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Editing for Sickle Cell

People with sickle cell disease are hopeful that the gene-editing tool CRISPR might be able to offer them relief, Technology Review reports.

"Patients are very intrigued by [CRISPR]. They think it sounds wonderful," Biree Andemariam, director of the New England Sickle Cell Institute at the University of Connecticut Health Center, tells Tech Review.

Because sickle cell is caused by a single, well-studied gene mutation in the hemoglobin-encoding HBB gene, Tech Review says it is a viable candidate for editing and could be one of the first conditions to be treated that way.

In particular, it reports that CRISPR Therapeutics is pursuing a gene-editing treatment for sickle cell in which stem cells are isolated from patients' blood and are then edited to activate the fetal form of hemoglobin before being returned to the patient. The fetal hemoglobin then raises the level of healthy hemoglobin in red blood cells. In early tests, CRISPR Therapeutics' Samarth Kulkarni says they successfully modified 85 percent of cells.

Tech Review notes that St. Jude Children's Research Hospital, Editas Medicine, and Intellia Therapeutics are all exploring similar approaches. In addition, it says Stanford University researchers are exploring an approach that instead modifies the HBB gene and hope to begin a clinical trial at the end of the year or early next.