Core B: Vector Core. This Vector Core will provide high quality preparations of viral and plasmid vectors to facilitate research involving the development of therapeutic strategies in both small and large animal models. Services offered by the Core include providing consulting, reagents and training related to vector production. This includes production of 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 Vector Core will be directed by Dr. Jeff Chamberlain with assistance from Dr. James Allen. Dr. Allen, in conjunction with the Chamberlain lab has established protocols for the production of adeno-associated viral vectors, particularly AAV6 but also several other pseudotyped vectors including AAVS and 9, and 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 a recent 5 year period these reagents were provided via our Wellstone Center Vector Core lab. We also have numerous dystrophin and utrophin cDNAs and antibodies that will be made available via this Core lab. We will provide high quality, high titer preparations of adeno-associated viral (AAV) vectors based primarily on AAV serotype 6, but also for AAV 1, 8, 9 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 dystrophin cDNAs and antibodies that will be made available via this Core lab. Training will allow investigators to apply methods for production and purification of viral vectors (AAV and lentiviral) in their own labs as desired. 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 the Chamberlain group or members of this Wellstone Center to National groups studying muscular dystrophy.
This Core represents an important vector resource, as production uses highly specialized techniques not available to most labs. Gene therapy is an important technique for a variety of muscular dystrophies, including DMD and FSHD. AAV vectors are useful for gene replacement as well as gene knockdown. Lentiviral vectors are an important tool for modifying stem cells and studying disease mechanisms. However the cost and human resources needed to bring production into a lab are considerable
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