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Takenari Yamashita, Hui Lin Chai, Sayaka Teramoto, Shoji Tsuji, Kuniko Shimazaki, Shin-ichi Muramatsu and Shin Kwak Rescue of amyotrophic lateral sclerosis phenotype in a mouse model by intravenous AAV9-ADAR2 delivery to motor neurons EMBO Molecular Medicine 5

Version of Record online: 24 SEP 2013 | DOI: 10.1002/emmm.201302935

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A single intravenous injection of AAV9-ADAR2 in a mechanistic mouse model of sporadic ALS prevents progressive motor neurons dysfunction and death by enhancing ADAR2 enzymatic activity and expression of GluA2R instead of GluA2Q, marker of disease.

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