Key result
AAV2/1 was the optimal vector for in utero cochlear gene transfer, efficiently transducing progenitors without adverse effects on cell differentiation or auditory function.
Why the study?
Does in utero delivery of recombinant viruses efficiently and safely transduce cochlear progenitor cells in fetal mice?
Does in utero delivery of recombinant viruses efficiently and safely transduce cochlear progenitor cells in fetal mice?
AAV2/1 is an optimal and safe vector for in utero cochlear gene transfer in mice, showing promise for developing gene-based therapies for congenital hearing loss.
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Does not yet support clinical use of in utero cochlear gene therapy; extends vector optimization data in fetal mouse models.
Bedrosian et al. (2006) studied Congenital hearing deficits. In utero delivery of recombinant viruses (AAV2/1, AAV2/8, and lentivirus) was evaluated on Efficiency and cellular specificity of transgene expression, stability, and safety. AAV2/1 was the optimal vector for in utero cochlear gene transfer, efficiently transducing progenitors without adverse effects on cell differentiation or auditory function.
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