In vivo CRISPR editing with no detectable genome-wide off-target mutations
Akcakaya P, Bobbin ML, Guo JA, Malagon-Lopez J, Clement K, Garcia SP, Fellows MD, Porritt MJ, Firth MA, Carreras A, Baccega T, Seeliger F, Bjursell M, Tsai SQ, Nguyen NT, Nitsch R, Mayr LM, Pinello L, Bohlooly-Y M, Aryee MJ, Maresca M, Joung JK. Nature. ...
Structural Basis for the RNA-Guided Ribonuclease Activity of CRISPR-Cas13d
Zhang C, Konermann S, Brideau NJ, Lotfy P, Wu X, Novick SJ, Strutzenberg T, Griffin PR, Hsu PD, Lyumkis D. Cell. 2018 Sep 20;175(1):212-223.e17. PubMed
A Synthetic Cross-Species CD200R1 Agonist Suppresses Inflammatory Immune Responses In Vivo
Prodeus A, Sparkes A, Fischer NW, Cydzik M, Huang E, Khatri I, Young A, Woo L, Chow CW, Gorczynski R, Gariépy J. Mol Ther Nucleic Acids. 2018 Sep 7;12:350-358. PubMed
Chimeric XNA: An Unconventional Design for Orthogonal Informational Systems
Efthymiou T, Gavette J, Stoop M, De Riccardis F, Froeyen M, Herdewijn P, Krishnamurthy R. Chemistry. 2018 Sep 3;24(49):12811-12819. PubMed
A strongly pairing fifth base: oligonucleotides with a C-nucleoside replacing thymidine
Walter TJ, Richert C. Nucleic Acids Res. 2018 Sep 19;46(16):8069-8078. PubMed
Exosome-Mediated Small RNA Delivery: A Novel Therapeutic Approach for Inflammatory Lung Responses
Zhang D, Lee H, Wang X, Rai A, Groot M, Jin Y. Mol Ther. 2018 Sep 5;26(9):2119-2130. PubMed
Intratracheal Administration of siRNA Dry Powder Targeting Vascular Endothelial Growth Factor Inhibits Lung Tumor Growth in Mice
Miwata K, Okamoto H, Nakashima T, Ihara D, Horimasu Y, Masuda T, Miyamoto S, Iwamoto H, Fujitaka K, Hamada H, Shibata A, Ito T, Okuda T, Hattori N. Mol Ther Nucleic Acids. 2018 Sep 7;12:698-706. PubMed
Inhaled TLR9 Agonist Renders Lung Tumors Permissive to PD-1 Blockade by Promoting Optimal CD4+ and CD8+ T-cell Interplay
Gallotta M, Assi H, Degagné É, Kannan SK, Coffman RL, Guiducci C. Cancer Res. 2018 Sep 1;78(17):4943-4956. PubMed
A synthetic microRNA-92a inhibitor (MRG-110) accelerates angiogenesis and wound healing in diabetic and nondiabetic wounds
Gallant-Behm CL, Piper J, Dickinson BA, Dalby CM, Pestano LA, Jackson AL. Wound Repair Regen. 2018 Aug 17. PubMed
Hepatic-targeted RNA interference provides robust and persistent knockdown of alpha-1 antitrypsin levels in ZZ patients
Turner AM, Stolk J, Bals R, Lickliter JD, Hamilton J, Christianson DR, Given BD, Burdon JG, Loomba R, Stoller JK, Teckman JH. J Hepatol. 2018 Aug;69(2):378-384. PubMed