New England Journal of Medicine· 2025Q1

DB-OTO Gene Therapy for Inherited Deafness

Vassili Valayannopoulos, Manohar Bance, Daniela Carvalho, John H. Greinwald et al.

66citations
Q1SCImago
2025year

Short summary

DB-OTO gene therapy, delivering the OTOF gene, restored hearing to a level enabling natural acoustic hearing in 75% (9 of 12) of children with congenital deafness due to otoferlin deficiency.

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Field: Sensory Systems

Sensory SystemsNeuroscience