Zlatev I, Castoreno A, Brown CR, Qin J, Waldron S, Schlegel MK, Degaonkar R, Shulga-Morskaya S, Xu H, Gupta S, Matsuda S, Akinc A, Rajeev KG, Manoharan M, Maier MA, Jadhav V. Nat Biotechnol. 2018 Jul;36(6):509-511. Clinical data from GalNAc-conjugated siRNAs demonstrate long-term persistence of their therapeutic effect (up to 6 months after a single…
Sinha R, Kim YJ, Nomakuchi T, Sahashi K, Hua Y, Rigo F, Bennett CF, Krainer AR. Nucleic Acids Res. 2018 Jun 1;46(10):4833-4844. The three joint first authors: Currently, the most successful oligonucleotide therapeutic in the market is the splice modulating antisense oligonucleotide nusinersen/Sprinraza (see this previous OTS Perspective and the November 2017 Paper of the…
Reebye V, Huang KW, Lin V, Jarvis S, Cutilas P, Dorman S, Ciriello S, Andrikakou P, Voutila J, Saetrom P, Mintz PJ, Reccia I, Rossi JJ, Huber H, Habib R, Kostomitsopoulos N, Blakey DC, Habib NA. Oncogene. 2018 Jun;37(24):3216-3228 Small activating RNAs (saRNA) are the newest oligonucleotide therapeutic modality and have so far remained relatively…
Finn JD, Smith AR, Patel MC, Shaw L, Youniss MR, van Heteren J, Dirstine T, Ciullo C, Lescarbeau R, Seitzer J, Shah RR, Shah A, Ling D, Growe J, Pink M, Rohde E, Wood KM, Salomon WE, Harrington WF, Dombrowski C, Strapps WR, Chang Y, Morrissey DV. Cell Rep. 2018 Feb 27;22(9):2227-2235 Genome editing using…