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Advanced filters: Author: Rrita Daci Clear advanced filters

  • A phase 1/2 trial of dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis, administered by bilateral intrathalamic, cisterna magna and intrathecal delivery found a dose-dependent biochemical correction of the disease.

    • Florian Eichler
    • Oguz I. Cataltepe
    • Terence R. Flotte
    ResearchOpen Access
    Nature Medicine
    Volume: 31, P: 2927-2935