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Related Concept Videos

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...
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...
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...
Experimental RNAi02:15

Experimental RNAi

RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...

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Related Experiment Video

Updated: May 13, 2026

A Reporter Based Cellular Assay for Monitoring Splicing Efficiency
08:53

A Reporter Based Cellular Assay for Monitoring Splicing Efficiency

Published on: September 15, 2021

Targeting RNA splicing for disease therapy.

Mallory A Havens1, Dominik M Duelli, Michelle L Hastings

  • 1Department of Cell Biology and Anatomy, Chicago Medical School, Rosalind Franklin University of Medicine and Science, North Chicago, IL, USA.

Wiley Interdisciplinary Reviews. RNA
|March 21, 2013
PubMed
Summary

Aberrant pre-messenger RNA splicing causes genetic diseases. Therapeutic strategies targeting splicing offer a promising approach to correct these defects without altering the genome.

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Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
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Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts

Published on: May 11, 2018

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Last Updated: May 13, 2026

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

Published on: September 15, 2021

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
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Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts

Published on: May 11, 2018

Area of Science:

  • Molecular Biology
  • Genetics
  • Therapeutics

Background:

  • Pre-messenger RNA splicing is crucial for gene expression in eukaryotes.
  • Splicing defects lead to cellular dysfunction and human genetic diseases.
  • Some diseases arise from splicing abnormalities not linked to direct mutations.

Purpose of the Study:

  • To review clinically relevant splicing defects in diseases.
  • To discuss current tools for modulating splicing.
  • To highlight emerging splice-modulating therapeutics for genetic disorders.

Main Methods:

  • Review of literature on splicing defects and therapeutic strategies.
  • Analysis of tools like antisense oligonucleotides, modified snRNAs, trans-splicing, and small molecules.
  • Examination of specific mutations and diseases targeted by splice-modulating approaches.

Main Results:

  • Splicing defects are implicated in numerous human diseases.
  • Various tools effectively manipulate splicing to correct aberrant gene expression.
  • Targeting splicing offers a therapeutic avenue for genetic disorders.

Conclusions:

  • Splicing is a viable therapeutic target for genetic diseases.
  • Advances in splice-modulating tools show significant promise.
  • Emerging therapeutics aim to correct splicing defects, offering new treatment options.