A new study shows that gene therapy can significantly improve hearing in both children and adults with congenital deafness caused by mutations in the OTOF gene.

Re-administration of adeno-associated virus (AAV)-mediated gene therapy remains challenging due to neutralizing antibodies (NAbs) induced by the initial dose. We previously conducted a single-arm trial showing that single-dose administration of AAV-hOTOF gene therapy in individuals with OTOF-related deafness is safe and leads to hearing improvements. Here we initially demonstrate that AAV1-hOTOF re-administration to the contralateral ear in Otof−/− mice with peak serum NAb titers successfully rescued hearing with limited immune activation. After a protocol amendment of our trial, four patients (aged 2.2–3.4 years) with pre-existing NAbs (titers 1:135–1:3,645), who had previously received a single dose of the gene therapy, were enrolled to receive a second dose in the contralateral ear, as part of the ongoing trial, with a follow-up ranging from 26 weeks to 52 weeks. The primary endpoint was the occurrence of dose-limiting toxicities at 6 weeks and secondary endpoints included safety an

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Etwa zwei von 1.000 Kindern in Deutschland kommen gehörlos zur Welt. Meist ist die Ursache ein Gendefekt. Das gilt auch bei der sogenannten OTOF-Taubheit. Wie Studien zeigen, lässt diese sich jetzt mi...

In a multicentre trial of AAV1-hOTOF gene therapy involving 42 participants aged 0.8–32.3 years with autosomal recessive deafness 9, treatment demonstrated safety, tolerability, and improved hearing a...

In a multicentre trial of AAV1-hOTOF gene therapy involving 42 participants aged 0.8–32.3 years with autosomal recessive deafness 9, treatment demonstrated safety, tolerability, and improved hearing and speech perception, with age and distortion product otoacoustic emissions associated with treatment outcomes.

The authors describe the scientific foundations of a study of gene therapy for hearing loss caused by damaging variants in the gene OTOF.

Children’s Wisconsin says it is the first U.S. center to deliver FDA-approved Otarmeni gene therapy for OTOF-related genetic hearing loss outside a clinical trial.

Rund zwei von 1.000 Kindern in Deutschland werden gehörlos geboren. Ursache ist häufig ein Gendefekt, so wie bei der OTOF-Taubheit. Für diese gibt es nun eine Gentherapie, die laut Studien erstmals Er...

A new study shows that gene therapy can significantly improve hearing in both children and adults with congenital deafness caused by mutations in the OTOF gene.

YouTube video by Киноконцерн "Мосфильм"

Preliminary results from an investigator-initiated clinical trial showed that an AAV-OTOF gene therapy was safe and led to hearing improvements in ten patients with congenital deafness with 6–12 month...

One 7-year-old girl who received a traditional cochlear implant in one ear and the gene therapy in another, recovered almost all her hearing.

Gene therapy can improve hearing in children and adults with congenital deafness or severe hearing impairment, a new study involving researchers at Karolinska Institutet reports. Hearing improved in a...