Related Experiment Video
Updated: Nov 5, 2025

Using the E1A Minigene Tool to Study mRNA Splicing Changes
Published on: April 22, 2021
Therapeutic Modulation of RNA Splicing in Malignant and Non-Malignant Disease
Ettaib El Marabti1, Omar Abdel-Wahab2
1Clinical Transplant Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.
Abstract:
RNA splicing is the enzymatic process by which non-protein coding sequences are removed from RNA to produce mature protein-coding mRNA. Splicing is thereby a major mediator of proteome diversity as well as a dynamic regulator of gene expression. Genetic alterations disrupting splicing of individual genes or altering the function of splicing factors contribute to a wide range of human genetic diseases as well as cancer. These observations have resulted in the development of therapies based on oligonucleotides that bind to RNA sequences and modulate splicing for therapeutic benefit. In parallel, small molecules that bind to splicing factors to alter their function or modify RNA processing of individual transcripts are being pursued for monogenic disorders as well as for cancer.
Related Concept Videos
RNA Splicing
Alternative RNA Splicing
There are five types of alternative RNA splicing that vary in the ways the pre-mRNA segments are removed or retained in the mature mRNA. The first...
Alternative RNA Splicing
Chromatin Structure Regulates pre-mRNA Processing
The chromatin structure, especially...
What is Gene Expression?
Regulation of Expression at Multiple Steps

