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Active Grants and Sponsored Research

Sponsor Grant ID: 1U54DK110805-01 Project Date: August 05, 2016 - July 31, 2021
Sponsoring Organization: NIH/NIDD

A Center of Molecular Hematology

Description of Major Goals
Support use of mice and zebrafish as model organisms for hematopoietic development.
Sponsor Grant ID: T32 CA136432 Project Date: September 22, 2014 - August 31, 2019
Sponsoring Organization: National Institute of Health (NIH)

Research Training in Pediatric Oncology

Description of Major Goals
The major goal of this T32 program is to train physicians who will be the future academic leaders in basic and/or clinical pediatric oncology research
Sponsor Grant ID: 2R01HL032259-34 Project Date: September 15, 2014 - August 31, 2019
Sponsoring Organization: National Institutes Of Health (NIH)

Molecular Analysis of Normal and Thalassemic DNA

Description of Major Goals
The major goals of this project are to understand how FOG-1 participates in GATA-dependent gene regulation, how GATA-1 functions in gene transcription, and how BCL11A protein controls HbF.
Sponsor Grant ID: 5R01HL119099-03 Project Date: September 1, 2014 - May 31, 2019
Sponsoring Organization: National Institute of Health (NIH)

Systems Biology Analysis of Human Erythropoiesis

Description of Major Goals
The major goals of this project are to predict and validate the developmental stage-specific gene regulatory networks in human primary erythroid precursors by integrating genomic and epigenomic data-types; to perturb the gene regulatory networks using molecular and genetic experiments and further integrate such information to refine our network model; and to characterize the role of genetic variants in influencing chromatin state, gene expression, and erythroid traits.
Sponsor Grant ID: 70792 Project Date: January 1, 2017 - December 31, 2018
Sponsoring Organization: Harrington Discovery Institute

Discovery of natural products for induction of fetal hemoglobin for sickle cell disease

Description of Major Goals
The goal is to identify new agents for reactivation of fetal hemoglobin (HbF) in adults as treatment for the major beta-hemoglobin disorders, sickle cell disease and beta-thalassemia.
Project Date: September 01, 2015 - August 31, 2018
Sponsoring Organization: Doris Duke Charitable Foundation

High-resolution and high through-put genome editing to determine minimal repressive sequences within the beta-globin gene cluster

Description of Major Goals
The study will identify a set of CRISPR sgRNAs sufficient to cause substantial disruption of the B-globulin cluster HbF repressive element
Sponsor Grant ID: U01HL117720-04 Project Date: August 15, 2013 - May 31, 2018
Sponsoring Organization: National Institutes of Health (NIH)

Translational and clinical studies targeting gamma-globin modulation

Description of Major Goals
The goal is to exploit the observation that individuals with propionic acidemia (PA) have enhanced HbF expression, in 3 specific aims, the PI proposes to 1) extend the observation that PA is associated specifically with HbF induction, 2) determine the unique metabolites that may be associated with said induction and 3) examine the effects of candidate metabolites on in vitro erythroid cell cultures. Specific globin gene expression, global gene expression, epigenetic modification of the beta globin locus, and a specific analysis of the effects of these metabolites on HDAC activity are proposed.
Project Date: January 01, 2016 - December 31, 2017
Sponsoring Organization: Hyundai Hope on Wheels

Epigenetic Eradication of Chronic Leukemia

Description of Major Goals
the major goals of this award are to explore the Ezh2-dependence of CML leukemic stem cells, and to determine that small molecule inhibition of Ezh2 is functionally equivalent to Ezh2 loss.
Project Date: October 01, 2014 - September 30, 2017
Sponsoring Organization: Biogen

Hematopoietic regulators and efficient genome engineering

Description of Major Goals
The goals of the project are to improve methods for gene engineering of hematopoietic stem cells in mouse and human in order to identify key regulators of blood cell development and self-renewal.
Sponsor Grant ID: 5P01HL032262-34 Project Date: August 15, 2011 - June 30, 2017
Sponsoring Organization: National Institutes Of Health (NIH)

Developmental Biology of Human Erythropoiesis: PPG

Description of Major Goals
The major goal of this project is to further an understanding of the genetic mechanisms that control the fetal to adult hemoglobin switch.
Sponsor Grant ID: 5U01HL100001-06 Project Date: September 30, 2009 - April 30, 2017
Sponsoring Organization: National Institutes Of Health (NIH)

Human Pluripotent Stem Cell and Progenitor Models of Cardiac and Blood Diseases (7 years)

Description of Major Goals
Dr. Orkin is PI of a project focused on the effects of trisomy 21 (Down syndrome) on normal hematopoiesis, leukemia in the setting of Down syndrome, and contributions of trisomy 21 on cardiac development, through the study of trisomy 21 ES and iPS cells.
Project Date: April 08, 2014 - April 07, 2017
Sponsoring Organization: Pfizer

Identification of a development for the treatment of sickle cell

Description of Major Goals
The major goals of this project are to test the feasibility of discovering small molecules with good potential to progress to leads that reduce the activity of BCL11A in repressing γ−globin expression, progress two series through SDS and LD to deliver lead and back-up for candidate selection, and identify one new target within the BCL11A / γ−globin expression pathway for possible entrance into the RDRU drug discover portfolio.