[3P] 83 Genome Editing and Gene Therapy

[3P-385] Gene suppressing therapy for Pelizaeus-Merzbacher disease using AAV vectors engineered with artificial miRNA

*Heng Li¹, Reiko Mishima¹, Ken Inoue¹ (1. Dept Biochemistry and Cellular Biology, National Center of Neurology and Psychiatry)

Keywords:Pelizaeus-Merzbacher disease, AAV, miRNA, gene therapy