S-EPMC12433340 - Single-dose GC101 gene therapy for spinal muscular atrophy types II and III: an open-label single-arm study. - OmicsDI

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Single-dose GC101 gene therapy for spinal muscular atrophy types II and III: an open-label single-arm study.

ABSTRACT: QUICK TAKE.

SUBMITTER: Ma XW

PROVIDER: S-EPMC12433340 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature