With sickle cell disease, the mutations in a person’s two copies of the HBB gene cause red blood cells to become rigid and crescent-shaped, resembling a sickle, rather than the usual round ...
Researchers described a promising new approach for using gene therapy to treat sickle cell disease in the journal Human Gene ...
This protection against malaria has kept the sickle cell gene mutation in the population. Sickle cell trait is found in up to ...
Cyclosporin H boosts a new lentivirus-delivered gene therapy with enhanced anti-sickling properties in blood stem cells from ...
It may be possible to collect nearly three times as many stem cells for sickle cell gene therapy with motixafortide than standard plerixafor.
ST. LOUIS — All morning, nurses had been coming in, taking vitals, offering blankets, pressing buttons to silence alarms. It ...
This fundamental biologic process is defined by a series of complex metabolic processes, which are often dysregulated in ...
Blood stem cells develop through different stages to become fully mature red blood cells. This fundamental biologic process ...
Excitement around the sickle cell treatment market has been palpable. Around the beginning of the year, two new breakthrough gene editing medications — including the first FDA-approved CRISPR ...
Editas Medicine said Tuesday it will license out or partner its lead sickle cell treatment, the latest shift for a tarantella company that has repeatedly switched direction and leadership since ...
people with sickle cell anemia (SCA), the most severe form of SCD, can often have a greater risk of death from malaria, compared with people who have SCT or no hemoglobin gene mutation.
CAMBRIDGE, Mass., Oct. 23, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare ...