Fatty acid conjugation enhances potency of antisense oligonucleotides in muscle
Prakash TP, Mullick AE, Lee RG, Yu J, Yeh ST, Low A, Chappell AE, Østergaard ME, Murray S, Gaus HJ, Swayze EE, Seth PP. Nucleic Acids Res. 2019 May 25. PubMed
Targeting Huntingtin Expression in Patients with Huntington’s Disease
Tabrizi SJ, Leavitt BR, Landwehrmeyer GB, Wild EJ, Saft C, Barker RA, Blair NF, Craufurd D, Priller J, Rickards H, Rosser A, Kordasiewicz HB, Czech C, Swayze EE, Norris DA, Baumann T, Gerlach I, Schobel SA, Paz E, Smith AV, Bennett CF, Lane RM. N ...
Two-in-One Chemogene Assembled from Drug-Integrated Antisense Oligonucleotides To Reverse Chemoresistance
Mou Q, Ma Y, Ding F, Gao X, Yan D, Zhu X, Zhang C. J Am Chem Soc. 2019 May 1;141(17):6955-6966. PubMed
Site-specific replacement of phosphorothioate with alkyl phosphonate linkages enhances the therapeutic profile of gapmer ASOs by modulating interactions with cellular proteins
Migawa MT, Shen W, Wan WB, Vasquez G, Oestergaard ME, Low A, De Hoyos CL, Gupta R, Murray S, Tanowitz M, Bell M, Nichols JG, Gaus H, Liang XH, Swayze EE, Crooke ST, Seth PP. Nucleic Acids Res. 2019 Apr 29. pii: gkz247. PubMed
Duplex RNAs and ss-siRNAs Block RNA Foci Associated with Fuchs’ Endothelial Corneal Dystrophy
Hu J, Shen X, Rigo F, Prakash TP, Mootha VV, Corey DR. Nucleic Acid Ther. 2019 Apr;29(2):73-81. PubMed
An HK2 antisense oligonucleotide induces synthetic lethality in HK1-HK2+ multiple myeloma
Xu S, Zhou T, Doh HM, Trinh KR, Catapang A, Lee JT, Braas D, Bayley NA, Yamada RE, Vasuthasawat A, Sasine JP, Timmerman JM, Larson SM, Kim Y, Macleod AR, Morrison SL, Herschman HR. Cancer Res. 2019 Mar 18. pii: canres.2799.2018. PubMed
Differential uptake, kinetics and mechanisms of intracellular trafficking of next-generation antisense oligonucleotides across human cancer cell lines
Linnane E, Davey P, Zhang P, Puri S, Edbrooke M, Chiarparin E, Revenko AS, Macleod AR, Norman JC, Ross SJ. Nucleic Acids Res. 2019 Mar 30. PubMed
Pnpla3 silencing with antisense oligonucleotides ameliorates nonalcoholic steatohepatitis and fibrosis in Pnpla3 I148M knock-in mice
Lindén D, Ahnmark A, Pingitore P, Ciociola E, Ahlstedt I, Andréasson AC, Sasidharan K, Madeyski-Bengtson K, Zurek M, Mancina RM, Lindblom A, Bjursell M, Böttcher G, Ståhlman M, Bohlooly-Y M, Haynes WG, Carlsson B, Graham M, Lee R, Murray S, Valenti L, Bhanot S, Åkerblad ...
IONIS-PKKRx a Novel Antisense Inhibitor of Prekallikrein and Bradykinin Production
Ferrone JD, Bhattacharjee G, Revenko AS, Zanardi TA, Warren MS, Derosier FJ, Viney NJ, Pham NC, Kaeser GE, Baker BF, Schneider E, Hughes SG, Monia BP, MacLeod AR. Nucleic Acid Ther. 2019 Feb 28. PubMed
Antisense Inhibition of Glucagon Receptor by IONIS-GCGRRx Improves Type 2 Diabetes Without Increase in Hepatic Glycogen Content in Patients With Type 2 Diabetes on Stable Metformin Therapy
Morgan ES, Tai LJ, Pham NC, Overman JK, Watts LM, Smith A, Jung SW, Gajdošík M, Krššák M, Krebs M, Geary RS, Baker BF, Bhanot S. Diabetes Care. 2019 Feb 14. pii: dc181343. PubMed
