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Single-dose genome editing therapy rescues auditory and vestibular functions in adult mice with DFNA41 deafness
Journal article   Peer reviewed

Single-dose genome editing therapy rescues auditory and vestibular functions in adult mice with DFNA41 deafness

Wei Wei, Wenliang Zhu, Stewart Silver, Ariel M Armstrong, Fletcher S Robbins, Arun Prabhu Rameshbabu, Katherina Walz, Yizhou Quan, Wan Du, Yehree Kim, …
The Journal of clinical investigation, Vol.135(20), e187872
2025-10-15
PMID: 41090360

Abstract

Animals CRISPR-Cas Systems Deafness - genetics Deafness - therapy Dependovirus - genetics Disease Models, Animal Gene Editing Genetic Therapy Humans Mice Mutation, Missense Vestibule, Labyrinth - physiopathology

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