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 …

AAV‐mediated gene therapy for hereditary deafness: progress and perspectives

L Zhang, F Tan, J Qi, Y Lu, X Wang, X Yang… - Advanced …, 2024 - Wiley Online Library
Hereditary deafness is the most prevalent sensory deficit disorder, with over 100 identified
deafness‐related genes. Clinical treatment options are currently limited to external devices …

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 …

Rescue of hearing by adenine base editing in a humanized mouse model of Usher syndrome type 1F

CW Peters, KS Hanlon, MV Ivanchenko, E Zinn… - Molecular Therapy, 2023 - cell.com
Usher syndrome type 1F (USH1F), characterized by congenital lack of hearing and balance
and progressive loss of vision, is caused by mutations in the PCDH15 gene. In the …

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 …

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 …

Optimization strategies and advances in the research and development of AAV‐based gene therapy to deliver large transgenes

VV Kolesnik, RF Nurtdinov… - Clinical and …, 2024 - Wiley Online Library
Abstract Adeno‐associated virus (AAV)‐based therapies are recognized as one of the most
potent next‐generation treatments for inherited and genetic diseases. However, several …

Defects in hair cells disrupt the development of auditory peripheral circuitry

RT Bottom, Y Xu, C Siebald, J Jung, U Müller - Nature Communications, 2024 - nature.com
Deafness is the most common form of sensory impairment in humans and frequently caused
by defects in hair cells of the inner ear. Here we demonstrate that in male mice which model …

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 …

Finding a window for gene therapy for hereditary deafness

MV Ivanchenko, DP Corey - Proceedings of the National …, 2023 - National Acad Sciences
About one in 500 children is born with significant hearing impairment, and at least half of the
cases have genetic causes (1). Two-or threefold more children have normal hearing at birth …