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

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.