A man and woman who are both carriers of the sickle cell anemia mutation can have a healthy child by fertilizing eggs in a lab, then deciding which of multiple embryos that develop from the eggs will become the baby, a case study indicates. It also showed that umbilical cord…
Crizanlizumab (SEG101), manufactured by Switzerland’s Novartis, delays the time to first sickle cell pain crisis (SCPC) in adults with sickle cell disease, according to results from a subgroup analysis of the Phase II SUSTAIN study (NCT01895361). Researchers presented the abstract, “Crizanlizumab 5.0 Mg/Kg Increased the Time to First on-Treatment…
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…
Howard University researchers have discovered a link between increases in Veillonella gut bacteria and higher levels of pain in sickle cell disease patients. The team presented their findings at the American Physiological Society’s Physiological and Pathophysiological Consequences of Sickle Cell Disease conference in Washington, Nov. 6-8. Sickle cell disease is a blood…
The Medical University of South Carolina (MUSC) was recently awarded a Doris Duke Charitable Foundation grant to develop a new gene-modifying therapy for the treatment of sickle cell disease. The project, “Epigenetic Modulators for the Treatment of Sickle Cell Disease,” was awarded more than $750,000 as part of the…
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