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Optimistic, Though Cautious

Some sickle cell patients are concerned that new gene-editing approaches for treating the disease might not be widely available or affordable, MIT's Technology Review reports.

As the New York Times noted earlier this year, sickle cell disease has been an attractive candidate for gene therapies and there are three broad approaches companies are taking to address the disease: genetically modifying immature blood cells from patients' bone marrow, activating fetal hemoglobin, and using CRISPR for gene editing. This summer, Vertex Pharmaceuticals and CRISPR Therapeutics began a US trial in which they are using CRISPR to modify cells from patients' bone marrow to produce fetal hemoglobin.

Vince Bonham, senior advisor to the NHGRI director on genomics and health disparities, said at Tech Review's EmTech conference that while it is an "it's an exciting time" from a scientific view for people with sickle cell, more work is needed from a sociological view. He and his colleagues recently surveyed individuals from within the sickle cell community and as they reported in Genetics in Medicine last December, they found that while patients, parents, and physicians are cautiously optimistic about gene editing, they are also concerned that after testing in sickle cell patients, the tool would then be applied to treat other diseases at sickle cell's expense.

"The potential is great, but we must ask the question: Who will benefit?" Bonham said, according to Tech Review.

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