Title David G. Nathan Distinguished Professor of Pediatrics Institution Boston Children's Hospital Address Dana Farber Cancer Institute
Dept. of Pediatric Oncology, 16th Floor
44 Binney St
Boston MA 02115
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Mentoring
Available: 12/01/22, Expires: 01/01/24
Over the past 15 years we have determined the mechanism by which fetal hemoglobin (HbF) is silenced. Increased HbF ameliorates the severity of the hemoglobin disorders, sickle cell disease and beta-thalassemia. Therefore, reactivation of HbF in patients with these disorders has been a long sought goal. Using a variety of genetic and molecular approaches, we determined how to reactivate HbF expression in adult erythroid cells using gene editing by CRISPR/Cas9, and this work has been translated to patients in successful clinical trials by pharma. In order to reduce the burden of disease, however, small molecules (drugs) that mimic the effects of genetic manipulations in turning on HbF are in great need. We are working in the laboratory on discovery of small molecules that modulate the level of BCL11A, the critical silencer of HbF. Students with particular interests in chemical biology are encouraged to consider this project.
Characterizing the interactions of Bcl11a and mammalian SWI/SNF (BAF) chromatin remodeling complexes
Summer, 06/08/15 - 08/03/15
A Genetically Engineered Mouse Model of Osteosarcoma that Closely Mimics the Human Disease
Summer, 06/12/07 - 08/31/07
Research
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U54DK110805
(ORKIN, STUART H)
Aug 5, 2016 - Jul 31, 2021
A CENTER OF MOLECULAR HEMATOLOGY
Role: Principal Investigator
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R01HL119099
(YUAN, GUO-CHENG)
Sep 1, 2014 - May 31, 2020
Systems Biology Analysis of Human Erythropoiesis
Role: Principal Investigator
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U01HL100001
(DALEY, GEORGE Q)
Sep 30, 2009 - Apr 30, 2017
Human Pluripotent Stem Cell and Progenitor Models of Cardiac and Blood Diseases
Role: Co-Principal Investigator
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RC2HL101553
(ORKIN, STUART H)
Sep 30, 2009 - Aug 31, 2012
Extending GWAS at the BCL11A locus to novel therapeutics for HbF induction
Role: Principal Investigator
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T32CA136432
(ARMSTRONG, SCOTT A)
Jul 13, 2009 - Jun 30, 2025
Research Training in Pediatric Oncology
Role: Principal Investigator
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U01CA105423
(ORKIN, STUART H)
Sep 10, 2004 - Jul 31, 2014
Exploiting mouse models of pediatric cancers and epigenetics for therapy
Role: Principal Investigator
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R01HL075735
(ORKIN, STUART H)
Dec 1, 2003 - Nov 30, 2013
Gfi-proteins in Hematopoietic Development
Role: Principal Investigator
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R01HL069142
(ORKIN, STUART H)
Sep 30, 2001 - Aug 31, 2005
Molecular Mechanisms of Tissue Stem Cell Plasticity
Role: Principal Investigator
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P01CA068484
(SALLAN, STEPHEN E)
Sep 30, 1995 - Apr 30, 2013
Therapeutic Index of Acute Lymphoblastic Leukemia
Role: Co-Principal Investigator
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P30DK049216
(ORKIN, STUART H)
Sep 30, 1994 - Jun 30, 2016
A CENTER OF MOLECULAR DEVELOPMENTAL HEMATOPOIESIS
Role: Principal Investigator
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R01HL051290
(ORKIN, STUART H)
Dec 1, 1993 - Nov 30, 2003
HEMATOPOIESIS IN NF E2 GENE TARGETED ES CELLS
Role: Principal Investigator
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P20DK047466
(ORKIN, STUART H)
Sep 30, 1993 - Aug 31, 1995
MOLECULAR DEVELOPMENTAL HEMATOPOIESIS
Role: Principal Investigator
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S15MH050795
(ORKIN, STUART H)
Sep 30, 1992 - Aug 31, 1993
ASIP-CHILDREN'S HOSPITAL-BOSTON
Role: Principal Investigator
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R13GM038845
(ORKIN, STUART H)
Jun 12, 1987 - May 31, 1988
GORDON RESEARCH CONFERENCE ON MOLECULAR GENETICS
Role: Principal Investigator
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R37HD018661
(ORKIN, STUART H)
Jul 1, 1983 - Jun 30, 1998
STRUCTURE-FUNCTION ANALYSIS OF SPECIFIC HUMAN GENES
Role: Principal Investigator
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R01HD018661
(ORKIN, STUART H)
Jul 1, 1983 - Jun 30, 1988
STRUCTURE-FUNCTION ANALYSIS OF SPECIFIC HUMAN GENES
Role: Principal Investigator
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P01HL032262
(ORKIN, STUART H)
Jan 1, 1983 - Mar 31, 2022
DEVELOPMENTAL BIOLOGY OF HUMAN ERYTHROPOIESIS
Role: Principal Investigator
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R01HL032259
(ORKIN, STUART H)
Apr 1, 1982 - Apr 30, 2023
Molecular Analysis of Normal and Thalassemic DNA
Role: Principal Investigator
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T32GM007748
(MORTON, CYNTHIA CASSON)
Jul 1, 1979 - Jun 30, 2023
Training Grant in Genetics
Role: Co-Principal Investigator
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P50DK049216
(ORKIN, STUART H)
Aug 31, 1999
CENTER FOR MOLECULAR DEVELOPMENT HEMATOPOIESIS
Role: Co-Principal Investigator
Bibliographic
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