Despite scientific discoveries in the field of gene and cell therapy, some diseases still have
no effective treatment. Advances in genetic engineering methods have enabled 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 …

Most genetic diseases arise from recessive point mutations that require correction, rather
than disruption, of the pathogenic allele to benefit patients. Base editing has the potential to …

Parvoviruses are a diverse family of small, non-enveloped DNA viruses that infect a wide
variety of species, tissues and cell types. For over half a century, their intriguing biology and …

Normal hearing and synaptic transmission at afferent auditory inner hair cell (IHC) synapses
require otoferlin. Deafness DFNB 9, caused by mutations in the OTOF gene encoding …

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 …

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 …

Hereditary hearing loss often results from mutation of genes expressed by cochlear hair
cells. Gene addition using AAV vectors has shown some efficacy in mouse models, but …

During embryonic development, hierarchical cascades of transcription factors interact with
lineage-specific chromatin structures to control the sequential steps in the differentiation of …

The mechanoreceptive sensory hair cells in the inner ear are selectively vulnerable to
numerous genetic and environmental insults. In mammals, hair cells lack regenerative …