Mutant Huntingtin Slows Cells’ Protein-Building Machinery, Study Finds

Stalled protein-building may underlie Huntington’s disease, according to data from a recent study. The findings showed that the mutated huntingtin protein, which is at the heart of the disorder, slows down ribosomes, the small cell machinery structures responsible for building new proteins. According to investigators, knowing this and understanding…

Tiny Lipids as Delivery Vehicle May Make CRISPR-Cas9 More Effective Gene Editing Tool

Tiny fatty (lipid) particles can be used to enhance the delivery of gene editing tools, such as CRISPR-Cas9, to targeted cells and considerably improve their ability to possibly treat human disorders like Huntington’s disease, researchers reported. Their study, “Fast and Efficient CRISPR/Cas9 Genome Editing In Vivo Enabled by…

New CRISPR-Cas9 Technique Corrects Defects Linked to Huntington’s, UCSD Study Suggests

A new version of the CRISPR-Cas9 gene editing technique designed to target RNA molecules can fix the underlying cause of Huntington’s disease and similar disorders, say researchers at the University of California San Diego. Their study, “Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9,” appeared in…

Huntington’s Research, Treatment May Benefit from New Gene-Editing Tool

Researchers have developed a new gene-editing tool that may improve the precision of future gene therapy efforts in Huntington’s disease, as well as numerous other genetic conditions. The study, “A chemical-inducible CRISPR–Cas9 system for rapid control of genome editing,” published in the journal Nature Chemical Biology,…