Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis.

Wang H, Yang B, Qiu L, Yang C, Kramer J, Su Q, Guo Y, Brown RH Jr, Gao G, Xu Z
Hum Mol Genet. 2013 Oct 9. PubMed Article

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