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 …

Gene therapy would benefit from the effective editing of targeted cells with CRISPR-Cas9
tools. However, it is difficult to precisely assess the editing performance in vivo because the …

OTOF mutations are the principal causes of auditory neuropathy. There are reports on Otof‐
related gene therapy in mice, but there is no preclinical research on the drug evaluations …

The TDP-43 proteinopathies, which include amyotrophic lateral sclerosis and frontotemporal
dementia, are a devastating group of neurodegenerative disorders that are characterized by …

Manipulating gene expression within and outside the nervous system is useful for
interrogating gene function and developing therapeutic interventions for a variety of …

Adeno-associated viral (AAV) vectors are increasingly used as vehicles for gene delivery to
treat hearing loss. However, lack of specificity of the transgene expression may lead to …

Hearing loss stands as the most prevalent sensory deficit among humans, posing a
significant global health challenge. Projections indicate that by 2050, approximately 10% of …

Up to 1.5 billion people worldwide suffer from various forms of hearing loss, with an
additional 1.1 billion people at risk from various insults such as increased consumption of …

Inner ear gene therapy using adeno-associated viral vectors (AAVs) in neonatal mice can
alleviate hearing loss in mouse models of deafness. However, efficient and safe transgene …