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.
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
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. ...
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
Splice-Switching Antisense Oligonucleotides Reduce LRRK2 Kinase Activity in Human LRRK2 Transgenic Mice
Korecka JA, Thomas R, Hinrich AJ, Moskites AM, Macbain ZK, Hallett PJ, Isacson O, Hastings ML. Mol Ther Nucleic Acids. 2020 Sep 4;21:623-635. Pubmed
QR-313, an antisense oligonucleotide, shows therapeutic efficacy for treatment of dominant and recessive dystrophic epidermolysis bullosa: a preclinical study
Bornert O, Hogervorst M, Nauroy P, Bischof J, Swildens J, Athanasiou I, Tufa SF, Keene DR, Kiritsi D, Hainzl S, Murauer EM, Marinkovich MP, Platenburg G, Hausser I, Wally V, Ritsema T, Koller U, Haisma EM, Nyström A. J Invest Dermatol. 2020 Sep 15:S0022-202X(20)32061-3. 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
Therapeutic efficacy of antisense oligonucleotides in mouse models of CLN3 Batten disease.
Centa JL, Jodelka FM, Hinrich AJ, Johnson TB, Ochaba J, Jackson M, Duelli DM, Weimer JM, Rigo F, Hastings ML. Nat Med. 2020 Jul 27. Pubmed
