Association of Patisiran, an RNA Interference Therapeutic, With Regional Left Ventricular Myocardial Strain in Hereditary Transthyretin Amyloidosis: The APOLLO Study
Minamisawa M, Claggett B, Adams D, Kristen AV, Merlini G, Slama MS, Dispenzieri A, Shah AM, Falk RH, Karsten V, Sweetser MT, Chen J, Riese R, Vest J, Solomon SD. JAMA Cardiol. 2019 Mar 16. PubMed
Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates
Zhu X, Yin L, Theisen M, Zhuo J, Siddiqui S, Levy B, Presnyak V, Frassetto A, Milton J, Salerno T, Benenato KE, Milano J, Lynn A, Sabnis S, Burke K, Besin G, Lukacs CM, Guey LT, Finn PF, Martini PGV. Am J Hum Genet. 2019 ...
Proximity Ligation Assays for In Situ Detection of Innate Immune Activation: Focus on In Vitro-Transcribed mRNA
Blanchard EL, Loomis KH, Bhosle SM, Vanover D, Baumhof P, Pitard B, Zurla C, Santangelo PJ. Mol Ther Nucleic Acids. 2019 Mar 1;14:52-66. PubMed
Guidelines for Experiments Using Antisense Oligonucleotides and Double-Stranded RNAs
Gagnon KT, Corey DR. Nucleic Acid Ther. 2019 Mar 23. PubMed
Re-Engineering RNA Molecules into Therapeutic Agents
Egli M, Manoharan M. Acc Chem Res. 2019 Mar 26. PubMed
Chemical methods for the modification of RNA
Flamme M, McKenzie LK, Sarac I, Hollenstein M. Methods. 2019 Mar 21. pii: S1046-2023(18)30274-3. PubMed
Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria
Sardh E, Harper P, Balwani M, Stein P, Rees D, Bissell DM, Desnick R, Parker C, Phillips J, Bonkovsky HL, Vassiliou D, Penz C, Chan-Daniels A, He Q, Querbes W, Fitzgerald K, Kim JB, Garg P, Vaishnaw A, Simon AR, Anderson KE. N Engl J ...
2019 Dr. Alan M. Gewirtz Memorial Scholarship Winner: Bruno M.D.C. Godinho, PhD
Bruno M.D.C. Godinho, PhD RNA Therapeutics Institute, UMass Medical School Biography: Bruno Godinho received his PhD in Pharmacy from University College Cork (Ireland) in 2014, where he studied formulation-based approaches for siRNA delivery to the central nervous system (CNS). His work from graduate school ...
Interview with Daniel O’Reilly, PhD Candidate
Daniel O’Reilly MChem, PhD Candidate McGill University  How did you become interested in the field of oligonucleotides? My introduction to the world of genetic disease and oligonucleotides came at an early age when my mum was diagnosed with Huntington’s Disease (HD). Witnessing the ...
Pnpla3 silencing with antisense oligonucleotides ameliorates nonalcoholic steatohepatitis and fibrosis in Pnpla3 I148M knock-in mice
Lindén D, Ahnmark A, Pingitore P, Ciociola E, Ahlstedt I, Andréasson AC, Sasidharan K, Madeyski-Bengtson K, Zurek M, Mancina RM, Lindblom A, Bjursell M, Böttcher G, Ståhlman M, Bohlooly-Y M, Haynes WG, Carlsson B, Graham M, Lee R, Murray S, Valenti L, Bhanot S, Åkerblad ...
