Recent Advances in Targeting RNA Splicing Factors with Small Molecules

Area of Science:

• Molecular Biology
• Genetics
• Cancer Research

Background:

• Pre-messenger RNA (mRNA) splicing generates diverse proteins from a single gene.
• Dysregulation of splicing factors contributes to aberrant gene expression in cancer.
• Key splicing factors like SF3B1, U2AF1, SRSF2, and RBM39 are implicated in various cancers.

Purpose of the Study:

• To review recurrent mutations and dysregulated expressions of splicing factors in cancers.
• To provide perspectives on small-molecule compounds targeting splicing factors for cancer therapy.
• To discuss functional assays for hit/lead discovery in developing splicing factor-targeted drugs.

Main Methods:

• Literature review of mutations and dysregulated expressions of splicing factors in cancer.
• Analysis of recent developments in small-molecule compounds targeting splicing factors.
• Evaluation of functional assays for drug discovery and development.

Main Results:

• Splicing factor mutations and dysregulation are common in cancers.
• Small molecules targeting splicing factors are emerging as a novel cancer treatment strategy.
• Functional assays are crucial for identifying and developing effective targeted therapies.

Conclusions:

• Targeting splicing factors represents a promising therapeutic approach for cancer.
• Further research into splicing factor mutations and targeted small molecules is warranted.
• Advancements in functional assays will accelerate the development of novel cancer treatments.