- 专利标题: Methods for modulating KLHL1 levels, methods for modulating current activity in T-type calcium channels, molecules therefor, and methods for identifying molecules therefor
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申请号: US15273079申请日: 2016-09-22
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公开(公告)号: US10047377B2公开(公告)日: 2018-08-14
- 发明人: Erika Sofia Piedras-Renteria , Jody Lee Martin
- 申请人: Loyola University of Chicago
- 申请人地址: US IL Maywood
- 专利权人: Loyola University of Chicago
- 当前专利权人: Loyola University of Chicago
- 当前专利权人地址: US IL Maywood
- 代理机构: Hartman Global IP Law
- 代理商 Gary M. Hartman; Domenica N. S. Hartman
- 主分类号: C07H21/02
- IPC分类号: C07H21/02 ; C07H21/04 ; A61K31/70 ; C12N15/86 ; C12N15/113 ; C12Q1/6876 ; C12Q1/6883 ; C12N5/00 ; A61K48/00
摘要:
Methods for modulating T-type calcium channel activity without directly targeting the T-type calcium channels are provided that include modulating kelch-like protein 1 (KLHL1) levels in a subject by providing a small hairpin RNA (shRNA) that targets a KLHL1 gene, and then administering the shRNA to the subject in an amount sufficient to modulate KLHL1 gene expression. The KLHL1 level directly effects current activity in T-type calcium channels and therefore modulation of KLHL 1 gene expression indirectly modulates current activity in T-type calcium channels. The methods may be implemented with, for example, an shRNA molecule suitable for modulating a KLHL1 level in the subject which may be provided as a plasmid encoding the shRNA molecule or an adeno-associated virus vector encoding the shRNA molecule. Methods of identifying compounds that modulate current activity in T-type calcium channels by determining an effect of the compound on KLHL1 gene expression are also provided.
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