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Diseases such as sickle cell disease that are caused by autosomal recessive disorders for which a person's normal phenotype or cell function may be restored in cells that have the disease by a normal copy of the gene that is mutated, may be a good candidate for gene therapy treatment.
Rodgers is a physician-scientist who studies diseases of bone marrow and is best known for his work on the molecular genetics of hemoglobinopathies and on developing a treatment for sickle cell anemia. [1] He was a major contributor to the development of hydroxyurea therapy, [5] which was approved by the FDA in 1998. [6]
The treatment, however, also sparks mixed feelings among both Black sickle cell patients and doctors, who are concerned about potential side effects, costs and access to the therapy, which could ...
Sickle cell nephropathy is a type of kidney disease associated with sickle cell disease which causes kidney complications as a result of sickling of red blood cells in the small blood vessels. The hypertonic and relatively hypoxic environment of the renal medulla , coupled with the slow blood flow in the vasa recta , favors sickling of red ...
It is possible for a person to have both the gene for hemoglobin S (the form associated with sickle cell anemia) and the gene for hemoglobin C; this state is called hemoglobin SC disease, and is generally more severe than hemoglobin C disease, but milder than sickle cell anemia.
The FDA on Friday also approved a second treatment for sickle cell disease, called Lyfgenia, a gene therapy from drugmaker Bluebird Bio. Both treatments work by genetically modifying a patient’s ...
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