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 […]