The Molecular Biology Society of Japan

[1P-0687] Gene therapy using Adeno-Associated Virus Vectors in a Plp1-transgenic mouse model of Pelizaeus-Merzbacher disease

〇Heng Li1, Hironori Okada2, Kazuhisa Sakai3, Takashi Okada2, Noritaka Ichinohe3, Yu-ichi Goto1, Ken Inoue1 (1.Dept of Mental Retardation and Birth Defect Research, National Institute of Neuroscience, National Center of Neurology and Psychiatry, 2.Dept of Molecular and Medical Genetics, Graduate School of Medicine, Nippon Medical School, 3.Dept of Ultrastructural Research, National Institute of Neuroscience, National Center of Neurology and Psychiatry)

inherited hypomyelinaitng leukodystrophy, gene therapy, Adeno-associated virus, artificial miRNA

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