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
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
Base editors for simultaneous introduction of C-to-T and A-to-G mutations.
Sakata RC, Ishiguro S, Mori H, Tanaka M, Tatsuno K, Ueda H, Yamamoto S, Seki M, Masuyama N, Nishida K, Nishimasu H, Arakawa K, Kondo A, Nureki O, Tomita M, Aburatani H, Yachie N. Nat Biotechnol. 2020 Jul;38(7):865-869. Pubmed
Combinatorial Inhibition of Cell Surface Receptors Using Dual Aptamer-Functionalized Nanoconstructs for Cancer Treatment.
Lee H, Kim TH, Park D, Jang M, Chung JJ, Kim SH, Kim SH, Lee KH, Jung Y, Oh SJ. Pharmaceutics. 2020 Jul 21;12(7):E689. Pubmed
