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The successful pathway proved far more complex and challenging than initially imagined, says lead investigator gene therapist Philippe Leboulch, of the Massachusetts Institute of Technology and Harvard University. "Everybody thought it would be the first genetic disorder cured by gene therapy, that it would be simple, but it turned out to be completely different. It was a real challenge," says Leboulch, who has worked with SCD for more than 10 years.
Two key factors made SCD a promising candidate for gene therapy research. First, the sickle cell mutation is a single point alteration in the human bA-globin ...
