Investigating the pharmacodynamic durability of GalNAc-siRNA conjugates
Brown CR, Gupta S, Qin J, Racie T, He G, Lentini S, Malone R, Yu M, Matsuda S, Shulga-Morskaya S, Nair AV, Theile CS, Schmidt K, Shahraz A, Goel V, Parmar RG, Zlatev I, Schlegel MK, Nair JK, Jayaraman M, Manoharan M, Brown D, Maier ...
Direct Delivery of Antisense Oligonucleotides to the Middle and Inner Ear Improves Hearing and Balance in Usher Mice
Lentz JJ, Pan B, Ponnath A, Tran CM, Nist-Lund C, Galvin A, Goldberg H, Robillard KN, Jodelka FM, Farris HE, Huang J, Chen T, Zhu H, Zhou W, Rigo F, Hastings ML, Géléoc GSG. Mol Ther. 2020 Aug 5:S1525-0016(20)30412-3. Pubmed
Anti-α-synuclein ASO delivered to monoamine neurons prevents α-synuclein accumulation in a Parkinson’s disease-like mouse model and in monkeys.
Alarcón-ArÃs D, Pavia-Collado R, Miquel-Rio L, Coppola-Segovia V, Ferrés-Coy A, Ruiz-Bronchal E, Galofré M, Paz V, Campa L, Revilla R, Montefeltro A, Kordower JH, Vila M, Artigas F, Bortolozzi A. EBioMedicine. 2020 Aug 15;59:102944. Pubmed
Physiological impact and disease reversion for the severe form of centronuclear myopathy linked to Dynamin
Massana Muñoz X, Kretz C, Silva-Rojas R, Ochala J, Menuet A, Romero NB, Cowling BS, Laporte J. JCI Insight. 2020 Aug 18:137899. Pubmed
Prion protein lowering is a disease-modifying therapy across prion disease stages, strains and endpoints
Minikel EV, Zhao HT, Le J, O'Moore J, Pitstick R, Graffam S, Carlson GA, Kavanaugh MP, Kriz J, Kim JB, Ma J, Wille H, Aiken J, McKenzie D, Doh-Ura K, Beck M, O'Keefe R, Stathopoulos J, Caron T, Schreiber SL, Carroll JB, Kordasiewicz HB, Cabin ...
2020 OTS Young Investigator Award Winner Maja M. Janas De Angelis, PhD
Maja M. Janas De Angelis, PhD, Alnylam Pharmaceuticals, Inc. Advancing the Safety and Reach of RNAi Therapeutics Biography: Maja Janas De Angelis, PhD, DABT is currently an Associate Director, Investigative Toxicology, at Alnylam Pharmaceuticals. She earned her Bachelor of Arts in Chemistry and Biochemistry ...
2020 Dr. Alan M. Gewirtz Memorial Scholarship Award Winner Annabelle Biscans, PhD
Annabelle Biscans, PhD, AstraZeneca Designing New Chemically Engineered siRNAs for Extrahepatic Delivery Biography: Annabelle Biscans obtained her PhD in chemistry from the University of Montpellier (France) in 2015, where her work focused on the chemical synthesis of RNA prodrugs for the development of new ...
2020 Dr. Alan M. Gewirtz Memorial Scholarship Award Winner Chantal M. Ferguson
Chantal M. Ferguson, RNA Therapeutics Institute, University of Massachusetts Medical School RNAi modulation of APOE as a potential therapeutic for Alzheimer Disease Biography: Chantal joined the Khvorova Lab in 2017 with an interest in developing new tools to understand and treat neurological disorders. Her ...
July 2020 Joint Papers of the Month
Phase 1/2 study of COVID-19 RNA vaccine BNT162b1 in adults. Mulligan MJ, Lyke KE, Kitchin N, Absalon J, Gurtman A, Lockhart S, Neuzil K, Raabe V, Bailey R, Swanson KA, Li P, Koury K, Kalina W, Cooper D, Fontes-Garfias C, Shi PY, Türeci Ö, ...
Directed RNase H cleavage of nascent transcripts causes transcription termination
Lai F, Damle SS, Ling KK, Rigo F Read More
