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May 31, 2022 News by Lindsey Shapiro, PhD

2 Proteins That Silence Fetal Hemoglobin May Be Therapy Target

Interfering with two newly identified proteins — NFIA and NFIX — involved both directly and indirectly in silencing fetal hemoglobin in red blood cells, could be a new therapeutic approach for sickle cell disease (SCD), according to researchers. Their hypothesis is that inhibiting the production or activity of these…

April 14, 2022 News by Margarida Maia, PhD

Researcher Awarded for Work That Could ‘Revolutionize’ SCD Therapy

A renowned stem cell biologist has been honored with the 2022 Canada Gairdner International Award for his work that set the grounds for a new approach to treating sickle cell disease (SCD) and beta-thalassemia. Stuart H. Orkin, MD, is the David G. Nathan Distinguished Professor of Pediatrics at…

December 12, 2017 News by Iqra Mumal, MSc

Intellia Uses Gene Editing to Replace Defective Blood Component in Sickle Cell Disease

Intellia Therapeutics has used gene editing to replace an oxygen-carrying component in blood that is defective in sickle cell disease. The work involved editing the BCL11A gene in a mouse model of the disease to produce an effective version of the oxygen-carrying component, hemoglobin. Intellia discussed the project at the…

July 6, 2017 News by Alice Melão

Researchers Identify Genetic Predictors of Sickle Cell Anemia-Related Complications

Patients with sickle cell anemia who do not have alpha-thalassemia and the genetic rs1427407 T variant of the BCL11A gene may be at higher risk for hemolysis (rupture of red blood cells) and stroke, according to the results of studies of three independent groups. These findings were featured in…

Recent Posts

  • SCD, sickle cell trait may increase risk of diabetic eye disease: Study
  • On sickle cell habitus, the physical symptoms of severe cases
  • Top-line data from Phase 3 trial of mitapivat expected this year
  • Hydroxyurea may help ease sleep issues in children: Study
  • Why some SCD patients give birth to children with the disease
  • You just found out your child has sickle cell disease. Now what?
  • PIONEER trial of pociredir still recruiting sickle cell disease patients
  • Guest Voice: 3 coping tools help my fight against sickle cell disease
  • CT scans for lung blood clots in sickle cell increased after 2020
  • Could gene therapy improve my life as an adult with sickle cell?


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