(September 10, 2019 by Doris Duke Charitable Foundation) – The Doris Duke Charitable Foundation today announced the six projects receiving approximately $5 million through the second Sickle Cell Disease/Advancing Cures grant competition. Four of the researchers who won support for their projects will study aspects of gene editing that could improve its successful clinical application in patients with sickle cell disease. The other two researchers were awarded grants to investigate new drug-like molecules and potential new drug targets to promote expression of functional hemoglobin. Adult hemoglobin is a protein in red blood cells that is mutated in sickle cell disease, which disrupts its ability to transport oxygen through the body.
“With the rise of CRISPR gene editing and many other promising developments, this is an exciting time for sickle cell disease research and an opportunity to make important strides toward delivering a cure,” said Betsy Myers, Program Director for Medical Research at the Doris Duke Charitable Foundation. “We are thrilled to support these clinical researchers as they harness the power of recent progress in the field in efforts to develop new ways to advance gene therapies and restore red blood cell function.”
This announcement coincides with National Sickle Cell Awareness Month, which began in 1983 to foster public awareness about the genetic disease that researchers estimate affects between 90,000 to 100,000 Americans. It is the most common inherited blood disorder in the United States, and approximately 300,000 people worldwide are born with sickle cell disease each year. Patients with sickle cell disease carry dysfunctional red blood cells that alter regular blood flow, which translates into pain, poor organ oxygenation and organ damage, and a life span of only about 40 years.
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