Vupanorsen, an N-acetyl galactosamine-conjugated antisense drug to ANGPTL3 mRNA, lowers triglycerides and atherogenic lipoproteins in patients with diabetes, hepatic steatosis, and hypertriglyceridaemia
Gaudet D, Karwatowska-Prokopczuk E, Baum SJ, Hurh E, Kingsbury J, Bartlett VJ, Figueroa AL, Piscitelli P, Singleton W, Witztum JL, Geary RS, Tsimikas S, St L O'Dea L. Eur Heart J. 2020 Aug 29:ehaa689. Pubmed
Convective forces increase rostral delivery of intrathecal radiotracers and antisense oligonucleotides in the cynomolgus monkey nervous system.
Sullivan JM, Mazur C, Wolf DA, Horky L, Currier N, Fitzsimmons B, Hesterman J, Pauplis R, Haller S, Powers B, Tayefeh L, DeBrosse-Serra B, Hoppin J, Kordasiewicz H, Swayze EE, Verma A. J Transl Med. 2020 Aug 8;18(1):309. Pubmed
The dose threshold for nanoparticle tumour delivery
Ouyang B, Poon W, Zhang YN, Lin ZP, Kingston BR, Tavares AJ, Zhang Y, Chen J, Valic MS, Syed AM, MacMillan P, Couture-Senécal J, Zheng G, Chan WCW. Nat Mater. 2020 Aug 10. Pubmed
Synthesis and Characterization of Thiophosphoramidate Morpholino Oligonucleotides and Chimeras
Langner HK, Jastrzebska K, Caruthers MH. J Am Chem Soc. 2020 Aug 31. Pubmed
A short de novo synthesis of nucleoside analogs
Meanwell M, Silverman SM, Lehmann J, Adluri B, Wang Y, Cohen R, Campeau LC, Britton R. Science. 2020 Aug 7;369(6504):725-730. Pubmed
Aptamers against mouse and human tumor-infiltrating myeloid cells as reagents for targeted chemotherapy
De La Fuente A, Zilio S, Caroli J, Van Simaeys D, Mazza EMC, Ince TA, Bronte V, Bicciato S, Weed DT, Serafini P. Sci Transl Med. 2020 Jun 17;12(548):eaav9760. Pubmed
Aptamer-mediated reversible transactivation of gene expression by light
Mayer G, Renzl C, Kakoti A. Angew Chem Int Ed Engl. 2020 Aug 31. Pubmed
Antisense oligonucleotides increase Scn1a expression and reduce seizures and SUDEP incidence in a mouse model of Dravet syndrome
Han Z, Chen C, Christiansen A, Ji S, Lin Q, Anumonwo C, Liu C, Leiser SC, Meena, Aznarez I, Liau G, Isom LL. Sci Transl Med. 2020 Aug 26;12(558):eaaz6100. Pubmed
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
