Article

PAM-flexible adenine base editing rescues hearing loss in a humanized MPZL2 mouse model harboring an East Asian founder mutation

2024-11-25

Abstract excerpt

<title>Abstract</title> <p>Hearing loss is one of the most prevalent sensory disorders, but no commercial biological treatments are currently available. Here, we identified an East Asia-specific founder mutation, the homozygous c.220C>T mutation in MPZL2, that contributes to a significant proportion of hereditary deafness cases in our cohort study. We found that the disease-causing mutation could be targetable by...

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Literature Corpus work: 4e9517fd-865d-5657-9357-2bf943379803
DOI: 10.21203/rs.3.rs-5353095/v1

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