Gene therapy : the Wellcome Trust lecture 1993 / Marcus Pembrey.

  • Pembrey, Marcus E.
Date:
1993
Catalogue details

Licence: Attribution 4.0 International (CC BY 4.0)

Credit: Gene therapy : the Wellcome Trust lecture 1993 / Marcus Pembrey. Source: Wellcome Collection.

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    GENE THERAPY is a new way of treating genetic disease that aims to tackle the root cause. If a gene is missing it aims to provide one. The Burden of Genetic Disease There are some 4,000 known, simply-inherited genetic disorders and, in aggregate, they are the cause of much suffering in 1-2% of the population. The commoner inherited disorders include cystic fibrosis, sickle cell disease, the thalassaemias, fragile X syndrome, Duchenne muscular dystrophy, haemophilia A, Huntington's disease, neurofibromatosis, and adult polycystic kidney disease. Traditional therapies are often of limited or short - lived benefit and a burden to the patient and their family