Drug development for the treatment of RyR1-related skeletal muscle diseases

被引：7
|
作者：
Murayama, Takashi [1 ]
Kurebayashi, Nagomi [1 ]
Ishida, Ryosuke [2 ]
Kagechika, Hiroyuki [2 ]
机构：
[1] Juntendo Univ, Dept Pharmacol, Sch Med, Tokyo, Japan
[2] Tokyo Med & Dent Univ, Inst Biomat & Bioengn, Tokyo, Japan
来源：
CURRENT OPINION IN PHARMACOLOGY | 2023年 / 69卷
关键词：
CENTRAL CORE DISEASE; MALIGNANT HYPERTHERMIA; RYANODINE RECEPTOR; CALCIUM-RELEASE; CONGENITAL MYOPATHIES; MOLECULAR-MECHANISM; DANTROLENE; RYR1; CHANNEL; MUTATIONS;
D O I：
10.1016/j.coph.2023.102356
中图分类号：
R9 [药学];
学科分类号：
1007 ;
摘要：
Type 1 ryanodine receptor (RyR1) is an intracellular Ca2+ release channel on the sarcoplasmic reticulum of skeletal muscle, and it plays a central role in excitation-contraction (E-C) coupling. Mutations in RyR1 are implicated in various muscle diseases including malignant hyperthermia, central core disease, and myopathies. Currently, no specific treatment exists for most of these diseases. Recently, high-throughput screening (HTS) assays have been developed for identifying potential candidates for treating RyR-related muscle diseases. Currently, two different methods, namely a FRET-based assay and an endoplasmic reticulum Ca2+-based assay, are avail-able. These assays identified several compounds as novel RyR1 inhibitors. In addition, the development of a reconstituted platform permitted HTS assays for E-C coupling modulators. In this review, we will focus on recent progress in HTS assays and discuss future perspectives of these promising approaches.
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页数：7
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