AAV gene therapy for autosomal recessive deafness 9: a single-arm trial

2025/07/02 by Jieyu Qi, Liyan Zhang, Ling Lu +20 · 1 voice · 11 citations
Neuroscience · Biochemistry, Genetics and Molecular Biology · #Hearing, Cochlea, Tinnitus, Genetics #RNA regulation and disease #Virus-based gene therapy research

paper · doi:10.1038/s41591-025-03773-w

Cited by

• CochleaNet: deep learning-based image analysis for cochlear connectomics and gene therapy
• OTOF Gene Therapy: From Breakthroughs to Roadmaps
• Dual AAV gene therapy achieves recovery of hearing and auditory processing in a DFNB16 mouse model. [europepmc]
• Compensatory Interplay Between Clarin-1 and Clarin-2 Deafness-Associated Proteins Governs Phenotypic Variability in Hearing. [europepmc]
• OTOF Gene Therapy: From Breakthroughs to Roadmaps. [europepmc]
• The landscape of gene mutations in a cohort of 3353 Han Chinese children with nonsyndromic hearing loss. [europepmc]
• Pvalb8, a Type of Oncomodulin, Regulates Neuromast Development and Auditory Function in Zebrafish. [europepmc]
• Human promoter-driven AAV tools enable precision gene therapy targeting cochlear hair cells. [europepmc]
• Long-term restoration of auditory function in a DFNA2 mouse model by adenine base editing. [europepmc]
• Genomic and Epigenomic Advances in Hearing Loss: Molecular Mechanisms, Diagnostics, and Emerging Therapies. [europepmc]
• Generation of marmoset monkeys with a non-mosaic disruption of the OTOF gene as a model of human deafness. [europepmc]

Discussions

• Deafness reversed: Single injection brings hearing back within weeks | ScienceDaily [lemmy, 115 points, 3 comments]