Delegates at the American Heart Association s annual conference in Chicago heard about research from China which suggested pills containing crushed bugs could be beneficial to patients.
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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.
This is the first time such a drug - which edits DNA in the body without any tissue having to be removed - has yielded results in patients. Just a single dose was required.