Deafness: from genetic architecture to gene therapy - Institut Pasteur Access content directly
Journal Articles Nature Reviews Genetics Year : 2023

Deafness: from genetic architecture to gene therapy


Progress in deciphering the genetic architecture of human sensorineural hearing impairment (SNHI) or loss, and multidisciplinary studies of mouse models, have led to the elucidation of the molecular mechanisms underlying auditory system function, primarily in the cochlea, the mammalian hearing organ. These studies have provided unparalleled insights into the pathophysiological processes involved in SNHI, paving the way for the development of inner-ear gene therapy based on gene replacement, gene augmentation or gene editing. The application of these approaches in preclinical studies over the past decade has highlighted key translational opportunities and challenges for achieving effective, safe and sustained inner-ear gene therapy to prevent or cure monogenic forms of SNHI and associated balance disorders.
Fichier principal
Vignette du fichier
PRR_Petit_clean_1678126492_35-111023.pdf (1016.62 Ko) Télécharger le fichier
Origin Files produced by the author(s)

Dates and versions

pasteur-04097478 , version 1 (10-10-2023)




Christine Petit, Crystel Bonnet, Saaïd Safieddine. Deafness: from genetic architecture to gene therapy. Nature Reviews Genetics, 2023, 24, pp.665-686. ⟨10.1038/s41576-023-00597-7⟩. ⟨pasteur-04097478⟩
420 View
269 Download



Gmail Mastodon Facebook X LinkedIn More