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New hope (for familial ALS)

Injection of virus-delivered gene silencer blocks ALS degeneration, saves motor function

“The new approach involves injecting shRNA—an artificial RNA molecule capable of silencing or turning off a targeted gene—that is delivered to cells via a harmless adeno-associated virus. In the new research, single injections of the shRNA-carrying virus were placed at two sites in the spinal cord of adult mice expressing an ALS-causing mutation of the SOD1 gene, either just before disease onset or when the animals had begun showing symptoms”.

“In adult mice already displaying ALS-like symptoms, the injection effectively blocked further disease progression and degeneration of motor neurons”.

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