Several forms of stem cell modificafion are currently envisioned that will facilitate the development of the next generafion of cell-based therapeutics. Integrafing viral vectors are commonly used to permanently and most efficienfiy insert genes of interest, or to introduce libraries of genes to conduct screens. A current major research emphasis and area of expertise of invesfigators at CHOP is to characterize genetic disorders in stem cells. One of the next areas of high priority of research will be to develop methods to correct monogenic genefic disorders. For this purpose, ZFNs are currenfiy the only method that has sufficient efficiency to modify stem cells at levels necessary to support therapy. We will be providing these services in a fimely, cost-efficient manner that will provide high quality large-scale products that avoids the need for each investigator to develop the necessary skill sets. By providing the best state-of-the-art backbone for the vectors and the latest in ZFN technology we anticipate that we will not only allow better standardization of product on the UPENN/CHOP campus, but also enhance the quality of available products, leading to enhanced producfivity and synergy on campus in the field of benign hematopoiesis.

National Institute of Health (NIH)
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Center Core Grants (P30)
Project #
Application #
Study Section
Special Emphasis Panel (ZDK1-GRB-G)
Project Start
Project End
Budget Start
Budget End
Support Year
Fiscal Year
Total Cost
Indirect Cost
Children's Hospital of Philadelphia
United States
Zip Code
Wunderlich, Mark; Brooks, Ryan A; Panchal, Rushi et al. (2014) OKT3 prevents xenogeneic GVHD and allows reliable xenograft initiation from unfractionated human hematopoietic tissues. Blood 123:e134-44
Sullivan, Spencer K; Mills, Jason A; Koukouritaki, Sevasti B et al. (2014) High-level transgene expression in induced pluripotent stem cell-derived megakaryocytes: correction of Glanzmann thrombasthenia. Blood 123:753-7
Kamat, Viraj; Paluru, Prasuna; Myint, Melissa et al. (2014) MicroRNA screen of human embryonic stem cell differentiation reveals miR-105 as an enhancer of megakaryopoiesis from adult CD34+ cells. Stem Cells 32:1337-46
Thom, Christopher S; Traxler, Elizabeth A; Khandros, Eugene et al. (2014) Trim58 degrades Dynein and regulates terminal erythropoiesis. Dev Cell 30:688-700
Paralkar, Vikram R; Mishra, Tejaswini; Luan, Jing et al. (2014) Lineage and species-specific long noncoding RNAs during erythro-megakaryocytic development. Blood 123:1927-37
Shin, Jae-Won; Buxboim, Amnon; Spinler, Kyle R et al. (2014) Contractile forces sustain and polarize hematopoiesis from stem and progenitor cells. Cell Stem Cell 14:81-93
Paluru, Prasuna; Hudock, Kristin M; Cheng, Xin et al. (2014) The negative impact of Wnt signaling on megakaryocyte and primitive erythroid progenitors derived from human embryonic stem cells. Stem Cell Res 12:441-51
Tiyaboonchai, Amita; Mac, Helen; Shamsedeen, Razveen et al. (2014) Utilization of the AAVS1 safe harbor locus for hematopoietic specific transgene expression and gene knockdown in human ES cells. Stem Cell Res 12:630-7
Crispino, John D; Weiss, Mitchell J (2014) Erythro-megakaryocytic transcription factors associated with hereditary anemia. Blood 123:3080-8
Buxboim, Amnon; Swift, Joe; Irianto, Jerome et al. (2014) Matrix elasticity regulates lamin-A,C phosphorylation and turnover with feedback to actomyosin. Curr Biol 24:1909-17

Showing the most recent 10 out of 21 publications