Gene Therapy: An Historical Overview for Familial Hearing Loss.

Publisher: MDPI Country of Publication: Switzerland NLM ID: 101092791 Publication Model: Electronic Cited Medium: Internet ISSN: 1422-0067 (Electronic) Linking ISSN: 14220067 NLM ISO Abbreviation: Int J Mol Sci Subsets: MEDLINE

Original Publication: Basel, Switzerland : MDPI, [2000-

Genetic Therapy*/methods ; Genetic Therapy*/history ; Hearing Loss*/therapy ; Hearing Loss*/genetics; Humans ; History, 21st Century ; History, 20th Century ; Animals

Gene therapy is a promising molecular approach for the management of familial hearing loss. This type of molecular therapy is the physical manifestation of genetic determinism-the notion that individual genes cause individual phenotypes. The current composition weaves through various branches of the biomedical sciences to uncover the molecular biologic premise for genetic determinism and the impetus behind gene therapy. Consequently, it is revealed that the underlying molecular biologic premise was scaffolded on successful observations from simple biologic assays that were devoid of the complexities of human disease biology. Furthermore, modern successful gene therapies are largely driven by commercial and academic incentives at the cost of scientific rigor. This poses several perverse challenges for patients, clinicians and the public at large. Issues concerning safety, efficacy, and ethics are far from resolved despite regulatory agency approvals, the media's bias for gene therapy and the many lucrative investor positions. Lastly, the therapeutic claims regarding gene therapy are the most ambitious claims made within the hearing sciences. Therefore, scientists, clinicians, and patients must be equipped with the tools needed to appropriately consume and appraise such claims. These and other issues are also directly addressed, with the aim of providing a realistic sense of whether current human gene therapies are ready to be positioned within our routine clinical armamentarium against hearing loss.
Competing Interests: The author declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article.

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Keywords: CRISPR-Cas9; congenital; deafness; ear; gene editing; genetic; hearing; hereditary; sensorineural; treatment

Date Created: 20250226 Date Completed: 20250226 Latest Revision: 20250228

20250228

PMC11855000

10.3390/ijms26041469

40003934