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Systemic delivery of a splice-switching oligonucleotide heteroduplex corrects splicing in central nervous system and muscle in spinal muscular atrophy mice. https://www.biorxiv.org/content/10.1101/2024.01.24.577012v1 #Morpholino
Systemic delivery of a splice-switching oligonucleotide heteroduplex corrects splicing in central nervous system and muscle in spinal muscular atrophy mice. https://www.biorxiv.org/content/10.1101/2024.01.24.577012v1 #Morpholino