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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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Pleiotropy01:33

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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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Hearing01:31

Hearing

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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.
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Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

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Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
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iPS Cell Differentiation01:22

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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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Related Experiment Video

Updated: Jan 13, 2026

Posterior Semicircular Canal Approach for Inner Ear Gene Delivery in Neonatal Mouse
03:52

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

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Gene therapy for hereditary deafness: progress, achievements and future challenges.

Kaifan Xu1, Yun Xiao1, Xiujuan Xia1

  • 1Department of Department of Otolaryngology-Head and Neck Surgery, Shandong Provincial ENT Hospital, Shandong University, Jinan, Shandong, China.

Acta Oto-Laryngologica
|January 6, 2026
PubMed
Summary

Gene therapy is revolutionizing hearing restoration for genetic sensorineural hearing loss. Adeno-associated virus (AAV)-OTOF gene therapy has successfully restored hearing in children, marking a new era for treating deafness.

Keywords:
AAVscochleargene therapiesgenetic hearing loss

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Canalostomy As a Surgical Approach to Local Drug Delivery into the Inner Ears of Adult and Neonatal Mice
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Last Updated: Jan 13, 2026

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Area of Science:

  • Genetics
  • Otolaryngology
  • Regenerative Medicine

Background:

  • Sensorineural hearing loss, primarily genetic, affects over 50% of congenital cases.
  • Current treatments like hearing aids and cochlear implants improve audibility but don't restore cochlear biology.

Purpose of the Study:

  • Review the evolution of inner-ear gene therapy from preclinical to clinical stages.
  • Summarize gene therapy strategies and technological challenges.
  • Highlight advances in therapeutic approaches for hearing loss.

Main Methods:

  • Surveyed preclinical studies in rodents and non-human primates.
  • Analyzed adeno-associated virus (AAV) delivery vectors and surgical routes.
  • Reviewed clinical data from AAV-mediated OTOF gene therapy trials.

Main Results:

  • Efficient viral vectors can transduce various cochlear cell types.
  • Safe surgical routes for inner-ear delivery are established in primate models.
  • AAV-OTOF gene therapy restored hearing in children with DFNB9 congenital deafness.

Conclusions:

  • Inner-ear gene therapy is now a clinically validated treatment for hearing loss.
  • Successful AAV-OTOF trials pave the way for treating diverse auditory and vestibular disorders.
  • This heralds a new era of restorative medicine for deafness.