Treating otoferlin deficiency with gene therapy

Nawal Ouzren, Sensorion, Montpellier, France, gives an overview of the rationale behind developing a gene therapy for patients with hearing loss who have an otoferlin deficiency. Otoferlin is a protein expressed in inner hair cells, which is sensitive to a certain frequency. Initial in vivo studies have successfully restored expression of otoferlin in animal models, who have regained functional hearing as a result of the gene therapy. This interview was conducted during Meeting on the Mediterranean 2022.