Gene therapy restores auditory functions in an adult Vglut3 knockout mouse model

X Zhao, H Liu, H Liu, R Cai, H Wu - Human Gene Therapy, 2022 - liebertpub.com
Adeno-associated virus (AAV)-based gene therapy has been demonstrated to be extremely
effective for treating genetic hearing loss over the past several years. However, successful …

Restoration of hearing in the VGLUT3 knockout mouse using virally mediated gene therapy

O Akil, RP Seal, K Burke, C Wang, A Alemi, M During… - Neuron, 2012 - cell.com
Mice lacking the vesicular glutamate transporter-3 (VGLUT3) are congenitally deaf due to
loss of glutamate release at the inner hair cell afferent synapse. Cochlear delivery of …

AAV-mediated gene delivery to the inner ear

O Akil, L Lustig - Adeno-Associated Virus Vectors: Design and Delivery, 2019 - Springer
Cochlear gene therapy has made tremendous strides over the past 5 years. The first study
documenting successful restoration of congenital hearing loss using AAV-mediated gene …

Single and dual vector gene therapy with AAV9-PHP. B rescues hearing in Tmc1 mutant mice

J Wu, P Solanes, C Nist-Lund, S Spataro… - Molecular Therapy, 2021 - cell.com
AAV-mediated gene therapy is a promising approach for treating genetic hearing loss.
Replacement or editing of the Tmc1 gene, encoding hair cell mechanosensory ion channels …

AAV-ie-K558R mediated cochlear gene therapy and hair cell regeneration

Y Tao, X Liu, L Yang, C Chu, F Tan, Z Yu, J Ke… - … and Targeted Therapy, 2022 - nature.com
The cochlea consists of multiple types of cells, including hair cells, supporting cells and
spiral ganglion neurons, and is responsible for converting mechanical forces into electric …

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 …

AAV8BP2 and AAV8 transduce the mammalian cochlear lateral wall and endolymphatic sac with high efficiency

K Isgrig, Y Ishibashi, HJ Lee, J Zhu, M Grati… - … Therapy-Methods & …, 2022 - cell.com
Inner ear gene therapy using adeno-associated viruses (AAVs) has been successfully
applied to several mouse models of hereditary hearing loss to improve their auditory …

Recent development of AAV-based gene therapies for inner ear disorders

Y Lan, Y Tao, Y Wang, J Ke, Q Yang, X Liu, B Su, Y Wu… - Gene Therapy, 2020 - nature.com
Gene therapy for auditory diseases is gradually maturing. Recent progress in gene therapy
treatments for genetic and acquired hearing loss has demonstrated the feasibility in animal …

Surgical method for virally mediated gene delivery to the mouse inner ear through the round window membrane

O Akil, SL Rouse, DK Chan, LR Lustig - JoVE (Journal of Visualized …, 2015 - jove.com
Gene therapy, used to achieve functional recovery from sensorineural deafness, promises to
grant better understanding of the underlying molecular and genetic mechanisms that …

Approaches and vectors for efficient cochlear gene transfer in adult mouse models

Y Zhao, L Zhang, D Wang, B Chen, Y Shu - Biomolecules, 2022 - mdpi.com
Inner ear gene therapy using adeno-associated viral vectors (AAVs) in neonatal mice can
alleviate hearing loss in mouse models of deafness. However, efficient and safe transgene …