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Gene Therapy
Phase III
AAV9 Gene Transfer in Spinal Muscular Atrophy
Perspectivum
619,
31-41 (2023)
·
10.1038/s41586-023-06328-2
·
NCT03505073
·
4:48
Intravenous administration, retrograde motor neuron tropism, nuclear import, and episomal SMN1 transgene expression in lumbar anterior horn cells following onasemnogene abeparvovec delivery.
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