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Researchers from the Leiden University Medical Center (LUMC) have successfully used stem cell gene therapy to treat a baby with the severe congenital immune disorder SCID. An important milestone: it is the first time stem cell gene therapy of Dutch o
Share Researchers and clinicians at Leiden University Medical Centre (LUMC) are about to begin a milestone clinical study. It will be the first time a stem cell gene therapy developed in the Netherlands is used in a clinic. The therapy will be used to treat children with SCID, a rare disorder where children are born without essential immune cells. ‘We are the first group worldwide to have developed stem cell gene therapy for this type of SCID, RAG1-SCID to be precise. It is also the first stem cell gene therapy initiated by the Netherlands that will actually be used in patients,’ says Pro...