Time matters! – Why PK/PD is important for oligo development
Presenter:Â Carl Amilon (AstraZeneca) Date:Â June 10, 2020 Description: This webinar will give an overview of how incorporating quantitative PK/PD aspects of an oligonucleotide will help you designing more informative experiments and help in translating to a human. We have all struggled with questions around the ...
Interview with Amalia Sapag, PhD
Science: Q: Paul Zamecnik and Mary Stephenson in 1978 reported the very first studies on antisense mechanisms of gene silencing. Since the inception of this field – how far have we come – both in basic and therapeutic research & development? A: The original antisense mechanism has been joined
Imaging small molecule-induced endosomal escape of siRNA
Du Rietz H, Hedlund H, Wilhelmson S, Nordenfelt P, Wittrup A. Nat Commun. 2020 Apr 14;11(1):1809. Joint First Authors Hampus Du Rietz and Dr. Anders Wittrup Delivery is currently the biggest hurdle oligonucleotide therapeutics face to become effective treatments. ...
Re-Evaluating the Conventional Wisdom about Binding Assays
Wilson BD, Soh HT. Trends Biochem Sci. 2020 May 10:S0968-0004(20)30108-0. Pubmed
Emerging siRNA Design Principles and Consequences for Biotransformation and Disposition in Drug Development
Humphreys SC, Thayer MB, Campbell J, Chen WLK, Adams D, Lade JM, Rock BM. J Med Chem. 2020 May 26. Pubmed
Technical Considerations for Use of Oligonucleotide Solution API
Muslehiddinoglu J, Simler R, Hill ML, Mueller C, Amery JHA, Dixon L, Watson A, Storch K, Gazziola C, Gielen F, Lange SA, Prail JD, Nesta DP. Nucleic Acid Ther. 2020 May 6. Pubmed
Amplified Self-Immolative Release of Small Molecules by Spatial Isolation of Reactive Groups on DNA-Minimal Architectures
Prinzen AL, Saliba D, Hennecker C, Trinh T, Mittermaier A, Sleiman HF. Angew Chem Int Ed Engl. 2020 Apr 11. Pubmed
Structural disruption of exonic stem-loops immediately upstream of the intron regulates mammalian splicing
Saha K, England W, Fernandez MM, Biswas T, Spitale RC, Ghosh G. Nucleic Acids Res. 2020 May 13:gkaa358. Pubmed
Structure-Guided Engineering of the Homodimeric Mango-IV Fluorescence Turn-on Aptamer Yields an RNA FRET Pair
Trachman RJ 3rd, Cojocaru R, Wu D, Piszczek G, Ryckelynck M, Unrau PJ, Ferré-D'Amaré AR. Structure. 2020 May 1:S0969-2126(20)30126-X. Pubmed
Safety, Tolerability, and Efficacy of Viltolarsen in Boys with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial
Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, Smith EC, McDonald CM, Zaidman CM, Morgenroth LP, Osaki H, Satou Y, Yamashita T, Hoffman EP; CINRG DNHS Investigators. JAMA Neurol. 2020 May 26 Pubmed
