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 …
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 …
subpopulations of neurons using acetylcholine, GABA, or serotonin as neurotransmitters. In …
Adeno-associated virus gene replacement for recessive inner ear dysfunction: Progress and challenges
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 …
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 …
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 …
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 …
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
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 …
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 …
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 …
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 …
normal gene transfer in the early developmental stage to prevent deafness. The delivery of …