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Updated: Jul 11, 2025

Next Generation Sequencing for the Detection of Actionable Mutations in Solid and Liquid Tumors
Published on: September 20, 2016
U2AF1 in various neoplastic diseases and relevant targeted therapies for malignant cancers with complex mutations
Qing Nian1, Yihui Li2, Jingwei Li1
1Department of Transfusion, Sichuan Provincial People's Hospital, University of Electronic Science and Technology of China, Chengdu, Sichuan 610072, P.R. China.
Abstract:
U2 small nuclear RNA auxiliary factor 1 (U2AF1) is a multifunctional protein that plays a crucial role in the regulation of RNA splicing during eukaryotic gene expression. U2AF1 belongs to the SR family of splicing factors and is involved in the removal of introns from mRNAs and exon-exon binding. Mutations in U2AF1 are frequently observed in myelodysplastic syndrome, primary myelofibrosis, chronic myelomonocytic leukaemia, hairy cell leukaemia and other solid tumours, particularly in lung, pancreatic, and ovarian carcinomas. Therefore, targeting U2AF1 for therapeutic interventions may be a viable strategy for treating malignant diseases. In the present review, the pathogenic mechanisms associated with U2AF1 in different malignant diseases were summarized, and the potential of related targeting agents was discussed. Additionally, the feasibility of natural product-based therapies directed against U2AF1 was explored.
Insights
U2 small nuclear RNA auxiliary factor 1 (U2AF1) is crucial for RNA splicing and frequently mutated in cancers. Targeting U2AF1 presents a promising therapeutic strategy for malignant diseases.
Area of Science:
- Molecular Biology
- Cancer Biology
- Genetics
Background:
- U2 small nuclear RNA auxiliary factor 1 (U2AF1) is a key splicing factor regulating gene expression.
- U2AF1 mutations are prevalent in hematologic malignancies and solid tumors, including lung, pancreatic, and ovarian cancers.
Approach:
- This review summarizes pathogenic mechanisms of U2AF1 in various cancers.
- It discusses the therapeutic potential of targeting U2AF1 and related agents.
- The feasibility of natural product-based therapies against U2AF1 is explored.
Key Points:
- U2AF1's role in RNA splicing and its association with diverse malignancies.
- The significance of U2AF1 mutations in disease pathogenesis.
- Potential therapeutic strategies targeting U2AF1.
Conclusions:
- Targeting U2AF1 offers a viable therapeutic avenue for cancer treatment.
- Natural products may provide novel agents for U2AF1-directed therapies.
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