The Human Pluripotent Stem Cell Core was established in 2008 as part of the Center for Cellular and Molecular Therapeutics. Our mission is to provide expertise and quality-control reagents for the culture and differentiation of human embryonic stem cells (ESC) and induced pluripotent stem cells (iPSC) to CHOP, the University of Pennsylvania, and outside academic communities.
An infrastructure and solid foundation has been created for the generation of iPSC lines that are used by investigators worldwide for modeling human disease to study mechanism, development, and establish new therapeutic modalities. A recent addition to the core has been the establishment of the genome editing technology using CRISPR/Cas. This technology is being utilized on iPSCs established by the Core for creating isogenic lines that eliminate the clonal heterogeneity and variability in downstream applications. We provide enrichment courses to actively train investigators with the necessary tools and expertise to maximize successful outcomes.
Maguire JA, Gagne A, Mills JA, Gadue P, French DL. Generation of human control iPS cell line CHOPWT9 from healthy adult peripheral blood mononuclear cells. Stem Cell Res 16:14-16, 2016.
Maguire JA, Lu L, Mills JA, Sullivan LM, Gagne A, Gadue P, French DL. Generation of Hermansky Pudlak Syndrome Type 1 (HPS1) induced pluripotent stem cells (iPSCs). Stem Cell Res 16:233-235, 2016.
Disease modeling and Genome editing:
Tiyaboonchai A, Cardenas-Diaz FL, Ying L, Maguire JA, Sim X, Jobaliya C, Gagne A, Kishore S, Stanescu DE, Hughes N, De Leon DD, French DL, Gadue P. GATA6 plays an important role in the induction of human definitive endoderm, development of the pancreas and functionality of pancreatic cells. Stem Cell Reports, 2017.
Sim X, Vardenas-Diaz FL, French DL, Gadue P. A doxycycline-inducible system for genetic correction of iPSC disease models. Springer Protocols, Methods Mol Biol 1353:13-23, 2016.
Byrska-Bishop M, VanDorn D, Campbell AE, Betensky M, Arca PR, Yao Y, Gadue P, Costa FF, Nemiroff RL, Blobel GA, French DL, Hardison RC, Weiss MJ, Chou ST. Pluripotent stem cells reveal novel erythroid activities of the GATA1 N-terminus. JCI 125:993-1005, 2015.
Sullivan SK, Mills JA, Koukouritaki SB, Vo KK, Lyde RB, Paluru P, Zhao G, Sullivan LM, Wang Y, Kishore S, Gharaibeh EZ, Lambert MP, Wilcox DA, French DL, Poncz M, Gadue P. High-level transgene expression in induced pluripotent stem cell-derived megakaryocytes: correction of Glanzmann thrombasthenia. Blood 123:753-757, 2014.
Tiyaboonchai A, Mac H, Shamsedeen R, Mills JA, Kishore S, French DL, Gadue P. Utilization of the AAVS1 safe harbor locus for hematopoietic specific transgene expression and gene knockdown in human ES cells. Stem Cell Res 12:630-637, 2014.