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Items: 1 to 20 of 116

1.

A Glu-urea-Lys Ligand-conjugated Lipid Nanoparticle/siRNA System Inhibits Androgen Receptor Expression In Vivo.

Lee JB, Zhang K, Tam YY, Quick J, Tam YK, Lin PJ, Chen S, Liu Y, Nair JK, Zlatev I, Rajeev KG, Manoharan M, Rennie PS, Cullis PR.

Mol Ther Nucleic Acids. 2016;5:e348. doi: 10.1038/mtna.2016.43.

2.

Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate.

Zimmermann TS, Karsten V, Chan A, Chiesa J, Boyce M, Bettencourt BR, Hutabarat R, Nochur S, Vaishnaw A, Gollob J.

Mol Ther. 2017 Jan 4;25(1):71-78. doi: 10.1016/j.ymthe.2016.10.019.

PMID:
28129130
3.

siRNA carrying an (E)-vinylphosphonate moiety at the 5' end of the guide strand augments gene silencing by enhanced binding to human Argonaute-2.

Elkayam E, Parmar R, Brown CR, Willoughby JL, Theile CS, Manoharan M, Joshua-Tor L.

Nucleic Acids Res. 2016 Dec 15. pii: gkw1298. [Epub ahead of print] No abstract available.

PMID:
27986853
4.

A Highly Durable RNAi Therapeutic Inhibitor of PCSK9.

Fitzgerald K, White S, Borodovsky A, Bettencourt BR, Strahs A, Clausen V, Wijngaard P, Horton JD, Taubel J, Brooks A, Fernando C, Kauffman RS, Kallend D, Vaishnaw A, Simon A.

N Engl J Med. 2017 Jan 5;376(1):41-51. doi: 10.1056/NEJMoa1609243.

5.

Impact of Oligonucleotide Structure, Chemistry, and Delivery Method on In Vitro Cytotoxicity.

Janas MM, Jiang Y, Schlegel MK, Waldron S, Kuchimanchi S, Barros SA.

Nucleic Acid Ther. 2017 Feb;27(1):11-22. doi: 10.1089/nat.2016.0639.

PMID:
27923110
6.

siRNA carrying an (E)-vinylphosphonate moiety at the 5' end of the guide strand augments gene silencing by enhanced binding to human Argonaute-2.

Elkayam E, Parmar R, Brown CR, Willoughby JL, Theile CS, Manoharan M, Joshua-Tor L.

Nucleic Acids Res. 2016 Nov 29. pii: gkw1171. [Epub ahead of print] Erratum in: Nucleic Acids Res. 2016 Dec 15;:.

PMID:
27903888
7.

Strategies to Determine Assay Format for the Assessment of Neutralizing Antibody Responses to Biotherapeutics.

Wu B, Chung S, Jiang XR, McNally J, Pedras-Vasconcelos J, Pillutla R, White JT, Xu Y, Gupta S.

AAPS J. 2016 Nov;18(6):1335-1350.

PMID:
27495119
8.

Effect of terminal 3'-hydroxymethyl modification of an RNA primer on nonenzymatic primer extension.

Pal A, Das RS, Zhang W, Lang M, McLaughlin LW, Szostak JW.

Chem Commun (Camb). 2016 Sep 29;52(80):11905-11907.

PMID:
27722518
9.

The RNA-binding protein vigilin regulates VLDL secretion through modulation of Apob mRNA translation.

Mobin MB, Gerstberger S, Teupser D, Campana B, Charisse K, Heim MH, Manoharan M, Tuschl T, Stoffel M.

Nat Commun. 2016 Sep 26;7:12848. doi: 10.1038/ncomms12848.

10.

Evaluation of Therapeutic Oligonucleotides for Familial Amyloid Polyneuropathy in Patient-Derived Hepatocyte-Like Cells.

Niemietz CJ, Sauer V, Stella J, Fleischhauer L, Chandhok G, Guttmann S, Avsar Y, Guo S, Ackermann EJ, Gollob J, Monia BP, Zibert A, Schmidt HH.

PLoS One. 2016 Sep 1;11(9):e0161455. doi: 10.1371/journal.pone.0161455.

11.
12.

Proliferation and Recruitment Contribute to Myocardial Macrophage Expansion in Chronic Heart Failure.

Sager HB, Hulsmans M, Lavine KJ, Moreira MB, Heidt T, Courties G, Sun Y, Iwamoto Y, Tricot B, Khan OF, Dahlman JE, Borodovsky A, Fitzgerald K, Anderson DG, Weissleder R, Libby P, Swirski FK, Nahrendorf M.

Circ Res. 2016 Sep 16;119(7):853-64. doi: 10.1161/CIRCRESAHA.116.309001.

PMID:
27444755
13.

An Investigational RNAi Therapeutic Targeting Glycolate Oxidase Reduces Oxalate Production in Models of Primary Hyperoxaluria.

Liebow A, Li X, Racie T, Hettinger J, Bettencourt BR, Najafian N, Haslett P, Fitzgerald K, Holmes RP, Erbe D, Querbes W, Knight J.

J Am Soc Nephrol. 2017 Feb;28(2):494-503. doi: 10.1681/ASN.2016030338.

PMID:
27432743
14.

Exposure to siRNA-GalNAc Conjugates in Systems of the Standard Test Battery for Genotoxicity.

Janas MM, Jiang Y, Duncan RG, Hayes AN, Liu J, Kasperkovitz PV, Placke ME, Barros SA.

Nucleic Acid Ther. 2016 Dec;26(6):363-371.

PMID:
27387336
15.

Sequence-Defined Oligomers from Hydroxyproline Building Blocks for Parallel Synthesis Applications.

Kanasty RL, Vegas AJ, Ceo LM, Maier M, Charisse K, Nair JK, Langer R, Anderson DG.

Angew Chem Int Ed Engl. 2016 Aug 8;55(33):9529-33. doi: 10.1002/anie.201602748.

PMID:
27365192
16.

Pharmacokinetics of Tacrolimus and Cyclosporine in Liver Transplant Recipients Receiving 3 Direct-Acting Antivirals as Treatment for Hepatitis C Infection.

Badri PS, Parikh A, Coakley EP, Ding B, Awni WM, Dutta S, Menon RM.

Ther Drug Monit. 2016 Oct;38(5):640-5. doi: 10.1097/FTD.0000000000000315.

PMID:
27310199
17.

RNAi targeting multiple cell adhesion molecules reduces immune cell recruitment and vascular inflammation after myocardial infarction.

Sager HB, Dutta P, Dahlman JE, Hulsmans M, Courties G, Sun Y, Heidt T, Vinegoni C, Borodovsky A, Fitzgerald K, Wojtkiewicz GR, Iwamoto Y, Tricot B, Khan OF, Kauffman KJ, Xing Y, Shaw TE, Libby P, Langer R, Weissleder R, Swirski FK, Anderson DG, Nahrendorf M.

Sci Transl Med. 2016 Jun 8;8(342):342ra80. doi: 10.1126/scitranslmed.aaf1435.

18.

Cutaneous Adverse Events in the Randomized, Double-Blind, Active-Comparator DECIDE Study of Daclizumab High-Yield Process Versus Intramuscular Interferon Beta-1a in Relapsing-Remitting Multiple Sclerosis.

Krueger JG, Kircik L, Hougeir F, Friedman A, You X, Lucas N, Greenberg SJ, Sweetser M, Castro-Borrero W, McCroskery P, Elkins J.

Adv Ther. 2016 Jul;33(7):1231-45. doi: 10.1007/s12325-016-0353-2.

19.

Continuous intranigral infusion is not associated with observable behavioral deficits or marked pathology: a preclinical safety study.

Grondin R, Ai Y, Hardy PA, Butt MT, Nelson BD, Lemmon JD, Bumcrot D, Gash DM, Gerhardt GA, Zhang Z.

J Neurosurg. 2016 May 27:1-10. [Epub ahead of print]

PMID:
27231974
20.

5'-(E)-Vinylphosphonate: A Stable Phosphate Mimic Can Improve the RNAi Activity of siRNA-GalNAc Conjugates.

Parmar R, Willoughby JL, Liu J, Foster DJ, Brigham B, Theile CS, Charisse K, Akinc A, Guidry E, Pei Y, Strapps W, Cancilla M, Stanton MG, Rajeev KG, Sepp-Lorenzino L, Manoharan M, Meyers R, Maier MA, Jadhav V.

Chembiochem. 2016 Jun 2;17(11):985-9. doi: 10.1002/cbic.201600130.

PMID:
27121751
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