Delivery of therapeutics to the inner ear: The challenge of the blood-labyrinth barrier

S Nyberg, NJ Abbott, X Shi, PS Steyger… - Science translational …, 2019 - science.org
Permanent hearing loss affects more than 5% of the world's population, yet there are no
nondevice therapies that can protect or restore hearing. Delivery of therapeutics to the …

Inner ear gene therapies take off: current promises and future challenges

S Delmaghani, A El-Amraoui - Journal of clinical medicine, 2020 - mdpi.com
Hearing impairment is the most frequent sensory deficit in humans of all age groups, from
children (1/500) to the elderly (more than 50% of the over-75 s). Over 50% of congenital …

[PDF][PDF] 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 …

Gene therapy for hearing loss

R Omichi, SB Shibata, CC Morton… - Human molecular …, 2019 - academic.oup.com
Sensorineural hearing loss (SNHL) is the most common sensory disorder. Its underlying
etiologies include a broad spectrum of genetic and environmental factors that can lead to …

Choice of vector and surgical approach enables efficient cochlear gene transfer in nonhuman primate

E Andres-Mateos, LD Landegger, C Unzu… - Nature …, 2022 - nature.com
Inner ear gene therapy using adeno-associated viral vectors (AAV) promises to alleviate
hearing and balance disorders. We previously established the benefits of Anc80L65 in …

Emerging gene therapies for genetic hearing loss

H Ahmed, O Shubina-Oleinik, JR Holt - … of the Association for Research in …, 2017 - Springer
Gene therapy, or the treatment of human disease using genetic material, for inner ear
dysfunction is coming of age. Recent progress in developing gene therapy treatments for …

Characterization of the transcriptome of nascent hair cells and identification of direct targets of the Atoh1 transcription factor

T Cai, HI Jen, H Kang, TJ Klisch, HY Zoghbi… - Journal of …, 2015 - Soc Neuroscience
Hair cells are sensory receptors for the auditory and vestibular system in vertebrates. The
transcription factor Atoh1 is both necessary and sufficient for the differentiation of hair cells …

Disease mechanisms and gene therapy for Usher syndrome

GGS Géléoc, A El-Amraoui - Hearing research, 2020 - Elsevier
Usher syndrome (USH) is a major cause of deaf-blindness in humans, affecting∼ 400 000
patients worldwide. Three clinical subtypes, USH1-3, have been defined, with 10 USH …

[HTML][HTML] Gene therapy for human sensorineural hearing loss

Y Ren, LD Landegger, KM Stankovic - Frontiers in Cellular …, 2019 - frontiersin.org
Hearing loss is the most common sensory impairment in humans and currently disables 466
million people across the world. Congenital deafness affects at least 1 in 500 newborns, and …

Delivery of adeno-associated virus vectors in adult mammalian inner-ear cell subtypes without auditory dysfunction

Y Tao, M Huang, Y Shu, A Ruprecht, H Wang… - Human gene …, 2018 - liebertpub.com
Hearing loss, including genetic hearing loss, is one of the most common forms of sensory
deficits in humans with limited options of treatment. Adeno-associated virus (AAV)-mediated …