The biochemical basis of microRNA targeting efficacy.
McGeary SE, Lin KS, Shi CY, Pham T, Bisaria N, Kelley GM, Bartel DP. Science. 2019 Dec 5. pii: eaav1741. PubMed
Interfering with long non-coding RNA MIR22HG processing inhibits glioblastoma progression through suppression of Wnt/β-catenin signalling.
Han M, Wang S, Fritah S, Wang X, Zhou W, Yang N, Ni S, Huang B, Chen A, Li G, Miletic H, Thorsen F, Bjerkvig R, Li X, Wang J. Brain. 2019 Dec 31. pii: awz406. PubMed
A selective Aurora-A 5′-UTR siRNA inhibits tumor growth and metastasis.
Lai CH, Chen RY, Hsieh HP, Tsai SJ, Chang KC, Yen CJ, Huang YC, Liu YW, Lee JC, Lai YC, Hung LY, Lin BW. Cancer Lett. 2019 Dec 23;472:97-107. PubMed
Liver Activation of Hepatocellular Nuclear Factor-4α by Small Activating RNA Rescues Dyslipidemia and Improves Metabolic Profile.
Huang KW, Reebye V, Czysz K, Ciriello S, Dorman S, Reccia I, Lai HS, Peng L, Kostomitsopoulos N, Nicholls J, Habib RS, Tomalia DA, Sætrom P, Wilkes E, Cutillas P, Rossi JJ, Habib NA. Mol Ther Nucleic Acids. 2019 Nov 21;19:361-370. PubMed ...
Knockdown of GADD34 in neonatal mutant SOD1 mice ameliorates ALS.
Ghadge GD, Sonobe Y, Camarena A, Drigotas C, Rigo F, Ling KK, Roos RP. Neurobiol Dis. 2019 Dec 16;136:104702. PubMed
Targeting Repeated Regions Unique to a Gene Is an Effective Strategy for Discovering Potent and Efficacious Antisense Oligonucleotides.
Pedersen L, Hagedorn P, Vikeså J, Karlsen ST, Særmark P, Koch T, Lindow M. Mol Ther Nucleic Acids. 2019 Nov 15;19:124-131. PubMed
Golgi-endosome transport mediated by M6PR facilitates release of antisense oligonucleotides from endosomes
Liang XH, Sun H, Hsu CW, Nichols JG, Vickers TA, De Hoyos CL, Crooke ST. Nucleic Acids Res. 2019 Dec 16. pii: gkz1171. PubMed
Interview with the 2019 OTS Lifetime Achievement Award Winner Muthiah (Mano) Manoharan
Dr. Alan M. Gewirtz Memorial Scholarship Award: Bruno M.D.C. Godinho, PhD, RNA Therapeutics Institute, UMass Medical School
Nusinersen Initiated in Infants During the Presymptomatic Stage of Spinal Muscular Atrophy
De Vivo DC, Bertini E, Swoboda KJ, Hwu WL, Crawford TO, Finkel RS, Kirschner J, Kuntz NL, Parsons JA, Ryan MM, Butterfield RJ, Topaloglu H, Ben-Omran T, Sansone VA, Jong YJ, Shu F, Staropoli JF, Kerr D, Sandrock AW, Stebbins C, Petrillo M, Braley ...
Delivery of Macromolecular Therapeutics: Tackling a Billion Year Old Barrier
Presenter:Â Steven Dowdy (UCSD School of Medicine) Date:Â December 17, 2019 Description: All macromolecular therapeutics, including ASOs, siRNAs, peptides, proteins, CRISPR, mRNA and non-viral DNA vectors, are taken up into cells by endocytosis. However, <1% to none of the endocytosed therapeutic cargo escapes into the cytoplasm ...
