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Scientists at Weill Cornell Medical College have developed a gene therapy strategy that could feasibly treat both beta-thalassemia and sickle cell disease. The technology is based on delivery of a lentiviral vector carrying both the human β-globin gene and an ankyrin insulator to improve gene transcription and translation, and boost levels of ...
Hydroxycarbamide, also known as hydroxyurea, is a medication used in sickle-cell disease, essential thrombocythemia, chronic myelogenous leukemia, polycythemia vera, and cervical cancer. [4] [5] In sickle-cell disease it increases fetal hemoglobin and decreases the number of attacks. [4] It is taken by mouth. [4]
The person may need to be monitored for several days in the hospital after the transfusion, but the length of stay generally depends on the condition for which the exchange transfusion was performed. Sickle Cell Disease patients may be exchanged in an outpatient setting and can be sent home the very same day. [13]
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.
Hereditary xerocytosis occurs more commonly in African populations, [2] [4] and it exhibits complex interactions with other hereditary alterations of red blood cells, including sickle cell disease [4] [5] and malaria resistance. [4] [6] Osmosis leads to the red blood cell having a constant tendency to swell and burst.
A similar relationship exists between malaria and sickle-cell disease. One theory to explain this is that cells infected with the Plasmodium parasite are cleared more rapidly by the spleen . This phenomenon might give G6PD deficiency carriers an evolutionary advantage by increasing their fitness in malarial endemic environments.
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