mCherry on Top: A Positive Read-Out Cellular Platform for Screening DMD Exon Skipping Xenopeptide-PMO Conjugates

Lessl, AL; Pöhmerer, J; Lin, Y; Wilk, U; Höhn, M; Hörterer, E; Wagner, E; Lächelt, U

Lächelt, U (通讯作者),Ludwig Maximilians Univ Munchen, Pharmaceut Biotechnol Dept Pharm, D-81377 Munich, Germany.;Lächelt, U (通讯作者),Ludwig Maximilians Univ Munchen, Ctr Nanosci CeNS, D-80799 Munich, Germany.;Lächelt, U (通讯作者),Univ Vienna, Dept Pharmaceut Sci, A-1090 Vienna, Austria.

BIOCONJUGATE CHEMISTRY, 2023; 34 (12): 2263

Abstract

Phosphorodiamidate morpholino oligomers (PMOs) are a special type of antisense oligonucleotides (ASOs) that can be used as therapeutic modulators of p......

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