Tweaked CRISPR in Neurons Gives Scientists New Power to Probe Brain Diseases


By Jason Alvarez (UCSF News)

A team of scientists at UC San Francisco and the National Institutes of Health have achieved another CRISPR first, one which may fundamentally alter the way scientists study brain diseases.

In a paper published Aug. 15 in the journal Neuron, the researchers describe a technique that uses a special version of CRISPR developed at UCSF to systematically alter the activity of genes in human neurons generated from stem cells, the first successful merger of stem cell-derived cell types and CRISPR screening technologies.

Though mutations and other genetic variants are known to be associated with an increased risk for many neurological diseases, technological bottlenecks have thwarted the efforts of scientists working to understand exactly how these genes cause disease. 

“Prior to this study, there were significant limitations that restricted what scientists could do with human neurons in the lab,” said Martin Kampmann, PhD, associate professor in UCSF’s Institute for Neurodegenerative Diseases, a CZ Biohub Investigator, and co-senior author of the new study.

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