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Hemoglobin F - sickle cell

BCL11A-based gene therapy for sickle cell disease passes key preclinical test

September 06, 2016
A precision-engineered gene therapy virus, inserted into blood stem cells that are then transplanted, markedly reduced sickle-induced red-cell damage in mice with sickle cell disease, according to a study in the Journal of Clinical Investigation. The work sets the stage for bringing a decades-old discovery about sickle cell disease to the bedside. A clinical gene therapy trial, using a virus rendered harmless in the laboratory, is expected to launch in 2017.
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.
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.
StuartOrkin_Alt

Gene therapy offers hope for treatment of sickle cell anemia

October 01, 2016
Scientists are finalizing plans to use gene therapy to treat one of the world’s most widespread inherited diseases – sickle cell anemia, the Guardian reports. The technique could begin trials next year, say researchers.
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.
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

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.
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.
David Williams, MD

New gene therapy clinical trial offers hope for sickle cell disease

November 14, 2017
David Williams, MD, discusses Dana-Farber/Boston Children's new gene therapy clinical trial for sickle cell disease in a live webchat.
David Williams, MD

New gene therapy strategy for sickle cell disease shows early promise in humans

December 01, 2018
A patient with a severe form of sickle cell disease is symptom-free after gene therapy knocks down BCL11A and restores fetal hemoglobin production.
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