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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...
Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...

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Related Experiment Video

Updated: Jun 24, 2026

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
07:02

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

Published on: May 11, 2018

Progress toward therapy with antisense-mediated splicing modulation.

Liutao Du1, Richard A Gatti

  • 1Department of Pathology and Laboratory Medicine, The David Geffen School of Medicine at UCLA, 675 Charles Young Drive South, CA 90095-1732, USA. ldu@mednet.ucla.edu

Current Opinion in Molecular Therapeutics
|March 31, 2009
PubMed
Summary
This summary is machine-generated.

Antisense oligonucleotides (AO) offer novel therapeutic strategies by modulating gene splicing to restore function or inhibit expression. This review highlights AO advances in targeting splicing for treating genetic disorders, cancers, and infections.

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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

Related Experiment Videos

Last Updated: Jun 24, 2026

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
07:02

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

Published on: May 11, 2018

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

Area of Science:

  • Molecular biology
  • Genetics
  • Pharmacology

Background:

  • Antisense oligonucleotides (AO) are short nucleic acid sequences designed to bind target mRNA.
  • Gene splicing is a critical process in gene expression, and its dysregulation is implicated in various diseases.
  • Modulating gene splicing offers a promising avenue for therapeutic intervention.

Purpose of the Study:

  • To review recent advancements in antisense-mediated splicing modulation for molecular therapy.
  • To discuss the applications of AO in treating diseases by targeting gene splicing.
  • To highlight progress in AO delivery systems.

Main Methods:

  • Review of current literature on antisense oligonucleotides and gene splicing.
  • Analysis of therapeutic strategies employing AO for splicing modulation.
  • Examination of disease applications and delivery methods for AO.

Main Results:

  • AO can be designed to reprogram gene splicing, restoring normal gene function.
  • AO can inhibit gene expression by disrupting aberrant splicing.
  • Significant progress has been made in AO design, targeting specificity, and delivery.

Conclusions:

  • Antisense-mediated splicing modulation represents a powerful tool in molecular therapy.
  • AO offer versatile therapeutic potential for genetic disorders, cancers, and infectious diseases.
  • Continued research in AO delivery and targeting will expand their clinical utility.