申请人:Ramot at Tel-Aviv University Ltd.
公开号:US20200061076A1
公开(公告)日:2020-02-27
Methods of treating leukodystrophy are provided. Accordingly there is provided a method of treating leukodystrophy in a subject, the method comprising administering to the subject a therapeutically effective amount of an agent capable of up-regulating activity and/or expression of a component participating in a Sigma-1 Receptor (Sig-1R) signaling pathway. Also provided are agents and methods of up-regulating activity of Sig-1R in a cell and treating a disease that can benefit from up-regulating activity of Sig-1R. Also provided are agents and methods of modulating activity of sonic hedgehog (SHH) in a cell and treating a disease that can benefit from modulating activity of SHH.