Archive for the tag: Batten

Batten Disease and Gene Therapy

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Batten disease occurs when at least one of the thirteen known ceroid lipofuscinosis (CLN) genes is mutated. Gene therapy may offer a one-time treatment to slow or ideally stop the progression of these disorders by delivering a working gene to target the cause of the disease.

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What is Batten Disease

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Sara Mole, PhD at the University College of London describes Batten disease and the difficulty in educating clinicians about this extremely rare condition.

We talked with Dr Mole at the 15th International Conference on Neuronal Ceroid Lipfuscinosis (Batten Disease) held in Boston, MA; October 5-8, 2016.
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