medicalxpress.com· Medical Xpress - Genetics·· 17 hours agoPickScore67
New gene-editing approach shows promise in treating Huntington's disease in mice
In brief
A Cardiff University team used Cas9 nickase, which cuts a single DNA strand, in mouse models of Huntington's disease. The approach shortened CAG/CTG repeats in the majority of targeted neurons and improved motor function.
Why it matters
Provides a mechanistic comparison of Cas9 nickase single-strand cutting with conventional double-strand cutting and reports animal results on CAG/CTG repeats, protein aggregation and motor function.
Source: medicalxpress.com · medicalxpress.com