CRISPR corrects genetic defect so cells can normalize blood sugar Using induced pluripotent stem cells produced from the skin of a patient with a rare, genetic form of insulin-dependent diabetes called Wolfram syndrome, researchers transformed the human stem cells into insulin-producing cells and used the gene-editing tool CRISPR-Cas9 to correct a genetic defect that had … Read more
Gene therapies have been a point of discussion during the last several years as a potential curative option for a variety of disease indications. The majority of the cardiovascular and metabolic disorder (CVMD) gene therapies in development are targeting cardiovascular indications, indicating immense opportunity for biotech companies to pursue drug candidates that target metabolic disorders, … Read more
DelveInsight’s Metabolic disorders based Gene Therapy Reports Metabolic disorders are collectively common, frequently severe and in many instances difficult or impossible to treat. Accordingly, there is a compelling need to explore novel therapeutic modalities, including gene therapy, and examine multiple phenotypes where the risks of experimental therapy are outweighed by potential benefits to trial participants. … Read more