Science Spotlight: Base editing to reduce toxic HTT fragments
BioCentury’s translational roundup also includes a 5-HT2AR agonist for CRC and ‘SECmeres’ for Alzheimer’s liquid biopsy
A University of Illinois at Urbana-Champaign team found a way to edit HTT so cells make a version of the protein that’s resistant to the cleavage that drives Huntington’s disease pathology.
Using CRISPR base editors, the authors of the Nature Biomedical Engineering study disrupted the splice acceptor in HTT exon 13, causing the spliceosome to skip a small stretch of the gene that includes a caspase-6 cleavage site. The resulting protein therefore can’t be chopped into the toxic, aggregation-prone fragments implicated in Huntington’s...