The future of medicine: Gene therapy and IMDs
Overview Gene therapy involves altering the genes inside your body's cells in an effort to treat or stop disease. Genes contain your DNA — the code that controls much of your body's form and function, and in most Inherited Metabolic Disorders, faulty genes results in an enzyme deficiency.
How does gene therapy work? Gene therapy is the transfer or editing of a genetic material to cure a disease. Depending on the delivery strategy chosen, gene therapy can be performed in vivo (inside the body) or ex vivo (outside the body) with integrating (i.e. permanent modification of the host genome) or non-integrating (e.g. through viral vectors which are modified versions of a virus that is different from the virus being targeted to deliver important instructions to our cells.
