Aptamer-mediated reversible transactivation of gene expression by light
Mayer G, Renzl C, Kakoti A. Angew Chem Int Ed Engl. 2020 Aug 31. Pubmed
Antisense oligonucleotides increase Scn1a expression and reduce seizures and SUDEP incidence in a mouse model of Dravet syndrome
Han Z, Chen C, Christiansen A, Ji S, Lin Q, Anumonwo C, Liu C, Leiser SC, Meena, Aznarez I, Liau G, Isom LL. Sci Transl Med. 2020 Aug 26;12(558):eaaz6100. Pubmed
Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression
Lim KH, Han Z, Jeon HY, Kach J, Jing E, Weyn-Vanhentenryck S, Downs M, Corrionero A, Oh R, Scharner J, Venkatesh A, Ji S, Liau G, Ticho B, Nash H, Aznarez I. Nat Commun. 2020 Jul 9;11(1):3501. Pubmed
Position-specific oxidation of miR-1 encodes cardiac hypertrophy
Seok H, Lee H, Lee S, Ahn SH, Lee HS, Kim GD, Peak J, Park J, Cho YK, Jeong Y, Gu D, Jeong Y, Eom S, Jang ES, Chi SW. Nature. 2020 Aug;584(7820):279-285. Pubmed
Introducing an In Vitro Liver Stability Assay Capable of Predicting the In Vivo Pharmacodynamic Efficacy of siRNAs for IVIVC
Basiri B, Xie F, Wu B, Humphreys SC, Lade JM, Thayer MB, Yamaguchi P, Florio M, Rock BM. Mol Ther Nucleic Acids. 2020 Sep 4;21:725-736. Pubmed
Single-Stranded Phosphorothioated Regions Enhance Cellular Uptake of Cholesterol-Conjugated siRNA but Not Silencing Efficacy
Ly S, Echeverria D, Sousa J, Khvorova A. Mol Ther Nucleic Acids. 2020 Sep 4;21:991-1005. Pubmed
Investigating the pharmacodynamic durability of GalNAc-siRNA conjugates
Brown CR, Gupta S, Qin J, Racie T, He G, Lentini S, Malone R, Yu M, Matsuda S, Shulga-Morskaya S, Nair AV, Theile CS, Schmidt K, Shahraz A, Goel V, Parmar RG, Zlatev I, Schlegel MK, Nair JK, Jayaraman M, Manoharan M, Brown D, Maier ...
Direct Delivery of Antisense Oligonucleotides to the Middle and Inner Ear Improves Hearing and Balance in Usher Mice
Lentz JJ, Pan B, Ponnath A, Tran CM, Nist-Lund C, Galvin A, Goldberg H, Robillard KN, Jodelka FM, Farris HE, Huang J, Chen T, Zhu H, Zhou W, Rigo F, Hastings ML, Géléoc GSG. Mol Ther. 2020 Aug 5:S1525-0016(20)30412-3. Pubmed