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

被引：9

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.

引用

页数：7

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