Sustitución enzimática mediante el uso de portadores de liposomas en los fibroblastos de gangliosidosis Gm1 felinos

To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.