Treatment of influenza and SARS-CoV-2 infections via mRNA-encoded Cas13a in rodents
Blanchard EL, Vanover D, Bawage SS, Tiwari PM, Rotolo L, Beyersdorf J, Peck HE, Bruno NC, Hincapie R, Michel F, Murray J, Sadhwani H, Vanderheyden B, Finn MG, Brinton MA, Lafontaine ER, Hogan RJ, Zurla C, Santangelo PJ. Nat Biotechnol. 2021 Feb 3. Pubmed
Systemic delivery of antagomirs during blood brain barrier disruption is disease-modifying in experimental epilepsy
Reschke CR, Silva LFA, Vangoor VR, Rosso M, David B, Cavanagh BL, Connolly NMC, Brennan GP, Sanz-Rodriguez A, Mooney C, Batool A, Greene C, Brennan M, Conroy RM, Rüber T, Prehn JHM, Campbell M, Pasterkamp RJ, Henshall DC. Mol Ther. 2021 Feb 17:S1525-0016(21)00088-5. Pubmed
The HIF-1α antisense long non-coding RNA drives a positive feedback loop of HIF-1α mediated transactivation and glycolysis
Zheng F, Chen J, Zhang X, Wang Z, Chen J, Lin X, Huang H, Fu W, Liang J, Wu W, Li B, Yao H, Hu H, Song E. Nat Commun. 2021 Feb 26;12(1):1341. Pubmed
Magnesium accumulation upon cyclin M4 silencing activates microsomal triglyceride transfer protein improving NASH
Simón J, Goikoetxea-Usandizaga N, Serrano-Maciá M, Fernández-Ramos D, Sáenz de Urturi D, Gruskos JJ, Fernández-Tussy P, Lachiondo-Ortega S, González-Recio I, Rodríguez-Agudo R, Gutiérrez-de-Juan V, Rodríguez-Iruretagoyena B, Varela-Rey M, Gimenez-Mascarell P, Mercado-Gomez M, Gómez-Santos B, Fernandez-Rodriguez C, Lopitz-Otsoa F, Bizkarguenaga M, Dames S, Schaeper U, Martin ...
Binding of phosphorothioate oligonucleotides with RNase H1 can cause conformational changes in the protein and alter the interactions of RNase H1 with other proteins
Zhang L, Vickers TA, Sun H, Liang XH, Crooke ST. Nucleic Acids Res. 2021 Feb 12:gkab078. Pubmed
Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models
Liu Y, Dodart JC, Tran H, Berkovitch S, Braun M, Byrne M, Durbin AF, Hu XS, Iwamoto N, Jang HG, Kandasamy P, Liu F, Longo K, Ruschel J, Shelke J, Yang H, Yin Y, Donner A, Zhong Z, Vargeese C, Brown RH Jr. Nat Commun. ...
Short antisense oligonucleotides alleviate the pleiotropic toxicity of RNA harboring expanded CGG repeats
Derbis M, Kul E, Niewiadomska D, Sekrecki M, Piasecka A, Taylor K, Hukema RK, Stork O, Sobczak K. Nat Commun. 2021 Feb 24;12(1):1265. Pubmed
November 2020 Paper of the Month
Building on previous work, here Asami, Nagata, Yoshioka and colleagues from the Tokyo Medical and Dental University and Ionis Pharmaceuticals investigate new heteroduplex oligonucleotide (HDO) designs for antisense oligonucleotides.
The Emerging Era of N-of-1 Drugs for Ultra-Rare Genetic Diseases
Imagine if a medication or therapy could be created to treat just one individual with an ultra-rare disease. There are nearly 7,000 known rare diseases (1) and many are debilitating and ultimately fatal. In the U.S., a disease is considered rare when it affects ...
A Hitchhiker’s Guide to Click-Chemistry with Nucleic Acids
Fantoni NZ, El-Sagheer AH, Brown T. Chem Rev. 2021 Jan 14. Pubmed
