In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafness
Yeh WH, Shubina-Oleinik O, Levy JM, Pan B, Newby GA, Wornow M, Burt R, Chen JC, Holt JR, Liu DR. Sci Transl Med. 2020 Jun 3;12(546):eaay9101. Pubmed
A rationally engineered cytosine base editor retains high on-target activity while reducing both DNA and RNA off-target effects
Zuo E, Sun Y, Yuan T, He B, Zhou C, Ying W, Liu J, Wei W, Zeng R, Li Y, Yang H. Nat Methods. 2020 Jun;17(6):600-604. Pubmed
Antisense oligonucleotide-mediated correction of CFTR splicing improves chloride secretion in cystic fibrosis patient-derived bronchial epithelial cells.
Michaels WE, Bridges RJ, Hastings ML. Nucleic Acids Res. 2020 Jun 10:gkaa490. Pubmed
Exon-Skipping Oligonucleotides Restore Functional Collagen VI by Correcting a Common COL6A1 Mutation in Ullrich CMD
Aguti S, Bolduc V, Ala P, Turmaine M, Bönnemann CG, Muntoni F, Zhou H. Mol Ther Nucleic Acids. 2020 Jun 1;21:205-216. Pubmed
Argonaute binding within 3′-untranslated regions poorly predicts gene repression
Chu Y, Kilikevicius A, Liu J, Johnson KC, Yokota S, Corey DR. Nucleic Acids Res. 2020 Jun 5:gkaa478. Pubmed
MicroRNA-206 Regulates Stress-Provoked Aggressive Behaviors in Post-weaning Social Isolation Mice
Chang CH, Kuek EJW, Su CL, Gean PW. Mol Ther Nucleic Acids. 2020 Jun 5;20:812-822. Pubmed
A systems approach delivers a functional microRNA catalog and expanded targets for seizure suppression in temporal lobe epilepsy
Venø MT, Reschke CR, Morris G, Connolly NMC, Su J, Yan Y, Engel T, Jimenez-Mateos EM, Harder LM, Pultz D, Haunsberger SJ, Pal A, Heller JP, Campbell A, Langa E, Brennan GP, Conboy K, Richardson A, Norwood BA, Costard LS, Neubert V, Del Gallo F, ...
Reversing a model of Parkinson’s disease with in situ converted nigral neurons
Qian H, Kang X, Hu J, Zhang D, Liang Z, Meng F, Zhang X, Xue Y, Maimon R, Dowdy SF, Devaraj NK, Zhou Z, Mobley WC, Cleveland DW, Fu XD. Nature. 2020 Jun;582(7813):550-556. Pubmed
Academia or Industry? A career discussion
Presenter: Prof. Annemieke Aartsma-Rus, (OTS President; Leiden University Medical Center) and Dr. Shalini Andersson (Chief Scientist New Therapeutic Modalities and Head of Oligonucleotide Discovery, AstraZeneca) Date: July 15, 2020 Description: To start off, Prof. Annemieke Aartsma-Rus (OTS President; Leiden University Medical Center) and Dr. ...
Base-pair Conformational Switch Modulates miR-34a Targeting of Sirt1 mRNA
Baronti L, Guzzetti I, Ebrahimi P, Friebe Sandoz S, Steiner E, Schlagnitweit J, Fromm B, Silva L, Fontana C, Chen AA, Petzold K. Nature. 2020 May 27 Lorenzo Baronti The first author: As part of RNA-induced silencing complexes (RISCs), microRNAs bind to target ...
