Manuscript update alert!
Correcting expanded repeats with the #CRISPR Cas9 nickase.
In the brain!
Of a #Huntington’s disease model!
With improvements to molecular phenotypes!
And rescued locomotion!
A massive team effort.
www.biorxiv.org/content/10.1...
biorxiv.org
Cas9 Nickase-Mediated Contraction of CAG/CTG Repeats in Vivo is Accompanied by Improvements in Huntington's Disease Pathology
Expanded CAG/CTG repeats cause over 15 different diseases that all remain without a disease-modifying treatment. Because repeat length accounts for most of the variation in disease severity, contracti...