Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy
Amoasii L, Hildyard JCW, Li H, Sanchez-Ortiz E, Mireault A, Caballero D, Harron R, Stathopoulou TR, Massey C, Shelton JM, Bassel-Duby R, Piercy RJ, Olson EN. Science. 2018 Oct 5;362(6410):86-91. PubMed
Treatment of a metabolic liver disease by in vivo genome base editing in adult mice
Villiger L, Grisch-Chan HM, Lindsay H, Ringnalda F, Pogliano CB, Allegri G, Fingerhut R, Häberle J, Matos J, Robinson MD, Thöny B, Schwank G. Nat Med. 2018 Oct;24(10):1519-1525. PubMed
In utero CRISPR-mediated therapeutic editing of metabolic genes
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Genetic Modulation of RNA Splicing with a CRISPR-Guided Cytidine Deaminase
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Several structural motifs cooperate in determining the highly effective anti-thrombin activity of NU172 aptamer
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Osteopontin mediates glioblastoma-associated macrophage infiltration and is a therapeutic target
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Evaluation of the effect of 2′-O-methyl, fluoro hexitol, bicyclo and Morpholino nucleic acid modifications on potency of GalNAc conjugated antisense oligonucleotides in mice
Prakash TP, Yu J, Kinberger GA, Low A, Jackson M, Rigo F, Swayze EE, Seth PP. Bioorg Med Chem Lett. 2018 Oct PubMed
Targeted delivery of antisense oligonucleotides to pancreatic β-cells
Ämmälä C, Drury WJ 3rd, Knerr L, Ahlstedt I, Stillemark-Billton P, Wennberg-Huldt C, Andersson EM, Valeur E, Jansson-Löfmark R, Janzén D, Sundström L, Meuller J, Claesson J, Andersson P, Johansson C, Lee RG, Prakash TP, Seth PP, Monia BP, Andersson S. Sci Adv. 2018 Oct ...
STAT3 Inhibition Combined with CpG Immunostimulation Activates Antitumor Immunity to Eradicate Genetically Distinct Castration-Resistant Prostate Cancers
Moreira D, Adamus T, Zhao X, Su YL, Zhang Z, White SV, Swiderski P, Lu X, DePinho RA, Pal SK, Kortylewski M. Clin Cancer Res. 2018 Oct 18. PubMed
Inferring Half-Lives at the Effect Site of Oligonucleotide Drugs
Jansson-Löfmark R, Gennemark P. Nucleic Acid Ther. 2018 Oct 31. PubMed