Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …

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 …

Mutations to the OTOF gene are among the most common reasons for auditory neuropathy.
Although cochlear implants are often effective in restoring sound transduction, there are …

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 …

In vivo therapeutic gene transfer has emerged as a novel class of medicines. Its feasibility
relies on the safe and efficacious delivery of genetic cargo to the appropriate targets. The …

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 …

Pathogenic mutations in the OTOF gene cause autosomal recessive hearing loss (DFNB9),
one of the most common forms of auditory neuropathy. There is no biological treatment for …

Inner ear gene therapy using adeno-associated viral vectors (AAV) promises to alleviate
hearing and balance disorders. We previously established the benefits of Anc80L65 in …

The cochlea consists of multiple types of cells, including hair cells, supporting cells and
spiral ganglion neurons, and is responsible for converting mechanical forces into electric …