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Discovery of natural adeno-associated virus variants and their characterization as potential gene therapy vectors

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Recombinant adeno-associated viruses (rAAVs) are the ideal vectors for human gene therapy because of their excellent safety profiles and therapeutic transgene delivery efficacies. Efforts to discover novel AAVs with improved transduction efficiencies and reduced host immunities continue to be major focuses for the gene therapy field.  Using single molecule, real-time (SMRT) sequencing of proviral capsid libraries isolated from >800 human surgical specimens, we discovered a collection of novel clade B, C, and E variants.  One of our lab’s current research pursuits is to test these variants in mice and cynomolgus macaques under various routes of administration to target a range of tissue and cell types of interest.

A variant we have named AAVv66, was recently identified as the most abundant pro-viral capsid variant in a clinical pancreatic neoplasm sample that was observed to harbor a high diversity of capsid sequences when profiled by high-throughput sequencing approaches. The AAVv66 capsid harbors 13 residues that are unique from AAV2, most of which are within defined variable regions of VP3. Aside from exhibiting favorable tropism in the CNS following intrahippocampal injections, we found that AAVv66 displays better packaging efficiencies than prototypical AAV2; thus demonstrating another important facet as a promising gene therapy capsid.



Prof. Philip Tai
University of Massachusetts Medical School

Journey Description


E - Main Entrance/Reception
1 - Managing Board; sections: Infection Biology Unit, Unit of Infection Models, Laboratory Animal Science Unit, Primate Genetics Laboratory, Behavioral Ecology and Sociobiology Unit, Cognitive Ethology Laboratory, Neurobiology Laboratory, Research Coordination, Public Relations, Information Technology, Administration, Technical Support, Library.
2 - Material Delivery/Purchase
3 - Platform Degenerative Diseases
4 - Cognitive Neuroscience Laboratory
5 - Animal Husbandry
6 - Imaging Center, Functional Imaging Laboratory

Arrival by car

Leave the interstate A7 at exit "Göttingen Nord". Follow the B27 straight ahead in the direction of "Braunlage". After the third traffic light intersection turn right towards "Kliniken". Afterwards turn left onto "Robert-Koch-Straße" direction "Universität Nordbereich/Polizei". At the end of "Robert-Koch-Straße" turn right onto "Otto-Hahn-Straße", direction "Nikolausberg". The first street on the left turn onto "Kellnerweg", follow the signs "Deutsches Primatenzentrum".

Arrival by bus

The footpath from the bus stop "Kellnerweg" to the Main Entrance/Reception: 
From Bus stop "Kellnerweg" (line 21/22 and 23) Cross the road, go in the direction of the bus. At the mailbox, turn left into the footpath and proceed to the end. Turn right into the Kellnerweg. The main entrance of the DPZ is on the left side.

Date and Time 29.07.21 - 15:00 - 17:00 Signup is not required

Location online-Veranstaltung


Deutsches Primatenzentrum GmbH

Leibniz-Institut für Primatenforschung

Kellnerweg 4

37077 Göttingen

Contact Shabnam Ghazanfarisarabi
Plattform Degenerative Erkrankungen
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