Scn8a antisense oligonucleotide is protective in mouse models of SCN8A Encephalopathy and Dravet Syndrome.
Lenk GM, Jafar-Nejad P, Hill SF, Huffman LD, Smolen CE, Wagnon JL, Petit H, Yu W, Ziobro J, Bhatia K, Parent J, Giger RJ, Rigo F, Meisler MH. Ann Neurol. 2020 Jan 14. PubMed
Understanding the effect of controlling phosphorothioate chirality in the DNA gap on the potency and safety of gapmer antisense oligonucleotides.
Østergaard ME, De Hoyos CL, Wan WB, Shen W, Low A, Berdeja A, Vasquez G, Murray S, Migawa MT, Liang XH, Swayze EE, Crooke ST, Seth PP. Nucleic Acids Res. 2020 Jan 25. PubMed
Antisense-Mediated Transcript Knockdown Triggers Premature Transcription Termination.
Lee JS, Mendell JT. Mol Cell. 2020 Jan 2. pii: S1097-2765(19)30924-4. PubMed
Directed RNase H Cleavage of Nascent Transcripts Causes Transcription Termination.
Lai F, Damle SS, Ling KK, Rigo F. Mol Cell. 2020 Jan 3. pii: S1097-2765(19)30959-1. PubMed
Interview with Marcin Kortylewski, PhD
Marcin Kortylewski, Ph.D. Associate Professor Department of Immuno-Oncology Beckman Research Institute City of Hope National Medical Center How did you become interested in the field of oligonucleotides? Rather late, after about ten years of graduate and postdoctoral research on signal transduction and tumor immunology ...
mRNA Structure Regulates Protein Expression Through Changes in Functional Half-Life
Mauger DM, Cabral BJ, Presnyak V, Su SV, Reid DW, Goodman B, Link K, Khatwani N, Reynders J, Moore MJ, McFadyen IJ. Proc Natl Acad Sci U S A. 2019 Nov 26;116(48):24075-24083. Melissa J. Moore Iain J. McFadyen ...
RNA Splicing by the Spliceosome.
Wilkinson ME, Charenton C, Nagai K. Annu Rev Biochem. 2019 Dec 3. PubMed
A reversible RNA on-switch that controls gene expression of AAV-delivered therapeutics in vivo.
Zhong G, Wang H, He W, Li Y, Mou H, Tickner ZJ, Tran MH, Ou T, Yin Y, Diao H, Farzan M. Nat Biotechnol. 2019 Dec 23. PubMed
mRNA structure regulates protein expression through changes in functional half-life.
Mauger DM, Cabral BJ, Presnyak V, Su SV, Reid DW, Goodman B, Link K, Khatwani N, Reynders J, Moore MJ, McFadyen IJ. Proc Natl Acad Sci U S A. 2019 Nov 26;116(48):24075-24083. PubMed
HER3-targeted protein chimera forms endosomolytic capsomeres and self-assembles into stealth nucleocapsids for systemic tumor homing of RNA interference in vivo.
Alonso-Valenteen F, Pacheco S, Srinivas D, Rentsendorj A, Chu D, Lubow J, Sims J, Miao T, Mikhael S, Hwang JY, Abrol R, Medina Kauwe LK. Nucleic Acids Res. 2019 Dec 2;47(21):11020-11043. PubMed
