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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...

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: Jul 5, 2026

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA

Published on: February 1, 2019

Cationic liposome systems in gene therapy.

A D Miller¹

¹Imperial College of Science, Technology & Medicine, Department of Chemistry, South Kensington, London, SW7 2AY, UK. a.miller@ic.ac.uk

Idrugs : the Investigational Drugs Journal

|May 10, 2008

Summary

Cationic liposomes show promise for gene therapy delivery in patients. However, significant technical challenges remain for their widespread use as gene delivery vehicles in clinical applications.

Area of Science:

Biotechnology
Molecular Biology
Gene Therapy

Background:

Cationic liposomes are established agents for facilitating nucleic acid delivery to cells in laboratory settings.
Gene therapy offers a potential treatment modality for various diseases by delivering therapeutic genes or nucleic acids.

Purpose of the Study:

To evaluate the feasibility and current limitations of using cationic liposomes as vectors for in vivo gene therapy.
To assess the potential of cationic liposome systems for future clinical gene therapy applications.

Main Methods:

Review of existing literature on cationic liposome-mediated nucleic acid delivery in vitro and in vivo.
Analysis of technical challenges and research progress in the field of gene therapy vectors.

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Last Updated: Jul 5, 2026

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA

Published on: February 1, 2019

Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes

Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes

Published on: July 23, 2016

Formulating and Characterizing Lipid Nanoparticles for Gene Delivery using a Microfluidic Mixing Platform

Formulating and Characterizing Lipid Nanoparticles for Gene Delivery using a Microfluidic Mixing Platform

Published on: February 25, 2021

Main Results:

Cationic liposomes have demonstrated efficacy in delivering nucleic acids in vivo for gene therapy research.
Numerous technical problems persist, hindering the direct clinical translation of this technology.

Conclusions:

While cationic liposomes hold potential as gene therapy vectors, substantial advancements in basic research are required.
Further development is essential to overcome current obstacles and realize the future clinical utility of cationic liposomes in gene therapy.