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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...

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Related Experiment Video

Updated: Dec 25, 2025

Surgical Method for Virally Mediated Gene Delivery to the Mouse Inner Ear through the Round Window Membrane

Surgical Method for Virally Mediated Gene Delivery to the Mouse Inner Ear through the Round Window Membrane

Published on: March 16, 2015

Adeno-associated virus gene replacement for recessive inner ear dysfunction: Progress and challenges.

Charles Askew¹, Wade W Chien²

¹Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.

Hearing Research

|April 6, 2020

Summary

Gene replacement therapy offers a promising new treatment for hereditary hearing loss, using viral vectors to deliver corrective genes. This approach shows potential for improving auditory function and is nearing human clinical trials.

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Last Updated: Dec 25, 2025

Surgical Method for Virally Mediated Gene Delivery to the Mouse Inner Ear through the Round Window Membrane

Surgical Method for Virally Mediated Gene Delivery to the Mouse Inner Ear through the Round Window Membrane

Published on: March 16, 2015

Posterior Semicircular Canal Approach for Inner Ear Gene Delivery in Neonatal Mouse

Posterior Semicircular Canal Approach for Inner Ear Gene Delivery in Neonatal Mouse

Published on: March 2, 2018

Canalostomy As a Surgical Approach to Local Drug Delivery into the Inner Ears of Adult and Neonatal Mice

Canalostomy As a Surgical Approach to Local Drug Delivery into the Inner Ears of Adult and Neonatal Mice

Published on: May 25, 2018

Area of Science:

Otolaryngology
Genetics
Molecular Biology

Background:

Hereditary hearing loss affects approximately 3 in 1000 children under 4 in the US.
Current treatments like cochlear implants are limited; no targeted drug therapies exist.
Gene replacement therapy is a potential therapeutic strategy for hereditary hearing loss.

Purpose of the Study:

To review the current status of adeno-associated virus (AAV) gene replacement therapy for recessive hereditary hearing loss.
To discuss challenges and opportunities in translating this therapy for patients.

Main Methods:

Focuses on gene replacement using viral vectors (specifically AAV) to deliver functional cDNA.
Reviews proof-of-concept studies in mouse models and discusses human clinical trial readiness.
Examines the biological basis of defective genes and therapeutic strategy design.

Main Results:

Gene replacement has shown success in improving auditory function in animal models.
The effectiveness depends on the specific gene's biology and timely intervention before sensory structure degeneration.
Therapeutic windows may vary, with post-lingual onset genes offering longer treatment opportunities.

Conclusions:

Adeno-associated virus (AAV) gene replacement therapy is a promising approach for hereditary hearing loss.
Successful translation requires addressing gene delivery challenges and identifying optimal therapeutic windows.
Future research into post-lingual onset genes could expand treatment possibilities.