Enzymatic synthesis of hypermodified DNA polymers for sequence-specific display of four different hydrophobic groups
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Intracellular delivery of therapeutic antisense oligonucleotides targeting mRNA coding mitochondrial proteins by cell-penetrating peptides
Cerrato CP, Kivijärvi T, Tozzi R, Lehto T, Gestin M, Langel Ăœ. J Mater Chem B. 2020 Epub 2020 Nov 11. Pubmed
Immunogenicity Assessment of Inotersen, a 2′-O-(2-Methoxyethyl) Antisense Oligonucleotide in Animals and Humans: Effect on Pharmacokinetics, Pharmacodynamics, and Safety
Yu RZ, Wang Y, Norris DA, Kim TW, Narayanan P, Geary RS, Monia BP, Henry SP. Nucleic Acid Ther. 2020 Oct;30(5):265-275. Pubmed
Synthesis, chirality-dependent conformational and biological properties of siRNAs containing 5′-(R)- and 5′-(S)-C-methyl-guanosine
Mikami A, Erande N, Matsuda S, Kel'in A, Woods LB, Chickering T, Pallan PS, Schlegel MK, Zlatev I, Egli M, Manoharan M. Nucleic Acids Res. 2020 Sep 29:gkaa750. Pubmed
Synthesis and Characterization of Thiophosphoramidate Morpholino Oligonucleotides and Chimeras
Langner HK, Jastrzebska K, Caruthers MH. J Am Chem Soc. 2020 Aug 31. Pubmed
A short de novo synthesis of nucleoside analogs
Meanwell M, Silverman SM, Lehmann J, Adluri B, Wang Y, Cohen R, Campeau LC, Britton R. Science. 2020 Aug 7;369(6504):725-730. Pubmed
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
Wei T, Cheng Q, Min YL, Olson EN, Siegwart DJ. Nat Commun. 2020 Jun 26;11(1):3232. Pubmed
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
Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, Smith EC, McDonald CM, Zaidman CM, Morgenroth LP, Osaki H, Satou Y, Yamashita T, Hoffman EP; CINRG DNHS Investigators. JAMA Neurol. 2020 May 26 Pubmed