Plans for a Moonshot

The American Society of Hematology (ASH) plans to call for a sickle cell moonshot this fall—an all-out effort that has been long in coming.

Researchers in 1956 discovered the mutation in the gene for hemoglobin that causes sickle cell. It has led to neither treatments nor cures for the 300,000 babies worldwide—1/3 of them in the US—born annually with the painful disease. “Sickle cell patients have never been at the front of the line,” said David Nathan, a past president of Dana-Farber, citing a paucity of support from scientists, funders and the government.

Thanks in part to the development of genome-editing technology, a cure is within reach, said ASH vice president Dr. Alexis Thompson of Children’s Hospital of Chicago.
 

STAT

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