Article

Systemic delivery of a splice-switching oligonucleotide heteroduplex corrects splicing in central nervous system and muscle in spinal muscular atrophy mice

2024-01-25

Abstract excerpt

<h4>ABSTRACT</h4> Oligonucleotide therapeutics are an established class of drugs for the treatment of genetic disorders. Their clinical development is challenging, however, as they typically distribute poorly to extra-hepatic tissues after systemic injection. Here we tested the heteroduplex oligonucleotide (HDO) platform for systemic delivery of SMN2 splice-switching oligonucleotides of 2’- O- methoxyethyl phos...

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Literature Corpus work: 91d158fe-9d61-5705-b6b6-4f52d5e3d692
DOI: 10.1101/2024.01.24.577012

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