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Trends Mol Med. 2017 Jun;23(6):483-485. doi: 10.1016/j.molmed.2017.04.003. Epub 2017 May 4.

A CRISPR Approach to Neurodegenerative Diseases.

Author information

1
Institute for Neurodegenerative Diseases, Weill Institute for Neurosciences, University of California, San Francisco, San Francisco, CA, USA; Department of Biochemistry and Biophysics, University of California, San Francisco, San Francisco, CA, USA; Chan-Zuckerberg Biohub, San Francisco, CA, USA; California Institute for Quantitative Biomedical Research (QB3), University of California, San Francisco, San Francisco, CA, USA; Quantitative Biosciences Institute (QBI), University of California, San Francisco, San Francisco, CA, USA. Electronic address: martin.kampmann@ucsf.edu.

Abstract

A major barrier to developing effective therapies for neurodegenerative diseases is our incomplete understanding of the underlying cellular mechanisms. Genetic screens in human-induced pluripotent stem cell-derived neurons can elucidate such mechanisms. Genome-wide screens using CRISPR interference and CRISPR activation provide complementary biological insights and may reveal potential therapeutic targets.

PMID:
28478951
PMCID:
PMC5604856
DOI:
10.1016/j.molmed.2017.04.003
[Indexed for MEDLINE]
Free PMC Article

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