Induced Pluripotent Stem Cells and CRISPR-Cas9 Gene-Editing on Transthyretin Amyloid Cardiomyopathy

Abstract

Transthyretin amyloid cardiomyopathy is a fatal disease of the myocardium causing a protein buildup of Transthyretin. Over 120,000 people in the United States suffer from transthyretin amyloid cardiomyopathy, and half of those diagnosed will die within four years of the onset of symptoms. However, applying CRISPR-Cas9 gene editing will reduce amounts of transthyretin produced by the liver by up to 96% and minimize transthyretin expression by 91%. Induced Pluripotent Stem Cell therapy shows signs of at least 20 years or greater in life expectancy. It puts 39% of recipients into complete remission. Using CRISPR-Cas9 technology, an IV is placed and lipid nanoparticles deliver mRNA with Cas9 production and a single guide RNA for targeting the transthyretin production in hepatocytes. Gathering stem cells has never been easier, using adult somatic cells and returning them to an embryotic state is more efficient and ethical than ever.