Recent Therapeutic Progress and Future Perspectives for the Treatment of Hearing Loss

J Lye, DS Delaney, FK Leith, VS Sardesai… - Biomedicines, 2023 - mdpi.com
Up to 1.5 billion people worldwide suffer from various forms of hearing loss, with an
additional 1.1 billion people at risk from various insults such as increased consumption of …

The opportunities and challenges of gene therapy for treatment of inherited forms of vision and hearing loss

EJ Simons, I Trapani - Human Gene Therapy, 2023 - liebertpub.com
Inherited forms of blindness and deafness are highly prevalent and severe conditions that
significantly impact the lives of millions of people worldwide. The lack of therapeutic options …

PCDH15 dual-AAV gene therapy for deafness and blindness in Usher syndrome type 1F models

MV Ivanchenko, DM Hathaway, EM Mulhall… - The Journal of clinical …, 2024 - jci.org
Usher syndrome type 1F (USH1F), resulting from mutations in the protocadherin-15
(PCDH15) gene, is characterized by congenital lack of hearing and balance, and …

A base editor for the long-term restoration of auditory function in mice with recessive profound deafness

C Cui, S Wang, D Wang, J Zhao, B Huang… - Nature Biomedical …, 2024 - nature.com
A prevalent recessive mutation (c. 2485C> T, p. Q829X) within the OTOF gene leads to
profound prelingual hearing loss. Here we show that in Otof mice harbouring a mutation (c …

[HTML][HTML] Biotechnological applications of purine and pyrimidine deaminases

J Del Arco, J Acosta, J Fernández-Lucas - Biotechnology Advances, 2024 - Elsevier
Deaminases, ubiquitous enzymes found in all living organisms from bacteria to humans,
serve diverse and crucial functions. Notably, purine and pyrimidine deaminases, while …

PCDH15 dual-AAV gene therapy for deafness and blindness in usher syndrome type 1F

MV Ivanchenko, DM Hathaway, EM Mulhall, KT Booth… - bioRxiv, 2023 - biorxiv.org
Usher syndrome type 1F (USH1F), resulting from mutations in the protocadherin-15
(PCDH15) gene, is characterized by congenital lack of hearing and balance, and …

Extracellular vesicles for developing targeted hearing loss therapy

X Pan, Y Li, P Huang, H Staecker, M He - Journal of Controlled Release, 2024 - Elsevier
Substantial efforts have been made for local administration of small molecules or biologics
in treating hearing loss diseases caused by either trauma, genetic mutations, or drug …

CRISPR-based editing strategies to rectify EYA1 complex genomic rearrangement linked to haploinsufficiency

H Yi, Y Yun, WH Choi, HY Hwang, JH Cha… - … Therapy-Nucleic Acids, 2024 - cell.com
Pathogenic structure variations (SVs) are associated with various types of cancer and rare
genetic diseases. Recent studies have used Cas9 nuclease with paired guide RNAs …

Canonical MAPK signaling in auditory neuropathy

Y Wang, L Huang, X Cen, Y Liang, K Chen - Biochimica et Biophysica Acta …, 2024 - Elsevier
Auditory neuropathy (AN) is an under-recognized form of hearing loss characterized by
lesions in inner hair cells (IHCs), ribbon synapses and spiral ganglion neurons (SGNs). The …

Base editing correction of OCRL in Lowe syndrome: ABE-mediated functional rescue in patient-derived fibroblasts

S Chen, CH Lo, Z Liu, Q Wang, K Ning… - Human Molecular …, 2024 - academic.oup.com
Lowe syndrome, a rare X-linked multisystem disorder presenting with major abnormalities in
the eyes, kidneys, and central nervous system, is caused by mutations in OCRL gene …