Search research articles

ABOUT JoVE

Overview Leadership Blog JoVE Help Center

AUTHORS

Publishing Process Editorial Board Scope & Policies Peer Review FAQ Submit

LIBRARIANS

Testimonials Subscriptions Access Resources Library Advisory Board FAQ

RESEARCH

JoVE Journal Methods Collections JoVE Encyclopedia of Experiments Archive

EDUCATION

JoVE Core JoVE Business JoVE Science Education JoVE Lab Manual Faculty Resource Center Faculty Site

Terms & Conditions of Use

Related Concept Videos

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...

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by

Same author

Self-synergizing mutual prodrug liposomes for targeted cancer therapy <i>via</i> redox-amplified Pin1 inhibition.

Theranostics·2026

Same author

Prognostic impact of multi-divisional trigeminal neuralgia on pain outcomes following microvascular decompression.

Clinical neurology and neurosurgery·2026

Same author

FOXC2 and WT1 regulate transcriptional reprogramming during the podocyte response to injury.

JCI insight·2026

Same author

Convergence of neuroinflammation across major neurotropic viral exposomes in AD and ADRD.

Journal of neuroinflammation·2026

Same author

A Self-Deliverable H<sub>2</sub>O<sub>2</sub>-Responsive Tocopherol Dimer for Enhanced Antioxidant and Liposomal Delivery.

Molecules (Basel, Switzerland)·2026

Same author

Protein C Activity and Atrial Fibrillation-Related Thrombogenic Phenotypes in Patients With Ischemic Stroke.

American journal of hematology·2026

Same journal

Tracking Synthetic Adhesins on Bacterial Surfaces with Immunofluorescence Microscopy.

Methods in molecular biology (Clifton, N.J.)·2026

Same journal

Post-Selection Methods for Analyzing mRNA Display Selections and Optimization of Hits.

Methods in molecular biology (Clifton, N.J.)·2026

Same journal

High-Performance Computing in Tandem Mass Spectrometry (MS/MS) Peptide Identification.

Methods in molecular biology (Clifton, N.J.)·2026

Same journal

Engineering and Adapting Disulfide-Containing Proteins to Enable Intracellular Functionality.

Methods in molecular biology (Clifton, N.J.)·2026

Same journal

AI-Driven Protein Research: From Prediction to Design.

Methods in molecular biology (Clifton, N.J.)·2026

Same journal

Methods for the In Vitro Selection of Protein and Peptide Libraries Using mRNA Display.

Methods in molecular biology (Clifton, N.J.)·2026

See all related articles

Search research articles

Related Experiment Video

Updated: Jul 3, 2026

Chitosan/Interfering RNA Nanoparticle Mediated Gene Silencing in Disease Vector Mosquito Larvae

Chitosan/Interfering RNA Nanoparticle Mediated Gene Silencing in Disease Vector Mosquito Larvae

Published on: March 25, 2015

Chitosan nanoparticle-mediated gene transfer.

Dongwon Lee¹, Shyam S Mohapatra

¹College of Medicine, Department of Internal Medicine, University of South Florida, Tampa, FL, USA.

Methods in Molecular Biology (Clifton, N.J.)

|August 6, 2008

Summary

This summary is machine-generated.

Chitosan nanocomplexes offer a promising solution for gene therapy, overcoming challenges like poor DNA uptake and degradation. This natural polymer enhances DNA delivery for effective gene expression in cells and in vivo.

More Related Videos

Enhanced Gene Delivery and Expression using Intraosseous Injection of Chitosan Nanoparticles Encapsulated Adenine Base Editor Plasmids

Enhanced Gene Delivery and Expression using Intraosseous Injection of Chitosan Nanoparticles Encapsulated Adenine Base Editor Plasmids

Published on: May 16, 2025

Gene Transfection toward Spheroid Cells on Micropatterned Culture Plates for Genetically-modified Cell Transplantation

Gene Transfection toward Spheroid Cells on Micropatterned Culture Plates for Genetically-modified Cell Transplantation

Published on: July 31, 2015

Related Experiment Videos

Last Updated: Jul 3, 2026

Chitosan/Interfering RNA Nanoparticle Mediated Gene Silencing in Disease Vector Mosquito Larvae

Chitosan/Interfering RNA Nanoparticle Mediated Gene Silencing in Disease Vector Mosquito Larvae

Published on: March 25, 2015

Enhanced Gene Delivery and Expression using Intraosseous Injection of Chitosan Nanoparticles Encapsulated Adenine Base Editor Plasmids

Enhanced Gene Delivery and Expression using Intraosseous Injection of Chitosan Nanoparticles Encapsulated Adenine Base Editor Plasmids

Published on: May 16, 2025

Gene Transfection toward Spheroid Cells on Micropatterned Culture Plates for Genetically-modified Cell Transplantation

Gene Transfection toward Spheroid Cells on Micropatterned Culture Plates for Genetically-modified Cell Transplantation

Published on: July 31, 2015

Area of Science:

Biotechnology
Genomics
Proteomics

Background:

Gene therapy faces challenges with DNA therapeutics, including poor cellular uptake and rapid degradation.
Viral and nonviral vectors are used to improve DNA delivery and preserve therapeutic activity.

Purpose of the Study:

To investigate the potential of chitosan as a nonviral vector for gene therapy.
To evaluate the efficacy of chitosan-DNA nanocomplexes in protecting DNA and facilitating cellular uptake.

Main Methods:

Formation of chitosan-DNA nanocomplexes.
Assessment of DNA protection against degradation.
Evaluation of cellular uptake and gene transfer efficiency in vitro and in vivo.

Main Results:

Chitosan effectively forms nanocomplexes with DNA.
These nanocomplexes protect DNA from DNase degradation.
Chitosan facilitates effective and safe DNA transfer into cells, both in culture and in vivo.

Conclusions:

Chitosan is a biocompatible and biodegradable natural polysaccharide with potential as a gene delivery vector.
Chitosan-DNA nanocomplexes represent a viable strategy to overcome major drawbacks in current gene therapy approaches.