Abstract
A multitude of maleimides are grafted onto the backbone of a phosphorothioate antisense oligonucleotide (ASO) to generate the construct of maleimide-grafted ASO (Mal-g-ASO). Through click conjugation with cell membrane thiols that triggers endocytosis-independent cellular internalization, Mal-g-ASO exhibited enhanced cellular uptake efficiency, resulting in a remarkable improvement of ASO-based gene silencing.
MeSH terms
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Animals
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Biocompatible Materials / chemistry
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Biocompatible Materials / metabolism
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Cell Line
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Endocytosis
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Fluorescent Dyes / chemistry
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Gene Silencing*
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Humans
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Maleimides / chemistry*
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Mice
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Microscopy, Confocal
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Oligonucleotides, Antisense / chemistry
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Oligonucleotides, Antisense / metabolism*
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Phosphates / chemistry
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Rats
Substances
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Biocompatible Materials
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Fluorescent Dyes
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Maleimides
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Oligonucleotides, Antisense
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Phosphates
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phosphorodithioic acid