Sangamo's Downs Sydrome Approach

Sangamo  (SGMO) published data using their zinc finger DNA-binding protein technology to insert a gene that will essentially "silence" the extra copy of a gene that causes Downs Syndrome. The procedure provides a model for studing the basic biology of Downs syndrome, and could enable the development of drugs that can potentially rebalance the cellular processes and pathologies that are affected by the disorder.

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