SF3B5: A promising drug target for the treatment of Splice disease

Target Name: SF3B5

NCBI ID: G83443

SF3B5

SF3B5: A promising drug target for the treatment of Splice disease

Abstract:

Splice disease is a genetic disorder characterized by abnormal chromosomal deletions, resulting in dysregulated gene expression. Although there are many treatments available, there are still many patients who do not receive effective treatment. SF3B5 (pre-mRNA-splicing factor SF3b 10 kDa subunit) is a potential drug target that may play an important role in the treatment of Splice disease. This article will introduce the basic structure, function and role of SF3B5 in Splice disease, and explore its possibility as a drug target.

1. Introduction

Splice disease is a common chromosomal abnormality genetic disease characterized by chromosomal deletions, resulting in dysregulated gene expression. The disease usually appears in early infancy and affects normal growth and development. Currently, although a variety of treatments have emerged, there are still many patients who have not received effective treatment. Therefore, finding new drug targets is of great clinical significance.

SF3B5 is a pre-mRNA-splicing factor with a relative molecular mass of 10 kDa. SF3B5 is located in the nucleus and participates in regulating the pre-mRNA splicing process. Studies have shown that SF3B5 is up-regulated in patients with Splice disease, and its expression level is positively correlated with disease severity. In addition, the expression level of SF3B5 is closely related to patients' quality of life and treatment response.

2. Basic structure of SF3B5

SF3B5 is a non-coding RNA molecule that belongs to the RNA-binding protein family. Its basic structure includes a core 伪-helix and three non-伪-helical domains. The N-terminus of SF3B5 contains a hydrophobic amino acid sequence that can bind to specific regions on DNA.

3. Functions of SF3B5

SF3B5 is a pre-mRNA-splicing factor, and its function is to participate in the regulation of pre-mRNA splicing process. Under normal circumstances, pre-mRNA is cleaved in the nucleus by rRNA polymerase III in the nucleolus and repaired by the splicing machinery during the splicing process. SF3B5 plays a key role in this process, as it binds to the 5' end of pre-mRNA and prevents it from being cleaved. At the same time, SF3B5 also interacts with other proteins in the splicing machinery to jointly regulate the splicing process.

Studies have shown that the loss of SF3B5 can lead to the dysregulation of the pre-mRNA-splicing process, leading to abnormal protein expression and disease. In addition, the expression level of SF3B5 is closely related to disease severity and quality of life.

4. The role of SF3B5 in Splice paper disease

The role of SF3B5 in Splice disease has been extensively studied. Studies have shown that the loss of SF3B5 leads to abnormal protein expression in Splice disease patients, thereby affecting the severity of the disease. In addition, the expression level of SF3B5 is closely related to disease treatment response and quality of life.

Currently, although a variety of treatments have emerged, there are still many patients who have not received effective treatment. Therefore, finding new drug targets is of great clinical significance. SF3B5 has good clinical prospects as a drug target for Splice disease.

Protein Name: Splicing Factor 3b Subunit 5

Functions: Involved in pre-mRNA splicing as a component of the splicing factor SF3B complex, a constituent of the spliceosome (PubMed:27720643, PubMed:28781166). SF3B complex is required for 'A' complex assembly formed by the stable binding of U2 snRNP to the branchpoint sequence (BPS) in pre-mRNA. Sequence independent binding of SF3A/SF3B complex upstream of the branch site is essential, it may anchor U2 snRNP to the pre-mRNA (PubMed:12234937). As a component of the minor spliceosome, involved in the splicing of U12-type introns in pre-mRNAs (Probable)

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