[HTML][HTML] Advances in gene therapy hold promise for treating hereditary hearing loss

L Jiang, D Wang, Y He, Y Shu - Molecular Therapy, 2023 - cell.com
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 …

[HTML][HTML] The genetic and phenotypic landscapes of Usher syndrome: from disease mechanisms to a new classification

S Delmaghani, A El-Amraoui - Human Genetics, 2022 - Springer
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 …

Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents

X Gao, Y Tao, V Lamas, M Huang, WH Yeh, B Pan… - Nature, 2018 - nature.com
Although genetic factors contribute to almost half of all cases of deafness, treatment options
for genetic deafness are limited,,,,. We developed a genome-editing approach to target a …

In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafness

WH Yeh, O Shubina-Oleinik, JM Levy, B Pan… - Science translational …, 2020 - science.org
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 …

[HTML][HTML] AAV-ie enables safe and efficient gene transfer to inner ear cells

F Tan, C Chu, J Qi, W Li, D You, K Li, X Chen… - Nature …, 2019 - nature.com
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 …

[HTML][HTML] Inner ear gene therapies take off: current promises and future challenges

S Delmaghani, A El-Amraoui - Journal of clinical medicine, 2020 - mdpi.com
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 …

Single-cell transcriptomic profiling of the mouse cochlea: An atlas for targeted therapies

P Jean, F Wong Jun Tai… - Proceedings of the …, 2023 - National Acad Sciences
Functional molecular characterization of the cochlea has mainly been driven by the
deciphering of the genetic architecture of sensorineural deafness. As a result, the search for …

[HTML][HTML] Improved TMC1 gene therapy restores hearing and balance in mice with genetic inner ear disorders

CA Nist-Lund, B Pan, A Patterson, Y Asai… - Nature …, 2019 - nature.com
Fifty percent of inner ear disorders are caused by genetic mutations. To develop treatments
for genetic inner ear disorders, we designed gene replacement therapies using synthetic …

Emerging approaches for restoration of hearing and vision

S Kleinlogel, C Vogl, M Jeschke, J Neef… - Physiological …, 2020 - journals.physiology.org
Impairments of vision and hearing are highly prevalent conditions limiting the quality of life
and presenting a major socioeconomic burden. For a long time, retinal and cochlear …

Towards the optical cochlear implant: optogenetic approaches for hearing restoration

A Dieter, D Keppeler, T Moser - EMBO molecular medicine, 2020 - embopress.org
Cochlear implants (CI s) are considered the most successful neuroprosthesis as they enable
speech comprehension in the majority of half a million CI users suffering from sensorineural …