What are Cell and Gene Therapies?
In cell therapy, diseases are treated with cells grown or modified outside of the body. Stem cells are most often used for this therapy as they can mature into many specialized types of cells.
In gene therapy, diseases are treated at the genetic level by either deactivating, replacing or repairing the faulty DNA that causes the disease.
Functional DNA is delivered into the patient’s cells by a vector, usually of viral origin. These vectors carry the DNA into the host cell and insert the material into the patient’s DNA, restoring normal gene function and protein production.
CGTs have many potential applications in cancer, degenerative and congenital diseases. As of now, nineteen CGT therapies have been approved by FDA and 16 by EMA. More than 2,000 cell and gene therapy trials have been conducted to date.