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Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial

Ad-hoc AnalysisCell TherapyBone Marrow FailureAutoimmune DiseaseEarly-onset Metachromatic LeukodystrophyMedicineHematologyPathologyStem-cell TherapyStem CellsHealth Sciences