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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...

Site-Targeted Drug Delivery Systems: Polymeric Carriers

Site-Targeted Drug Delivery Systems: Polymeric Carriers

Polymeric carriers enhance targeted drug delivery by increasing efficacy while minimizing off-target effects. These carriers comprise a biodegradable polymeric backbone integrated with functional elements that enable targeting, improve physicochemical properties, and regulate drug release.Targeting MechanismsThe targeting ability of polymeric carriers is mediated by a homing device, which is a molecular recognition component designed to selectively bind to specific tissues or cells. Monoclonal...

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: Jun 16, 2026

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

Multifunctional nanocomplexes for gene transfer and gene therapy.

Stephen L Hart¹

¹Wolfson Centre for Gene Therapy of Childhood Disease, UCL Institute of Child Health, London, UK. shart@ich.ucl.ac.uk

Cell Biology and Toxicology

|February 4, 2010

Summary

Researchers are developing receptor-targeted nanocomplexes (RTNs) for advanced gene therapy. These synthetic nanoparticles mimic viruses for safer, more effective in vivo gene delivery, with applications in cystic fibrosis and vascular grafts.

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Evaluation of Polymeric Gene Delivery Nanoparticles by Nanoparticle Tracking Analysis and High-throughput Flow Cytometry

Evaluation of Polymeric Gene Delivery Nanoparticles by Nanoparticle Tracking Analysis and High-throughput Flow Cytometry

Published on: March 1, 2013

Related Experiment Videos

Last Updated: Jun 16, 2026

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

Evaluation of Polymeric Gene Delivery Nanoparticles by Nanoparticle Tracking Analysis and High-throughput Flow Cytometry

Evaluation of Polymeric Gene Delivery Nanoparticles by Nanoparticle Tracking Analysis and High-throughput Flow Cytometry

Published on: March 1, 2013

Area of Science:

Biotechnology
Gene Therapy
Nanomedicine

Background:

DNA aggregates with polycationic reagents are known as non-viral vectors, synthetic vectors, lipoplexes, polyplexes, and nanoparticles.
Nanoparticles offer potential for delivering multiple genes, genomic constructs, and siRNA, with improved in vivo gene delivery efficiency.

Purpose of the Study:

To review applications and challenges of nanoparticle technologies in gene therapy.
To focus on the development of receptor-targeted nanocomplexes (RTNs) for gene therapy research.

Main Methods:

Development of synthetic nanodelivery systems mimicking viral structures with synthetic safety.
Optimization of RTNs for airway epithelial and vascular transfection.
Modular design of RTN platform for structure-function hypothesis testing.

Main Results:

RTNs have been optimized for airway epithelial transfection, advancing gene therapy for cystic fibrosis.
RTNs are developed for vascular transfection in vein grafts for bypass surgery.
The modular RTN platform facilitates investigation into particle formation, stability, and nuclear disassembly.

Conclusions:

Nanoparticle technologies, particularly RTNs, show significant promise for future gene therapy applications.
RTNs offer a versatile platform for targeted gene delivery, mimicking viruses while maintaining safety.
Further research into RTN design and function will drive advancements in gene therapy for various diseases.