ArticleNature communications2025
PAM-flexible adenine base editing rescues hearing loss in a humanized MPZL2 mouse model harboring an East Asian founder mutation.
Article in Nature communications, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.
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Who cites it
8 citing papers in PubMed.
- Functionalized Biomimetic Scaffolds for Human-Derived Auditory Neural Circuit Construction.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Gene Editing Technologies for Hereditary Hearing Loss: Prospects and Challenges.Neuroscience bulletin · 2026Review
- Genomic and Epigenomic Advances in Hearing Loss: Molecular Mechanisms, Diagnostics, and Emerging Therapies.Journal of personalized medicine · 2026Review
- Long-term restoration of auditory function in a DFNA2 mouse model by adenine base editing.EMBO molecular medicine · 2026Article
- Empirical optimization of dual-sgRNA design for in vivo CRISPR/Cas9-mediated exon deletion in mice.Computational and structural biotechnology journal · 2026Article
- CRISPR-based functional genomics tools in vertebrate models.Experimental & molecular medicine · 2025Review
- Advancing precision ear medicine: leveraging animal models for disease insights and therapeutic innovations.Mammalian genome : official journal of the International Mammalian Genome Society · 2025Review
- Autophagy induction enhances homologous recombination-associated CRISPR-Cas9 gene editing.Nucleic acids research · 2025Article
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Authors and funding
30 authors.
Funding
Abstract
Hearing loss is one of the most prevalent sensory disorders, but no commercial biological treatments are currently available. Here, we identify an East Asia-specific founder mutation, the homozygous c.220 C > T mutation in MPZL2, that contributes to a significant proportion of hereditary deafness cases in our cohort study. We find that the disease-causing mutation can be targetable by adenine base editors (ABEs) that enable A·T-to-G·C base corrections without DNA double-strand breaks. To demonstrate this, we develop a humanized mouse model (hMPZL2
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Registered trials
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