Novel 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 ...
Nov 7, 2022
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