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 …
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 …
remarkable success in neurosensory restoration, particularly in the auditory system …
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 …
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 …
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
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 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 …
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 …
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 …
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 …
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 …
worldwide disabling condition and dysregulation of redox homeostasis is the hallmark of …