[HTML][HTML] AAV2. 7m8 is a powerful viral vector for inner ear gene therapy

K Isgrig, DS McDougald, J Zhu, HJ Wang… - Nature …, 2019 - nature.com
Adeno-associated virus (AAV) has been successfully used to deliver gene therapy to
improve auditory function in mouse models of hereditary hearing loss. Many forms of …

[HTML][HTML] Precise detection of CRISPR-Cas9 editing in hair cells in the treatment of autosomal dominant hearing loss

C Cui, D Wang, B Huang, F Wang, Y Chen, J Lv… - … Therapy-Nucleic Acids, 2022 - cell.com
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 …

Gene therapy for hearing loss

R Omichi, SB Shibata, CC Morton… - Human molecular …, 2019 - academic.oup.com
Sensorineural hearing loss (SNHL) is the most common sensory disorder. Its underlying
etiologies include a broad spectrum of genetic and environmental factors that can lead to …

Emerging gene therapies for genetic hearing loss

H Ahmed, O Shubina-Oleinik, JR Holt - … of the Association for Research in …, 2017 - Springer
Gene therapy, or the treatment of human disease using genetic material, for inner ear
dysfunction is coming of age. Recent progress in developing gene therapy treatments for …

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 …

Helios is a key transcriptional regulator of outer hair cell maturation

L Chessum, MS Matern, MC Kelly, SL Johnson… - Nature, 2018 - nature.com
The sensory cells that are responsible for hearing include the cochlear inner hair cells
(IHCs) and outer hair cells (OHCs), with the OHCs being necessary for sound sensitivity and …

Toward cochlear therapies

J Wang, JL Puel - Physiological reviews, 2018 - journals.physiology.org
Sensorineural hearing impairment is the most common sensory disorder and a major health
and socio-economic issue in industrialized countries. It is primarily due to the degeneration …

[HTML][HTML] Gene therapy for regenerative medicine

H Hosseinkhani, AJ Domb, G Sharifzadeh, V Nahum - Pharmaceutics, 2023 - mdpi.com
The development of biological methods over the past decade has stimulated great interest in
the possibility to regenerate human tissues. Advances in stem cell research, gene therapy …

Disease mechanisms and gene therapy for Usher syndrome

GGS Géléoc, A El-Amraoui - Hearing research, 2020 - Elsevier
Usher syndrome (USH) is a major cause of deaf-blindness in humans, affecting∼ 400 000
patients worldwide. Three clinical subtypes, USH1-3, have been defined, with 10 USH …

Local gene therapy durably restores vestibular function in a mouse model of Usher syndrome type 1G

A Emptoz, V Michel, A Lelli, O Akil… - Proceedings of the …, 2017 - National Acad Sciences
Our understanding of the mechanisms underlying inherited forms of inner ear deficits has
considerably improved during the past 20 y, but we are still far from curative treatments. We …