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Man Wang1,2, Ziyu Zhang1, Xiaohan Wang1

  • 1Department of Otolaryngology Head and Neck Surgery, Zhongda Hospital, State Key Laboratory of Digital Medical Engineering, Jiangsu Provincial Key Laboratory of Critical Care Medicine, School of Life Sciences and Technology, School of Medicine, Advanced Institute for Life and Health, Southeast University, Nanjing, China.

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Gene editing successfully restored hearing in a mouse model of hereditary deafness (DFNA15) by precisely correcting mutations in the POU4F3 gene. This adenine base editor therapy offers a promising new treatment for genetic hearing loss.

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Area of Science:

  • Genetics
  • Molecular Biology
  • Otolaryngology

Background:

  • Hereditary deafness, including autosomal dominant non-syndromic deafness 15 (DFNA15), is caused by genetic mutations, notably in the POU4F3 gene.
  • Currently, no effective clinical treatments exist for DFNA15, highlighting a significant unmet medical need.

Purpose of the Study:

  • To develop and evaluate a gene editing strategy for treating DFNA15 by targeting the mutated POU4F3 allele.
  • To establish a Pou4f3 mutant mouse model that accurately mimics human DFNA15.

Main Methods:

  • Development and screening of adenine base editors (ABEs) fused to Cas9, specifically targeting the Pou4f3 Q113* allele.
  • In vitro testing of ABE efficiency and specificity, followed by in vivo delivery using adeno-associated virus (AAV) in neonatal Pou4f3 mutant mice.
  • Assessment of hearing recovery and long-term efficacy through auditory functional tests and biosafety analyses.

Main Results:

  • A specific adenine base editor, SchABE8e, demonstrated high precision and efficiency (up to 48.5%) in targeting the mutated Pou4f3 allele in vitro.
  • In vivo treatment of neonatal mice with AAV-delivered SchABE8e-sgRNA3 resulted in near-complete hearing recovery.
  • The therapeutic effect of the gene editing persisted for at least four months, with supportive biosafety data.

Conclusions:

  • Adenine base editing represents a feasible and effective therapeutic strategy for DFNA15.
  • This approach holds significant promise for treating hereditary hearing loss caused by specific genetic mutations.