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.

Public Health Relevance

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

National Institute of Health (NIH)
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Specialized Center--Cooperative Agreements (U54)
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Special Emphasis Panel (ZAR1-KM (M1))
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University of Washington
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Halbert, Christine L; Allen, James M; Chamberlain, Jeffrey S (2018) AAV6 Vector Production and Purification for Muscle Gene Therapy. Methods Mol Biol 1687:257-266
Adams, Marvin E; Odom, Guy L; Kim, Min Jeong et al. (2018) Syntrophin binds directly to multiple spectrin-like repeats in dystrophin and mediates binding of nNOS to repeats 16-17. Hum Mol Genet 27:2978-2985
Mack, David L; Poulard, Karine; Goddard, Melissa A et al. (2017) Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs. Mol Ther 25:839-854
Bengtsson, Niclas E; Hall, John K; Odom, Guy L et al. (2017) Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy. Nat Commun 8:14454
Amoasii, Leonela; Long, Chengzu; Li, Hui et al. (2017) Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy. Sci Transl Med 9:
Chamberlain, Joel R; Chamberlain, Jeffrey S (2017) Progress toward Gene Therapy for Duchenne Muscular Dystrophy. Mol Ther 25:1125-1131
Elverman, Matthew; Goddard, Melissa A; Mack, David et al. (2017) Long-term effects of systemic gene therapy in a canine model of myotubular myopathy. Muscle Nerve 56:943-953
Whitehead, Nicholas P; Bible, Kenneth L; Kim, Min Jeong et al. (2016) Validation of ultrasonography for non-invasive assessment of diaphragm function in muscular dystrophy. J Physiol 594:7215-7227
Kolwicz Jr, Stephen C; Odom, Guy L; Nowakowski, Sarah G et al. (2016) AAV6-mediated Cardiac-specific Overexpression of Ribonucleotide Reductase Enhances Myocardial Contractility. Mol Ther 24:240-250
Statland, Jeffrey M; Tawil, Rabi (2016) Facioscapulohumeral Muscular Dystrophy. Continuum (Minneap Minn) 22:1916-1931

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