Publications

Selected Publications with contributions from Axolabs' scientists:

2024

Conformation matters: siRNAs with antisense strands with 5'-(E)-vinyl-phosphonate-∝-L-LNA elicit stronger RNAi-mediated gene silencing than those with 5'-(E)-vinyl-phosphonate-LNA. Datta D, Kumar P, Mandal S , Krampert M , Egli M, Hrdlicka P, Manoharan M
Chem. Commun. (RSC) 2024, PMID: 39431399, Read Article

The Tautomeric State of N4-Hydroxycytidine within Base-Paired RNA. Besse I, Stiller C, Schroeder T, Schäd B, Grüne M, Dietsch J,  and Höbartner C
ACS Central Science 2024 Article in Press

Illuminating Neuropeptide Y Y4 Receptor Binding: Fluorescent Cyclic Peptides with Subnanomolar Binding Affinity as Novel Molecular Tools. Gleixner J, Kopanchuk S, Grätz L, Tahk M, Laasfeld T, Veikšina S, Höring C, Gattor A O, Humphrys L, Müller C, Archipowa N, Köckenberger J, Heinrich M, Kutta R, Rinken A, and Keller M
ACS Pharmacology & Translational Science. Article in Press.

Atomic Mutagenesis of N6-Methyladenosine Reveals Distinct Recognition Modes by Human m6A Reader and Eraser Proteins. Seitz F, Jungnickel T, Kleiber N, Kretschmer J, Dietzsch J, Adelmann J, Bohnsack K, Bohnsack M and Höbartner C Journal of the American Chemical Society, Read Abstract.

2023

Lipid nanoparticle-encapsulated, chemically modified anti-adenoviral siRNAs inhibit hepatic adenovirus infection in immunosuppressed Syrian hamsters. Geisler A, Dieringer B, Elsner L, Klingel K, Klopfleisch R, Vornlocher HP, Kurreck J, Fechner H. Molecular Therapy Nucleis Acids, 2023, Volume 32, P923-936. Read Article.

2022

Treatment of the metabolic syndrome by siRNA targeting apolipoprotein CIII. Recio-López P, Valladolid-Acebes I, Hadwiger P, Hossbach M, Krampert M, Prata C, Berggren PO, & Juntti-Berggren L. BioFactors (Oxford, England), 2022, Online ahead of print. Read Article.

2021

Access to 1'-Amino Carbocyclic Phosphoramidite to Enable Postsynthetic Functionalization of Oligonucleotides. Rydzik AM, Balk R, Koegler M, Steinle T, Riether D, Gottschling D, Org Lett. 2021, 23(17),6735. View Abstract.

Chemical and Enzymatic Synthesis of Sialylated Glycoforms of Human Erythropoietin. Unverzagt C, Hessefort H, Gross A, Seeleithner S, Hessefort M, Kirsch T, Perkams L, Bundgaard KO, Gottwald K, Rau D, Graf CG, Rozanski E, Weidler S. Angew Chem Int Ed Engl. 2021. Online ahead of print. Read Article.

2020

CDR132L improves systolic and diastolic function in a large animal model of chronic heart failure. Batkai S, Genschel C, Viereck J, Rump S, Bär C, Borchert T, Traxler D, Riesenhuber M, Spannbauer A , Lukovic D, Zlabinger K, Hašimbegović E, Winkler J, Garamvölgyi R, Neitzel S, Gyöngyösi M, Thum T, European Herat Journal. 2020, PMID: 33089304 DOI: 10.1093/eurheartj/ehaa791. Read Article.

Preclinical development of a miR-132 inhibitor for heart failure treatment. Foinquinos A, Batkai S, Genschel C, Viereck J, Rump S, Gyöngyösi M, Traxler D, Riesenhuber M, Spannbauer A, Lukovic D, Weber N, Zlabinger K, Hašimbegović E, Winkler J, Jan Fiedler, Dangwal S, Fischer M, de la Roche J, Wojciechowski D, Kraft T, Garamvölgyi R, Neitzel S, Chatterjee S, Yin X, Bär C, Mayr M, Ke Xiao K & Thum T, Nature Communications. 2020, (11), 633. Read Article.

Immune Sensing of Synthetic, Bacterial, and Protozoan RNA by Toll-like Receptor 8 Requires Coordinated Processing by RNase T2 and RNase 2. Ostendorf T, Zillinger T, Andryka K, Schlee-Guimaraes TM, Schmitz S, Marx S, Bayrak K, Linke R, Salgert S, Wegner J, Grasser T, Bauersachs S, Soltesz L, Hübner MP, Nastaly M, Coch C, Kettwig M, Roehl I, Henneke M, Hoerauf A, Barchet W, Gärtner J, Schlee M, Hartmann G, Bartok E, Immunity. 2020, 52, (4), 591. Read Abstract.

Targeted Inhibition of the NUP98-NSD1 Fusion Oncogene in Acute Myeloid Leukemia. Mohanty S, Jyotsana N, Sharma A, Kloos A, Gabdoulline R, Othman B, Lai CK, Schottmann R, Mandhania M, Schmoellerl J, Grebien F, Ramsay E, Thomas A, Vornlocher HP, Ganser A, Thol F and Heuser M, Cancers. 2020, 12(10), 2766. Read Article.

2019

Determination of low-picomolar affinities of sgRNAs and crRNA/tracrRNAs for Cas9. Theer T, Deckert J, Hadwiger P, Röhl I, Breitsprecher D, Tschammer N. NanoTemper Application Notes. 2019, NT-MO-030-01. Read Article.

Ligand Conjugated Multimeric siRNAs Enable Enhanced Uptake and Multiplexed Gene Silencing. Brown JM, Dahlman JE, Neuman KK, Prata CAH, Krampert MC, Hadwiger PM, Vornlocher HP. Nucleic Acid Ther. 2019, in press. Read article.

Lipid nanoparticle-mediated siRNA delivery for safe targeting of human CML in vivo. Jyotsana N, Sharma A, Chaturvedi A, Budida R, Scherr M, Kuchenbauer F, Lindner R, Noyan F, Sühs KW, Stangel M, Grote-Koska D, Brand K, Vornlocher HP, Eder M, Thol F, Ganser A, Humphries RK,0, Ramsay E, Cullis P, Heuser M. Ann Hematol. 2019 May 18. doi: 10.1007/s00277-019-03713-y. Read abstract.

2018

2018 White Paper on Recent Issues in Bioanalysis: 'A global bioanalytical community perspective on last decade of incurred samples reanalysis (ISR)' (Part 1 - small molecule regulated bioanalysis, small molecule biomarkers, peptides & oligonucleotide bioanalysis). Welink J, Xu Y, Yang E, Wilson A, Henderson N, Luo L, Fraser S, Kavita U, Musuku A, James C, Fraier D, Zhang Y, Goykhman D, Summerfield S, Woolf E, Verhaeghe T, Hughes N, Behling A, Brown K, Bulychev A, Buonarati M, Cherry E, Cho SJ, Cludts I, Dillen L, Dodge R, Edmison A, Garofolo F, Green R, Haidar S, Hottenstein C, Ishii-Watabe A, Jang HG, Ji A, Jones B, Kassim S, Ma M, Lima Santos GM, Norris DA, Owen T, Piccoli S, Ramanathan R, Roehl I, Rosenbaum AI, Saito Y, Sangster T, Savoie N, Stebbins C, Sydor J, de Merbel NV, Verthelyi D, Vinter S, Whale E. Bioanalysis. 2018 Nov 1;10(22):1781-1801. Read article.

RNA interference efficiently targets human leukemia driven by a fusion oncogene in vivo. Jyotsana N, Sharma A, Chaturvedi A, Scherr M, Kuchenbauer F, Sajti L, Barchanski A, Lindner R, Noyan F, Sühs KW, Grote-Koska D, Brand K, Vornlocher HP, Stanulla M, Bornhauser B, Bourquin JP, Eder M, Thol F, Ganser A, Humphries RK, Ramsay E, Cullis P, Heuser M. Leukemia. 2018 Jan;32(1):224-226.  Read article

Activity of nucleic acid polymers in rodent models of HBV infection. Schöneweis K, Motter N, Roppert PL, Lu M, Wang B, Roehl I, Glebe D, Yang D, Morrey JD, Roggendorf M, Vaillant A. Antiviral Research. 2018 Jan;149:26-33. Read article

Silencing the CSF-1 axis using nanoparticle encapsulated siRNA mitigates viral and autoimmune myocarditis. Meyer IS, Goetzke CC, Kespohl M, Sauter M, Heuser A, Eckstein V, Vornlocher HP, Anderson AG, Haas J, Meder B, Katus HA, Klingel K, Beling A and Leuschner F. Frontiers in Immunology. 2018; 9: 2303. Read article.

2017

Nucleic acid polymers with accelerated plasma and tissue clearance for chronic hepatitis B therapy. Roehl I, Seiffert S, Brikh C, Quinet J, Jamard C, Dörfler N, Lockridge JA, Cova L, Vaillant A. Molecular Therapy - Nucleic Acids. 2017, 8:1-12. Read Article

Identification of novel fibrosis modifiers by in vivo siRNA silencing. Vollmann EH, Cao L, Amatucci A, Reynolds T, Hamann S, Dalkilic-Liddle I, Cameron TO, Hossbach M, Kauffman KJ, Mir FF, Anderson DG, Novobrantseva T, Koteliansky V, Kisseleva T, Brenner D, Duffield J, Burkly LC. Molecular Therapy - Nucleic Acids. 2017, 7:314-323. Read Article

Monitoring integrity and localization of modified single-stranded RNA oligonucleotides using ultrasensitive fluorescence methods. Heissig P, Schrimpf W, Hadwiger P, Wagner E, Lamb DC. PLoS One. 2017, 12(3):e0173401. Read Article

2016

Hepatitis B virus genome replication triggers toll-like receptor 3-dependent interferon responses in the absence of hepatitis B surface antigen. Real CI, Lu M, Liu J, Huang X, Trippler M, Hossbach M, Deckert J, Jahn-Hofmann K, Ickenstein LM, John MJ, Gibbert K, Dittmer U, Vornlocher HP, Schirmbeck R, Gerken G, Schlaak JF, Broering R. Scientific Reports. 2016, 6:24865 Read Article.

DNA as Tunable Adaptor for siRNA Polyplex Stabilization and Functionalization. Heissig P, Klein PM, Hadwiger P, Wagner E. Molecular Therapy—Nucleic Acids. 2016, 5: e288 Read Article

2015

Isoforms of Melanopsin Mediate Different Behavioral Responses to Light. Jagannath A, Hughes S, Abdelgany A, Pothecary CA, Di Pretoro S, Pires SS, Vachtsevanos A, Pilorz V, Brown LA, Hossbach M, MacLaren RE, Halford S, Gatti S, Hankins MW, Wood MJ, Foster RG, Peirson SN. Current Biology. 2015, Sep 21;25(18):2430-2434. Read Article

2014

Silver Arrays Inside DNA Duplexes Constructed from Silver(I)-Mediated Pyrrolo-dC–Pyrrolo-dC Base Pairs. Mei H, Yang H, Roehl I, Seela F. ChemPlusChem 2014, DOI: 10.1002/cplu.201402060. Read Abstract

Enzyme-triggered PEGylated siRNA-nanoparticles for controlled release of siRNA.Yingyuad P, Mével M, Prata C, Kontogiorgis C, Thanou M, Miller AD. J RNAi Gene Silencing 2014, Jan 28;10:490-499. Read Article

In vitro and in vivo efficacy of SYL040012, a novel siRNA compound for treatment of glaucoma. Martínez T, González V, Roehl I, Wright N, Pañeda C, Jiménez AI. Molecular Therapy. 2014 Jan; 22(1) :81-91 Read Article

2013

The CRTC1-SIK1 Pathway Regulates Entrainment of the Circadian Clock. Jagannath A, Butler R, Godinho SIH, Couch Y, Brown LA, Vasudevan SR, Flanagan KC, Anthony D, Churchill GC, Wood MJA, Steiner G, Ebeling M, Hossbach M, Wettstein JG, Duffield GE, Gatti S, Hankins MW, Foster RG and Peirson SN. Cell. 2013 Aug 29; 154(5):1100-1111 View Summary

Ag+-mediated DNA base pairing: extraordinary stable pyrrolo-dC - pyrrolo-dC pairs binding two silver ions. Mei H, Roehl I, Seela F. JOrgChem. 2013 Aug 21. View Abstract

Separation of Oligonucleotides and Related Substances. Noll B and Roehl I. Analysis of Oligonucleotides and their Related Substances. 2013 Jun 5; 157-200. ISBN: 9781906799144.Content

cGAS produces a 2'-5'-linked cyclic dinucleotide second messenger that activates STING. Ablasser A, Goldeck M, Cavlar T, Deimling T, Witte G, Roehl I, Hopfner KP, Ludwig J, Hornung V. Nature. 2013 May 30. doi: 10.1038/nature12306. View Abstract

Hepatocyte-targeted RNAi Therapeutics for the Treatment of Chronic Hepatitis B Virus Infection. Wooddell CI, Rozema DB, Hossbach M, John M, Hamilton HL, Chu Q, Hegge JO, Klein JJ, Wakefield DH, Oropeza CE, Deckert J, Roehl I, Jahn-Hofmann K, Hadwiger P, Vornlocher HP, McLachlan A & Lewis DL. Molecular Therapy. 2013 doi:10.1038/mt.2013.31View Article

IFNs and ISGs are up-regulated in HBV-transgenic mice lacking HBsAg. Real CI, Broering R, Trippler M, Jahn-Hofmann K, Ickenstein L, John M, Kleinehr K, Driftmann S, Kemper T, Vornlocher HP, Reimann J, Schirmbeck R, Lu M, Gerken G, Schlaak JF. Z Gastroenterol. 2013; 51-P_5_56 View Abstract

2012

Assessing unintended hybridization-induced biological effects of oligonucleotides.Lindow M, Vornlocher HP, Riley D, Kornbrust DJ, Burchard J, Whiteley LO, Kamens J, Thompson JD, Nochur S, Younis H, Bartz S, Parry J, Ferrari N, Henry SP, Levin AA. Nat Biotechnol. 2012 Oct 10;30(10):920-3. View Abstract

Nano Sized Multifunctional Polyplexes for Receptor Mediated SiRNA Delivery. Dohmen C, Edinger D, Froehlich T, Schreiner L, Laechelt U, Troiber C, Rädler J, Hadwiger P, Vornlocher HP, Wagner E. ACS Nano. 2012 May 30. View Abstract on PubMed

Structure-activity relationships of siRNA carriers based on sequence-defined oligo (ethane amino) amides. Froehlich T, Edinger D, Klaeger R, Troiber C, Salcher E, Badgujar N, Martin I, Schaffert D, Cengizeroglu A, Hadwiger P, Vornlocher HP, Wagner E. J Control Release. 2012; 160(3):532-541. Epub 2012 Mar 24. View Abstract on PubMed

Defined Folate-PEG-siRNA Conjugates for Receptor-specific Gene Silencing. Dohmen C, Froehlich T, Laechelt U, Roehl I, Vornlocher HP, Hadwiger P and Wagner E. Molecular Therapy–Nucleic Acids. 2012; 1, e7. View Article

Delivery of siRNA to the Mouse Lung via a Functionalized Lipopolyamine. Polach KJ, Matar M, Rice J, Slobodkin G, Sparks J, Congo R, Rea-Ramsey A, McClure D, Brunhoeber E, Krampert M, Schuster A, Jahn-Hofmann K, John M, Vornlocher HP, Fewell JG, Anwer K, Geick A. Mol Ther. 2012 Jan;20(1):91-100. Epub 2011 Oct 11. View Abstract on PubMed

2011

Synthesis, characterization, and in vitro transfection activity of charge-reversal amphiphiles for DNA delivery. Zhang XX, Prata CA, Berlin JA, McIntosh TJ, Barthelemy P, Grinstaff MW. Bioconjug Chem. 2011 Apr 20;22(4):690-9. Epub 2011 Apr 1. View Abstract on PubMed

EGFRvIII promotes glioma angiogenesis and growth through the NF-κB, interleukin-8 pathway. Bonavia R, Inda MM, Vandenberg S, Cheng SY, Nagane M, Hadwiger P, Tan P, Sah DW, Cavenee WK, Furnari FB. Oncogene. 2011 Dec 5. View Abstract on PubMed

Purification of small interfering RNA using nondenaturing anion-exchange chromatography. Noll B, Seiffert S, Hertel F, Debelak H, Hadwiger P, Vornlocher HP, Roehl I.Nucleic Acid Ther. 2011; 21, 383-93. Epub 2011 Oct 21. View Abstract on PubMed

Solid-phase synthesized sequence-defined t-shape, i-shape and u-shape polymers for pDNA and siRNA delivery. Schaffert D, Troiber C, Salcher EE, Fröhlich T, Martin I, Badgujar N, Dohmen C, Edinger D, Kläger R, Maiwald G, Farkasova K, Seeber S, Jahn-Hofmann K, Hadwiger P, Wagner E. Angewandte Chemie International Edition. 2011; 50, 8986-8989.View Abstract on PubMed

Unique gene-silencing and structural properties of 2’-fluoro modified siRNAs. Manoharan M, Akinc A, Pandey RK, Quin J, Hadwiger P, John M, Mills K, Charisse K, Maier M, Nechev L, Greene EM, Pallan, PS, Rozners E, Rajeev KG, Egli M. Angewandte Chemie. 2011; 123(10), 2332-2336. View Abstract on PubMed

Characterization of Side Reactions During the Annealing of Small Interfering RNAs. Seiffert S, Debelak H, Hadwiger P, Jahn-Hofmann K, Roehl I, Vornlocher HP, Noll B. Analytical Biochemistry. 2011; 414(1), 47-57. View Abstract on PubMed

Characterization of small interfering RNA by non-denaturing ion-pair reversed-phase liquid chromatography. Noll B, Seiffert S, Vornlocher HP, Roehl I. J Chromatogr A. 2011; 1218(33):5609-17. Epub 2011 Jun 22. View Abstract on PubMed

2010

Cationic nucleoside lipids derived from universal bases: A rational approach for siRNA transfection. Ceballos C, Khiati S, Prata CA, Zhang XX, Giorgio S, Marsal P, Grinstaff MW, Barthélémy P, Camplo M. Bioconjug Chem. 2010 Jun 16;21(6):1062-9. View Abstract on PubMed

The effect of charge-reversal amphiphile spacer composition on DNA and siRNA delivery.Zhang XX, Prata CA, McIntosh TJ, Barthélémy P, Grinstaff MW. Bioconjug Chem. 2010 May 19;21(5):988-93. View Abstract on PubMed

Modulation of thermal stability can enhance the potency of siRNA. Addepalli H, Meena, Peng CG, Wang G, Fan Y, Charisse K, Jayaprakash KN, Rajeev KG, Pandey RK, Lavine G, Zhang L, Jahn-Hofmann K, Hadwiger P, Manoharan M, Maier MA. Nucleic Acids Research. 2010; 38(20), 7320-7331. View Abstract on PubMed

Tumor heterogeneity is an active process maintained by a mutant EGFR-induced cytokine circuit in glioblastoma. Inda M, Bonavia R, Mukasa A, Narita Y, Sah DWY, Vandenberg S, Brennan C, Johns TG, Bachoo R, Hadwiger P, Tan P, De Pinho RA, Cavenee W, Furnari F. Genes & Development. 2010; 24(16), 1731-1745. View Abstract on PubMed

Polyethylenimine/small interfering RNA-mediated knockdown of vascular endothelial growth factor in vivo exerts anti-tumor effects synergistically with Bevacizumab. Hoebel S, Koburger I, John M, Czubayko F, Hadwiger P, Vornlocher HP, Aigner A. Journal of Gene Medicine. 2010; 12(3), 287-300. View Abstract on PubMed

2009

RNA interference-mediated silencing of the respiratory syncytial virus nucleocapsid defines a potent antiviral strategy. Alvarez R, Elbashir S, Borland T, Toudjarska I, Hadwiger P, John M, Roehl I, Morskaya SS, Martinello R, Kahn J, Van Ranst M, Tripp RA, DeVincenzo JP, Pandey R, Maier M, Nechev L, Manoharan M, Kotelianski V, Meyers R. Antimicrobial Agents and Chemotherapy. 2009; 53(9), 3952-3962. View Abstract on PubMed

Cationic nucleoside lipids based on a 3-nitropyrrole universal base for siRNA delivery.Ceballos C, Prata CA, Giorgio S, Garzino F, Payet D, Barthélémy P, Grinstaff MW, Camplo M.Bioconjug Chem. 2009 Feb;20(2):193-6. View Abstract on PubMed

FAPP2 gene downregulation increases tumor cell sensitivity to Fas-induced apoptosis.Tritz R, Hickey MJ, Lin AH, Hadwiger P, Sah DWY, Neuwelt EA, Mueller BM, Kruse CA.Biochemical and Biophysical Research Communications. 2009; 383(2), 167-171. View Abstract on PubMed

2008

siRNA Down-regulation of the PATZ1 Gene in Human Glioma Cells Increases Their Sensitivity to Apoptotic Stimuli. Tritz R, Mueller BM, Hickey MJ, Lin AH, Gomez GG, Hadwiger P, Sah DW, Muldoon L, Neuwelt EA, Kruse CA. Cancer Ther. 2008;6(B):865-876. View Abstract on PubMed

Host scavenger receptor SR-BI plays a dual role in the establishment of malaria parasite liver infection. Rodrigues CD, Hannus M, Prudêncio M, Martin C, Gonçalves LA, Portugal S, Epiphanio S, Akinc A, Hadwiger P, Jahn-Hofmann K, Röhl I, van Gemert GJ, Franetich JF, Luty AJ, Sauerwein R, Mazier D, Koteliansky V, Vornlocher HP, Echeverri CJ, Mota MM. Cell Host Microbe. 2008 Sep 11;4(3):271-82. View Abstract on PubMed

Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates. Frank-Kamenetsky M, Grefhorst A, Anderson NN, Racie TS, Bramlage B, Akinc A, Butler D, Charisse K, Dorkin R, Fan Y, Gamba-Vitalo C, Hadwiger P, Jayaraman M, John M, Jayaprakash KN, Maier M, Nechev L, Rajeev KG, Read T, Röhl I, Soutschek J, Tan P, Wong J, Wang G, Zimmermann T, de Fougerolles A, Vornlocher HP, Langer R, Anderson DG, Manoharan M, Koteliansky V, Horton JD, Fitzgerald K. Proc Natl Acad Sci U S A. 2008 Aug 19;105(33):11915-20. Epub 2008 Aug 11. View Abstract on PubMed

A combinatorial library of lipid-like materials for delivery of RNAi therapeutics. Akinc A, Zumbuehl A, Goldberg M, Leshchiner ES, Busini V, Hossain N, Bacallado SA, Nguyen DN, Fuller J, Alvarez R, Borodovsky A, Borland T, Constien R, de Fougerolles A, Dorkin JR, Narayanannair Jayaprakash K, Jayaraman M, John M, Koteliansky V, Manoharan M, Nechev L, Qin J, Racie T, Raitcheva D, Rajeev KG, Sah DW, Soutschek J, Toudjarska I, Vornlocher HP, Zimmermann TS, Langer R, Anderson DG. Nat Biotechnol. 2008 May;26(5):561-9. Epub 2008 Apr 27. View Abstract on PubMed

A new helper phospholipid for gene delivery. Prata CA, Li Y, Luo D, McIntosh TJ, Barthelemy P, Grinstaff MW. Chem Commun (Camb). 2008 Apr 7;(13):1566-8. Epub 2008 Jan 29. View Abstract on PubMed

Lipophilic peptides for gene delivery. Prata CA, Zhang XX, Luo D, McIntosh TJ, Barthelemy P, Grinstaff MW. Bioconjug Chem. 2008 Feb;19(2):418-20. Epub 2008 Jan 11. View Abstract on PubMed

siRNA knock-down of mutant torsinA restores processing through secretory pathway in DYT1 dystonia cells. Hewett JW, Nery FC, Niland B, Ge P, Tan P, Hadwiger P, Tannous BA, Sah DW, Breakefield XO. Hum Mol Genet. 2008 May 15;17(10):1436-45. Epub 2008 Feb 7. View Abstract on PubMed

Heme oxygenase-1 is an anti-inflammatory host factor that promotes murine plasmodium liver infection. Epiphanio S, Mikolajczak SA, Gonçalves LA, Pamplona A, Portugal S, Albuquerque S, Goldberg M, Rebelo S, Anderson DG, Akinc A, Vornlocher HP, Kappe SH, Soares MP, Mota MM. Cell Host Microbe. 2008; 3,331-8. View Abstract on PubMed

2007

Effective RNAi-mediated gene silencing without interruption of the endogenous microRNA pathway. John M, Constien R, Akinc A, Goldberg M, Moon YA, Spranger M, Hadwiger P, Soutschek J, Vornlocher HP, Manoharan M, Stoffel M, Langer R, Anderson DG, Horton JD, Koteliansky V, Bumcrot D. Nature. 2007 Oct 11;449(7163):745-7. Epub 2007 Sep 26. View Abstract on PubMed

Bcl-2 downregulation sensitizes nonsmall cell lung cancer cells to cisplatin, but not to docetaxel. Losert D, Pratscher B, Soutschek J, Geick A, Vornlocher HP, Müller M, Wacheck V.Anticancer Drugs. 2007 Aug;18(7):755-61. View Abstract on PubMed

Interfering with disease: a progress report on siRNA-based therapeutics. de Fougerolles A, Vornlocher HP, Maraganore J, Lieberman J. Nat Rev Drug Discov. 2007 Jun;6(6):443-53. Review. View Abstract on PubMed

2006

Hyperoxia causes angiopoietin 2-mediated acute lung injury and necrotic cell death.Bhandari V, Choo-Wing R, Lee CG, Zhu Z, Nedrelow JH, Chupp GL, Zhang X, Matthay MA, Ware LB, Homer RJ, Lee PJ, Geick A, de Fougerolles AR, Elias JA. Nat Med. 2006 Nov;12(11):1286-93. Epub 2006 Nov 5. View Abstract on PubMed

Effective silencing of EGFR with RNAi demonstrates non-EGFR dependent proliferation of glioma cells. Vollmann A, Vornlocher HP, Stempfl T, Brockhoff G, Apfel R, Bogdahn U. Int J Oncol. 2006 Jun;28(6):1531-42. View Abstract on PubMed

RNAi-mediated gene silencing in non-human primates. Zimmermann TS, Lee AC, Akinc A, Bramlage B, Bumcrot D, Fedoruk MN, Harborth J, Heyes JA, Jeffs LB, John M, Judge AD, Lam K, McClintock K, Nechev LV, Palmer LR, Racie T, Röhl I, Seiffert S, Shanmugam S, Sood V, Soutschek J, Toudjarska I, Wheat AJ, Yaworski E, Zedalis W, Koteliansky V, Manoharan M, Vornlocher HP, MacLachlan I. Nature. 2006 May 4;441(7089):111-4. Epub 2006 Mar 26. View Abstract on PubMed 

Non-viral charge reversal vectors for pDNA delivery. Prata CA, Li Y, Luo D, McIntosh TJ, Grinstaff MW. Conf Proc IEEE Eng Med Biol Soc. 2006;1:4347-9. View Abstract on PubMed

Gene silencing with siRNA duplexes composed of target-mRNA-complementary and partially palindromic or partially complementary single-stranded siRNAs. Hossbach M, Gruber J, Osborn M, Weber K, Tuschl T. RNA Biol. 2006 Apr;3(2):82-9. Epub 2006 Apr 19.View Abstract on PubMed

2005

Antibody-directed cell-type-specific delivery of siRNA. Vornlocher HP. Trends Mol Med.2006 Jan;12(1):1-3. Epub 2005 Nov 14. View Abstract on PubMed

Nucleic acid complexing glycosyl nucleoside-based amphiphile. Arigon J, Prata CA, Grinstaff MW, Barthélémy P. Bioconjug Chem. 2005 Jul-Aug;16(4):864-72. View Abstract on PubMed

Supramolecular assemblies of DNA with neutral nucleoside amphiphiles. Barthelemy P, Prata CA, Filocamo SF, Immoos CE, Maynor BW, Hashmi SA, Lee SJ,
Grinstaff MW. Chem Commun (Camb). 2005 Mar 14;(10):1261-3. Epub 2005 Jan 21. View Abstract on PubMed

Nucleoside phosphocholine amphiphile for in vitro DNA transfection. Moreau L, Barthélémy P, Li Y, Luo D, Prata CA, Grinstaff MW. Mol Biosyst. 2005 Sep;1(3):260-4. Epub 2005 Jul 22. View Abstract on PubMed

Dendritic supramolecular assemblies for drug delivery. Morgan MT, Carnahan MA, Finkelstein S, Prata CA, Degoricija L, Lee SJ, Grinstaff MW. Chem Commun (Camb). 2005 Sep 14;(34):4309-11. Epub 2005 Jul 14. View Abstract on PubMed

Targeting MLL-AF4 with short interfering RNAs inhibits clonogenicity and engraftment of t(4;11)-positive human leukemic cells. Thomas M, Gessner A, Vornlocher HP, Hadwiger P, Greil J, Heidenreich O. Blood. 2005 Nov 15;106(10):3559-66. Epub 2005 Jul 26. View Abstract on PubMed

RNA interference in vivo: toward synthetic small inhibitory RNA-based therapeutics. de Fougerolles A, Manoharan M, Meyers R, Vornlocher HP. Methods Enzymol. 2005;392:278-96.View Abstract on PubMed

Chemical modifications to achieve increased stability and sensitive detection of siRNA.Hadwiger P, and Vornlocher HP. In: RNA Interference: From Basic Science to Drug Development (K. Appasani, ed.), Cambridge University Press, Cambridge. 2005; 194-206.View Book Summary

Gene silencing methods for mammalian cells: application of synthetic short interfering RNAs. John M, Geick A, Hadwiger P, Vornlocher HP, Heidenreich O. Handbook of RNA Biochemistry. 2005; 897-909. View Book Summary

2004

Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Soutschek J, Akinc A, Bramlage B, Charisse K, Constien R, Donoghue M, Elbashir S, Geick A, Hadwiger P, Harborth J, John M, Kesavan V, Lavine G, Pandey RK, Racie T, Rajeev KG, Röhl I, Toudjarska I, Wang G, Wuschko S, Bumcrot D, Koteliansky V, Limmer S, Manoharan M, Vornlocher HP. Nature. 2004 Nov 11;432(7014):173-8. View Abstract on PubMed

Steroid and lipid conjugates of siRNAs to enhance cellular uptake and gene silencing in liver cells. Lorenz C, Hadwiger P, John M, Vornlocher HP, Unverzagt C. Bioorg Med Chem Lett. 2004 Oct 4;14(19):4975-7. View Abstract on PubMed

siRNA Selection Server: an automated siRNA oligonucleotide prediction server. Yuan B, Latek R, Hossbach M, Tuschl T, Lewitter F. Nucleic Acids Res. 2004 Jul1;32 (Web Server issue):W130-4. View Abstract on PubMed

RNAi knockdown of hPrp31 leads to an accumulation of U4/U6 di-snRNPs in Cajal bodies.Schaffert N, Hossbach M, Heintzmann R, Achsel T, Luehrmann R. EMBO J. 2004 Aug 4;23(15):3000-9. View Abstract on PubMed

All common p210 and p190 Bcr-abl variants can be targeted by RNA-interference.Wohlbold L, van der Kuip H, Moehring A, Granot G, Oren M, Vornlocher HP, and Aulitzky W.Leukemia. 2004; 19, 290-292. View Abstract on PubMed

c-FLIP proteins mediate resistance of Hodgkin-/Reed-Sternberg cells to death receptor induced apoptosis. Mathas S, Lietz A, Anagnostopoulos I, Hummel F, Janz M, Jundt F, Jöhrens-Leder K, Vornlocher HP, Bommert K, Stein H, Dörken B. J. Exp. Med. 2004; 199:1041-1052. View Abstract on PubMed

Charge-reversal amphiphiles for gene delivery. Prata CA, Zhao Y, Barthelemy P, Li Y, Luo D, McIntosh TJ, Lee SJ, Grinstaff MW. J Am Chem Soc. 2004 Oct 6;126(39):12196-7. View Abstract on PubMed

The oncogenic fusion protein RUNX1-CBFA2T1 supports proliferation and inhibits senescence in t(8;21)-positive leukaemic cells. Martinez N, Drescher B, Thomas M, Riehle H, Cullmann C, Vornlocher HP, Greil J, Ganser A, Heil G, Nordheim A, Krauter J, Heidenreich O. BMC Cancer. 2004; 4:44. View Abstract on PubMed

2003

Small interfering RNA targeting bcl-2 sensitizes malignant melanoma. Wacheck V, Losert D, Günsberg P, Vornlocher HP, Hadwiger P, Geick A, Pehamberger H, Müller M, Jansen B.Oligonucleotides. 2003;13(5):393-400. View Abstract on PubMed

The apparent uptake of fluorescently labeled siRNAs by electroporated cells depends on the fluorochrome. Dunne J, Drescher B, Riehle H, Hadwiger P, Young BD, Krauter J, Heidenreich O. Oligonucleotides. 2003;13(5):375-80. View Abstract on PubMed

Inhibition of bcr-abl gene expression by small interfering RNA sensitizes for imatinib mesylate (STI571). Wohlbold L, van der Kuip H, Miething C, Vornlocher HP, Knabbe C, Duyster J, Aulitzky WE. Blood. 2003 Sep 15;102(6):2236-9. Epub 2003 May 15. View Abstract on PubMed

Gene silencing by RNAi in mammalian cells. John M, Geick A, Hadwiger P, Vornlocher HP, Heidenreich O. Curr Protoc Mol Biol. 2003 May;Chapter 26:Unit 26.2. View Abstract on PubMed

A new class of Spiegelmers containing 2'-fluoro-nucleotides. Helmling S, Moyroud E, Schroeder W, Roehl I, Kleinjung F, Stark S, Bahrenberg G, Gillen C, Klussmann S, Vonhoff S.Nucleosides Nucleotides Nucleic Acids. 2003 May-Aug;22(5-8):1035-8. View Abstract on PubMed

Aminomodified nucleobases: functionalized nucleoside triphosphates applicable for SELEX. Schoetzau T, Langner J, Moyroud E, Roehl I, Vonhoff S, Klussmann S. Bioconjug Chem.2003 Sep-Oct;14(5):919-26. View Abstract on PubMed

AML1/MTG8 oncogene suppression by small interfering RNAs supports myeloid differentiation of t(8;21)-positive leukemic cells. Heidenreich O, Krauter J, Riehle H, Hadwiger P, John M, Heil G, Vornlocher HP, Nordheim A. Blood. 2003 Apr 15;101(8):3157-63. Epub 2002 Dec 12. View Abstract on PubMed

siRNA als Plattform für die Entwicklung innovativer Therapeutika in der Onkologie.Wacheck V und Vornlocher HP. Der Onkologe. 2003; 9:1121-1129. View Abstract

Gene silencing by RNAi in mammalian cells. John M, Geick A, Hadwiger P, Vornlocher HP, Heidenreich O. In: Current Protocols in Molecular Biology, John Wiley & Sons, New York.(2003) pp. 26.2.1-26.2.14. View Abstract on PubMed

2002

Selective inhibition of FLICE-like inhibitory protein expression with small interfering RNA oligonucleotides is sufficient to sensitize tumor cells for TRAIL-induced apoptosis.Siegmund D, Hadwiger P, Pfizenmaier K, Vornlocher HP, Wajant H. Mol Med. 2002 Nov;8(11):725-32.
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