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Until now, the only known cure for sickle cell disease was a bone marrow transplant from a donor, which carries the risk of rejection by the immune system, in addition to the difficult process of ...
114,800 (2015) [8] Sickle cell disease ( SCD ), also simply called sickle cell, is a group of hemoglobin-related blood disorders typically inherited. [2] The most common type is known as sickle cell anemia. [2] It results in an abnormality in the oxygen-carrying protein haemoglobin found in red blood cells. [2]
The post U.S. approves two sickle cell gene therapies, which doctors hope can cure the painful disorder appeared first on TheGrio. “Sickle cell disease is a rare, debilitating and life ...
Sickle cell is the most common disease to receive approval for gene therapy treatment, following decades of development and years of approvals for therapy for people with rarer conditions.
The risks and benefits related to gene therapy for sickle cell disease are not known. Gene therapy has been used in the eye. The eye is especially suitable for adeno-associated virus vectors. Luxturna is an approved gene therapy to treat Leber's hereditary optic neuropathy.
Voxelotor, sold under the brand name Oxbryta, is a medication used for the treatment of sickle cell disease. Voxelotor is the first hemoglobin oxygen-affinity modulator. Voxelotor has been shown to have disease-modifying potential by increasing hemoglobin levels and decreasing hemolysis indicators in sickle cell patients.
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