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Related Concept Videos

Gene Therapy00:59

Gene Therapy

26.6K
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...
26.6K

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Related Experiment Video

Updated: Nov 17, 2025

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

Published on: March 2, 2018

10.3K

Inner Ear Gene Delivery: Vectors and Routes.

Chris Valentini1, Betsy Szeto1, Jeffrey W Kysar1,2

  • 1Department of Otolaryngology -- Head and Neck Surgery, Columbia University Vagelos College of Physicians and Surgeons, New York, NY.

Hearing, Balance and Communication
|February 19, 2021
PubMed
Summary
This summary is machine-generated.

Gene therapy offers a promising approach to restore hearing by addressing underlying defects. Advances in viral and non-viral vectors, along with inner ear delivery methods, are key to overcoming challenges in treating hearing loss.

Keywords:
Gene therapyhearing lossinner ear deliverynon-viral vectorviral vector

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Related Experiment Videos

Last Updated: Nov 17, 2025

Posterior Semicircular Canal Approach for Inner Ear Gene Delivery in Neonatal Mouse
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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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Area of Science:

  • Otolaryngology
  • Ophthalmology
  • Molecular Biology

Background:

  • Current hearing loss treatments provide limited functional improvement.
  • Restoring normal hearing remains an unmet clinical need.
  • Gene therapy presents a potential solution for innate hearing defects.

Purpose of the Study:

  • Review recent advancements in viral and non-viral vectors for gene therapy.
  • Discuss strategies to overcome inner ear delivery barriers for gene products.
  • Highlight the potential of gene therapy for hearing restoration.

Main Methods:

  • Comprehensive literature search of Medline, EMBASE, Web of Science, and Google Scholar.
  • Utilized specific search terms related to inner ear anatomy and gene therapy.
  • Included reference lists of identified articles for study selection.

Main Results:

  • Both viral and non-viral vectors are crucial for advancing inner ear gene delivery.
  • The round window membrane presents a significant barrier to gene delivery.
  • Intratympanic and intracochlear delivery methods aim to overcome these barriers.

Conclusions:

  • Gene therapy holds promise for restoring hearing by correcting genetic defects.
  • Technological progress in inner ear drug delivery enhances gene therapy prospects.
  • Further research into delivery techniques is essential for successful gene therapy applications.