Novel CRISPR/Cas9-based gene-editing therapy shows promise for patients with transthyretin amyloid cardiomyopathy
A single IV infusion of NTLA-2001, a novel CRISPR/Cas9-based gene editing therapy, significantly reduced circulating transthyretin (TTR) protein levels in patients with ATTR amyloid cardiomyopathy, a progressive and fatal cause of heart failure, according to late-breaking research presented today at the American Heart Association’s Scientific Sessions 2022. The meeting, held in person in Chicago and