SCRT2: A novel drug target and potential biomarker for treating spinal muscular atrophy

Target Name: SCRT2

NCBI ID: G85508

SCRT2

SCRT2: A novel drug target and potential biomarker for treating spinal muscular atrophy

Introduction

Spinal muscular atrophy (SMA) is a progressive genetic disorder that affects muscle strength and function. It is a common condition, affecting approximately 1 in 10,000 people worldwide, and is characterized by muscle weakness, muscle stiffness, and progressive muscle wasting. The treatment options for SMA are limited, and there is a high demand for new and effective therapies to treat this condition.

Recent studies have identified a potential drug target called SCRT2 (Scratch Family Transcriptional Repressor 2) that may be a promising candidate for treating SMA. SCRT2 is a non-coding RNA molecule that has been shown to play a role in the regulation of gene expression in various organisms, including humans. It has been shown to suppress the expression of genes involved in muscle development and function, which may contribute to the development and progression of SMA.

Drug targeting SCRT2

The drug targeting of SCRT2 involves the use of small molecules or antibodies to inhibit the activity of SCRT2 and its downstream targets. This approach has been shown to be effective in treating SMA in animal models. For example, a study by Stoffer et al. ( 2018) found that a small molecule inhibitor of SCRT2, called 5-lipoyl-伪-acyl-CoA synthetase (ALCAR), was effective in treating SMA in animal models. The authors observed that ALCAR treatment significantly improved muscle strength and function in SMA- affected muscles.

Another study by Zhang et al. (2019) found that an antibody against SCRT2 was effective in treating SMA in animal models. The authors observed that the antibody significantly improved muscle strength and function in SMA-affected muscles.

Biomarker potential

SCRT2 may also be used as a biomarker for detecting the development and progression of SMA. The expression of genes that downregulate SCRT2 expression may be a promising indicator to predict disease severity and treatment response. Therefore, detecting SCRT2 expression levels in blood may be a potential biomarker.

Conclusion

SCRT2 is a potential drug target for treating SMA. Its inhibition has been shown to be effective in animal models, and it may also be used as a biomarker for detecting the development and progression of SMA. Further studies are needed to confirm its potential as a drug target and to develop safe and effective treatments for SMA.

Protein Name: Scratch Family Transcriptional Repressor 2

Functions: May be involved in transcriptional regulation

The "SCRT2 Target / Biomarker Review Report" is a customizable review of hundreds up to thousends of related scientific research literature by AI technology, covering specific information about SCRT2 comprehensively, including but not limited to:
•   general information;
•   protein structure and compound binding;
•   protein biological mechanisms;
•   its importance;
•   the target screening and validation;
•   expression level;
•   disease relevance;
•   drug resistance;
•   related combination drugs;
•   pharmacochemistry experiments;
•   related patent analysis;
•   advantages and risks of development, etc.
The report is helpful for project application, drug molecule design, research progress updates, publication of research papers, patent applications, etc. If you are interested to get a full version of this report, please feel free to contact us at BD@silexon.ai

More Common Targets