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Related Concept Videos

RNA Splicing01:32

RNA Splicing

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Splicing is the process by which eukaryotic RNA is edited before its translation into protein. The RNA strand transcribed from eukaryotic DNA is called the primary transcript. The primary transcripts that become mRNAs are called precursor messenger RNAs (pre-mRNAs). Eukaryotic pre-mRNA contains alternating sequences of exons and introns. Exons are nucleotide sequences that code for proteins, whereas introns are the non-coding regions. In RNA splicing, introns are removed and exons are bonded...
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Pre-mRNA Processing: RNA Splicing01:36

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Alternative RNA Splicing02:18

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Alternative RNA splicing is the regulated splicing of exons and introns to produce different mature mRNAs from a single pre-mRNA. Unlike in constitutive splicing where a single gene produces a single type of mRNA, alternative splicing allows an organism to produce multiple proteins from a single gene and plays an important role in protein diversity.
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...
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Chromatin Structure and RNA Splicing02:41

Chromatin Structure and RNA Splicing

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Pre-mRNA Processing: Modification of pre-mRNA Ends01:35

Pre-mRNA Processing: Modification of pre-mRNA Ends

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In eukaryotic cells, transcripts made by RNA polymerase are modified and processed before exiting the nucleus. Unprocessed RNA is called precursor mRNA or pre-mRNA to distinguish it from mature mRNA.
Once about 20-40 ribonucleotides have been joined together by RNA polymerase, a group of enzymes adds a cap to the 5' end of the growing transcript. In this process, a 5' phosphate is replaced by modified guanosine that has a methyl group attached (7-methyl guanosine). This 5' cap helps...
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Chromatin Structure Regulates pre-mRNA Processing02:41

Chromatin Structure Regulates pre-mRNA Processing

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In eukaryotic cells, nascent mRNA transcripts need to undergo many post-transcriptional modifications to reach the cell cytoplasm and translate into functional proteins. For a long time, transcription and pre-mRNA processing were considered two independent events that occur sequentially in the cell. However, it has now been well established that transcription and pre-mRNA processing are two simultaneous processes that are precisely regulated inside the cell.
The chromatin structure, especially...
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Related Experiment Video

Updated: Oct 18, 2025

A Reporter Based Cellular Assay for Monitoring Splicing Efficiency
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A Reporter Based Cellular Assay for Monitoring Splicing Efficiency

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Promoting spliceosome assembly for therapeutic intent.

Bin Lu1, Omar Abdel-Wahab1

  • 1Human Oncology and Pathogenesis Program, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY 10065, USA.

Trends in Pharmacological Sciences
|October 4, 2021
PubMed
Summary

Researchers found a new way to control RNA splicing by boosting spliceosome binding to pre-mRNA. This discovery offers a novel therapeutic target for modulating splicing processes.

Keywords:
RBM39RNASF3B1U2AF1myelodysplastic syndromessplicing

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Area of Science:

  • Molecular Biology
  • RNA Processing
  • Drug Discovery

Background:

  • RNA splicing is a critical cellular process for gene expression, converting precursor messenger RNA (pre-mRNA) into mature mRNA.
  • The spliceosome, a large molecular machine, catalyzes RNA splicing.
  • Dysregulation of RNA splicing is implicated in various diseases, highlighting the need for therapeutic interventions.

Purpose of the Study:

  • To identify novel pharmacologic strategies for modulating RNA splicing.
  • To investigate methods for perturbing spliceosome activity.
  • To establish a new chemical target for therapeutic development.

Main Methods:

  • The study by Chatrikhi et al. focused on identifying compounds that can alter spliceosome function.
  • Investigated the enhancement of spliceosome binding to pre-mRNA as a mechanism to perturb splicing.
  • Utilized pharmacologic approaches to target the splicing machinery.

Main Results:

  • Chatrikhi et al. successfully identified pharmacologic agents capable of perturbing RNA splicing.
  • Demonstrated that enhancing spliceosome binding to pre-mRNA is a viable strategy.
  • Established a novel mechanism for modulating splicing through chemical intervention.

Conclusions:

  • The findings present a new chemical target and mechanism for the therapeutic modulation of RNA splicing.
  • Pharmacologic enhancement of spliceosome-pre-mRNA binding offers a promising avenue for future drug development.
  • This research opens doors for novel treatments targeting diseases associated with aberrant splicing.