Therapeutic AAV gene transfer to the nervous system: a clinical reality

E Hudry, LH Vandenberghe - Neuron, 2019 - cell.com
Gene transfer has long been a compelling yet elusive therapeutic modality. First mainly
considered for rare inherited disorders, gene therapy may open treatment opportunities for …

Neurocognitive factors in sensory restoration of early deafness: a connectome model

A Kral, WG Kronenberger, DB Pisoni… - The Lancet …, 2016 - thelancet.com
Progress in biomedical technology (cochlear, vestibular, and retinal implants) has led to
remarkable success in neurosensory restoration, particularly in the auditory system …

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 …

PRDX1 activates autophagy via the PTEN-AKT signaling pathway to protect against cisplatin-induced spiral ganglion neuron damage

W Liu, L Xu, X Wang, D Zhang, G Sun, M Wang… - Autophagy, 2021 - Taylor & Francis
Spiral ganglion neurons (SGNs) are auditory neurons that relay sound signals from the inner
ear to the brainstem. The ototoxic drug cisplatin can damage SGNs and thus lead to …

Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo

Y Tao, V Lamas, W Du, W Zhu, Y Li… - Nature …, 2023 - nature.com
Mutations in Atp2b2, an outer hair cell gene, cause dominant hearing loss in humans. Using
a mouse model Atp2b2 Obl/+, with a dominant hearing loss mutation (Oblivion), we show …

A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear

LD Landegger, B Pan, C Askew, SJ Wassmer… - Nature …, 2017 - nature.com
Efforts to develop gene therapies for hearing loss have been hampered by the lack of safe,
efficient, and clinically relevant delivery modalities,. Here we demonstrate the safety and …

Generation of inner ear organoids containing functional hair cells from human pluripotent stem cells

KR Koehler, J Nie, E Longworth-Mills, XP Liu… - Nature …, 2017 - nature.com
The derivation of human inner ear tissue from pluripotent stem cells would enable in vitro
screening of drug candidates for the treatment of hearing and balance dysfunction and may …

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 …

Gene expression by mouse inner ear hair cells during development

DI Scheffer, J Shen, DP Corey… - Journal of …, 2015 - Soc Neuroscience
Hair cells of the inner ear are essential for hearing and balance. As a consequence,
pathogenic variants in genes specifically expressed in hair cells often cause hereditary …

Targeting dysregulation of redox homeostasis in noise-induced hearing loss: oxidative stress and ROS signaling

AR Fetoni, F Paciello, R Rolesi, G Paludetti… - Free Radical Biology and …, 2019 - Elsevier
Hearing loss caused by exposure to recreational and occupational noise remains a
worldwide disabling condition and dysregulation of redox homeostasis is the hallmark of …