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.

Agency
National Institute of Health (NIH)
Institute
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Type
Research Program Projects (P01)
Project #
5P01DK088760-03
Application #
8532892
Study Section
Special Emphasis Panel (ZDK1-GRB-6)
Project Start
Project End
Budget Start
2013-08-01
Budget End
2014-07-31
Support Year
3
Fiscal Year
2013
Total Cost
$176,864
Indirect Cost
Name
University of California San Francisco
Department
Type
DUNS #
094878337
City
San Francisco
State
CA
Country
United States
Zip Code
94143
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
Ye, Lin; Wang, Jiaming; Beyer, Ashley I et al. (2014) Seamless modification of wild-type induced pluripotent stem cells to the natural CCR5?32 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
Xie, Fei; Ye, Lin; Chang, Judy C et al. (2014) Seamless gene correction of ?-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac. Genome Res 24:1526-33
Sargent, R Geoffrey; Suzuki, Shingo; Gruenert, Dieter C (2014) Nuclease-mediated double-strand break (DSB) enhancement of small fragment homologous recombination (SFHR) gene modification in human-induced pluripotent stem cells (hiPSCs). Methods Mol Biol 1114:279-90
Fomin, Marina E; Zhou, Yanchen; Beyer, Ashley I et al. (2013) Production of factor VIII by human liver sinusoidal endothelial cells transplanted in immunodeficient uPA mice. PLoS One 8:e77255
Ng, Terry Fei Fan; Kondov, Nikola O; Hayashimoto, Nobuhito et al. (2013) Identification of an astrovirus commonly infecting laboratory mice in the US and Japan. PLoS One 8:e66937
Ye, Lin; Muench, Marcus O; Fusaki, Noemi et al. (2013) Blood cell-derived induced pluripotent stem cells free of reprogramming factors generated by Sendai viral vectors. Stem Cells Transl Med 2:558-66