Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression
Lim KH, Han Z, Jeon HY, Kach J, Jing E, Weyn-Vanhentenryck S, Downs M, Corrionero A, Oh R, Scharner J, Venkatesh A, Ji S, Liau G, Ticho B, Nash H, Aznarez I. Nat Commun. 2020 Jul 9;11(1):3501. Pubmed
Position-specific oxidation of miR-1 encodes cardiac hypertrophy
Seok H, Lee H, Lee S, Ahn SH, Lee HS, Kim GD, Peak J, Park J, Cho YK, Jeong Y, Gu D, Jeong Y, Eom S, Jang ES, Chi SW. Nature. 2020 Aug;584(7820):279-285. Pubmed
Introducing an In Vitro Liver Stability Assay Capable of Predicting the In Vivo Pharmacodynamic Efficacy of siRNAs for IVIVC
Basiri B, Xie F, Wu B, Humphreys SC, Lade JM, Thayer MB, Yamaguchi P, Florio M, Rock BM. Mol Ther Nucleic Acids. 2020 Sep 4;21:725-736. Pubmed
Single-Stranded Phosphorothioated Regions Enhance Cellular Uptake of Cholesterol-Conjugated siRNA but Not Silencing Efficacy
Ly S, Echeverria D, Sousa J, Khvorova A. Mol Ther Nucleic Acids. 2020 Sep 4;21:991-1005. Pubmed
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.
AlarcĂ³n-ArĂs D, Pavia-Collado R, Miquel-Rio L, Coppola-Segovia V, FerrĂ©s-Coy A, Ruiz-Bronchal E, GalofrĂ© 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 ...
