In a clinical trial, a single intravenous infusion of NTLA-2001, a novel gene-editing therapy based on CRISPR/Cas9, significantly reduced abnormal levels of the protein transthyretin in patients with ATTR amyloid cardiomyopathy, a progressive and fatal cause of heart failure
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.
CHICAGO — A novel CRISPR-based in vivo gene editing therapy reduced transthyretin levels in patients with hereditary transthyretin amyloidosis with cardiomyopathy, researchers reported at the American Heart Association Scientific Sessions. Transthyretin amyloid cardiomyopathy (ATTR-CM) is a “progressive and fatal disease,” during which amyloid deposits cause impaired
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