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[Gene therapy progress: hopes for Usher syndrome].

Charlotte Calvet1, Ghizlene Lahlou1, Saaid Safieddine2

  • 1Institut Pasteur, Unité de génétique et physiologie de l'audition, 25, rue du Docteur Roux, 75724 Paris, Cedex 15, France - Inserm UMRS 1120, 75015 Paris, France - Sorbonne Universités, 75005 Paris, France.

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Summary

Gene therapy offers a promising approach to cure genetic hearing and balance impairments. This review focuses on viral gene therapy strategies for Usher syndrome, aiming for clinical application.

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Area of Science:

  • Ototolaryngology
  • Genetics
  • Molecular Biology

Background:

  • Hearing and balance impairments represent a significant global health issue.
  • Current treatments for genetic deafness are limited, lacking effective curative options.
  • Viral gene therapy shows potential for treating genetic diseases.

Purpose of the Study:

  • To review recent advances in gene therapy for restoring cochlear and vestibular functions.
  • To focus on mouse models of Usher syndrome, a leading genetic cause of combined deafness and blindness.
  • To highlight therapeutic approaches with high clinical application potential.

Main Methods:

  • Review of preclinical and clinical studies on gene therapy for hearing and balance disorders.
  • Analysis of molecular bases of genetic deafness.
  • Focus on viral vector-mediated gene delivery in mouse models.

Main Results:

  • Gene therapy approaches are being developed to correct, replace, or modify defective genes causing deafness.
  • Significant progress has been made in restoring cochlear and vestibular functions in mouse models.
  • Usher syndrome models are crucial for evaluating gene therapy efficacy.

Conclusions:

  • Local gene therapy holds promise for treating genetic hearing and balance impairments, particularly Usher syndrome.
  • Further research and development are needed to translate these findings into clinical practice.
  • Understanding the molecular basis of genetic deafness is key to advancing gene therapy.