Guidelines for Experiments Using Antisense Oligonucleotides and Double-Stranded RNAs

Paper of the MonthBy OTSMay 23, 2019Leave a comment

Gagnon KT, Corey DR. Nucleic Acid Ther. 2019 Mar 23. In addition to the widespread use of synthetic oligonucleotides to manipulate RNA levels in basic research, interest in oligonucleotide therapeutics has increased substantially due to the commercial success of recently approved nucleic acid drugs. In turn, this has led to an influx of scientists largely…

Safety evaluation of 2′-deoxy-2′-fluoro nucleotides in GalNAc-siRNA conjugates

Paper of the MonthBy Petra DistererApril 26, 2019Leave a comment

Janas MM, Zlatev I, Liu J, Jiang Y, Barros SA, Sutherland JE, Davis WP, Liu J, Brown CR, Liu X, Schlegel MK, Blair L, Zhang X, Das B, Tran C, Aluri K, Li J, Agarwal S, Indrakanti R, Charisse K, Nair J, Matsuda S, Rajeev KG, Zimmermann T, Sepp-Lorenzino L, Xu Y, Akinc A, Fitzgerald…

Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria

Paper of the MonthBy Petra DistererMarch 26, 2019Leave a comment

Sardh E, Harper P, Balwani M, Stein P, Rees D, Bissell DM, Desnick R, Parker C, Phillips J, Bonkovsky HL, Vassiliou D, Penz C, Chan-Daniels A, He Q, Querbes W, Fitzgerald K, Kim JB, Garg P, Vaishnaw A, Simon AR, Anderson KE. N Engl J Med. 2019 Feb 7;380(6):549-558. The three lead authors: Acute intermittent…

RNA inhibitors of nuclear proteins responsible for multiple organ dysfunction syndrome

Paper of the MonthBy Petra DistererFebruary 25, 2019Leave a comment

Urak KT, Blanco GN, Shubham S, Lin LH, Dassie JP, Thiel WH, Chen Y, Sonkar VK, Lei B, Murthy S, Gutierrez WR, Wilson ME, Stiber JA, Klesney-Tait J, Dayal S, Miller FJ Jr, Giangrande PH. Nat Commun. 2019 Jan 10;10(1):116. The three joint first authors: Multiple organ dysfunction syndrome(MODS) is a clinical complication caused by…