Human pluripotent stem cells that can give rise to almost any type of cell were originally isolated from embryonic cells and their utility as tools fo research and therapeutic development was severely restricted due to ethical considerations as well as the inability to derive stem cells from subjects with known disorders. The relatively recent development of technologies for deriving induced pluripotent stem cells (iPSCs) from cells that were previously thought to be terminally differentiated, such as skin or blood cells, has opened up nearly limitless new avenues for understanding human disease and developing novel approaches for treatment. By converting cells collected from affected individuals into iPSCs, and then to relevant cell types such as neurons for central nervous system disorders, we can create disease models in a dish, cells that recapitulate the disorder and can be used to screen therapeutic drugs or study the manifestation of the disorder and elucidate its origins. The NINDS Repository was created to collect and distribute a variety of human cells such as skin fibroblasts and blood cells and convert these to iPSCs for research in the areas of neurodegenerative disease such as Parkinson's disease, Alzheimer's disease, Huntington's disease, amyotrophic lateral sclerosis, Dystonia and Frontotemporal Degeneration. Given the complexity and cost of generating iPSC lines of high quality, and the emerging importance of disease modeling and regenerative medicine, there is a tremendous need in the neuroscience field for a reliable centralized source of these materials. RUCDR Infinite Biologics (RUCDR) is uniquely positioned to provide this resource for the NINDS. RUCDR has a long and successful track record as a repository and genetic resource since its inception in 1998. RUCDR manages several major repositories for the NIH, including the NIMH Repository and Genomics Resource (NRGR) which encompasses the NIMH Stem Cell Resource. The mission of the RUCDR NINDS Stem Cell Repository will be to work with NINDS investigators and staff on all aspects of study management to collect, process and distribute the highest quality cell samples from participants in its studies. These will include the culturing of fibroblasts from skin biopsies, th isolation of lymphocytes from blood, and the derivation of iPSC lines from both cell types. RUCDR will then perform a comprehensive range of tests on the iPSCs to ensure their quality, pluripotency and identity. RUCDR will provide additional services to the global community, including the generation of neural stem cells (NSCs), different types of neurons, and genome editing to repair or introduce in iPSCs specific mutations that are implicated in human disease.

Public Health Relevance

Twenty first century medicine will increasingly rely on emerging technologies such as disease modeling, tissue on a chip drug screening and regenerative medicine to develop novel treatments for human diseases such as Parkinson's disease, Alzheimer's disease, Huntington's disease, amyotrophic lateral sclerosis, Dystonia and Frontotemporal Degeneration. The RUCDR NINDS Repository will play a crucial role in providing high quality specimens and support for these studies to the global research community. These specimens include unmodified and genome edited Induced Pluripotent Stem Cells (iPSCs), neural stem cells (NSCs) and neurons from affected individuals.

National Institute of Health (NIH)
National Institute of Neurological Disorders and Stroke (NINDS)
Resource-Related Research Projects--Cooperative Agreements (U24)
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Special Emphasis Panel (ZNS1-SRB-T (05))
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Sutherland, Margaret L
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Rutgers University
Schools of Arts and Sciences
New Brunswick
United States
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Sakurai, Kunie; Kurtz, Andreas; Stacey, Glyn et al. (2016) First Proposal of Minimum Information About a Cellular Assay for Regenerative Medicine. Stem Cells Transl Med 5:1345-1361