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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: May 26, 2026

Preparation of rAAV9 to Overexpress or Knockdown Genes in Mouse Hearts

Preparation of rAAV9 to Overexpress or Knockdown Genes in Mouse Hearts

Published on: December 17, 2016

Advances in Gene Delivery Systems.

Kenya Kamimura¹, Takeshi Suda, Guisheng Zhang

¹Division of Gastroenterology and Hepatology, Graduate School of Medical and Dental Sciences, Niigata University, Niigata, Japan.

Pharmaceutical Medicine

|December 28, 2011

Summary

This summary is machine-generated.

Gene delivery methods, including viral vectors, nonviral vectors, and physical approaches, are crucial for gene therapy and function studies. Research focuses on optimizing these techniques for safe, effective, and targeted gene transfer.

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Last Updated: May 26, 2026

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Published on: October 30, 2018

Area of Science:

Biotechnology
Molecular Biology
Gene Therapy

Background:

Gene transfer into cells is fundamental for gene function research and therapeutic applications.
Current methods involve viral vectors, nonviral vectors, and physical approaches.

Purpose of the Study:

To review the rationale behind viral, nonviral, and physical gene delivery systems.
To summarize recent advancements in gene transfer technology.
To discuss the pros and cons of common gene delivery methods and offer future perspectives.

Main Methods:

Viral vector-mediated gene transfer (retrovirus, adenovirus, AAV, HSV).
Nonviral vector-mediated gene transfer (cationic liposomes, polymers, peptides).
Physical gene transfer methods (electroporation, hydrodynamic force).

Main Results:

Viral vectors offer high gene delivery efficiency.
Nonviral vectors are effective in vitro but less so in vivo.
Physical methods are simple and effective for localized delivery.

Conclusions:

Significant progress has been made in developing safe and effective gene delivery methods.
Each method has distinct advantages and disadvantages impacting its application.
Future research aims for enhanced target specificity and safety in gene transfer.