OTS President’s Paper Pick – August 2021
By: Annemieke Aartsma-Rus, Ph.D. The paper information: CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis Julian D Gillmore, Ed Gane, Jorg Taube, Justin Kao, Marianna Fontana, Michael L Maitland, Jessica Seitzer, Daniel O’Connell, Kathryn R Walsh, Kristy Wood, Jonathan Phillips, Yuanxin Xu, Adam Amaral, Adam ...
Presentations & Networking in a Virtual World
Presenter: Aurélie Lacroix, PhD, Sixfold Bioscience Keith T. Gagnon, Ph.D, Southern Illinois University School of Medicine Date: 25 August 2021 Time: 8am Pacific / 11am Eastern / 5m Central Description: Join us for a presentation of tips & tricks for making the most of ...
Maximize Your Involvement in the OTS Community
Presenter: Erin M. McConnell, Ph.D.,University of Ottawa Annemieke Aartsma-Rus, PhD, OTS President,Leiden University Medical Center Date: June 24, 2021 Time: 8am Pacific/11am Eastern/5pm Central European Time  Description: Do you study how oligonucleotides can be used as therapeutics? Are you new to the OTS? ...
Lipid Nanoparticles: Nanomedicine’s Triumph
Oligonucleotide therapeutics have been making headlines this past year, between the groundbreaking new mRNA vaccines and the recent news of phenomenal interim results for Intellia’s investigational genome editing treatment for ATTR amyloidosis. Neither of these would have been possible without a lesser-known component – ...
Interview with Tom Brown, PhD
Tom Brown Ph.D. Professor of Nucleic Acid Chemistry University of Oxford, Founder of ATDBio Ltd (and now Chief consultant). Â How did you become interested in the field of oligonucleotides? All my family members were manual workers, and I was the first to go ...
A Momentous CRISPR Milestone was Achieved with Remarkable Results
Intellia Therapeutics and Regeneron Pharmaceuticals’ recent announcement is a major milestone worth celebrating. They shared interim data from the first ever human study using systemically administered CRISPR as a gene editing therapy. Early results that surpassed expectations show that not only is the CRISPR-based ...
OTS President’s Paper Pick– July 2021
By: Annemieke Aartsma-Rus, Ph.D. Safety, Immunogenicity, and Efficacy of the BNT162b2 Covid-19 Vaccine in Adolescents. Frenck et al. N Engl J Med. 2021 May 27; DOI: 10.1056/NEJMoa2107456 While waiting in line to receive my second Moderna Covid-19 mRNA vaccine, I read the recent paper on ...
Oligonucleotide Therapeutics – Restoring Sight to the Blind
Vision is one of our most priceless senses. Without it, we miss out on precious visuals, such as witnessing a child’s face light up with joy or seeing a glorious sunset, and even the important relational activity of reading the expressions on someone’s face ...
Can Oligonucleotides Target RNA Splicing to Treat Disease?
The human body is phenomenally complex, and we are continually discovering incredible facts about it. One fascinating aspect lies in splicing of RNA as it is processed in the cell. Alternate splicing of a single pre-mRNA molecule can create tens of thousands of different ...
How to test for immune activation by your therapeutic oligonucleotide
Presenter: Arthur M. Krieg, MD, Checkmate Pharmaceuticals Date: 19 April 2018 Description: RNA and DNA oligonucleotides with charged backbones commonly cause immune activation, which can be recognized by appropriate in vitro and/or in vivo testing. Eukaryotic innate immune systems have evolved multiple receptors to ...
