Enzymatic synthesis of hypermodified DNA polymers for sequence-specific display of four different hydrophobic groups
OndruÅ¡ M, SĂ½korovĂ¡ V, BednĂ¡rovĂ¡ L, Pohl R, Hocek M. Nucleic Acids Res. 2020 Dec 2;48(21):11982-11993. Pubmed
Accelerating Post-SELEX Aptamer Engineering Using Exonuclease Digestion
Canoura J, Yu H, Alkhamis O, Roncancio D, Farhana R, Xiao Y. J Am Chem Soc. 2021 Jan 20;143(2):805-816. Pubmed
Evaluation of Engineered CRISPR-Cas-Mediated Systems for Site-Specific RNA Editing
Marina RJ, Brannan KW, Dong KD, Yee BA, Yeo GW. Cell Rep. 2020 Nov 3;33(5):108350. Pubmed
Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease
Esrick EB, Lehmann LE, Biffi A, Achebe M, Brendel C, Ciuculescu MF, Daley H, MacKinnon B, Morris E, Federico A, Abriss D, Boardman K, Khelladi R, Shaw K, Negre H, Negre O, Nikiforow S, Ritz J, Pai SY, London WB, Dansereau C, Heeney MM, Armant ...
Development of Prodrug Type Circular siRNA for In Vivo Knockdown by Systemic Administration
Hagiwara K, Honma M, Harumoto T, Harada K, Sawada T, Yamamoto J, Shinohara F. Nucleic Acid Ther. 2020 Dec;30(6):346-364 Pubmed
The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration
Jafar-Nejad P, Powers B, Soriano A, Zhao H, Norris DA, Matson J, DeBrosse-Serra B, Watson J, Narayanan P, Chun SJ, Mazur C, Kordasiewicz H, Swayze EE, Rigo F. Nucleic Acids Res. 2020 Dec 24:gkaa1235. Pubmed
Amido-Bridged Nucleic Acid-Modified Antisense Oligonucleotides Targeting SYT13 to Treat Peritoneal Metastasis of Gastric Cancer
Kanda M, Kasahara Y, Shimizu D, Miwa T, Umeda S, Sawaki K, Nakamura S, Kodera Y, Obika S. Mol Ther Nucleic Acids. 2020 Oct 6;22:791-802. Pubmed
Design and application of circular RNAs with protein-sponge function
Schreiner S, Didio A, Hung LH, Bindereif A. Nucleic Acids Res. 2020 Nov 24:gkaa1085 Pubmed
Dual mRNA therapy restores metabolic function in long-term studies in mice with propionic acidemia.
Jiang L, Park JS, Yin L, Laureano R, Jacquinet E, Yang J, Liang S, Frassetto A, Zhuo J, Yan X, Zhu X, Fortucci S, Hoar K, Mihai C, Tunkey C, Presnyak V, Benenato KE, Lukacs CM, Martini PGV, Guey LT. Nat Commun. 2020 Oct 21;11(1):5339. ...
Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study
Adams D, Polydefkis M, GonzĂ¡lez-Duarte A, Wixner J, Kristen AV, Schmidt HH, Berk JL, Losada LĂ³pez IA, Dispenzieri A, Quan D, ConceiĂ§Ă£o IM, Slama MS, Gillmore JD, Kyriakides T, Ajroud-Driss S, Waddington-Cruz M, Mezei MM, PlantĂ©-Bordeneuve V, Attarian S, Mauricio E, Brannagan TH 3rd, Ueda ...