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DavidDWilliams_Blue

Potential gene therapy approach to sickle cell disease highlighted at ASH

December 11, 2012
Proof-of-concept animal study selected as one of the best presentations at 54th American Society of Hematology Annual Meeting and Exposition
Sickle Cell Photo

Newly Discovered Gene Regulator Could Precisely Target Sickle Cell Disease

October 10, 2013
(News release) A research team from Dana-Farber/Boston Children's and other institutions has discovered a new genetic target for potential therapy of sickle cell disease. The target, called an enhancer, controls a molecular switch in red blood cells called BCL11A that, in turn, regulates hemoglobin production. The researchers – led by Daniel Bauer, MD, PhD, and Stuart Orkin, MD, of Dana-Farber/Boston Children's – reported their findings in Science.
DavidNathan

Hope Grows for Patients with Sickle Cell Disease

March 03, 2014
Advances in research and treatment are changing sickle cell disease from an inherited condition that often condemned children to painful and short lives into a condition that can be managed with less pain and a longer life expectancy, the Washington Post reports.
Venee Tubman, MD

Blood Work

August 11, 2014
Harvard Medical School interviewed Venee Tubman, MD, a pediatric hematologist, about her sickle cell work in Liberia.
Venee Tubman, MD

Liberia’s Response to Ebola

October 14, 2014
New England Cable News interviewed Venee Tubman, MD, about the Ebola outbreak in Liberia, where she has done extensive work on sickle cell disease.
Sickle Cell Photo

Gene Editing Study Reveals Possible "Achilles Heel" of Sickle Cell Disease

September 16, 2015
Researchers from Dana-Farber/Boston Children's, using CRISPR-based gene editing tools, have found that changes to a small stretch of DNA may circumvent the genetic defect behind sickle cell disease (SCD). The discovery, published in the journal Nature, creates a path for developing gene editing approaches for treating SCD and other hemoglobin disorders, such as thalassemia.
Matthew Heeney, sickle cell program director

Therapy Directed Against Platelets Does Not Significantly Reduce Painful Crises in Sickle Cell Disease

December 08, 2015
Treatment with the antiplatelet agent prasugrel does not significantly reduce the rate of pain crises or severe lung complications in children with sickle cell disease, according to a report published in the New England Journal of Medicine describing one of the largest and most geographically diverse international clinical trials on sickle cell disease to date.
Venee Tubman, MD

Making a difference in global sickle cell disease care

March 31, 2016
Venee Tubman, MD, has deep roots in Liberia, where her grandfather was the nation’s longest serving president. The pediatric hematologist/oncologist is bringing her expertise in sickle cell disease to the African country, where she has been instrumental in establishing newborn screening and follow-up care, Penn Medicine reports.
sickle cell disease blood cells

Genetic treatments for sickle cell

May 01, 2016
Researchers at Dana-Farber/Boston Children's and other centers are focusing on controlling the switch that regulates hemoglobin production to prevent the devastation of sickle cell disease, Scientific American reports.
Sickle Cell Photo

We’ve known for 50 years what causes sickle cell disease. Where’s the cure?

May 19, 2016
Boston Globe Media’s Stat examines efforts to use gene therapy or gene editing – or develop a pill – to harness the latest research on sickle cell disease. “This is the right time for a sickle cell moonshot,” says Dana-Farber/Boston Children’s President David Williams, MD.
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