Advances in cochlear gene therapies.
Journal
Current opinion in pediatrics
ISSN: 1531-698X
Titre abrégé: Curr Opin Pediatr
Pays: United States
ID NLM: 9000850
Informations de publication
Date de publication:
01 12 2023
01 12 2023
Historique:
medline:
2
11
2023
pubmed:
7
7
2023
entrez:
7
7
2023
Statut:
ppublish
Résumé
Hearing loss is the most common sensory deficit and in young children sensorineural hearing loss is most frequently genetic in etiology. Hearing aids and cochlear implant do not restore normal hearing. There is significant research and commercial interest in directly addressing the root cause of hearing loss through gene therapies. This article provides an overview of major barriers to cochlear gene therapy and recent advances in preclinical development of precision treatments of genetic deafness. Several investigators have recently described successful gene therapies in many common forms of genetic hearing loss in animal models. Elegant strategies that do not target a specific pathogenic variant, such as mini gene replacement and mutation-agnostic RNA interference (RNAi) with engineered replacement, facilitate translation of these findings to development of human therapeutics. Clinical trials for human gene therapies are in active recruitment. Gene therapies for hearing loss are expected to enter clinical trials in the immediate future. To provide referral for appropriate trials and counseling regarding benefits of genetic hearing loss evaluation, specialists serving children with hearing loss such as pediatricians, geneticists, genetic counselors, and otolaryngologists should be acquainted with ongoing developments in precision therapies.
Identifiants
pubmed: 37417821
doi: 10.1097/MOP.0000000000001273
pii: 00008480-990000000-00107
doi:
Types de publication
Review
Journal Article
Research Support, N.I.H., Extramural
Langues
eng
Sous-ensembles de citation
IM
Pagination
631-640Subventions
Organisme : NIDCD NIH HHS
ID : R01 DC002842
Pays : United States
Organisme : NIDCD NIH HHS
ID : R01 DC012049
Pays : United States
Organisme : NIDCD NIH HHS
ID : T32 DC000040
Pays : United States
Organisme : NIDCD NIH HHS
ID : R01 DC017955
Pays : United States
Informations de copyright
Copyright © 2023 Wolters Kluwer Health, Inc. All rights reserved.
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