Enzyme replacement in the CSF to treat metachromatic leukodystrophy in mouse model using single intracerebroventricular injection of self-complementary AAV1 vector

by Hironaka, Kohei, Yamazaki, Yoshiyuki, Hirai, Yukihiko, Yamamoto, Motoko, Miyake, Noriko, Miyake, Koichi, Okada, Takashi, Morita, Akio, Shimada, Takashi
Published in Sci Rep (2015)