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Surgical Method for Virally Mediated Gene Delivery to the Mouse Inner Ear through the Round Window Membrane
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Genetic Medicine for Hearing Loss: OTOF as Exemplar
Ann E Hickox1, Michelle D Valero1, James T McLaughlin1
1Akouos Inc., Boston, Massachusetts.
Journal of the American Academy of Audiology
|May 24, 2022
Summary
Genetic medicine offers hope for disabling hearing loss caused by faulty genes. Delivering correct genes, like otoferlin (OTOF), to the inner ear may restore hearing function.
Area of Science:
- Otolaryngology
- Genetics
- Molecular Biology
Background:
- Disabling hearing loss affects millions globally, often stemming from genetic mutations.
- Specific gene mutations lead to incorrect protein production essential for auditory function.
- Restoring proper gene function in the inner ear presents a potential therapeutic avenue.
Purpose of the Study:
- To outline a strategy for developing genetic medicines to treat hearing loss.
- To focus on otoferlin (OTOF) gene-mediated hearing loss as a model.
- To explore the potential of gene delivery for restoring cochlear function.
Main Methods:
- Reviewing current genetic medicine strategies.
- Analyzing the otoferlin gene (OTOF) and its role in hearing.
- Identifying target cells within the inner ear for gene delivery.
Main Results:
- Genetic medicine holds promise for treating sensorineural hearing loss.
- Targeted gene delivery can potentially restore cochlear function.
- The OTOF gene is a key target for treating specific inherited hearing impairments.
Conclusions:
- A strategic approach to genetic medicine development is crucial for treating hearing loss.
- Gene therapy offers a viable pathway to restore physiologic hearing.
- Focusing on genes like OTOF can advance treatments for inherited auditory disorders.
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