Contracting CAG/CTG repeats using the CRISPR-Cas9 nickase
CAG/CTG repeat expansions cause over 13 neurological diseases that remain without a cure. Because longer tracts cause more severe phenotypes, contracting them may provide a therapeutic avenue. No currently known agent can specifically generate contractions. Using a GFP-based chromosomal reporter tha...
Main Authors: | , , , |
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Format: | Online |
Language: | English |
Published: |
Nature Publishing Group
2016
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Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5105158/ |