Tay-Sachs disease is a rare hereditary disorder ... accumulate in the child's brain until they reach toxic levels. They cause ...

Mucopolysaccharidosis is a family of diseases caused by an enzyme deficiency that leads to the accumulation of glycosaminoglycans (GAGs) within the lysosomes of cells. In one particular variant of ...

View Full Profile. Learn about our Editorial Policies. In their 2019 paper, Liu’s team used prime editing to alter the gene mutations causing sickle cell disease and Tay-Sachs disease. Liu explained ...