AAV‐Mediated Gene Therapy Restores Hearing in Patients with DFNB9 Deafness
J Qi, F Tan, L Zhang, L Lu, S Zhang, Y Zhai… - Advanced …, 2024 - Wiley Online Library
Mutations in OTOFERLIN (OTOF) lead to the autosomal recessive deafness 9 (DFNB9). The
efficacy of adeno‐associated virus (AAV)‐mediated OTOF gene replacement therapy is …
efficacy of adeno‐associated virus (AAV)‐mediated OTOF gene replacement therapy is …
Rodent models in sensorineural hearing loss research: A comprehensive review
W Li, B Xu, Y Huang, X Wang, D Yu - Life Sciences, 2024 - Elsevier
Sensorineural hearing loss (SNHL) constitutes a major global health challenge, affecting
millions of individuals and substantially impairing social integration and quality of life. The …
millions of individuals and substantially impairing social integration and quality of life. The …
Ototoxicity: a high risk to auditory function that needs to be monitored in drug development
MP Pasdelou, L Byelyayeva, S Malmström… - Frontiers in Molecular …, 2024 - frontiersin.org
Hearing loss constitutes a major global health concern impacting approximately 1.5 billion
people worldwide. Its incidence is undergoing a substantial surge with some projecting that …
people worldwide. Its incidence is undergoing a substantial surge with some projecting that …
AAV-regulated Serpine2 overexpression promotes hair cell regeneration
Q Sun, F Tan, X Wang, X Gu, X Chen, Y Lu, N Li… - … Therapy Nucleic Acids, 2024 - cell.com
Inner ear hair cell (HC) damage is irreversible in mammals, but it has been shown that
supporting cells (SCs) have the potential to differentiate into HCs. Serpine2, a serine …
supporting cells (SCs) have the potential to differentiate into HCs. Serpine2, a serine …
Gene therapy for hereditary deafness
Gene therapy for hereditary deafness | Nature Medicine Skip to main content Thank you for
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[HTML][HTML] Heterogeneous group of genetically determined auditory neuropathy spectrum disorders
AA Buianova, MV Bazanova, VA Belova… - International Journal of …, 2024 - mdpi.com
Auditory neuropathy spectrum disorder (ANSD) is often missed by standard hearing tests,
accounting for up to 10% of hearing impairments (HI) and commonly linked to variants in 23 …
accounting for up to 10% of hearing impairments (HI) and commonly linked to variants in 23 …
DNA Sequencing in Newborn Screening: Opportunities, Challenges, and Future Directions
Background Newborn screening is a public health system designed to identify infants at risk
for conditions early in life to facilitate timely intervention and treatment to prevent or mitigate …
for conditions early in life to facilitate timely intervention and treatment to prevent or mitigate …
[HTML][HTML] Auditory Neuropathy Caused by a Structural Variation in the OTOF Gene, Identified Using Oxford Nanopore Adaptive Sampling
Background/Objectives: The OTOF gene is reported to be the causative gene for non-
syndromic recessive sensorineural hearing loss and auditory neuropathy spectrum disorder …
syndromic recessive sensorineural hearing loss and auditory neuropathy spectrum disorder …
Hearing loss secondary to variants in the OTOF gene
C Morales-Angulo, J Gallo-Terán… - International Journal of …, 2024 - Elsevier
Objective Genetic variants in the OTOF gene are responsible for non-syndromic hearing loss
with an autosomal recessive inheritance pattern. The objective of our work was to evaluate …
with an autosomal recessive inheritance pattern. The objective of our work was to evaluate …
Otoferlin gene therapy restores hearing in deaf children
JV Brigande - Molecular Therapy, 2024 - cell.com
The atmosphere prior to the start of the Late-Breaking Presidential Symposium on Otoferlin
Gene Therapy Clinical Trials at the Association for Research in Otolaryngology MidWinter …
Gene Therapy Clinical Trials at the Association for Research in Otolaryngology MidWinter …