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Stuart Holland Orkin


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

The research activities and funding listed below are automatically derived from NIH ExPORTER and other sources, which might result in incorrect or missing items. Faculty can login to make corrections and additions.
  1. U54DK110805 (ORKIN, STUART H) Aug 5, 2016 - Jul 31, 2021
    Role: Principal Investigator
  2. R01HL119099 (YUAN, GUO-CHENG) Sep 1, 2014 - May 31, 2020
    Systems Biology Analysis of Human Erythropoiesis
    Role: Principal Investigator
  3. 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
  4. 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
  5. T32CA136432 (ARMSTRONG, SCOTT A) Jul 13, 2009 - Jun 30, 2025
    Research Training in Pediatric Oncology
    Role: Principal Investigator

Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Faculty can login to make corrections and additions.
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Funded by the NIH National Center for Advancing Translational Sciences through its Clinical and Translational Science Awards Program, grant number UL1TR002541.