Gene transfer has long been a compelling yet elusive therapeutic modality. First mainly
considered for rare inherited disorders, gene therapy may open treatment opportunities for …

Progress in deciphering the genetic architecture of human sensorineural hearing
impairment (SNHI) or loss, and multidisciplinary studies of mouse models, have led to the …

Gene therapy focuses on genetic modification to produce therapeutic effects or treat
diseases by repairing or reconstructing genetic material, thus being expected to be the most …

Usher syndrome (USH) is the most common cause of deaf–blindness in humans, with a
prevalence of about 1/10,000 (~ 400,000 people worldwide). Cochlear implants are …

Hearing loss is the most common sensory disorder. While gene therapy has emerged as a
promising treatment of inherited diseases like hearing loss, it is dependent on the …

Mutations in OTOFERLIN (OTOF) lead to the autosomal recessive deafness 9 (DFNB9). The
efficacy of adeno‐associated virus (AAV)‐mediated OTOF gene replacement therapy is …

Hearing impairment is the most frequent sensory deficit in humans of all age groups, from
children (1/500) to the elderly (more than 50% of the over-75 s). Over 50% of congenital …

Usher syndrome has three subtypes, each being clinically and genetically heterogeneous
characterised by sensorineural hearing loss and retinitis pigmentosa (RP), with or without …

Usher syndrome type 1 F (USH1F), caused by mutations in the protocadherin-15 gene
(PCDH15), is characterized by congenital deafness, lack of balance, and progressive …

Outer hair cell (OHC) degeneration is a major cause of progressive hearing loss and
presbycusis. Despite the high prevalence of these disorders, targeted therapy is currently …