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Items: 32

1.

EXPLORE: A Prospective, Multinational, Natural History Study of Patients with Acute Hepatic Porphyria with Recurrent Attacks.

Gouya L, Ventura P, Balwani M, Bissell DM, Rees DC, Stölzel U, Phillips JD, Kauppinen R, Langendonk JG, Desnick RJ, Deybach JC, Bonkovsky HL, Parker C, Naik H, Badminton M, Stein PE, Minder E, Windyga J, Bruha R, Cappellini MD, Sardh E, Harper P, Sandberg S, Aarsand AK, Andersen J, Alegre F, Ivanova A, Talbi N, Chan A, Querbes W, Ko J, Penz C, Liu S, Lin T, Simon A, Anderson KE.

Hepatology. 2019 Sep 12. doi: 10.1002/hep.30936. [Epub ahead of print]

PMID:
31512765
2.

Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria.

Sardh E, Harper P, Balwani M, Stein P, Rees D, Bissell DM, Desnick R, Parker C, Phillips J, Bonkovsky HL, Vassiliou D, Penz C, Chan-Daniels A, He Q, Querbes W, Fitzgerald K, Kim JB, Garg P, Vaishnaw A, Simon AR, Anderson KE.

N Engl J Med. 2019 Feb 7;380(6):549-558. doi: 10.1056/NEJMoa1807838.

PMID:
30726693
3.

Patient Perspective on Acute Intermittent Porphyria with Frequent Attacks: A Disease with Intermittent and Chronic Manifestations.

Simon A, Pompilus F, Querbes W, Wei A, Strzok S, Penz C, Howe DL, Hungate JR, Kim JB, Agarwal S, Marquis P.

Patient. 2018 Oct;11(5):527-537. doi: 10.1007/s40271-018-0319-3.

4.

Role of angiopoietin-like 3 (ANGPTL3) in regulating plasma level of low-density lipoprotein cholesterol.

Xu YX, Redon V, Yu H, Querbes W, Pirruccello J, Liebow A, Deik A, Trindade K, Wang X, Musunuru K, Clish CB, Cowan C, Fizgerald K, Rader D, Kathiresan S.

Atherosclerosis. 2018 Jan;268:196-206. doi: 10.1016/j.atherosclerosis.2017.08.031. Epub 2017 Sep 21.

5.

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. Epub 2016 Jul 18.

6.

Metabolism of (13)C5-hydroxyproline in mouse models of Primary Hyperoxaluria and its inhibition by RNAi therapeutics targeting liver glycolate oxidase and hydroxyproline dehydrogenase.

Li X, Knight J, Fargue S, Buchalski B, Guan Z, Inscho EW, Liebow A, Fitzgerald K, Querbes W, Todd Lowther W, Holmes RP.

Biochim Biophys Acta. 2016 Feb;1862(2):233-9. doi: 10.1016/j.bbadis.2015.12.001. Epub 2015 Dec 2.

7.

Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification.

Chan A, Liebow A, Yasuda M, Gan L, Racie T, Maier M, Kuchimanchi S, Foster D, Milstein S, Charisse K, Sehgal A, Manoharan M, Meyers R, Fitzgerald K, Simon A, Desnick RJ, Querbes W.

Mol Ther Nucleic Acids. 2015 Nov 3;4:e263. doi: 10.1038/mtna.2015.36.

8.

Identification of siRNA delivery enhancers by a chemical library screen.

Gilleron J, Paramasivam P, Zeigerer A, Querbes W, Marsico G, Andree C, Seifert S, Amaya P, Stöter M, Koteliansky V, Waldmann H, Fitzgerald K, Kalaidzidis Y, Akinc A, Maier MA, Manoharan M, Bickle M, Zerial M.

Nucleic Acids Res. 2015 Sep 18;43(16):7984-8001. doi: 10.1093/nar/gkv762. Epub 2015 Jul 28.

9.

Hepatocyte-specific delivery of siRNAs conjugated to novel non-nucleosidic trivalent N-acetylgalactosamine elicits robust gene silencing in vivo.

Rajeev KG, Nair JK, Jayaraman M, Charisse K, Taneja N, O'Shea J, Willoughby JL, Yucius K, Nguyen T, Shulga-Morskaya S, Milstein S, Liebow A, Querbes W, Borodovsky A, Fitzgerald K, Maier MA, Manoharan M.

Chembiochem. 2015 Apr 13;16(6):903-8. doi: 10.1002/cbic.201500023. Epub 2015 Mar 18.

PMID:
25786782
10.

siRNA conjugates carrying sequentially assembled trivalent N-acetylgalactosamine linked through nucleosides elicit robust gene silencing in vivo in hepatocytes.

Matsuda S, Keiser K, Nair JK, Charisse K, Manoharan RM, Kretschmer P, Peng CG, V Kel'in A, Kandasamy P, Willoughby JL, Liebow A, Querbes W, Yucius K, Nguyen T, Milstein S, Maier MA, Rajeev KG, Manoharan M.

ACS Chem Biol. 2015 May 15;10(5):1181-7. doi: 10.1021/cb501028c. Epub 2015 Mar 2.

PMID:
25730476
11.

RNAi-mediated silencing of hepatic Alas1 effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice.

Yasuda M, Gan L, Chen B, Kadirvel S, Yu C, Phillips JD, New MI, Liebow A, Fitzgerald K, Querbes W, Desnick RJ.

Proc Natl Acad Sci U S A. 2014 May 27;111(21):7777-82. doi: 10.1073/pnas.1406228111. Epub 2014 May 12.

12.

Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates.

Dong Y, Love KT, Dorkin JR, Sirirungruang S, Zhang Y, Chen D, Bogorad RL, Yin H, Chen Y, Vegas AJ, Alabi CA, Sahay G, Olejnik KT, Wang W, Schroeder A, Lytton-Jean AK, Siegwart DJ, Akinc A, Barnes C, Barros SA, Carioto M, Fitzgerald K, Hettinger J, Kumar V, Novobrantseva TI, Qin J, Querbes W, Koteliansky V, Langer R, Anderson DG.

Proc Natl Acad Sci U S A. 2014 Mar 18;111(11):3955-60. doi: 10.1073/pnas.1322937111. Epub 2014 Feb 10. Erratum in: Proc Natl Acad Sci U S A. 2014 Apr 15;111(15):5753.

13.

Biodegradable lipids enabling rapidly eliminated lipid nanoparticles for systemic delivery of RNAi therapeutics.

Maier MA, Jayaraman M, Matsuda S, Liu J, Barros S, Querbes W, Tam YK, Ansell SM, Kumar V, Qin J, Zhang X, Wang Q, Panesar S, Hutabarat R, Carioto M, Hettinger J, Kandasamy P, Butler D, Rajeev KG, Pang B, Charisse K, Fitzgerald K, Mui BL, Du X, Cullis P, Madden TD, Hope MJ, Manoharan M, Akinc A.

Mol Ther. 2013 Aug;21(8):1570-8. doi: 10.1038/mt.2013.124. Epub 2013 Jun 25.

14.

Image-based analysis of lipid nanoparticle-mediated siRNA delivery, intracellular trafficking and endosomal escape.

Gilleron J, Querbes W, Zeigerer A, Borodovsky A, Marsico G, Schubert U, Manygoats K, Seifert S, Andree C, Stöter M, Epstein-Barash H, Zhang L, Koteliansky V, Fitzgerald K, Fava E, Bickle M, Kalaidzidis Y, Akinc A, Maier M, Zerial M.

Nat Biotechnol. 2013 Jul;31(7):638-46. doi: 10.1038/nbt.2612. Epub 2013 Jun 23.

PMID:
23792630
15.

Efficiency of siRNA delivery by lipid nanoparticles is limited by endocytic recycling.

Sahay G, Querbes W, Alabi C, Eltoukhy A, Sarkar S, Zurenko C, Karagiannis E, Love K, Chen D, Zoncu R, Buganim Y, Schroeder A, Langer R, Anderson DG.

Nat Biotechnol. 2013 Jul;31(7):653-8. doi: 10.1038/nbt.2614. Epub 2013 Jun 23.

16.

Systemic RNAi-mediated Gene Silencing in Nonhuman Primate and Rodent Myeloid Cells.

Novobrantseva TI, Borodovsky A, Wong J, Klebanov B, Zafari M, Yucius K, Querbes W, Ge P, Ruda VM, Milstein S, Speciner L, Duncan R, Barros S, Basha G, Cullis P, Akinc A, Donahoe JS, Narayanannair Jayaprakash K, Jayaraman M, Bogorad RL, Love K, Whitehead K, Levins C, Manoharan M, Swirski FK, Weissleder R, Langer R, Anderson DG, de Fougerolles A, Nahrendorf M, Koteliansky V.

Mol Ther Nucleic Acids. 2012 Jan 24;1:e4. doi: 10.1038/mtna.2011.3.

17.

Modular 'click-in-emulsion' bone-targeted nanogels.

Heller DA, Levi Y, Pelet JM, Doloff JC, Wallas J, Pratt GW, Jiang S, Sahay G, Schroeder A, Schroeder JE, Chyan Y, Zurenko C, Querbes W, Manzano M, Kohane DS, Langer R, Anderson DG.

Adv Mater. 2013 Mar 13;25(10):1449-54. doi: 10.1002/adma.201202881. Epub 2012 Dec 27.

18.

Focused ultrasound for targeted delivery of siRNA and efficient knockdown of Htt expression.

Burgess A, Huang Y, Querbes W, Sah DW, Hynynen K.

J Control Release. 2012 Oct 28;163(2):125-9. doi: 10.1016/j.jconrel.2012.08.012. Epub 2012 Aug 19.

19.

Molecularly self-assembled nucleic acid nanoparticles for targeted in vivo siRNA delivery.

Lee H, Lytton-Jean AK, Chen Y, Love KT, Park AI, Karagiannis ED, Sehgal A, Querbes W, Zurenko CS, Jayaraman M, Peng CG, Charisse K, Borodovsky A, Manoharan M, Donahoe JS, Truelove J, Nahrendorf M, Langer R, Anderson DG.

Nat Nanotechnol. 2012 Jun 3;7(6):389-93. doi: 10.1038/nnano.2012.73.

20.

Treatment of erythropoietin deficiency in mice with systemically administered siRNA.

Querbes W, Bogorad RL, Moslehi J, Wong J, Chan AY, Bulgakova E, Kuchimanchi S, Akinc A, Fitzgerald K, Koteliansky V, Kaelin WG Jr.

Blood. 2012 Aug 30;120(9):1916-22. doi: 10.1182/blood-2012-04-423715. Epub 2012 May 18.

21.

Combinatorial synthesis of chemically diverse core-shell nanoparticles for intracellular delivery.

Siegwart DJ, Whitehead KA, Nuhn L, Sahay G, Cheng H, Jiang S, Ma M, Lytton-Jean A, Vegas A, Fenton P, Levins CG, Love KT, Lee H, Cortez C, Collins SP, Li YF, Jang J, Querbes W, Zurenko C, Novobrantseva T, Langer R, Anderson DG.

Proc Natl Acad Sci U S A. 2011 Aug 9;108(32):12996-3001. doi: 10.1073/pnas.1106379108. Epub 2011 Jul 22.

22.

Synergistic silencing: combinations of lipid-like materials for efficacious siRNA delivery.

Whitehead KA, Sahay G, Li GZ, Love KT, Alabi CA, Ma M, Zurenko C, Querbes W, Langer RS, Anderson DG.

Mol Ther. 2011 Sep;19(9):1688-94. doi: 10.1038/mt.2011.141. Epub 2011 Jul 12.

23.

Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms.

Akinc A, Querbes W, De S, Qin J, Frank-Kamenetsky M, Jayaprakash KN, Jayaraman M, Rajeev KG, Cantley WL, Dorkin JR, Butler JS, Qin L, Racie T, Sprague A, Fava E, Zeigerer A, Hope MJ, Zerial M, Sah DW, Fitzgerald K, Tracy MA, Manoharan M, Koteliansky V, Fougerolles Ad, Maier MA.

Mol Ther. 2010 Jul;18(7):1357-64. doi: 10.1038/mt.2010.85. Epub 2010 May 11.

24.

Lipophilic siRNAs mediate efficient gene silencing in oligodendrocytes with direct CNS delivery.

Chen Q, Butler D, Querbes W, Pandey RK, Ge P, Maier MA, Zhang L, Rajeev KG, Nechev L, Kotelianski V, Manoharan M, Sah DW.

J Control Release. 2010 Jun 1;144(2):227-32. doi: 10.1016/j.jconrel.2010.02.011. Epub 2010 Feb 17.

PMID:
20170694
25.

Lipid-like materials for low-dose, in vivo gene silencing.

Love KT, Mahon KP, Levins CG, Whitehead KA, Querbes W, Dorkin JR, Qin J, Cantley W, Qin LL, Racie T, Frank-Kamenetsky M, Yip KN, Alvarez R, Sah DW, de Fougerolles A, Fitzgerald K, Koteliansky V, Akinc A, Langer R, Anderson DG.

Proc Natl Acad Sci U S A. 2010 Feb 2;107(5):1864-9. doi: 10.1073/pnas.0910603106. Epub 2010 Jan 11. Erratum in: Proc Natl Acad Sci U S A. 2010 May 25;107(21):9915.

26.

Direct CNS delivery of siRNA mediates robust silencing in oligodendrocytes.

Querbes W, Ge P, Zhang W, Fan Y, Costigan J, Charisse K, Maier M, Nechev L, Manoharan M, Kotelianski V, Sah DW.

Oligonucleotides. 2009 Mar;19(1):23-29. doi: 10.1089/oli.2008.0165.

PMID:
19093781
27.
28.

The human polyomaviruses.

Eash S, Manley K, Gasparovic M, Querbes W, Atwood WJ.

Cell Mol Life Sci. 2006 Apr;63(7-8):865-76. Review.

PMID:
16501889
29.

The human polyomavirus, JCV, uses serotonin receptors to infect cells.

Elphick GF, Querbes W, Jordan JA, Gee GV, Eash S, Manley K, Dugan A, Stanifer M, Bhatnagar A, Kroeze WK, Roth BL, Atwood WJ.

Science. 2004 Nov 19;306(5700):1380-3.

30.

Infection of vero cells by BK virus is dependent on caveolae.

Eash S, Querbes W, Atwood WJ.

J Virol. 2004 Nov;78(21):11583-90.

31.

A JC virus-induced signal is required for infection of glial cells by a clathrin- and eps15-dependent pathway.

Querbes W, Benmerah A, Tosoni D, Di Fiore PP, Atwood WJ.

J Virol. 2004 Jan;78(1):250-6.

32.

Early events in the life cycle of JC virus as potential therapeutic targets for the treatment of progressive multifocal leukoencephalopathy.

Baum S, Ashok A, Gee G, Dimitrova S, Querbes W, Jordan J, Atwood WJ.

J Neurovirol. 2003;9 Suppl 1:32-7.

PMID:
12709869

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