Using Lipid Nanoparticles to Efficiently Deliver CRISPR/Cas9 for Genome Editing

S. C. Alexander et al. Transthyretin amyloidosis (ATTR) is a progressive disease caused by accumulation of amyloid deposits of misfolded transthyretin (TTR) protein in multiple tissues including the heart, nerves and gastrointestinal tract. Reduction of TTR monomer via stabilization of circulating tetramer and silencing of TTR gene expression in hepatocytes of ATTR patients have emerged […]

Gene-editing Therapy NTLA-2001 Given Orphan Drug Status by FDA The U.S. Food and Drug Administration (FDA) has given orphan drug designation to NTLA- 2001, an investigational gene-editing therapy for familial amyloid polyneuropathy (FAP) and other forms of transthyretin (ATTR) amyloidosis. … The aim of NTLA-2001 is to disrupt the TTR gene, thus decreasing transthyretin production and preventing the buildup of these harmful protein […]

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

Julian D. Gillmore et al. Transthyretin amyloidosis, also called ATTR amyloidosis, is a life-threatening disease characterized by progressive accumulation of misfolded transthyretin (TTR) protein in tissues, predominantly the nerves and heart. NTLA-2001 is an in vivo gene-editing therapeutic agent that is designed to treat ATTR amyloidosis by reducing the concentration of TTR in serum. It […]