A CRISPR Approach to Neurodegenerative Diseases

Martin Kampmann

doi:10.1016/j.molmed.2017.04.003

A CRISPR Approach to Neurodegenerative Diseases

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

Author

Martin Kampmann¹

Affiliation

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.

Publication types

Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't

MeSH terms

Animals
CRISPR-Cas Systems*
Cell Differentiation*
Humans
Induced Pluripotent Stem Cells / metabolism*
Neurodegenerative Diseases* / genetics
Neurodegenerative Diseases* / metabolism
Neurodegenerative Diseases* / therapy
Neurons / metabolism*

Abstract

Publication types

MeSH terms

Grants and funding