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PCR Cloning and Subcloning> | … The open reading frame of isoform 2 of Clrn2 was synthesized by GenScript (Piscataway, NJ, USA) and cloned into a ss AAV-expression (AAV-CBA-W) plasmid into the multiple cloning site to generate AAV-CBA-Clrn1-W. We also had HA-tagged Clrn1 constructs synthesized … | Get A Quote |
Hereditary hearing loss often results from mutation of genes expressed by cochlear hair cells. Gene addition using AAV vectors has shown some efficacy in mouse models, but clinical application requires two additional advances. First, new AAV capsids must mediate efficient transgene expression in both inner and outer hair cells of the cochlea. Second, to have the best chance of clinical translation, these new vectors must also transduce hair cells in non-human primates. Here, we show that an AAV9 capsid variant, PHP.B, produces efficient transgene expression of a GFP reporter in both inner and outer hair cells of neonatal mice. We show also that AAV9-PHP.B mediates almost complete transduction of i... More