Suppression of proteolipid protein rescues Pelizaeus-Merzbacher disease
Elitt MS, Barbar L, Shick HE, Powers BE, Maeno-Hikichi Y, Madhavan M, Allan KC, Nawash BS, Gevorgyan AS, Hung S, Nevin ZS, Olsen HE, Hitomi M, Schlatzer DM, Zhao HT, Swayze A, LePage DF, Jiang W, Conlon RA, Rigo F, Tesar PJ. Nature. 2020 Jul ...
Phosphorothioate modified oligonucleotide-protein interactions
Crooke ST, Vickers TA, Liang XH. Nucleic Acids Res. 2020 Jun 4;48(10):5235-5253. Pubmed
Novel antisense inhibition of diacylglycerol O-acyltransferase 2 for treatment of non-alcoholic fatty liver disease: a multicentre, double-blind, randomised, placebo-controlled phase 2 trial
Loomba R, Morgan E, Watts L, Xia S, Hannan LA, Geary RS, Baker BF, Bhanot S. Lancet Gastroenterol Hepatol. 2020 Jun 15:S2468-1253(20)30186-2. Pubmed
Reversing a model of Parkinson’s disease with in situ converted nigral neurons
Qian H, Kang X, Hu J, Zhang D, Liang Z, Meng F, Zhang X, Xue Y, Maimon R, Dowdy SF, Devaraj NK, Zhou Z, Mobley WC, Cleveland DW, Fu XD. Nature. 2020 Jun;582(7813):550-556. Pubmed
Technical Considerations for Use of Oligonucleotide Solution API
Muslehiddinoglu J, Simler R, Hill ML, Mueller C, Amery JHA, Dixon L, Watson A, Storch K, Gazziola C, Gielen F, Lange SA, Prail JD, Nesta DP. Nucleic Acid Ther. 2020 May 6. Pubmed
Selective Naked-Eye Detection of SARS-CoV-2 Mediated by N Gene Targeted Antisense Oligonucleotide Capped Plasmonic Nanoparticles
Moitra P, Alafeef M, Dighe K, Frieman M, Pan D. ACS Nano. 2020 May 28:acsnano.0c03822. Pubmed
Detailed cellular assessment of albumin-bound oligonucleotides: Increased stability and lower non-specific cell uptake
Lacroix A, Fakih HH, Sleiman HF. J Control Release. 2020 Apr 21. PubMed
Cationic Amphiphilic Drugs Boost the Lysosomal Escape of Small Nucleic Acid Therapeutics in a Nanocarrier-Dependent Manner
Van de Vyver T, Bogaert B, De Backer L, Joris F, Guagliardo R, Van Hoeck J, Merckx P, Van Calenbergh S, Ramishetti S, Peer D, Remaut K, De Smedt SC, Raemdonck K. ACS Nano. 2020 Apr 28;14(4):4774-4791. PubMed
Population Pharmacokinetic-Pharmacodynamic Modeling of Inotersen, an Antisense Oligonucleotide for Treatment of Patients with Hereditary Transthyretin Amyloidosis
Yu RZ, Collins JW, Hall S, Ackermann EJ, Geary RS, Monia BP, Henry SP, Wang Y. Nucleic Acid Ther. 2020 Apr 15. PubMed
An Antisense Oligonucleotide Leads to Suppressed Transcription of Hdac2 and Long-Term Memory Enhancement
Poplawski SG, Garbett KA, McMahan RL, Kordasiewicz HB, Zhao H, Kennedy AJ, Goleva SB, Sanders TH, Motley ST, Swayze EE, Ecker DJ, Sweatt JD, Michael TP, Greer CB. Mol Ther Nucleic Acids. 2020 Mar 6;19:1399-1412. PubMed