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AAV gene therapy for autosomal recessive deafness 9: a single-arm trial
Abstract Gene therapy for congenital deafness has shown promising results in children but lacks data in older populations. We conducted a single-arm trial of adeno-associated virus (AAV)-OTOF gene therapy using the Anc80L65 capsid in ten participants with autosomal recessive deafness 9 aged 1.5 to 23.9 years at five sites in China. The primary endpoints were safety and tolerability within 5 years, and secondary endpoints assessed auditory function.
Correction to “AAV‐Mediated Gene Cocktails Enhance Supporting Cell Reprogramming and Hair Cell Regeneration”
Adv. Sci. 2024, 11, e2304551. http://doi.org/10.1002/advs.202304551 Description of the errors: In the original published paper, we found that the “Apex” image of the AAV-Atoh1 group in Figure 5A was improperly used. The corrected figures are shown below. Corrected Figure 5A: This correction does not affect the overall findings and conclusions of this paper. We apologize for this error.
AAV‐mediated Gene Cocktails Enhance Supporting Cell Reprogramming and Hair Cell Regeneration
1 Introduction Auditory sensory hair cells (HCs) and surrounding nonsensory supporting cells (SCs) differentiate from the common pool of inner ear progenitor cells.
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