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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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Updated: Sep 9, 2025

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

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[Gene therapy for inner ear disorders: a step into the future].

T G Markova^1,2,3, V V Bakhshinyan^2,3

¹Sverzhevsky Research Clinical Institute of Otorhinolaryngology, Moscow, Russia.

Vestnik Otorinolaringologii

|September 3, 2025

Summary

Gene therapy is emerging in audiology for inner ear disorders, with ongoing clinical trials. Raising awareness and improving diagnostic tools are crucial for hereditary hearing loss and auditory neuropathy spectrum disorders.

Keywords:

GJB2 OTOF TMC1 genes auditory neuropathy spectrum disorders gene therapy for inner ear disorders hereditary hearing loss otoferlin

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Last Updated: Sep 9, 2025

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

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

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Published on: March 16, 2015

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Published on: May 25, 2018

Area of Science:

Genetics and Medicine
Audiology and Otolaryngology

Background:

Molecular genetics advances and new gene technologies herald a new era in medicine.
Gene therapy is becoming a significant medical field, impacting audiology and otolaryngology.
Innovative therapeutic solutions for inner ear disorders are being developed, with some gene therapy programs in clinical trials.

Purpose of the Study:

To present the current status of gene therapy research in the complex field of inner ear disorders.
To highlight the intersection of science and practice in treating inner ear conditions.
To emphasize the need for increased awareness among healthcare professionals and patients regarding hereditary hearing loss.

Main Methods:

Review of current advancements in molecular genetics and gene therapy technologies.
Analysis of ongoing clinical trials for inner ear gene therapy.
Discussion of diagnostic challenges in hereditary hearing loss and auditory neuropathy spectrum disorders.

Main Results:

Gene therapy represents a promising frontier for treating inner ear disorders.
Clinical trials for inner ear gene therapy are progressing.
There is a recognized need for enhanced diagnostic tools and greater awareness.

Conclusions:

Gene therapy holds significant potential for audiology and otolaryngology.
Further development of reliable diagnostic tools is essential for early detection.
Educating doctors and patients about hereditary hearing loss is critical for utilizing new therapeutic opportunities.