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Related Concept Videos

Alternative RNA Splicing02:18

Alternative RNA Splicing

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...
Alternative RNA Splicing02:18

Alternative RNA Splicing

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...
RNA Splicing01:32

RNA Splicing

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...
RNA Splicing01:32

RNA Splicing

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...
Chromatin Structure Regulates pre-mRNA Processing02:41

Chromatin Structure Regulates pre-mRNA Processing

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...
Pre-mRNA Processing: RNA Splicing01:32

Pre-mRNA Processing: RNA Splicing

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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Related Experiment Video

Updated: Jul 19, 2026

Engineering Artificial Factors to Specifically Manipulate Alternative Splicing in Human Cells
10:06

Engineering Artificial Factors to Specifically Manipulate Alternative Splicing in Human Cells

Published on: April 26, 2017

Splicing modulation as a modifier of the CFTR function.

Malka Nissim-Rafinia1, Batsheva Kerem

  • 1Department of Genetics, Life Sciences Institute, Hebrew University, Jerusalem 91904, Israel.

Progress in Molecular and Subcellular Biology
|November 2, 2006
PubMed
Summary

Splicing defects cause cystic fibrosis (CF) variations. Modulating splicing factors can restore CFTR function, offering a potential therapeutic strategy for CF patients with splicing mutations.

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Functional Reconstitution and Channel Activity Measurements of Purified Wildtype and Mutant CFTR Protein
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Functional Reconstitution and Channel Activity Measurements of Purified Wildtype and Mutant CFTR Protein

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Engineering Artificial Factors to Specifically Manipulate Alternative Splicing in Human Cells
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Published on: April 26, 2017

Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients
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Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients

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Functional Reconstitution and Channel Activity Measurements of Purified Wildtype and Mutant CFTR Protein
09:59

Functional Reconstitution and Channel Activity Measurements of Purified Wildtype and Mutant CFTR Protein

Published on: March 9, 2015

Area of Science:

  • Molecular Biology
  • Genetics
  • Medical Research

Background:

  • Cystic Fibrosis (CF) is often caused by mutations impacting pre-mRNA splicing.
  • Splicing mutations lead to variable levels of correct and aberrant CFTR transcripts, correlating with disease severity.

Purpose of the Study:

  • To investigate the role of splicing machinery in modifying CF disease severity.
  • To explore the therapeutic potential of modulating splicing factors for CF treatment.

Main Methods:

  • Overexpression of splicing factors in cell models with CF-causing splicing mutations.
  • Analysis of correctly spliced CFTR RNA levels.
  • Assessment of CFTR channel function restoration.
  • Treatment with sodium butyrate, a histone deacetylase inhibitor.

Main Results:

  • Overexpression of specific splicing factors increased correct CFTR transcript levels.
  • Restoration of CFTR channel function was observed.
  • Sodium butyrate also enhanced correct CFTR transcript levels and function.

Conclusions:

  • Splicing machinery acts as a genetic modifier in CF patients with splicing mutations.
  • Splicing modulation presents a promising therapeutic avenue for genetic diseases caused by splicing defects.