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Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice

Paper ID

PAPER000281

Year

2019

DOI

10.1172/jci128205

PMID

31479430

Journal

Journal of Clinical Investigation

Source URL

Experiments

• EXP001685 with Pip6a-PMO-CAG7 (PMO (morpholino antisense oligonucleotide))
• EXP001686 with Pip6a-PMO-CAG7 (PMO (morpholino antisense oligonucleotide))
• EXP001687 with Pip6a-PMO-Ctrl (reverse GAC7 control) (PMO (morpholino antisense oligonucleotide))