Intracellular delivery of therapeutic antisense oligonucleotides targeting mRNA coding mitochondrial proteins by cell-penetrating peptides
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Determination of isoform-specific RNA structure with nanopore long reads
Aw JGA, Lim SW, Wang JX, Lambert FRP, Tan WT, Shen Y, Zhang Y, Kaewsapsak P, Li C, Ng SB, Vardy LA, Tan MH, Nagarajan N, Wan Y. Nat Biotechnol. 2020 Oct 26 Pubmed
Immunogenicity Assessment of Inotersen, a 2′-O-(2-Methoxyethyl) Antisense Oligonucleotide in Animals and Humans: Effect on Pharmacokinetics, Pharmacodynamics, and Safety
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Synthesis, chirality-dependent conformational and biological properties of siRNAs containing 5′-(R)- and 5′-(S)-C-methyl-guanosine
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Synthesis and Characterization of Thiophosphoramidate Morpholino Oligonucleotides and Chimeras
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A short de novo synthesis of nucleoside analogs
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Targeted Delivery of Antisense Oligonucleotides Using Neurotensin Peptide.
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Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing
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Defining the Design Parameters for in Vivo Enzyme Delivery Through Protein Spherical Nucleic Acids
Kusmierz CD, Bujold KE, Callmann CE, Mirkin CA. ACS Cent Sci. 2020 May 27;6(5):815-822. Pubmed
Safety, Tolerability, and Efficacy of Viltolarsen in Boys with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial
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