.1016/j.lfs.2020.118165