Post-natal treatment by a blood-brain-barrier permeable calpain inhibitor, SNJ1945 rescued defective function in lissencephaly

S. Toba,Yasuhisa Tamura,Kanako Kumamoto,Masami Yamada,K. Takao,S. Hattori,T. Miyakawa,Y. Kataoka,M. Azuma,K. Hayasaka,Masano Amamoto,K. Tominaga,A. Wynshaw-Boris,H. Wanibuchi,Yuichiro Oka,Makoto Sato,Mitsuhiro Kato,S. Hirotsune

Published 2013 in Scientific Reports

ABSTRACT

Toward a therapeutic intervention of lissencephaly, we applied a novel calpain inhibitor, SNJ1945. Peri-natal or post-natal treatment with SNJ1945 rescued defective neuronal migration in Lis1+/− mice, impaired behavioral performance and improvement of 18F-FDG uptake. Furthermore, SNJ1945 improved the neural circuit formation and retrograde transport of NFG in Lis1+/− mice. Thus, SNJ1945 is a potential drug for the treatment of human lissencephaly patients.

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