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Completed Grants and Sponsored Research

Sponsor Grant ID: 1U54DK110805-01 Project Date: August 5, 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: 70792 Project Date: January 1, 2017 - December 31, 2019
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.
Sponsor Grant ID: 5R01HL119099-04 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: 2R01HL032259-34 Project Date: September 15, 2014 - April 30, 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.
Project Date: September 1, 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
Project Date: April 8, 2014 - April 7, 2018
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.
Project Date: January 1, 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.
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.