Preclinical evaluation of the efficacy and safety of AAV1-hOTOF in mice and nonhuman primates

L Zhang, H Wang, M Xun, H Tang, J Wang, J Lv… - … Therapy-Methods & …, 2023 - cell.com
Pathogenic mutations in the OTOF gene cause autosomal recessive hearing loss (DFNB9),
one of the most common forms of auditory neuropathy. There is no biological treatment for …

Characterization of a human point mutation of VGLUT3 (p. A211V) in the rodent brain suggests a nonuniform distribution of the transporter in synaptic vesicles

L Ramet, J Zimmermann, T Bersot, O Poirel… - Journal of …, 2017 - Soc Neuroscience
The atypical vesicular glutamate transporter type 3 (VGLUT3) is expressed by
subpopulations of neurons using acetylcholine, GABA, or serotonin as neurotransmitters. In …

Adeno-associated virus gene replacement for recessive inner ear dysfunction: Progress and challenges

C Askew, WW Chien - Hearing research, 2020 - Elsevier
Approximately 3 in 1000 children in the US under 4 years of age are affected by hearing
loss. Currently, cochlear implants represent the only line of treatment for patients with severe …

AAV-based gene replacement: The promise of gene therapy for deafness

J Qi, L Zhang, R Chai - Molecular Therapy-Nucleic Acids, 2024 - cell.com
A recent study published in Molecular Therapy Nucleic Acids has demonstrated the efficacy
of a tissue-specific gene therapy in a model of autosomal recessive hearing loss 9 …

VGLUTs:'exciting'times for glutamatergic research?

S Takamori - Neuroscience research, 2006 - Elsevier
Glutamate is the principal excitatory neurotransmitter in the mammalian central nervous
system (CNS). Glutamate is first synthesized in the cytoplasm of presynaptic terminals before …

Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8

W Du, V Ergin, C Loeb, M Huang, S Silver… - Molecular Therapy, 2023 - cell.com
Patients with mutations in the TMPRSS3 gene suffer from recessive deafness
DFNB8/DFNB10. For these patients, cochlear implantation is the only treatment option. Poor …

Advances and challenges in adeno-associated viral inner-ear gene therapy for sensorineural hearing loss

K Bankoti, C Generotti, T Hwa, L Wang… - … Therapy-Methods & …, 2021 - cell.com
There is growing attention and effort focused on treating the root cause of sensorineural
hearing loss rather than managing associated secondary characteristic features. With recent …

Gene transfer into guinea pig cochlea using adeno‐associated virus vectors

M Konishi, K Kawamoto, M Izumikawa… - The Journal of Gene …, 2008 - Wiley Online Library
Background Several genes are candidates for treating inner ear diseases. For clinical
applications, minimally invasive approaches to the inner ear are desirable along with …

Vesicular glutamatergic transmission in noise-induced loss and repair of cochlear ribbon synapses

KX Kim, S Payne, A Yang-Hood, SZ Li… - Journal of …, 2019 - Soc Neuroscience
Noise-induced excitotoxicity is thought to depend on glutamate. However, the excitotoxic
mechanisms are unknown, and the necessity of glutamate for synapse loss or regeneration …

Noninvasive In Vivo Delivery of Transgene via Adeno-Associated Virus into Supporting Cells of the Neonatal Mouse Cochlea

T Iizuka, S Kanzaki, H Mochizuki, A Inoshita… - Human gene …, 2008 - liebertpub.com
There are a number of genetic diseases that affect the cochlea early in life, which require
normal gene transfer in the early developmental stage to prevent deafness. The delivery of …