Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency
Truong B, Allegri G, Liu XB, Burke KE, Zhu X, Cederbaum SD, Häberle J, Martini PGV, Lipshutz GS. Proc Natl Acad Sci U S A. 2019 Oct 15;116(42):21150-21159. PubMed
Pseudouridinylation of mRNA coding sequences alters translation
Eyler DE, Franco MK, Batool Z, Wu MZ, Dubuke ML, Dobosz-Bartoszek M, Jones JD, Polikanov YS, Roy B, Koutmou KS. Proc Natl Acad Sci U S A. 2019 Oct 31. PubMed
Recurrent noncoding U1 snRNA mutations drive cryptic splicing in SHH medulloblastoma
Suzuki H, Kumar SA, Shuai S, Diaz-Navarro A, Gutierrez-Fernandez A, De Antonellis P, Cavalli FMG, Juraschka K, Farooq H, Shibahara I, Vladoiu MC, Zhang J, Abeysundara N, Przelicki D, Skowron P, Gauer N, Luu B, Daniels C, Wu X, Forget A, Momin A, Wang J, ...
Transcriptional landscape and clinical utility of enhancer RNAs for eRNA-targeted therapy in cancer
Zhang Z, Lee JH, Ruan H, Ye Y, Krakowiak J, Hu Q, Xiang Y, Gong J, Zhou B, Wang L, Lin C, Diao L, Mills GB, Li W, Han L. Nat Commun. 2019 Oct 8;10(1):4562. PubMed
A dimerization-based fluorogenic dye-aptamer module for RNA imaging in live cells
Bouhedda F, Fam KT, Collot M, Autour A, Marzi S, Klymchenko A, Ryckelynck M. Nat Chem Biol. 2019 Oct 21. PubMed
2019 OTS Young Investigator Award Winner Holly Kordasiewicz, PhD
Holly Kordasiewicz, PhD Ionis Pharmaceuticals Biography: Dr. Holly Kordasiewicz is an Executive Director in the Neuroscience Drug Discovery Departments at Ionis Pharmaceuticals. Holly heads a team focused on identifying antisense oligonucleotide therapeutics for neurodegenerative diseases, including drugs now in clinical trials for ALS, Alzheimer’s disease, ...
2019 OTS Lifetime Achievement Award Winner Muthiah (Mano) Manoharan
Muthiah (Mano) Manoharan, Ph.D Alnylam Pharmaceuticals Muthiah (Mano) Manoharan serves as a Senior Vice President, a Scientific Advisory Board Member, and a Distinguished Research Scientist at Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA. In 2003, he was the first chemist hired at Alnylam. He and his ...
Congratulations to our 2019 Award Winners!
Dr. Alan M. Gewirtz Memorial Scholarship Award: Bruno M.D.C. Godinho, PhD, RNA Therapeutics Institute, UMass Medical School