A noninflammatory mRNA vaccine for treatment of experimental autoimmune encephalomyelitis
Krienke C, Kolb L, Diken E, Streuber M, Kirchhoff S, Bukur T, Akilli-Öztürk Ö, Kranz LM, Berger H, Petschenka J, Diken M, Kreiter S, Yogev N, Waisman A, Karikó K, Türeci Ö, Sahin U. Science. 2021 Jan 8;371(6525):145-153. Pubmed
The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy
Scaglioni D, Catapano F, Ellis M, Torelli S, Chambers D, Feng L, Beck M, Sewry C, Monforte M, Harriman S, Koenig E, Malhotra J, Popplewell L, Guglieri M, Straub V, Mercuri E, Servais L, Phadke R, Morgan J, Muntoni Acta Neuropathol Commun. 2021 Jan 6;9(1):7. ...
Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology
Spronck EA, Vallès A, Lampen MH, Montenegro-Miranda PS, Keskin S, Heijink L, Evers MM, Petry H, Deventer SJV, Konstantinova P, Haan M. Brain Sci. 2021 Jan 20;11(2):E129. Pubmed .
Safety, Pharmacokinetic and Pharmacodynamic Evaluation of a 2′-MOE Antisense Oligonucleotide-GalNAc3 Conjugate that Targets the Human Transmembrane Protease Serine 6 (TMPRSS6)
Zanardi TA, Korbmacher B, Boone L, Engelhardt JA, Wang Y, Burel S, Prill B, Aghajan M, Guo S, Henry SP. J Pharmacol Exp Ther. 2021 Jan 11:JPET-AR-2020-000222 Pubmed
TLR9 expression in Chronic Lymphocytic Leukemia identifies a pro-migratory subpopulation and novel therapeutic target
Kennedy E, Coulter EM, Halliwell E, Profitos-Peleja N, Walsby E, Clark B, Phillips EH, Burley TA, Mitchell S, Devereux S, Fegan C, Jones CI, Johnston R, Chevassut TJ, Schulz R, Seiffert M, Agathanggelou A, Oldreive C, Davies NJ, Stankovic T, Liloglou T, Pepper C, Pepper ...
Replacement of L-amino acid peptides with D-amino acid peptides mitigates anti-PEG antibody generation against polymer-peptide conjugates in mice
Sylvestre M, Lv S, Yang LF, Luera N, Peeler DJ, Chen BM, Roffler SR, Pun SH. J Control Release. 2021 Jan 11;331:142-153. Pubmed
A DNA origami-based aptamer nanoarray for potent and reversible anticoagulation in hemodialysis
Zhao S, Tian R, Wu J, Liu S, Wang Y, Wen M, Shang Y, Liu Q, Li Y, Guo Y, Wang Z, Wang T, Zhao Y, Zhao H, Cao H, Su Y, Sun J, Jiang Q, Ding B. Nat Commun. 2021 Jan 13;12(1):358. Pubmed ...
In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice
Koblan LW, Erdos MR, Wilson C, Cabral WA, Levy JM, Xiong ZM, Tavarez UL, Davison LM, Gete YG, Mao X, Newby GA, Doherty SP, Narisu N, Sheng Q, Krilow C, Lin CY, Gordon LB, Cao K, Collins FS, Brown JD, Liu DR. Nature. 2021 Jan;589(7843):608-614. ...
iR-10b-5p Rescues Diabetes and Gastrointestinal Dysmotility
Singh R, Ha SE, Wei L, Jin B, Zogg H, Poudrier SM, Jorgensen BG, Park C, Ronkon CF, Bartlett A, Cho S, Morales A, Chung YH, Lee MY, Park JK, Gottfried-Blackmore A, Nguyen L, Sanders KM, Ro S. Gastroenterology. 2021 Jan 7:S0016-5085(21)00001-9. Pubmed ...
Endothelial-Derived miR-17∼92 Promotes Angiogenesis to Protect against Renal Ischemia-Reperfusion Injury
Chiba T, Cerqueira DM, Li Y, Bodnar AJ, Mukherjee E, Pfister K, Phua YL, Shaikh K, Sanders BT, Hemker SL, Pagano PJ, Wu YL, Ho J, Sims-Lucas S. J Am Soc Nephrol. 2021 Jan 29:ASN.2020050717. Pubmed
