Efficient In Utero Gene Transfer to the Mammalian Inner Ears by the Synthetic Adeno-Associated Viral Vector Anc80L65
Chin-Ju Hu, Ying-Chang Lu, Yi-Hsiu Tsai, Haw-Yuan Cheng, Hiroki Takeda, Chun-Ying Huang, Ru Xiao, Chuan‐Jen Hsu, Jin‐Wu Tsai, Luk H. Vandenberghe, Chen‐Chi Wu, Yen‐Fu Cheng
Abstract
. This embryonic delivery method achieved high transduction efficiency in both inner and outer hair cells of the cochlea. Additionally, the transduction efficiency was high in the hair cells of the vestibules and semicircular canals and in spiral ganglion neurons. Our results support the potential of Anc80L65 as a gene therapy vehicle for prenatal inner ear disorders.
Topics & Concepts
Adeno-associated virusGene transferViral vectorIn uteroVector (molecular biology)VirologyBiologyGeneGeneticsFetusPregnancyRecombinant DNAVirus-based gene therapy researchCRISPR and Genetic EngineeringRNA regulation and disease