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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...

Gene Therapy

Gene Therapy

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: Apr 5, 2026

Author Spotlight: Optimizing Digital Droplet PCR Method for Accurate Adeno-Associated Viral Genome Quantification

Author Spotlight: Optimizing Digital Droplet PCR Method for Accurate Adeno-Associated Viral Genome Quantification

Published on: October 11, 2024

Viral vector-mediated gene therapies.

Katrin Hollinger¹, Jeffrey S Chamberlain

¹Department of Neurology, Wellstone Muscular Dystrophy Research Center, University of Washington School of Medicine, Seattle, Washington, USA.

Current Opinion in Neurology

|August 12, 2015

Summary

Recent advancements in adeno-associated virus (AAV) gene therapy offer new hope for Duchenne muscular dystrophy (DMD). Strategies like immune modulation and gene editing are improving treatment efficacy and paving the way for clinical trials.

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Last Updated: Apr 5, 2026

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Published on: October 11, 2024

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Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine

Published on: November 4, 2018

Area of Science:

Biomedical Science
Molecular Biology
Genetics

Background:

Gene therapy has emerged as a promising treatment for neuromuscular diseases.
Adeno-associated virus (AAV) vectors are a key delivery system in gene therapy.
Duchenne muscular dystrophy (DMD) is a severe genetic disorder affecting muscle function.

Purpose of the Study:

To review the progress of AAV vector-mediated gene therapy for DMD over the past year.
To highlight recent findings and their implications for future research and clinical applications.

Main Methods:

Review of recent scientific literature on AAV gene therapy for DMD.
Analysis of strategies for modulating immune responses to AAV vectors and transgenes.
Evaluation of gene editing techniques for restoring dystrophin expression.

Main Results:

Modulating immune responses enhances stable transduction efficiency of AAV vectors.
Gene editing with targeted nucleases shows potential for full-length dystrophin expression.
Utilizing genes with homologous functions may alleviate DMD symptoms.

Conclusions:

Recent research has significantly advanced the understanding of AAV-mediated therapy for DMD.
New therapeutic approaches have been validated, informing the design of preclinical and clinical trials.
Continued progress in AAV gene therapy holds promise for improved DMD treatment outcomes.