Causes and drug development in ataxia telangiectasia

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06/24/23

Dr Richard Gatti – David Geffen School of Medicine UCLA, USA

Dr Richard Gatti meets with ecancertv to discuss Ataxia telangiectasia (AT), a rare, and some times lethal, disorder in children where one out of three develops malignancy by the age of ten.

Dr Gatti notes that children with AT have a very strong cancer predisposition because they are missing the ATM protein which causes cell death by apoptosis; however, it is still unclear has to how this results in the development of cancer cells. Dr Gatti is currently working on the development of a group of drugs that treat cells with the mutation and induce them to produce the ATM protein. These drugs also have the potential to treat other genetic diseases.