Gene therapy

Gene therapy is a modern medical technology that can be used against both inherited diseases, such as sickle cell disease, or acquired diseases such as cancer. It entails adding new copies of a gene that may be broken, replacing a gene that’s missing, or replacing a gene that’s defective.

A key term in gene therapy is “vector,” that is, the material used to deliver new DNA. Vectors are often based on viruses that have had the viral genes removed and replaced with therapeutic ones.

In ex vivo gene therapy, cells are removed cells from the patient, new genetic material is inserted via a vector, and then the cells are returned to the patient. With in vivo gene therapy, the vector is directly infused into the bloodstream or injected into an organ.

Stories in this archive include gene therapy research for congenital deafness and Duchenne muscular dystrophy, and an article on why the world’ most expensive drug may not be overpriced.