Genetic Therapies For Neurodegenerative Disease

(From the New Scientist). The effectiveness of gene silencing as a therapy for certain kinds of neurodegenerative disorder has been demonstrated: mice with a condition similar to Huntington's disease were effectively cured. "This is the first example of targeted gene silencing of a disease gene in the brains of live animals. It suggests that this approach may eventually be useful for human therapies." This is early stage work and there is much to be done before this sort of technique can be tried in humans, but the fact that it works so well in mice means that we should be seeing more of gene silencing therapies in the years ahead.



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