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Capsid engineered-AAV serotype 9 variant (AAV9-PHP.B) system - Introduction

GeneMedi's AAV-PHP.B Rep-Cap plasmid (specific serotype across (blood-brain barrier(BBB) AAV RC plamid), also named AAV-PHP.B RC plasmid,is part of AAV-PHP.B packaging system. AAV-PHP.B Rep-Cap plamid supplies the AAV2 Rep(replication) proteins and the AAV-PHP.B capsid protein.

You can produce AAV-PHP.B paticle in 293T cell line in high titer using GeneMedi's AAV-PHP.B Rep-Cap plasmid with GeneMedi's AAV expression vector(overexpression or shRNA) and AAV helper plasmid.

AAV-PHP.B (also called AAV9-PHP.B) is a variant from AAV9 with AAV capsid evolution (AAV capsid peptide display). With enhanced CNS tropism (neurotropic), the AAV-PHP.B variant vector has also been validated in gene delivery in vivo across the blood-brain barrier(BBB) following intravenous infusion.LY6A, a novel cellular receptor has been verified as an essential receptor for AAV-PHP.B vector.

With the blood-brain barrier(BBB) crossing propertiy, AAV-PHP.B shows potential applications in CNS (neuron) gene therapy, especially in neurodegenerative diseases such as Alzheimer's disease (AD) and Parkinson's disease (PD), or neurogenetic disease such as Spinal Muscular Atrophy (SMA) and Huntington's disease(HD).