Daley (BCH) Research Hub 03

George Q. Daley, MD, PhD

U01 Principal Investigator, 1-U01HL134812-01

Stem Cells for Therapeutics Discovery in Genetic Blood Disorders

Under the auspices of the Progenitor Cell Biology Consortium (PCBC), our hub at Boston Children's Hospital used unbiased chemical screens in zebrafish and human induced Pluripotent Stem (iPS) Cells to identify two drugs that rescue the hematopoietic defects in Diamond Blackfan Anemia, a genetic bone marrow failure syndrome that lacks adequate pharmacologic or cell-based treatments. In the initial years of the next phase— the Progenitor Cell Translation Consortium—we propose to initiate clinical trials with these agents, and to establish a comprehensive center that during the subsequent years will develop new drugs, progenitor-based cell therapies, and gene therapies for additional genetic blood diseases, including Shwachman Diamond Syndrome, Fanconi Anemia, Dyskeratosis Congenita, primary immune deficiency, and hemoglobinopathy. We will build competency for both internal discovery and clinical translation, as well as Core facilities and generic platforms that will invite collaborations from external investigators, thereby serving the broader mission of the PCTC. Success in our aims will address considerable unmet needs for both the investigation of fundamental mechanisms of genetic blood disease and the development of novel therapeutics for conditions that while individually rare, are collectively a major challenge for hematology. The blood affords considerable advantages as a discovery system, given that analytic reagents and molecular catalogues of blood lineages abound; the high probability of success for blood diseases will establish a powerful proof-of-principle for leveraging human iPS cell models in clinical translation for other diseases.

members: 
Lead Principal Investigator
User Name Full Name Institute Affiliation Topic tags People tags
gdaley's picture
gdaley George Q. Daley, M.D., Ph.D. Children's Hospital Boston cardiac and blood disorders, chronic myelogenous leukemia, CML, differentiation, embryonic stem (ES) cells, germ cell development, hematopoietic stem cells, iPSC, regenerative therapy, self-renewal, target-directed chemotherapy
Co-PI/Co-Investigator
User Name Full Name Institute Affiliation Topic tags People tags
jlipton's picture
jlipton Jeffrey M. Lipton, MD, PhD Hofstra Northwell School of Medicine
ashimaura's picture
ashimaura Akiko Shimamura, MD, PhD University of Washington School of Medicine
avlachos's picture
avlachos Adrianna Vlachos, MD Hofstra Northwell School of Medicine
lzon's picture
lzon Leonard I. Zon, M.D. Harvard University blood, cancer, cdx4, cell proliferation, congenital anemia, development, genetic mutations, genomics, hematopoiesis, hox, pathways, stem cell, transplant, zebrafish
Post-Doctoral Fellow/Trainee
User Name Full Name Institute Affiliation Topic tags People tags
Abhan's picture
Abhan Arunoday Bhan Ph.D. Boston Children's Hospital
JAhom's picture
JAhom Jimmy Hom Hofstra Northwell School of Medicine
vlundin's picture
vlundin Vanessa Lundin, Ph.D. Boston Children's Hospital
DAposocco's picture
DAposocco David Posocco The Children's Hospital of Philadelphia
Trosanwo's picture
Trosanwo Tolulope Rosanwo Boston Children's Hospital
Rsugimura's picture
Rsugimura Rio Sugimura M.D. Ph.D Boston Children's Hospital
Csoriavalles's picture
Csoriavalles Clara Soina Valles Ph.D. Boston Children's Hospital
Publication: 
Author(s) Title Link
Leonard Zon, Alicia McConnell, Jeffrey Mito Neural Crest State Activation in NRAS Driven Melanoma, but not in NRAS-driven Melanocyte Expansion view
Longfei Wang, R. Grant Rowe, George Daley Small-Molecule Inhibitors Disrupt let-7 Oligouridylation and Release the Selective Blockade of let-7 Processing by LIN28 view
Marcella Cesana, Michael Guo, Davide Cacchiarelli, Lara Wahlster, Jessica Barragan, Sergei Doulatov, Linda T. Vo, Beatrice Salvatori, Cole Trapnell, Kendell Clement, Patrick Cahan, Kaloyan M. Tsanov, Patricia M. Sousa, Barbara Tazon-Vega, Adriano Bolondi, Federico M. Giorgi, Andrea Califano, John L. Rinn, Alexander Meissner, Joel N. Hirschhorn, George Q. Daley A CLK3-HMGA2 Alternative Splicing Axis Impacts Human Hematopoietic Stem Cell Molecular Identity throughout Development view
Doulatov S, Daley GQ Autophagy: It's in Your Blood. view

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