Regulation of HERG proteins by chemical chaperones

描述（由申请人提供）：该项目的长期目标是研究无法正常传输到质膜的离子通道的药理学拯救。蛋白质运输失败是先天性长QT2综合征（LQT2）相关离子通道病变的基础。与高亲和力离子通道结合的药物可以恢复突变离子通道的运输缺陷。然而，药物拯救这些通道的机制仍然知之甚少。本研究在分子和功能水平上探讨了Long QT2转运缺陷的药理学修复差异。

DESCRIPTION (provided by applicant): The long-term goal of this project is to investigate the pharmacological rescue of ion channels that fail to traffic normally to the plasma membrane. Failure of protein trafficking underlies ion channelopathies associated with congenital Long QT2 syndrome (LQT2). Drugs that bind to channels with a high affinity can restore the trafficking defects of mutant ion channels. However, the mechanisms that underlie the drug rescue of these channels remain poorly understood. This proposal examines the differences in pharmacological rescue of Long QT2 trafficking defects at the molecular and functional level.