Induced Pluripotent Stem Cells and CRISPR-Cas9 Gene-Editing on Transthyretin Amyloid Cardiomyopathy
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. Can these two treatments combined cure and improve life expectancy as well as quality of life in patients with transthyretin amyloid cardiomyopathy? These two combined will decrease symptoms that patients have as well as increase the quality and longevity of life. People going into remission on a previously thought impossible disease is remarkable, and using the advancements of CRSIPR-Cas9 and Induced Pluripotent Stem Cells together to solve transthyretin amyloid cardiomyopathy is the future of the treatment plans for the disease
