[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 …
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 …
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
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 …
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 …
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 …
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 …
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 …
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 …
for genetic inner ear disorders, we designed gene replacement therapies using synthetic …
Emerging approaches for restoration of hearing and vision
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 …
and presenting a major socioeconomic burden. For a long time, retinal and cochlear …
Towards the optical cochlear implant: optogenetic approaches for hearing restoration
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 …
speech comprehension in the majority of half a million CI users suffering from sensorineural …