A single-arm clinical trial in China demonstrated that adeno-associated virus (AAV)-mediated OTOF gene therapy improved hearing in both children and young adults with autosomal recessive deafness 9. Utilizing the synthetic Anc80L65 capsid and inner ear delivery via cochlear round window injection, patients exhibited rapid hearing improvement within one month, sustained at six months post-treatment. This progress marks a significant advance for gene therapies in inherited hearing loss affecting a broader age range, published in Nature Medicine with expert commendation for its transformative potential.