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Advanced filters: Author: Douglas E. Feltner Clear advanced filters

  • Biodistribution analysis of two patients with spinal muscular atrophy shows widespread onasemnogene abeparvovec DNA, mRNA and SMN protein throughout the central nervous system and peripheral organs following intravenous gene therapy administration.

    • Gretchen Thomsen
    • Arthur H. M. Burghes
    • Kevin D. Foust
    Research
    Nature Medicine
    Volume: 27, P: 1701-1711