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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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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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The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
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The Cochlea01:13

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The cochlea is a coiled structure in the inner ear that contains hair cells—the sensory receptors of the auditory system. Sound waves are transmitted to the cochlea by small bones attached to the eardrum called the ossicles, which vibrate the oval window that leads to the inner ear. This causes fluid in the chambers of the cochlea to move, vibrating the basilar membrane.
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Related Experiment Video

Updated: Jul 24, 2025

Surgical Method for Virally Mediated Gene Delivery to the Mouse Inner Ear through the Round Window Membrane
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Advances in cochlear gene therapies.

Miles J Klimara1, Richard J H Smith

  • 1Molecular Otolaryngology & Renal Research Laboratories, Department of Otolaryngology - Head and Neck Surgery, University of Iowa, Iowa City, Iowa 52242, USA.

Current Opinion in Pediatrics
|July 7, 2023
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Summary

Gene therapy offers a promising approach to treating genetic hearing loss, addressing the root cause rather than just symptoms. Advances in precision treatments are paving the way for human clinical trials in genetic deafness.

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Area of Science:

  • Otolaryngology
  • Genetics
  • Molecular Biology

Background:

  • Sensorineural hearing loss, often genetic in origin, is a common childhood deficit.
  • Current treatments like hearing aids and cochlear implants do not restore normal auditory function.
  • Gene therapy presents a novel approach to directly address the genetic causes of hearing loss.

Approach:

  • Overview of challenges and recent breakthroughs in cochlear gene therapy.
  • Discussion of preclinical developments in precision treatments for genetic deafness.
  • Highlighting mutation-agnostic strategies like mini gene replacement and RNA interference (RNAi).

Key Points:

  • Successful gene therapies have been demonstrated in animal models for common genetic hearing loss forms.
  • Mutation-agnostic strategies simplify the translation of findings to human therapeutics.
  • Clinical trials for human gene therapies are actively recruiting patients.

Conclusions:

  • Gene therapies for hearing loss are nearing clinical application.
  • Specialists need to be informed about precision therapies for appropriate patient referral and counseling.
  • Genetic evaluation is crucial for children with hearing loss to identify potential gene therapy candidates.