Antisense oligonucleotides correct the familial dysautonomia splicing defect in IKBKAP transgenic mice

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…

small activating RNA (saRNA)

Gene activation of CEBPA using saRNA: preclinical studies of the first in human saRNA drug candidate for liver cancer

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…