sickle cell disease

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Sickle Cell Disease: Gene-Editing Tools Point to Possible Ultimate Cure

Posted on October 25, 2016 by Dr. Francis Collins Caption: An electron micrograph showing two red blood cells, one normal (right) and the other (left) deformed by crystalline hemoglobin into the “sickle” shape characteristic of patients with sickle cell disease. Credit: Frans Kuypers: RBClab.com, UCSF Benioff Children’s Hospital Oakland Scientists first described the sickle-shaped red

Investigational Therapy Could Attack Cause of Sickle Cell Crises

Mar. 2, 2015 By Duke Medicine News and Communications DURHAM, N.C. – Treatment for painful episodes of blood vessel obstruction in sickle cell anemia is currently limited to controlling pain, but an investigational therapy might be able to interfere with the underlying cause of these events, known as vaso-occlusion crises, researchers at Duke Medicine report.

Stem Cell Transplant Reverses Sickle Cell Disease in Adults

National Institutes of Health, by Staff ~ July 14, 2014 Sickle cell disease is an inherited blood disorder that affects more than 90,000 Americans, mostly of African descent. The condition arises from a genetic defect that alters the structure of hemoglobin, the oxygen-carrying protein found in red blood cells. The modified hemoglobin causes normally round

Studies uncover new insights into pathophysiology of sickle cell disease and thalassemia, may help improve standard of care

Medical News Today, by Staff ~ December 11, 2013 New research presented during the 55th American Society of Hematology Annual Meeting and Exposition in New Orleans uncovers several important insights into the pathophysiology of sickle cell disease and thalassemia that may soon translate into the development of better, more targeted treatments for hundreds of thousands

Newly Discovered Gene Regulator Could Precisely Target Sickle Cell Disease

Regenerative Medicine, by Staff ~ November 11, 2013 "Coupled with recent advances in technologies for gene engineering in intact cells, it could lead to powerful ways of manipulating hemoglobin production and new treatment options for hemoglobin diseases." –Dr. Stuart Orkin. A research team from Dana-Farber/Boston Children's Cancer and Blood Disorders Center and other institutions has

UCLA Stem Cell Therapy for Sickle Cell Disease Advances Toward Clinical Trials

Newswise, by Staff ~ July 1, 2013 Researchers at UCLA’s Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research have successfully established the foundation for using hematopoietic (blood-producing) stem cells (HSC) from the bone marrow of patients with sickle cell disease (SCD) to treat the disease. The study was led by Dr.