Gene therapy is the use of genetic material- DNA or RNA- as a medicine. Classical gene therapy involves the introduction of functional genes, in the form of DNA, to replace mutated genes. This approach has been used to successfully treat a number of diseases including X-linked SCID, ADA-SCID, haemophilia B, lipoproteinlipase deficiency and Leber’s congenital amaurosis with intensive research on-going for other inherited diseases. More recently scientists are looking at using gene technology to repair rather than replace mutated genes, silence overactive genes and to also provide our immune cells with the tools they need to recognise and kill cancer cells and infectious. Read more…

Cell therapy involves the introduction of living cells into the body to treat disease. Blood transfusions and bone marrow transplants are examples of well-established cell therapies. The combination of gene and cell therapies has been successful in the treatment of X-SCID and ADA-SCID. Here patient blood stem cells have been treated by gene therapy outside of the body and the corrected cells re-introduced to the patient to produce a working immune system.

In recent years, stem cells have often been portrayed by the news & media as a new wonder therapy that could regenerate virtually any damaged organs and tissues in the human body. But are stem cells really a new discovery and what is their impact in medicine? Read more…

In this section of genetics resources, you can find more information on gene and cell therapy, our public engagement events and outreach activities.

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