CRISPR-induced exon skipping is dependent on premature termination codon mutations
Sui T, Song Y, Liu Z, Chen M, Deng J, Xu Y, Lai L, Li Z. Genome Biol. 2018 Oct 17;19(1):164. PubMed
Systemic messenger RNA as an etiological treatment for acute intermittent porphyria
Jiang L, Berraondo P, Jericó D, Guey LT, Sampedro A, Frassetto A, Benenato KE, Burke K, Santamaría E, Alegre M, Pejenaute Á, Kalariya M, Butcher W, Park JS, Zhu X, Sabnis S, Kumarasinghe ES, Salerno T, Kenney M, Lukacs CM, Ávila MA, Martini PGV, Fontanellas ...
Cell specific delivery of modified mRNA expressing therapeutic proteins to leukocytes
Veiga N, Goldsmith M, Granot Y, Rosenblum D, Dammes N, Kedmi R, Ramishetti S, Peer D. Nat Commun. 2018 Oct 29;9(1):4493. PubMed
Mouse genome-wide association studies and systems genetics uncover the genetic architecture associated with hepatic pharmacokinetic and pharmacodynamic properties of a constrained ethyl antisense oligonucleotide targeting Malat1
Pirie E, Ray S, Pan C, Fu W, Powers AF, Polikoff D, Miller CM, Kudrna KM, Harris EN, Lusis AJ, Crooke RM, Lee RG. PLoS Genet. 2018 Oct 29;14(10):e1007732. PubMed
Therapeutic Oligonucleotides: State of the Art
Smith CIE, Zain R. Annu Rev Pharmacol Toxicol. 2018 Oct 4. PubMed
Cell-Penetrating Peptide Conjugates of Steric Blocking Oligonucleotides as Therapeutics for Neuromuscular Diseases from a Historical Perspective to Current Prospects of Treatment
Gait MJ, Arzumanov AA, McClorey G, Godfrey C, Betts C, Hammond S, Wood MJA. Nucleic Acid Ther. 2018 Oct 16. PubMed
Base editing: precision chemistry on the genome and transcriptome of living cells
Rees HA, Liu DR. Nat Rev Genet. 2018 Dec;19(12):770-788. PubMed
Investigations on the interface of nucleic acid aptamers and binding targets
Cai S, Yan J, Xiong H, Liu Y, Peng D, Liu Z. Analyst. 2018 Nov 5;143(22):5317-5338. PubMed
Emerging microRNA Therapeutic Approaches for Cystic Fibrosis
Bardin P, Sonneville F, Corvol H, Tabary O. Front Pharmacol. 2018 Oct 8;9:1113. PubMed
An RNA toolbox for cancer immunotherapy
Pastor F, Berraondo P, Etxeberria I, Frederick J, Sahin U, Gilboa E, Melero I. Nat Rev Drug Discov. 2018 Oct;17(10):751-767. PubMed