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Gene Therapy

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...

Gene Therapy

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...

DNA-only Transposons

DNA-only Transposons

DNA-only transposons are called autonomous transposons since they code for the enzyme transposase that is required for the transposition mechanism. Insertion of transposons can alter gene functions in multiple ways. They can mutate the gene, alter gene expression by introducing a novel promoter or insulator sequence, introduce new splice sites, and change the mRNA transcripts produced, or remodel chromatin structure.
The donor site from where the transposon is excised is either degraded or...

Transposons

Transposons

Transposons, or "jumping genes," are small mobile genetic elements (MGEs) that range from 700 to 40,000 base pairs in length. They are found in all organisms and can move within the same chromosome or transfer to different chromosomes. In some cases, transposons can also jump between different host DNA molecules, such as plasmids or viruses, contributing to genetic variability.Barbara McClintock first discovered these mobile genetic elements in the 1940s while studying maize genetics, and she...

Transgenic Organisms

Transgenic Organisms

Microorganisms in Medicine and Therapeutics

Microorganisms in Medicine and Therapeutics

Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.

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Related Experiment Video

Updated: May 21, 2026

Electroporation-Based Genetic Modification of Primary Human Pigment Epithelial Cells Using the Sleeping Beauty Transposon System

Electroporation-Based Genetic Modification of Primary Human Pigment Epithelial Cells Using the Sleeping Beauty Transposon System

Published on: February 4, 2021

Recent developments in transposon-mediated gene therapy.

Mario Di Matteo¹, Eyayu Belay, Marinee K Chuah

¹Free University of Brussels, Division of Gene Therapy & Regenerative Medicine, Laarbeeklaan 103, B-1090 Brussels, Belgium.

Expert Opinion on Biological Therapy

|June 12, 2012

Summary

Transposons offer a safer, non-viral alternative for gene therapy. Novel Sleeping Beauty and piggyBac systems show promise for stable genetic modification of stem cells, advancing treatments for genetic disorders.

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Published on: January 12, 2018

Transposon Mediated Integration of Plasmid DNA into the Subventricular Zone of Neonatal Mice to Generate Novel Models of Glioblastoma

Transposon Mediated Integration of Plasmid DNA into the Subventricular Zone of Neonatal Mice to Generate Novel Models of Glioblastoma

Published on: February 22, 2015

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Last Updated: May 21, 2026

Electroporation-Based Genetic Modification of Primary Human Pigment Epithelial Cells Using the Sleeping Beauty Transposon System

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Published on: February 4, 2021

An Efficient In Vitro Transposition Method by a Transcriptionally Regulated Sleeping Beauty System Packaged into an Integration Defective Lentiviral Vector

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Transposon Mediated Integration of Plasmid DNA into the Subventricular Zone of Neonatal Mice to Generate Novel Models of Glioblastoma

Transposon Mediated Integration of Plasmid DNA into the Subventricular Zone of Neonatal Mice to Generate Novel Models of Glioblastoma

Published on: February 22, 2015

Area of Science:

Gene Therapy
Stem Cell Biology
Molecular Biology

Background:

Viral vectors face safety and manufacturing hurdles in gene therapy.
Non-viral gene delivery systems offer improved safety and manufacturing profiles.
Continuous advancements in gene transfer technologies impact stem cell research and therapy.

Purpose of the Study:

To review the potential of transposons in gene and cell therapy.
To highlight the safety, efficiency, and biology of Sleeping Beauty (SB) and piggyBac (PB) transposon systems.
To discuss in vivo applications of transposon-based gene therapy.

Main Methods:

Exploration of novel hyperactive SB and PB transposon systems.
Focus on ex vivo gene therapy applications in adult stem/progenitor cells (HSCs, MSCs, myoblasts, iPS cells).
Discussion of in vivo transposon-based gene therapy strategies.

Main Results:

SB and PB transposons enable stable, non-viral genetic modification of primary cells.
These systems are particularly effective in adult stem cells.
Potential for correcting hereditary disorders, including hematopoietic system disorders.

Conclusions:

Latest generation SB and PB transposons are attractive for non-viral gene therapy.
Transposons offer a viable approach for treating genetic disorders.
Targeted integration into safe harbor loci may enhance transposon safety.