Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson-Gilford progeria syndrome
Papers of interest to society members in the field of oligonucleotide therapeutics. Antisense, aptamers, lncRNA, siRNA, splice modulation and ribozymes.
α-Synuclein antisense oligonucleotides as a disease-modifying therapy for Parkinson’s disease
Papers of interest to society members in the field of oligonucleotide therapeutics. Antisense, aptamers, lncRNA, siRNA, splice modulation and ribozymes.
Antisense oligonucleotide therapy in a humanized mouse model of MECP2 duplication syndrome
Papers of interest to society members in the field of oligonucleotide therapeutics. Antisense, aptamers, lncRNA, siRNA, splice modulation and ribozymes.
Binding of phosphorothioate oligonucleotides with RNase H1 can cause conformational changes in the protein and alter the interactions of RNase H1 with other proteins
Zhang L, Vickers TA, Sun H, Liang XH, Crooke ST. Nucleic Acids Res. 2021 Feb 12:gkab078. Pubmed
Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models
Liu Y, Dodart JC, Tran H, Berkovitch S, Braun M, Byrne M, Durbin AF, Hu XS, Iwamoto N, Jang HG, Kandasamy P, Liu F, Longo K, Ruschel J, Shelke J, Yang H, Yin Y, Donner A, Zhong Z, Vargeese C, Brown RH Jr. Nat Commun. ...
Short antisense oligonucleotides alleviate the pleiotropic toxicity of RNA harboring expanded CGG repeats
Derbis M, Kul E, Niewiadomska D, Sekrecki M, Piasecka A, Taylor K, Hukema RK, Stork O, Sobczak K. Nat Commun. 2021 Feb 24;12(1):1265. Pubmed
Safety, Pharmacokinetic and Pharmacodynamic Evaluation of a 2′-MOE Antisense Oligonucleotide-GalNAc3 Conjugate that Targets the Human Transmembrane Protease Serine 6 (TMPRSS6)
Zanardi TA, Korbmacher B, Boone L, Engelhardt JA, Wang Y, Burel S, Prill B, Aghajan M, Guo S, Henry SP. J Pharmacol Exp Ther. 2021 Jan 11:JPET-AR-2020-000222 Pubmed
Targeting the 5′ untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy
Winkelsas AM, Grunseich C, Harmison GG, Chwalenia K, Rinaldi C, Hammond SM, Johnson K, Bowerman M, Arya S, Talbot K, Wood MJ, Fischbeck KH. Mol Ther Nucleic Acids. 2021 Jan 5;23:731-742. Pubmed
Selective antisense oligonucleotide inhibition of human IRF4 prevents malignant myeloma regeneration via cell cycle disruption
Mondala PK, Vora AA, Zhou T, Lazzari E, Ladel L, Luo X, Kim Y, Costello C, MacLeod AR, Jamieson CHM, Crews LA. Cell Stem Cell. 2021 Jan 20:S1934-5909(20)30601-9. Pubmed
The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration
Jafar-Nejad P, Powers B, Soriano A, Zhao H, Norris DA, Matson J, DeBrosse-Serra B, Watson J, Narayanan P, Chun SJ, Mazur C, Kordasiewicz H, Swayze EE, Rigo F. Nucleic Acids Res. 2020 Dec 24:gkaa1235. Pubmed
