The Administrative Core serves as the central support and communication resource for MDCRC investigators and their research teams. The goals of this core are to: 1) Provide the leadership and clear lines of authority necessary to promote cooperative, successful scientific interaction among the MDCRC participants 2) Provide an organized, well directed advisory network to ease the burden on MDCRC investigators 3) Effectively plan and coordinate research activities to promote fiscal responsibility and collaborative success 4) Facilitate development of new investigators across multiple disciplines and promote MDCRC collaborations through a Cooperative Pilot program in the area of muscular dystrophy.
The overall goal of the MDCRC Training and Education Core is to provide trainees with the necessary skills to translate advances in basic science muscular disease research into well designed clinical trials. To be successful, trainees must have an understanding of the concepts, features and limitations of clinical treatments, biology of the target disease, ethics of human and animal research and importance of well designed clinical trials. These concepts will be taught through a series of formal courses and seminars, bringing together a cross-disciplinary group of basic science and clinical researchers. Mentored research under the direction of the MDCRC will present trainees with the practical skills needed to pursue careers in translational research.
The specific aims of this core are to: 1) Organize a didactic core support network providing the leadership, mentorship and interactive evaluation necessary to guide trainees throughout their MDCRC experience 2) Utilize a diverse pool of candidates to create a multi-level training core in an interdisciplinary setting 3) Provide established and future investigators, trainees, research personnel and patient and lay community members with appropriate educational forums to facilitate their role in team focused interdisciplinary muscular research 4) Address the shortage of well-trained investigators by providing pre and post doctoral and MD trainees with the skills necessary to become independent researchers. This training is organized to provide broad based training principles of muscle disease interventions theory, practice and application. This will be accomplished by providing a rich and flexible training curriculum that meets the needs of a broad research community.
The Canine Muscular Dystrophy Core (CMDC) of the MDCRC supports pilot projects on well characterized canine DMD models. Many investigators may develop only one therapy all the way to the investigational new drug (IND) phase during their careers. These investigators will often be familiar with and have ready access to expertise in rodent models. However, few will have a background with analogous large animal diseases. The CDMC provides this resource and facilitates the translation of preliminary studies into broader hypothesis and milestone driven research. The CDMC has three interrelated aims that collectively support research of Wellstone Center investigators and others wishing to utilize canine DMD models in pre-clinical research. These aims are: 1) Perpetuate canine models of DMD to meet investigator demands for this important pre-clinical model 2) Work with UNC and the National Center for Canine Models of DMD core facilities to develop and refine biomarkers that can be used to determine efficacy of proposed DMD treatments 3) Collaborate with Wellstone Center investigators and others to conduct pilot projects that bolster translational research focused on therapeutic strategies for DMD.
For more information on the Canine Muscular Dystrophy Core, please visit http://www.ncdmd.org/
The goal of the MDCRC National Vector Muscular Dystrophy Core (NVMDC) is to provide muscular dystrophy researchers with a highly efficient and cost effective means of obtaining viral and non-viral vectors for their research. As an integrated GLP/GMP vector production and testing facility, the NVMDC provides a wide range of services and vector development expertise. Investigators can obtain vectors from all the major viral platforms, in a wide range of quantities and at several levels of quality from basic research grade to clinical grade. Further, our extensive experience with translational research provides MD investigators with the resources to facilitate the rapid translation of promising new developments into the clinic. Lastly, the NVMDC's regulatory expertise is available to assist investigators with preparation of regulatory submissions and maintenance of regulatory compliance.
The specific aims of the NVMDC are: 1) Provide vector and drug development services to investigators 2) Produce AAV vectors to support the specific aims of the MDCRC Project 2 - Evaluate hydrodynamic limb vein (HLV) and isolated limb artery (ILA) injection methods for delivery of various AAV minidys vectors in a dog model of muscular dystrophy. 3) To produce AAV vectors to support the specific aims of the MDCRC Project 3 - Adeno-associated Virus Vectors for Targeted and Repeat Delivery.
