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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: Jan 23, 2026

Neuro-rehabilitation Approach for Sudden Sensorineural Hearing Loss

Neuro-rehabilitation Approach for Sudden Sensorineural Hearing Loss

Published on: January 25, 2016

Gene therapy for hearing loss.

Ryotaro Omichi^1,2, Seiji B Shibata^1,3, Cynthia C Morton^4,5,6

¹Molecular Otolaryngology and Renal Research Laboratories, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.

Human Molecular Genetics

|June 23, 2019

Summary

This summary is machine-generated.

Gene therapy offers new hope for sensorineural hearing loss (SNHL), the most common sensory disorder. Research explores molecular treatments like gene replacement and editing to restore hearing, moving towards clinical trials.

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Modified Experimental Conditions for Noise-Induced Hearing Loss in Mice and Assessment of Hearing Function and Outer Hair Cell Damage

Modified Experimental Conditions for Noise-Induced Hearing Loss in Mice and Assessment of Hearing Function and Outer Hair Cell Damage

Published on: February 10, 2023

Area of Science:

Otolaryngology
Genetics
Molecular Biology

Background:

Sensorineural hearing loss (SNHL) is a prevalent sensory disorder with diverse genetic and environmental causes.
Current management relies on amplification devices, but novel therapeutic strategies are emerging.
Gene therapy presents a promising avenue for restoring and preventing SNHL.

Purpose of the Study:

To review molecular therapies for SNHL, including gene replacement, antisense oligonucleotides, RNA interference, and CRISPR-based gene editing.
To discuss delivery methods, techniques, and viral vectors for inner ear gene therapy.
To highlight advancements enabling the transition of basic science discoveries to clinical applications.

Main Methods:

Literature review of molecular therapies for SNHL.
Analysis of gene therapy delivery systems for the inner ear.
Discussion of gene editing technologies like CRISPR.

Main Results:

Proof-of-principle studies show potential for molecular agents to ameliorate SNHL.
Gene replacement and editing are key strategies for treating genetic hearing loss.
Various delivery methods and viral vectors are being investigated for inner ear gene therapy.

Conclusions:

Molecular therapies, particularly gene-based strategies, hold significant potential for treating SNHL.
Advancements in gene therapy are paving the way for clinical trials.
Restoring or preventing genetic hearing loss through molecular interventions is becoming a tangible reality.