Characterization of the Activity and Distribution of a 2′-O-Methoxyethyl-modified Antisense Oligonucleotide in Models of Acute and Chronic Kidney Disease
Donner AJ, Bell TA, Greenlee S, Graham MJ, Crooke RM. Nucleic Acid Ther. 2018 Aug 22. PubMed
A platform for discovery of functional cell-penetrating peptides for efficient multi-cargo intracellular delivery
Hoffmann K, Milech N, Juraja SM, Cunningham PT, Stone SR, Francis RW, Anastasas M, Hall CM, Heinrich T, Bogdawa HM, Winslow S, Scobie MN, Dewhurst RE, Florez L, Ong F, Kerfoot M, Champain D, Adams AM, Fletcher S, Viola HM, Hool LC, Connor T, Longville ...
Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models
McCampbell A, Cole T, Wegener AJ, Tomassy GS, Setnicka A, Farley BJ, Schoch KM, Hoye ML, Shabsovich M, Sun L, Luo Y, Zhang M, Thankamony S, Salzman DW, Cudkowicz M, Graham DL, Bennett CF, Kordasiewicz HB, Swayze EE, Miller TM. J Clin Invest. 2018 Jul ...
Inhibiting neutral amino acid transport for the treatment of phenylketonuria
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Therapeutic vulnerability of multiple myeloma to MIR17PTi, a first-in-class inhibitor of pri-mir-17-92
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Complex consisting of antisense DNA and β-glucan promotes internalization into cell through Dectin-1 and hybridizes with target mRNA in cytosol
Fujiwara N, Izumi H, Morimoto Y, Sakurai K, Mochizuki S. Cancer Gene Ther. 2018 Jul 4. PubMed
Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis
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PTBP1-Mediated Alternative Splicing Regulates the Inflammatory Secretome and the Pro-tumorigenic Effects of Senescent Cells
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Liver-Targeted Anti-HBV Single-Stranded Oligonucleotides with Locked Nucleic Acid Potently Reduce HBV Gene Expression In Vivo
Javanbakht H, Mueller H, Walther J, Zhou X, Lopez A, Pattupara T, Blaising J, Pedersen L, Albæk N, Jackerott M, Shi T, Ploix C, Driessen W, Persson R, Ravn J, Young JAT, Ottosen S. Mol Ther Nucleic Acids. 2018 Jun 1;11:441-454. PubMed
