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Custom Vector Construction> | These constructs were cloned in pSilencer 3.1 (GenScript, Piscataway, NJ) under the human H1 promoter and tested in vitro. shRNA 130 along with H1 promoter was further cloned into an AAV vector along with a reporter GFP under chicken β-actin promoter to identify the transduced cells. | Get A Quote |
Mutations in superoxide dismutase 1 (SOD1) are linked to familial amyotrophic lateral sclerosis (ALS) resulting in progressive motor neuron death through one or more acquired toxicities. Involvement of wild-type SOD1 has been linked to sporadic ALS, as misfolded SOD1 has been reported in affected tissues of sporadic patients and toxicity of astrocytes derived from sporadic ALS patients to motor neurons has been reported to be reduced by lowering the synthesis of SOD1. We now report slowed disease onset and progression in two mouse models following therapeutic delivery using a single peripheral injection of an adeno-associated virus serotype 9 (AAV9) encoding an shRNA to reduce the synthesis of ALS-causing hum... More