Preclinical Development of a vWF Aptamer to Limit Thrombosis and Engender Arterial Recanalization of Occluded Vessels
Nimjee SM, Dornbos D 3rd, Pitoc GA, Wheeler DG, Layzer JM, Venetos N, Huttinger A, Talentino SE, Musgrave NJ, Moody H, Rempel RE, Jones C, Carlisle K, Wilson J, Bratton C, Joseph ME, Khan S, Hoffman MR, Sommerville L, Becker RC, Zweier JL, Sullenger BA. ...
Anti-PEG Antibodies Inhibit the Anticoagulant Activity of PEGylated Aptamers
Moreno A, Pitoc GA, Ganson NJ, Layzer JM, Hershfield MS, Tarantal AF, Sullenger BA. Cell Chem Biol. 2019 Feb 15. pii: S2451-9456(19)30035-2. PubMed
Nusinersen in later-onset spinal muscular atrophy: Long-term results from the phase 1/2 studies
Darras BT, Chiriboga CA, Iannaccone ST, Swoboda KJ, Montes J, Mignon L, Xia S, Bennett CF, Bishop KM, Shefner JM, Green AM, Sun P, Bhan I, Gheuens S, Schneider E, Farwell W, De Vivo DC; ISIS-396443-CS2/ISIS-396443-CS12 Study Groups; ISIS-396443-CS2/ISIS-396443-CS12 Study. Neurology. 2019 Apr 24. PubMed
A Facile Method for the Removal of dsRNA Contaminant from In Vitro-Transcribed mRNA
Baiersdörfer M, Boros G, Muramatsu H, Mahiny A, Vlatkovic I, Sahin U, Karikó K. Mol Ther Nucleic Acids. 2019 Apr 15;15:26-35. PubMed
Improving mRNA-Based Therapeutic Gene Delivery by Expression-Augmenting 3′ UTRs Identified by Cellular Library Screening
Orlandini von Niessen AG, Poleganov MA, Rechner C, Plaschke A, Kranz LM, Fesser S, Diken M, Löwer M, Vallazza B, Beissert T, Bukur V, Kuhn AN, Türeci Ö, Sahin U. Mol Ther. 2019 Apr 10;27(4):824-836. PubMed
Reassessment of Exosome Composition
Jeppesen DK, Fenix AM, Franklin JL, Higginbotham JN, Zhang Q, Zimmerman LJ, Liebler DC, Ping J, Liu Q, Evans R, Fissell WH, Patton JG, Rome LH, Burnette DT, Coffey RJ. Cell. 2019 Apr 4;177(2):428-445.e18. PubMed
Development and Application of Base Editors
Yang B, Yang L, Chen J. CRISPR J. 2019 Apr;2:91-104. PubMed
Safety evaluation of 2′-deoxy-2′-fluoro nucleotides in GalNAc-siRNA conjugates
Janas MM, Zlatev I, Liu J, Jiang Y, Barros SA, Sutherland JE, Davis WP, Liu J, Brown CR, Liu X, Schlegel MK, Blair L, Zhang X, Das B, Tran C, Aluri K, Li J, Agarwal S, Indrakanti R, Charisse K, Nair J, Matsuda S, Rajeev ...
Design of microRNA therapeutics
Presenter: Andrea Kasinski, Purdue University, USA Date: May 15, 2019 Description: MicroRNAs (miRNAs) have emerged as powerful regulators of the genome and, through concerted efforts to identify their function and evaluate their ability to alter cell growth in vitro and in vivo, some have ...
Targeted delivery using aptamers
Presenter: Donald H. Burke/David Porciani, University of Missouri, USA Date: July 25, 2019 Description: Currently, effective cell-targeted delivery tools for oligonucleotide therapeutics are limited. Aptamers that internalize into target cells have been shown to deliver a wide variety of nucleic acid therapeutics, from siRNAs ...
