Ex vivo gene therapy for cystinosis had acceptable safety and improved disease biomarkers
1. In this phase 1–2 open-label study, autologous lentiviral hematopoietic stem-cell gene therapy for cystinosis was associated with sustained engraftment ...
Tag: Lentiviral gene therapy
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Lentiviral gene therapy prevents functional deterioration in cerebral adrenoleukodystrophy patients
1. In this prospective cohort study, in boys with diagnosed cerebral adrenoleukodystrophy, it was found that lentiviral gene therapy containing ...
