Researchers use CRISPR to reverse Huntington's in mice

In a paper published in the Journal of Clinical Investigation, Emory University researchers showed CRISPR/Cas9 gene editing in brain cells reversed Huntington's disease (HD) pathology and symptoms in a mouse model of the disease.

In a knock-in mouse model of human mutant huntingtin (HTT), adeno-associated virus (AAV) vector-mediated delivery of Cas9 and single guide RNA (sgRNA) targeting HTT in the striatum decreased nuclear accumulation and aggregation of mutant HTT and alleviated motor deficits and neurological symptoms...