VIRAL VECTOR CORE: Summary/Abstract This Viral Vector Core has been in operation for nearly 12 years, and its main role has been to support the Wellstone Center in Seattle as a Scientific Research Resource Core. The Core also offers support to non-Center members on a recharge basis. The Core provides high quality preparations of viral and plasmid vectors to facilitate research related to the muscular dystrophies. Services offered by the Core include providing consulting, reagents and training related to vector production. This includes production of adeno-associated and lentiviral vectors and muscular dystrophy related reagents (cell lines, vector backbones, protocols, antibodies etc) for members of this Center as well as laboratories in the US and across the World to support their efforts related to gene therapy for the muscular dystrophies. The Principle Investigator of the Vector Core is now Dr. Joel Chamberlain, who has worked closely with Core personnel on AAV and retroviral vectors for more than 10 years. The manager of the Core is Dr. James Allen. Dr. Allen, in conjunction with the Chamberlain, Russel, Odom and Hauschka labs has established protocols for the production of adeno-associated viral vectors, particularly AAV6 and 9, but also several other pseudotyped vectors including AAV1, 5, 7,8 and 8i, and VSVG-lentiviral vectors. For many years such vectors and associated reagents have been supplied by us to laboratories around the world as part of collaborations, and for the past 3.5 years (and a previous 5 year period) these reagents have been provided via the Wellstone Center Vector Core lab. We also have numerous dystrophin and utrophin cDNAs and antibodies that will be made available via this Core lab, as well as DM1, DUX4 and associated RNAi vectors. We will provide high quality, high titer preparations of adeno-associated viral (AAV) vectors based primarily on AAV serotypes 6 & 9, but also for AAV 1, 2, 8 and emerging new and modified serotypes. These recombinant AAV (rAAV) vectors will be provided to members of the Wellstone Center using funds from this Core, and to other muscular dystrophy researchers on a recharge basis to cover costs. We also have numerous muscle disease related cDNAs and antibodies that will be made available via this Core lab for all members of our Center. Training will allow investigators to apply methods for production and purification of viral vectors (AAV and lentiviral) in their own labs as desired, and will be advertised via the Center Web site. Consulting will be available to guide the use of the AAV and lentiviral vectors. Finally, we will provide reagents (such as vector backbones, plasmids, antibodies and protocols) that have been developed by members of this Wellstone Center to outside labs.
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