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Updated: Apr 6, 2026

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
Published on: August 9, 2022
Developing stem cell therapies for juvenile and adult-onset Huntington's disease
Kyle D Fink1, Peter Deng1, Audrey Torrest1
1Stem Cell Program & Institute for Regenerative Cures, University of California Davis Health Systems, 2921 Stockton Blvd. Sacramento, CA, USA 95817.
Abstract:
Stem cell therapies have been explored as a new avenue for the treatment of neurologic disease and damage within the CNS in part due to their native ability to mimic repair mechanisms in the brain. Mesenchymal stem cells have been of particular clinical interest due to their ability to release beneficial neurotrophic factors and their ability to foster a neuroprotective microenviroment. While early stem cell transplantation therapies have been fraught with technical and political concerns as well as limited clinical benefits, mesenchymal stem cell therapies have been shown to be clinically beneficial and derivable from nonembryonic, adult sources. The focus of this review will be on emerging and extant stem cell therapies for juvenile and adult-onset Huntington's disease.
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