An efficient method for the delivery of uncharged polyA-tailed phosphorodiamidate morpholino sequences (PMO) in mammalian cells consists of employing a synthetic 8-mer amphipathic trans-acting poly-2′-O-methyluridylic thiophosphate triester element (2′-OMeUtaPS) as a transfection reagent. Unlike the dTtaPS DNA-based element, this RNA element is potent at delivering polyA-tailed PMO sequences to HeLa pLuc 705 cells or to myotube muscle cells. However, much like dTtaPS, the 2′-OMeUtaPS-mediated internalization of PMO sequences occurs through an energy-dependent mechanism; macropinocytosis appears to be the predominant endocytic pathway used for cellular uptake. The transfected PMO sequences induce alternate splicing of either the pre-mRNA enc...
For subsets of Duchenne muscular dystrophy (DMD) mutations, antisense oligoribonucleotide (AON)-medi...
<p>Phosphorodiamidate morpholino oligonucleotides (PMOs) are a class of antisense oligonucleotides u...
Antisense oligonucleotides (AONs) are being developed as RNA therapeutic molecules for Duchenne musc...
Phosphorodiamidate morpholino oligomers (PMOs) are oligonucleotide analogs that can be used for ther...
The mdx mouse model of muscular dystrophy arose due to a nonsense mutation in exon 23 of the dystrop...
Cell-penetrating peptides (CPPs), containing arginine (R), 6-aminohexanoic acid (X), and/or β-alanin...
Themdxmouse model of muscular dystrophy arose due to a nonsense mutation in exon 23 of the dystrophi...
For subsets of Duchenne muscular dystrophy (DMD) mutations, antisense oligoribonucleotide (AON)-medi...
For subsets of Duchenne muscular dystrophy (DMD) mutations, antisense oligoribonucleotide (AON)-medi...
One of the crucial aspects of screening antisense oligonucleotides destined for therapeutic applicat...
Recent advances in drug development have seen numerous successful clinical translations using synthe...
2′-O-Methyl (2′-OMe) antisense oligonucleotides (AOs) possessing a various number of 4-(trimethylamm...
We describe two new methods of parallel chemical synthesis of libraries of peptide conjugates of pho...
The potential for therapeutic application of splice-switching oligonucleotides (SSOs) to modulate pr...
Abstract Antisense oligonucleotide (AO)‐mediated exon‐skipping therapies show promise in Duchenne mu...
For subsets of Duchenne muscular dystrophy (DMD) mutations, antisense oligoribonucleotide (AON)-medi...
<p>Phosphorodiamidate morpholino oligonucleotides (PMOs) are a class of antisense oligonucleotides u...
Antisense oligonucleotides (AONs) are being developed as RNA therapeutic molecules for Duchenne musc...
Phosphorodiamidate morpholino oligomers (PMOs) are oligonucleotide analogs that can be used for ther...
The mdx mouse model of muscular dystrophy arose due to a nonsense mutation in exon 23 of the dystrop...
Cell-penetrating peptides (CPPs), containing arginine (R), 6-aminohexanoic acid (X), and/or β-alanin...
Themdxmouse model of muscular dystrophy arose due to a nonsense mutation in exon 23 of the dystrophi...
For subsets of Duchenne muscular dystrophy (DMD) mutations, antisense oligoribonucleotide (AON)-medi...
For subsets of Duchenne muscular dystrophy (DMD) mutations, antisense oligoribonucleotide (AON)-medi...
One of the crucial aspects of screening antisense oligonucleotides destined for therapeutic applicat...
Recent advances in drug development have seen numerous successful clinical translations using synthe...
2′-O-Methyl (2′-OMe) antisense oligonucleotides (AOs) possessing a various number of 4-(trimethylamm...
We describe two new methods of parallel chemical synthesis of libraries of peptide conjugates of pho...
The potential for therapeutic application of splice-switching oligonucleotides (SSOs) to modulate pr...
Abstract Antisense oligonucleotide (AO)‐mediated exon‐skipping therapies show promise in Duchenne mu...
For subsets of Duchenne muscular dystrophy (DMD) mutations, antisense oligoribonucleotide (AON)-medi...
<p>Phosphorodiamidate morpholino oligonucleotides (PMOs) are a class of antisense oligonucleotides u...
Antisense oligonucleotides (AONs) are being developed as RNA therapeutic molecules for Duchenne musc...