[4P-0448] Gene therapy for DRPLA model mice by AAV-delivered CRISPR / Cas9.

〇Shoichiro Ando¹, Taisuke Kato², Yuka Koike¹, Sachiko Hirokawa², Kenta Kobayashi³, Shoji Tsuji⁴, Osamu Onodera^1,2 (1.Dept. of Neurology, BRI, Niigata Univ., 2.Dept. of Mol. Neuroscience, BRI, Niigata Univ., 3.Dept. of Viral Vector Development, NIPS, 4.Dept. of Molecular Neurology, Univ. of Tokyo)

DRPLA, Polyglutamine, CRISPR/Cas9, Gene-silencing, AAV-vector