Oligonucleotide therapy mitigates disease in Spinocerebellar Ataxia Type 3 mice
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CHP1 reduction ameliorates spinal muscular atrophy pathology by restoring calcineurin activity and endocytosis
Janzen E, Mendoza-Ferreira N, Hosseinibarkooie S, Schneider S, Hupperich K, Tschanz T, Grysko V, Riessland M, Hammerschmidt M, Rigo F, Bennett CF, Kye MJ, Torres-Benito L, Wirth B. Brain. 2018 Jun 28. PubMed
Efficient RNA drug delivery using red blood cell extracellular vesicles
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Identifying and avoiding off-target effects of RNase H-dependent antisense oligonucleotides in mice
Hagedorn PH, Pontoppidan M, Bisgaard TS, Berrera M, Dieckmann A, Ebeling M, Møller MR, Hudlebusch H, Jensen ML, Hansen HF, Koch T, Lindow M. Nucleic Acids Res. 2018 May 22. PubMed
Therapeutic silencing of FSP27 reduces the progression of atherosclerosis in Ldlr-/- mice
Rajamoorthi A, Lee RG, Baldán Á. Atherosclerosis. 2018 May 24;275:43-49. PubMed
H19 Induces Abdominal Aortic Aneurysm Development and Progression
Li DY, Busch A, Jin H, Chernogubova E, Pelisek J, Karlsson J, Sennblad B, Liu S, Lao S, Hofmann P, Bäcklund A, Eken SM, Roy J, Eriksson P, Dacken B, Ramanujam D, Dueck A, Engelhardt S, Boon RA, Eckstein HH, Spin JM, Tsao PS, Maegdefessel ...
Machine Learning To Predict Cell-Penetrating Peptides for Antisense Delivery
Wolfe JM, Fadzen CM, Choo ZN, Holden RL, Yao M, Hanson GJ, Pentelute BL. ACS Cent Sci. 2018 Apr 25;4(4):512-520. PubMed
Chronic Toxicity Assessment of 2′-O-Methoxyethyl Antisense Oligonucleotides in Mice
Zanardi TA, Kim TW, Shen L, Serota D, Papagiannis C, Park SY, Kim Y, Henry SP. Nucleic Acid Ther. 2018 Apr 30. PubMed
Intracerebroventricular Administration of a 2′-O-Methyl Phosphorothioate Antisense Oligonucleotide Results in Activation of the Innate Immune System in Mouse Brain
Toonen LJA, Casaca-Carreira J, Pellisé-Tintoré M, Mei H, Temel Y, Jahanshahi A, van Roon-Mom WMC. Nucleic Acid Ther. 2018 Apr;28(2):63-73. PubMed
Structural Determinants for the Interactions of Chemically Modified Nucleic Acids with the Stabilin-2 Clearance Receptor
Gaus H, Miller CM, Seth PP, Harris EN. Biochemistry. 2018 Apr 10;57(14):2061-2064. PubMed
