Project Summary – Resource (Vector) Core The Viral Vector Core was established in 2003 to meet the emerging interest in using AAV & lentiviral vectors for muscle gene transfer. The required specialized techniques and reagents prevented most labs from being able to generate the high titer and enormous quantities of vectors needed for systemic gene delivery, challenges that remain today. Consequently, a Viral Vector Core was established to generate vectors for Wellstone Centers, but which has been expanded to allow access to a broader range of users via a recharge mechanism. This Vector Core is our Scientific Research Resource Core. 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 vectors, reagents, consulting, 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 transfer and gene therapy for the muscular dystrophies. The Principle Investigator is Dr. Joel Chamberlain, who has worked closely with Core personnel on AAV and retroviral vectors for more than 15 years. The Core has established protocols and acquired reagents for lentiviral and adeno- associated viral vectors, particularly AAV6, 8 and 9, but also for other pseudotyped vectors including AAV1, 2, 5, 7 and several recently identified myotropic (e.g. AAVMyo) capsids. We also have numerous muscle cDNAs, such as for dystrophin and utrophin, muscle regulatory cassettes and antibodies that are available via this Core lab, as well as DM1, DUX4 and associated RNAi vectors. We provide high quality, high titer preparations of AAV and lentiviral vectors and also encourage users to visit the Core to learn, or request, our methods. Vectors and reagents are provided to members of our Wellstone Center using funds from this Core, and to other muscular dystrophy researchers on a recharge basis at cost. Training services allow investigators to apply methods for production and purification of viral vectors in their own labs as desired, and will be advertised via the Center Web site. Consulting will be available to guide the use of AAV and lentiviral vectors. Finally, we provide reagents (such as vector backbones, plasmids, antibodies) that have been developed by members of this Wellstone Center to outside labs.