Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue.
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DOI: 10.1038/s41591-021-01483-7
Abstract: Spinal muscular atrophy type 1 (SMA1) is a debilitating neurodegenerative disease resulting from survival motor neuron 1 gene (SMN1) deletion/mutation. Onasemnogene abeparvovec (formerly AVXS-101) is a gene therapy that restores SMN production via one-time systemic… read more here.
Keywords: abeparvovec dna; administration; biodistribution; smn protein ... See more keywords