AAV-PHP.S vector system (AAV-PHP.S expression and packaging plasmid system, across the blood-brain barrier(BBB) )

GeneMedi’s AAV-PHP.S Vector System (AAV serotype 2 variant(Y444F) helper-free packaging plasmids system) is including AAV-PHP.S Rep-Cap plamid (AAV-PHP.S-RC plasmid), AAV helper plasmid and AAV expression vectors (overexpression or shRNA).

You can choose your suiltable AAV expression vector plasmids from GeneMedi’s AAV expression vector core.The AAV expression vector plasmids were constructed with differernt expression cassettes, containing kinds of verified protomters and reporters including GFP, zsgreen, RFP, mcherry and luciferase. The GeneMedi’s AAV expression vectors have been proved very suitalble for unique gene overexpression or shRNA-mediated knock-down (also called RNAi (RNA interference ). You can also achieve gene knock-out(KO) or gene editing using our Crispr-cas9-gRNA AAV expression vector.

AAV-PHP.S Rep-Cap plamid supplies the AAV2 Rep(replication) proteins and the AAV-PHP.Scapsid-engineered protein.

AAV-PHP.S (also called AAV9-PHP.S) is a variant from AAV9 with AAV capsid evolution (AAV capsid peptide display). With enhanced CNS tropism (neurotropic),the AAV-PHP.S 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.S vector.

With the blood-brain barrier(BBB) crossing propertiy, AAV-PHP.S 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).ne