Nasal Spray Sends CRISPR to the Brain

 Nasal Spray Sends CRISPR to the Brain - Blog

Mucopolysaccharidosis I is caused by mutations in the gene for the enzyme alpha-L-iduronidase (IDUA). This enzyme is required for the breakdown of substances called glycosaminoglycans (GAGs) that are by-products of chemical reactions in the body’s cells. In 2018, a team of researchers was able to demonstrate the feasibility of CRISPR gene editing in vivo in […]