Sickle Cell Treatments Improve; Genetic Cure On The Horizon

WORTH WAITING FOR: Kwaku Ohene-Frempong of the Sickle Cell Disease Association of America says "gene therapy is the ultimate cure of sickle cell disease." Back in 1949, Linus Pauling determined that sickle cell disease-an inherited blood disorder that afflicts 1 in 400 African Americans-is caused by a defect in one of the genes that codes for hemoglobin. Now people with sickle cell disease are finally reaping the benefits of a half-century of research. Within the last two years, several treat

Robert Finn

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WORTH WAITING FOR: Kwaku Ohene-Frempong of the Sickle Cell Disease Association of America says "gene therapy is the ultimate cure of sickle cell disease."

Back in 1949, Linus Pauling determined that sickle cell disease-an inherited blood disorder that afflicts 1 in 400 African Americans-is caused by a defect in one of the genes that codes for hemoglobin. Now people with sickle cell disease are finally reaping the benefits of a half-century of research.

Within the last two years, several treatments have been developed that attack the cause and the significant symptoms of the disease. Bone-marrow transplantation can now provide a true cure for a small percentage of people with sickle cell disease. Two animal models have been developed. And researchers harbor realistic hopes that genetic therapy could actually cure the majority of sufferers.

SIGN OF A SYNDROME: A scanning electron micrograph of a sickle cell For many years sickle cell ...

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