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 ...
Extracellular microRNAs in human circulation are associated with miRISC complexes that are accessible to anti-AGO2 antibody and can bind target mimic oligonucleotides
Geekiyanage H, Rayatpisheh S, Wohlschlegel JA, Brown R Jr, Ambros V. Proc Natl Acad Sci U S A. 2020 Sep 29;117(39):24213-24223. Pubmed
A mouse tissue atlas of small noncoding RNA.
Isakova A, Fehlmann T, Keller A, Quake SR. Proc Natl Acad Sci U S A. 2020 Sep 25:202002277. Pubmed
Knockdown of liver-derived factor XII by GalNAc-siRNA ALN-F12 prevents thrombosis in mice without impacting hemostatic function
Liu J, Cooley BC, Akinc A, Butler J, Borodovsky A. Thromb Res. 2020 Aug 28;196:200-205. Pubmed
2′-O-Methyl at 20-mer Guide Strand 3′ Termini May Negatively Affect Target Silencing Activity of Fully Chemically Modified siRNA
Davis SM, Sousa J, Vangjeli L, Hassler MR, Echeverria D, Knox E, Turanov AA, Alterman JF, Khvorova A. Mol Ther Nucleic Acids. 2020 Sep 4;21:266-277. Pubmed
Cell Type Impacts Accessibility of mRNA to Silencing by RNA Interference
Ferguson CM, Echeverria D, Hassler M, Ly S, Khvorova A. Mol Ther Nucleic Acids. 2020 Sep 4;21:384-393. Pubmed
Some ASOs that bind in the coding region of mRNAs and induce RNase H1 cleavage can cause increases in the pre-mRNAs that may blunt total activity
Liang XH, Nichols JG, De Hoyos CL, Crooke ST. Nucleic Acids Res. 2020 Sep 25;48(17):9840-9858 Pubmed
C9orf72 poly(GR) aggregation induces TDP-43 proteinopathy
Cook CN, Wu Y, Odeh HM, Gendron TF, Jansen-West K, Del Rosso G, Yue M, Jiang P, Gomes E, Tong J, Daughrity LM, Avendano NM, Castanedes-Casey M, Shao W, Oskarsson B, Tomassy GS, McCampbell A, Rigo F, Dickson DW, Shorter J, Zhang YJ, Petrucelli L. ...