Oligonucleotide Therapeutics: Technology Evolution for Clinically Relevant Tissue Delivery
Presenter: Anastasia Khvorova, PhD, RNA Therapeutics Institute, University of Massachusetts Medical School Date: 10 September 2018 Description: A decade of progress in oligonucleotide chemistry and formulation development has resulted in several compounds, both siRNAs and antisense, demonstrating robust clinical activity. Based on sequence, these ...
Identifying and avoiding off-target effects of RNase H-dependent antisense oligonucleotides in mice
Hagedorn PH, Pontoppidan M, Bisgaard TS, Berrera M, Dieckmann A, Ebeling M, Møller MR, Hudlebusch H, Jensen ML, Hansen HF, Koch T, Lindow M. Nucleic Acids Res. 2018 May 22. PubMed
Therapeutic silencing of FSP27 reduces the progression of atherosclerosis in Ldlr-/- mice
Rajamoorthi A, Lee RG, Baldán Á. Atherosclerosis. 2018 May 24;275:43-49. PubMed
Regeneration of Cochlear Hair Cells and Hearing Recovery through Hes1 Modulation with siRNA Nanoparticles in Adult Guinea Pigs
Du X, Cai Q, West MB, Youm I, Huang X, Li W, Cheng W, Nakmali D, Ewert DL, Kopke RD. Mol Ther. 2018 May 2;26(5):1313-1326. PubMed
Reversal of siRNA-mediated gene silencing in vivo
Zlatev I, Castoreno A, Brown CR, Qin J, Waldron S, Schlegel MK, Degaonkar R, Shulga-Morskaya S, Xu H, Gupta S, Matsuda S, Akinc A, Rajeev KG, Manoharan M, Maier MA, Jadhav V. Nat Biotechnol. 2018 May 14. PubMed
Effective silencing of ENaC by siRNA delivered with epithelial-targeted nanocomplexes in human cystic fibrosis cells and in mouse lung
Tagalakis AD, Munye MM, Ivanova R, Chen H, Smith CM, Aldossary AM, Rosa LZ, Moulding D, Barnes JL, Kafetzis KN, Jones SA, Baines DL, Moss GWJ, O'Callaghan C, McAnulty RJ, Hart SL. Thorax. 2018 May 10. pii: thoraxjnl-2017-210670. PubMed
Efficient Gene Silencing in Brain Tumors with Hydrophobically Modified siRNAs
Osborn MF, Coles AH, Golebiowski D, Echeverria D, Moazami MP, Watts JK, Sena-Esteves M, Khvorova A. Mol Cancer Ther. 2018 Apr 13. PubMed
Estrogen receptor β promotes renal cell carcinoma progression via regulating LncRNA HOTAIR-miR-138/200c/204/217 associated CeRNA network
Ding J, Yeh CR, Sun Y, Lin C, Chou J, Ou Z, Chang C, Qi J, Yeh S. Oncogene. 2018 May 23. PubMed
A dual-targeting reconstituted high density lipoprotein leveraging the synergy of sorafenib and antimiRNA21 for enhanced hepatocellular carcinoma therapy
Li M, Su Y, Zhang F, Chen K, Xu X, Xu L, Zhou J, Wang W. Acta Biomater. 2018 May 31. pii: S1742-7061(18)30328-3. PubMed
miR-223-3p Inhibits Human Osteosarcoma Metastasis and Progression by Directly Targeting CDH6
Ji Q, Xu X, Song Q, Xu Y, Tai Y, Goodman SB, Bi W, Xu M, Jiao S, Maloney WJ, Wang Y. Mol Ther. 2018 May 2;26(5):1299-1312. PubMed