2021 Basic Paper Of The Year Award – Winners
Investigating the pharmacodynamic durability of GalNAc-siRNA conjugates Givosiran, a GalNAc-conjugated siRNA, which was recently approved on the market, displays sustained knock-down over month-long periods. The following paper investigates the reasons behind those impressive pharmacodynamic properties. After looking into various mechanisms related to stability, accumulation or ...
Directed RNase H cleavage of nascent transcripts causes transcription termination
Lai F, Damle SS, Ling KK, Rigo F Read More
Inclisiran for the treatment of heterozygous familial hyper-cholesterolemia (Raal et al., 2020)
Raal FJ, Kallend D, Ray KK, Turner T, Koenig W, Wright RS, Wijngaard PLJ, Curcio D, Jaros MJ, Leiter LA, Kastelein JJP; ORION-9 Investigators. Lee JS, Mendell JT. Read More
Antisense-Mediated Transcript Knockdown Triggers Premature Transcription Termination.
Lee JS, Mendell JT. Mol Cell. 2020 Jan 2. pii: S1097-2765(19)30924-4. PubMed
2018 Paper of the Year Award Winner: Alejandra Gonzalez-Duarte, MD
Dr. Alejandra Gonzalez-Duarte, MDNational Institute of Medical Sciences and Nutrition–Salvador Zubiran, MexicoTalk Title: Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin AmyloidosisA.Gonzalez‑Duarte1, D. Adams2, W.D. O’Riordan3, C.-C. Yang4, M. Ueda5, A.V. Kristen6, I. Tournev7, H.H. Schmidt8, T. Coelho9, J.L. Berk10, K.-P. Lin11, G. Vita12, S. Attarian13, ...
2017 Paper of the Year Award Winner: Richard Finkel, MD
Richard Finkel, MD Nemours Children’s Hospital Talk Title: Treatment of Infantile-onset Spinal Muscular Atrophy with Nusinersen: a Phase 2, Open-label, Dose-escalation Study Biography: Richard S. Finkel, MD is Professor of Neurology at the University of Central Florida School of Medicine, and Chief, Division of Neurology ...
2016 Paper of the Year Award Winner: Victor Serebrov, PhD
Authors: William “Wes” Salomon, Samson Jolly, Victor Serebrov, Melissa Moore, Phillip Zamore – RNA Therapeutics Institute, University of Massachusetts Medical Center Presenter: Victor Serebrov, PhD RNA Therapeutics Institute, University of Massachusetts Medical Center Talk Title: Single-Molecule Imaging Reveals that Argonaute Reshapes the Binding Properties of ...
