Developing treatments for muscular dytrophies
hero_pattern.png

Supporting Cores

Seattle MDSRC Cores


administrative CORE
Highlights

  • Promotes a Center environment through enhanced communication and collaboration of investigators within and outside the Center.

  • Organizes the Center Advisory Committee made up of non-Center members.

  • Organizes research meetings and seminars with internal and external speakers.

  • Manages the Outreach program, which includes opportunities to visit the Center, meet investigators, provide speakers at events with patients and advocacy groups & present educational activities at local schools and science events.


SHARED RESOURCE Vector CORE
highlights

  • Provides consulting, reagents, and training related to adeno-associated (AAV) and lenti viral vector production.

  • Provides high-quality, high-titer AAV and lentivirus vectors.

  • Provides muscular dystrophy-related reagents (cell lines, cDNA, vector backbones, protocols, antibodies, etc).

  • Provides services to members of this Center and outside laboratories (on a recharge basis) to support national efforts related to gene therapy for the muscular dystrophies.

  • If interested in purchasing vectors please fill out the form on our contact page


training CORE
highlights

  • Promotes training and career development of students and fellows associated with the Center.

  • Inspires and prepares talented new investigators to address the critical needs of individuals with muscular dystrophy.

  • Provides a rich and collaborative environment that brings together basic and clinical researchers in muscular dystrophy with students and fellows in training.

  • Provides partial salary support for trainees to pursue mentored research training in muscular dystrophy research.


This Administrative Core has been managing the activities of the Seattle MDSRC since May 2014. The Core will continue to provide oversight, coordinate activities and manage budgetary activities. We will assume responsibilities for outreach activities, a seminar series, and in coordination with a Training Core, activities previously handled through an Investigator Development and Patient Outreach (IDPO) Core. Dr. Jeffrey S. Chamberlain will direct the Core with assistance from Dr. Stephen Tapscott. Ms. Roberta Miller will handle day-to-day operation of the Core.

The Core will be responsible for overseeing all activities of this Center. This includes budgetary oversight, ensuring compliance with regulatory requirements, coordinating travel, and managing progress reports. Coordination will be aided by a Center Executive Committee, which will be composed of the PIs and key members of each Core and Project. We will also seek advice and critiques from our Center Advisory Committee, which is in place. Integration and oversight will also be facilitated by a Center web site managed by this Core. This publicly accessible web site will promote the Center’s mission with summaries of activities and progress, provide links to publications and events, advertise opportunities related to training and research, provide a portal to affiliated lab web sites, and links to contact Center members with questions, requests for reagent/resources or information on methods and data. The Core will manage semi-monthly Center-wide research meetings that will focus on recent data, areas of collaborative opportunities, announcements and discussions of progress. The research meetings will also be integrated with a seminar series for outside speakers. These meetings and seminars will also be coordinated with relevant Grand Rounds within our Institutions. These activities will form part of our Outreach program, which will include opportunities to visit the Center, meet investigators, provide speakers at events with patients and advocacy groups & present educational activities at local schools and science events.


The Viral Vector Core in the Wellstone Center 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. 

Current costs for members of the Wellstone Center:
1-5 Roller bottles of a single vector (exact same vector): $800/RB
6-11 Roller bottles of a single vector (exact same vector): $610/RB
12+ Roller bottles of a single vector (exact same vector): $525/RB


A major obligation of the Paul D. Wellstone Muscular Dystrophy Special Research Centers is to develop the human resources necessary to accomplish translational research and clinical studies in muscular dystrophies. The broad and long-term goal for the Training Core, which is overseen by Dr. Michael Regnier, is to provide the environment for mentored research training in basic research, clinical research, and ethics that will inspire and prepare talented new investigators to address the critical needs of individuals with muscular dystrophy. The aims for reaching the stated objectives are: 1) provide a rich and collaborative environment that brings together basic and clinical researchers in muscular dystrophy with students and fellows in training through a regular seminar series focused on muscular dystrophy; 2) provide access to a broad group of basic, translational, and clinical researchers for trainees; 3) provide partial salary support for trainees to pursue mentored research training in muscular dystrophy research; and 4) provide oversight, evaluation, and guidance to foster successful careers in muscular dystrophy research.

The Training Core will leverage the broad and deep training programs at the University of Washington, Seattle Children's Hospital, and the Fred Hutchinson Cancer Research Center to train the next generation of researchers in the skills and insight necessary to advance therapies for muscular dystrophies.