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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 inserted. The...

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 inserted. The...

Hearing

Hearing

When we hear a sound, our nervous system is detecting sound waves—pressure waves of mechanical energy traveling through a medium. The frequency of the wave is perceived as pitch, while the amplitude is perceived as loudness.

Genetic Lingo

Genetic Lingo

Pleiotropy

Pleiotropy

Pleiotropy is the phenomenon in which a single gene impacts multiple, seemingly unrelated phenotypic traits. For example, defects in the SOX10 gene cause Waardenburg Syndrome Type 4, or WS4, which can cause defects in pigmentation, hearing impairments, and an absence of intestinal contractions necessary for elimination. This diversity of phenotypes results from the expression pattern of SOX10 in early embryonic and fetal development. SOX10 is found in neural crest cells that form melanocytes,...

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Related Experiment Video

Updated: May 9, 2026

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

Gene therapy for deafness.

D C Kohrman¹, Y Raphael

¹Department of Otolaryngology-Head and Neck Surgery, Kresge Hearing Research Institute, The University of Michigan, Ann Arbor, MI, USA.

|July 19, 2013

Summary

This summary is machine-generated.

Gene therapy offers new hope for hearing loss, the most common sensory deficit. Research explores genetic pathways and molecular mechanisms for hearing preservation and regeneration, with potential clinical applications.

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Related Experiment Videos

Last Updated: May 9, 2026

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

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

Published on: March 16, 2015

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
Regenerative Medicine

Background:

Hearing loss is a widespread sensory deficit caused by genetic or environmental factors affecting the cochlea.
Understanding cochlear development, maintenance, and cell death mechanisms is crucial for addressing hearing impairment.

Purpose of the Study:

To review recent advances in gene-based therapies for hearing loss.
To explore opportunities for modulating genetic and molecular pathways for hearing restoration.

Main Methods:

Review of recent animal studies utilizing gene-based therapies.
Analysis of molecular mechanisms underlying cochlear function and cell trauma.

Main Results:

Gene-based therapies show promise in correcting genetic mutations related to hearing loss.
Approaches to enhance protective pathways and regenerate sensory cells are under investigation.

Conclusions:

Gene therapy presents a promising avenue for treating hearing loss.
Further research and refinement of these methods may lead to clinical applications for hearing restoration.