A targeted antisense therapeutic approach 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.
Perspectives on the Designation of Oligonucleotide Starting Materials
Kiesman WF, McPherson AK, Diorazio LJ, Van den Bergh L, Smith PD, Northall JM, Fettes A, Wang T, Mehlmann M, Raza S, Held G. Nucleic Acid Ther. 2021 Feb 2. Pubmed
Treatment of influenza and SARS-CoV-2 infections via mRNA-encoded Cas13a in rodents
Blanchard EL, Vanover D, Bawage SS, Tiwari PM, Rotolo L, Beyersdorf J, Peck HE, Bruno NC, Hincapie R, Michel F, Murray J, Sadhwani H, Vanderheyden B, Finn MG, Brinton MA, Lafontaine ER, Hogan RJ, Zurla C, Santangelo PJ. Nat Biotechnol. 2021 Feb 3. Pubmed
Splicing-associated chromatin signatures: a combinatorial and position-dependent role for histone marks in splicing definition
Agirre E, Oldfield AJ, Bellora N, Segelle A, Luco RF. Nat Commun. 2021 Jan 29;12(1):682. Pubmed
The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy
Scaglioni D, Catapano F, Ellis M, Torelli S, Chambers D, Feng L, Beck M, Sewry C, Monforte M, Harriman S, Koenig E, Malhotra J, Popplewell L, Guglieri M, Straub V, Mercuri E, Servais L, Phadke R, Morgan J, Muntoni Acta Neuropathol Commun. 2021 Jan 6;9(1):7. ...
In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice
Koblan LW, Erdos MR, Wilson C, Cabral WA, Levy JM, Xiong ZM, Tavarez UL, Davison LM, Gete YG, Mao X, Newby GA, Doherty SP, Narisu N, Sheng Q, Krilow C, Lin CY, Gordon LB, Cao K, Collins FS, Brown JD, Liu DR. Nature. 2021 Jan;589(7843):608-614. ...
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
MOTS-c promotes phosphorodiamidate morpholino oligomer uptake and efficacy in dystrophic mice
Ran N, Lin C, Leng L, Han G, Geng M, Wu Y, Bittner S, Moulton HM, Yin H. EMBO Mol Med. 2020 Dec 18:e12993. Pubmed
Evaluation of Engineered CRISPR-Cas-Mediated Systems for Site-Specific RNA Editing
Marina RJ, Brannan KW, Dong KD, Yee BA, Yeo GW. Cell Rep. 2020 Nov 3;33(5):108350. Pubmed
Poison Exon Splicing Regulates a Coordinated Network of SR Protein Expression during Differentiation and Tumorigenesis
Leclair NK, Brugiolo M, Urbanski L, Lawson SC, Thakar K, Yurieva M, George J, Hinson JT, Cheng A, Graveley BR, Anczuków O. Mol Cell. 2020 Nov 19;80(4):648-665.e9. Pubmed
