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Supporting the boldest research ideas in the interdisciplinary labs under the direction of Robert H. Brown Jr., DPhil, MD, one of the world's foremost authorities on ALS.

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Silencing of an ALS gene safely delivered to patients in UMass Medical School study

Synthetic microRNA treatment targets mutant SOD1 gene that causes ALS

UMass Medical School and Massachusetts General Hospital are the first to safely treat two research participants with a synthetic microRNA, delivered into the spinal fluid, designed to silence a human disease-causing gene. Details of the treatment appear in the New England Journal of Medicine.

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