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

2024-10-31

Abstract excerpt

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 adenine base editors (ABE...

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Identifiers and source

Literature Corpus work
11830c06-f384-5339-9f3d-a58223278158
DOI
10.1101/2024.10.29.620803
Open publication

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PAM-flexible adenine base editing rescues hearing loss in a humanized <i>MPZL2</i> mouse model harboring an East Asian founder mutationDOI 10.1101/2024.10.29.620803
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