Gene therapy for inherited hearing loss: updates and remaining challenges

R Hahn, KB Avraham - Audiology Research, 2023 - mdpi.com
Hearing loss stands as the most prevalent sensory deficit among humans, posing a
significant global health challenge. Projections indicate that by 2050, approximately 10% of …

Advances in cochlear gene therapies

MJ Klimara, RJH Smith - Current opinion in pediatrics, 2023 - journals.lww.com
Gene therapies for hearing loss are expected to enter clinical trials in the immediate future.
To provide referral for appropriate trials and counseling regarding benefits of genetic …

[HTML][HTML] Hair cell-specific Myo15 promoter-mediated gene therapy rescues hearing in DFNB9 mouse model

H Wang, MZ Xun, H Tang, J Zhao, S Hu… - … Therapy-Nucleic Acids, 2024 - cell.com
Adeno-associated viral (AAV) vectors are increasingly used as vehicles for gene delivery to
treat hearing loss. However, lack of specificity of the transgene expression may lead to …

[HTML][HTML] TMC function, dysfunction, and restoration in mouse vestibular organs

EM Ratzan, J Lee, MA Madison, H Zhu, W Zhou… - Frontiers in …, 2024 - frontiersin.org
Tmc1 and Tmc2 are essential pore-forming subunits of mechanosensory transduction
channels localized to the tips of stereovilli in auditory and vestibular hair cells of the inner …

Selection of appropriate non-clinical animal models to ensure translatability of novel AAV-gene therapies to the clinic

M Singh, A Brooks, P Toofan, K McLuckie - Gene Therapy, 2024 - nature.com
Abstract Gene Therapy Medicinal Products consist of a recombinant nucleic acid intended
for the modulation or manipulation of a genetic sequence. A single administration of a novel …

The menace of severe adverse events and deaths associated with viral gene therapy and its potential solution

A Kachanov, A Kostyusheva, S Brezgin… - Medicinal Research …, 2024 - Wiley Online Library
Over the past decade, in vivo gene replacement therapy has significantly advanced,
resulting in market approval of numerous therapeutics predominantly relying on adeno …

The ultrasonically treated nanoliposomes containing PCV2 DNA vaccine expressing gC1qR binding site mutant Cap is efficient in mice

Q Du, T Shi, H Wang, C Zhu, N Yang, D Tong… - Frontiers in …, 2023 - frontiersin.org
Nowadays, vaccines are broadly used to prevent porcine circovirus type 2 (PCV2) infection-
induced expenditures, but the virus is still spreading among pigs. The current PCV2 …

Gene therapy: an emerging therapy for hair cells regeneration in the cochlea

J Wang, J Zheng, H Wang, H He, S Li… - Frontiers in …, 2023 - frontiersin.org
Sensorineural hearing loss is typically caused by damage to the cochlear hair cells (HCs)
due to external stimuli or because of one's genetic factors and the inability to convert sound …

The applications of CRISPR/Cas-mediated genome editing in genetic hearing loss

J Wu, Y Tao, D Deng, Z Meng, Y Zhao - Cell & Bioscience, 2023 - Springer
Hearing loss (HL) can be caused by a number of different genetic factors. Non-syndromic HL
refers that HL occurs as an isolated symptom in an individual, whereas syndromic HL refers …

Expression of the human usherin c. 2299delG mutation leads to early-onset auditory loss and stereocilia disorganization

R Crane, L Tebbe, ML Mwoyosvi, MR Al-Ubaidi… - Communications …, 2023 - nature.com
Usher syndrome (USH) is the leading cause of combined deafness and blindness, with
USH2A being the most prevalent form. The mechanisms responsible for this debilitating …