Making medicines out of pre-mRNA splicing modulators
Presenter: Isabel Aznarez, Ph.D. Stoke Therapeutics Date: April 29th, 2021 Description: Splicing is the process that edits precursor-messenger RNA (pre-mRNA) to generate the messenger RNA (mRNA) that carries the code from the nucleus to the cytoplasm where the ribosomes will translate it into a ...
20th Anniversary of RNA Interference in Mammalian Cells
The groundbreaking discovery of RNA interference by Craig Mello and Andrew Fire was swiftly followed by the discovery of RNAi as a gene-silencing mechanism in mammalian cells. Since these breakthroughs, members of our community have devoted their lives to building on these exciting discoveries, ...
October 2020 Paper of the Month
The chromosomal 2q21.3 locus has undergone positive selection in the last 5000 years in Europeans and this has long been attributed to the presence of the lactase gene.
Oligonucleotides and the OTS Community Provide Excellent Solutions to the COVID-19 Pandemic
Oligonucleotide therapies are relatively new in the field of medicine, but they have allowed us to make astounding progress in the fight against COVID-19. Rather than simply repurposing existing drugs or spending years to develop new drugs and vaccines, oligo therapies are quickly providing ...
Design and application of circular RNAs with protein-sponge function
Schreiner S, Didio A, Hung LH, Bindereif A. Nucleic Acids Res. 2020 Nov 24:gkaa1085 Pubmed
Dual mRNA therapy restores metabolic function in long-term studies in mice with propionic acidemia.
Jiang L, Park JS, Yin L, Laureano R, Jacquinet E, Yang J, Liang S, Frassetto A, Zhuo J, Yan X, Zhu X, Fortucci S, Hoar K, Mihai C, Tunkey C, Presnyak V, Benenato KE, Lukacs CM, Martini PGV, Guey LT. Nat Commun. 2020 Oct 21;11(1):5339. ...
Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study
Adams D, Polydefkis M, González-Duarte A, Wixner J, Kristen AV, Schmidt HH, Berk JL, Losada López IA, Dispenzieri A, Quan D, Conceição IM, Slama MS, Gillmore JD, Kyriakides T, Ajroud-Driss S, Waddington-Cruz M, Mezei MM, Planté-Bordeneuve V, Attarian S, Mauricio E, Brannagan TH 3rd, Ueda ...
Intracellular delivery of therapeutic antisense oligonucleotides targeting mRNA coding mitochondrial proteins by cell-penetrating peptides
Cerrato CP, Kivijärvi T, Tozzi R, Lehto T, Gestin M, Langel Ü. J Mater Chem B. 2020 Epub 2020 Nov 11. Pubmed
Do Aptamers Always Bind? The Need for a Multifaceted Analytical Approach When Demonstrating Binding Affinity between Aptamer and Low Molecular Weight Compounds
Bottari F, Daems E, de Vries AM, Van Wielendaele P, Trashin S, Blust R, Sobott F, Madder A, Martins JC, De Wael K. J Am Chem Soc. 2020 Nov 18;142(46):19622-19630. Pubmed
Assessment of Aptamer-Targeted Contrast Agents for Monitoring of Blood Clots in Computed Tomography and Fluoroscopy Imaging
Koudrina A, O'Brien J, Garcia R, Boisjoli S, Kan PTM, Tsai EC, DeRosa MC. Bioconjug Chem. 2020 Nov 24. Pubmed
