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…

IL-6 SOMAmer

Increasing chemical diversity to overcome the pharmacokinetic limitations of Aptamers

In 2004, the first and so far only therapeutic aptamer, pegaptanib for the treatment of neovascular (wet) age-related macular degeneration, was approved by the FDA. Since then, only a small number of therapeutic aptamers have entered clinical trials, largely because potential applications have been limited by the inherent physicochemical properties and ensuing pharmacokinetic profile of…

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…