The purpose of Core C, the Cell Transplantation and Analysis Core, is to provide critical support for cell sourcing, isolation and analysis needs of research performed under the three Projects and Core B, The specific service aims of Core C are:
Aim 1 : To procure specific cell types and tissues from human embryonic, fetal, neonatal and adult tissue specimens. The requirements of project scientists for primary human tissue specimens such as skin, hematopoietic and mesenchymal cells will be met by obtaining these tissues from fetal, neonatal and adult specimens.
Aim 2 : To provide flow cytometric analysis and sorting services to project scientists. Expertise and assistance with flow cytometric analysis and fluorescence-activated cell sorting (FACS) will be provided to project scientists.
Aim 3 : To assay teratoma formation in immunodeficient mice. One hallmark of induced pluripotent stem (IPS) cells is their capacity to form teratomas when transplanted into immunodeficient mice. IPS cells and differentiated cells generated by the 3 projects will be assayed for teratoma forming ability to measure pluripotentiality.
Aim 4 : To assay long-term multilineage hematopoietic reconstitution of candidate hematopoietic stem cells (HSCs) generated from IPS cells. The capacity of hematopoietic cells created from IPS cells under Project 3 to reconstitute erythroid, myeloid and lymphoid lineages for at least 12 weeks will be tested by transplantation into immunodeficient mice. The work will be performed under the guidance of Dr. Marcus Muench at Blood systems Research Institute utilizing the Core Immunology and Cell, Tissue and Vivarium Cores already established at that institution.

Public Health Relevance

This core provides services critical to accomplishing the research goals of the overall program project that aims to develop new methods of treating hemoglobinopathies using genetically correcting patient cells to generate hematopoietic stem cells for autologous transplantation.

National Institute of Health (NIH)
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Research Program Projects (P01)
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Special Emphasis Panel (ZDK1-GRB-6)
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University of California San Francisco
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Suzuki, Shingo; Sargent, R Geoffrey; Illek, Beate et al. (2016) TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs. Mol Ther Nucleic Acids 5:e273
Beyer, Ashley I; Muench, Marcus O (2016) Comparison of human hematopoietic reconstitution in different strains of immunodeficient mice. Stem Cells Dev :
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Muench, Marcus O; Beyer, Ashley I; Fomin, Marina E et al. (2014) The adult livers of immunodeficient mice support human hematopoiesis: evidence for a hepatic mast cell population that develops early in human ontogeny. PLoS One 9:e97312
Xie, Fei; Ye, Lin; Chang, Judy C et al. (2014) Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac. Genome Res 24:1526-33
Ye, Lin; Wang, Jiaming; Beyer, Ashley I et al. (2014) Seamless modification of wild-type induced pluripotent stem cells to the natural CCR5Δ32 mutation confers resistance to HIV infection. Proc Natl Acad Sci U S A 111:9591-6
Fomin, M E; Togarrati, P P; Muench, M O (2014) Progress and challenges in the development of a cell-based therapy for hemophilia A. J Thromb Haemost 12:1954-65

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