Ramot at Tel-Aviv University Ltd.;Bar-llan University
发明人:
Orna ELROY-STEIN,Hanoch SENDEROWITZ,Andrea ATZMON
申请号：
US16612452
公开号：
US20200061076A1
申请日:
2018.05.10
申请国别(地区):
US
年份:
2020
代理人:
摘要：
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.
