Modulation of MKNK2 alternative splicing by splice-switching oligonucleotides as a novel approach for glioblastoma treatment
Mogilevsky M, Shimshon O, Kumar S, Mogilevsky A, Keshet E, Yavin E, Heyd F, Karni R. Nucleic Acids Res. 2018 Oct 17. PubMed
Optimized base editors enable efficient editing in cells, organoids and mice
Zafra MP, Schatoff EM, Katti A, Foronda M, Breinig M, Schweitzer AY, Simon A, Han T, Goswami S, Montgomery E, Thibado J, Kastenhuber ER, SĂ¡nchez-Rivera FJ, Shi J, Vakoc CR, Lowe SW, Tschaharganeh DF, Dow LE. Nat Biotechnol. 2018 Oct;36(9):888-893. PubMed
Improving cytidine and adenine base editors by expression optimization and ancestral reconstruction
Koblan LW, Doman JL, Wilson C, Levy JM, Tay T, Newby GA, Maianti JP, Raguram A, Liu DR. Nat Biotechnol. 2018 Oct;36(9):843-846. PubMed
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
Rossidis AC, Stratigis JD, Chadwick AC, Hartman HA, Ahn NJ, Li H, Singh K, Coons BE, Li L, Lv W, Zoltick PW, Alapati D, Zacharias W, Jain R, Morrisey EE, Musunuru K, Peranteau WH. Nat Med. 2018 Oct;24(10):1513-1518. PubMed
Genetic Modulation of RNA Splicing with a CRISPR-Guided Cytidine Deaminase
Yuan J, Ma Y, Huang T, Chen Y, Peng Y, Li B, Li J, Zhang Y, Song B, Sun X, Ding Q, Song Y, Chang X. Mol Cell. 2018 Oct 18;72(2):380-394.e7. PubMed
Several structural motifs cooperate in determining the highly effective anti-thrombin activity of NU172 aptamer
Troisi R, Napolitano V, Spiridonova V, Russo Krauss I, Sica F. Nucleic Acids Res. 2018 Oct 24. PubMed
Osteopontin mediates glioblastoma-associated macrophage infiltration and is a therapeutic target
Wei J, Marisetty A, Schrand B, Gabrusiewicz K, Hashimoto Y, Ott M, Grami Z, Kong LY, Ling X, Caruso HG, Zhou S, Wang YA, Fuller GN, Huse JT, Gilboa E, Kang N, Huang X, Verhaak R, Li S, Heimberger AB. J Clin Invest. 2018 Oct ...
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