CRISPR Technology Directly Targeting RNA Improves Huntington’s Disease Symptoms in Models

A three-dimensional illustration of the huntingtin protein. Produced in overabundance by the mutated HTT gene, the mutant huntingtin protein causes Huntington’s disease. (Image courtesy of Cambridge Independent).

Huntington’s disease (HD) is a neurological disorder that causes progressive loss of movement, coordination, and cognitive function. It is caused by a mutation in a single gene called huntingtin or HTT. More than 200,000 people worldwide live with the dominantly inherited genetic condition, approximately 30,000 in the United States. More than a quarter of a million Americans are at risk of inheriting HD from an affected parent. There is currently no cure. But in a new study, published December 12, 2022 in Nature Neuroscience, researchers at the University of California San Diego (UCSD) School of Medicine, with colleagues elsewhere, describe using RNA-targeting CRISPR/Cas13d technology to develop a new therapeutic strategy that specifically eliminates toxic RNA that causes HD. The open-access article is titled “An RNA-Targeting CRISPR–Cas13d System Alleviates Disease-Related Phenotypes in Huntington’s Disease Models.”

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