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Next-generation of targeted AAVP vectors for systemic transgene delivery against cancer.

Suwan K, Yata T, Waramit S, Przystal JM, Stoneham CA, Bentayebi K, Asavarut P, Chongchai A, Pothachareon P, Lee KY, Topanurak S, Smith TL, Gelovani JG, Sidman RL, Pasqualini R, Arap W, Hajitou A.

Proc Natl Acad Sci U S A. 2019 Sep 10;116(37):18571-18577. doi: 10.1073/pnas.1906653116. Epub 2019 Aug 2.


Targeted AAVP-based therapy in a mouse model of human glioblastoma: a comparison of cytotoxic versus suicide gene delivery strategies.

Staquicini FI, Smith TL, Tang FHF, Gelovani JG, Giordano RJ, Libutti SK, Sidman RL, Cavenee WK, Arap W, Pasqualini R.

Cancer Gene Ther. 2019 May 27. doi: 10.1038/s41417-019-0101-2. [Epub ahead of print]


A ligand motif enables differential vascular targeting of endothelial junctions between brain and retina.

Tang FHF, Staquicini FI, Teixeira AAR, Michaloski JS, Namiyama GM, Taniwaki NN, Setubal JC, da Silva AM, Sidman RL, Pasqualini R, Arap W, Giordano RJ.

Proc Natl Acad Sci U S A. 2019 Feb 5;116(6):2300-2305. doi: 10.1073/pnas.1809483116. Epub 2019 Jan 22.


Emerging Pharmacologic Targets in Cerebral Cavernous Malformation and Potential Strategies to Alter the Natural History of a Difficult Disease: A Review.

Chohan MO, Marchiò S, Morrison LA, Sidman RL, Cavenee WK, Dejana E, Yonas H, Pasqualini R, Arap W.

JAMA Neurol. 2019 Apr 1;76(4):492-500. doi: 10.1001/jamaneurol.2018.3634.


Modeling Complex Neurological Diseases with Stem Cells: A Study of Bipolar Disorder.

Pernia CD, Nathan NH, Tobe BTD, Winquist AM, Sidman RL, Goshima Y, Snyder EY.

Results Probl Cell Differ. 2018;66:265-282. doi: 10.1007/978-3-319-93485-3_12.


Selection of phage-displayed accessible recombinant targeted antibodies (SPARTA): methodology and applications.

D'Angelo S, Staquicini FI, Ferrara F, Staquicini DI, Sharma G, Tarleton CA, Nguyen H, Naranjo LA, Sidman RL, Arap W, Bradbury AR, Pasqualini R.

JCI Insight. 2018 May 3;3(9). pii: 98305. doi: 10.1172/jci.insight.98305.


MLH1-rheMac hereditary nonpolyposis colorectal cancer syndrome in rhesus macaques.

Brammer DW, Gillespie PJ, Tian M, Young D, Raveendran M, Williams LE, Gagea M, Benavides FJ, Perez CJ, Broaddus RR, Bernacky BJ, Barnhart KF, Alauddin MM, Bhutani MS, Gibbs RA, Sidman RL, Pasqualini R, Arap W, Rogers J, Abee CR, Gelovani JG.

Proc Natl Acad Sci U S A. 2018 Mar 13;115(11):2806-2811. doi: 10.1073/pnas.1722106115. Epub 2018 Feb 28.


Therapeutic targeting of membrane-associated GRP78 in leukemia and lymphoma: preclinical efficacy in vitro and formal toxicity study of BMTP-78 in rodents and primates.

Staquicini DI, D'Angelo S, Ferrara F, Karjalainen K, Sharma G, Smith TL, Tarleton CA, Jaalouk DE, Kuniyasu A, Baze WB, Chaffee BK, Hanley PW, Barnhart KF, Koivunen E, Marchiò S, Sidman RL, Cortes JE, Kantarjian HM, Arap W, Pasqualini R.

Pharmacogenomics J. 2018 May 22;18(3):436-443. doi: 10.1038/tpj.2017.46. Epub 2017 Dec 5.


Going viral? Linking the etiology of human prostate cancer to the PCA3 long noncoding RNA and oncogenic viruses.

Teixeira AA, Marchiò S, Dias-Neto E, Nunes DN, da Silva IT, Chackerian B, Barry M, Lauer RC, Giordano RJ, Sidman RL, Wheeler CM, Cavenee WK, Pasqualini R, Arap W.

EMBO Mol Med. 2017 Oct;9(10):1327-1330. doi: 10.15252/emmm.201708072. No abstract available.


BMTP-11 is active in preclinical models of human osteosarcoma and a candidate targeted drug for clinical translation.

Lewis VO, Devarajan E, Cardó-Vila M, Thomas DG, Kleinerman ES, Marchiò S, Sidman RL, Pasqualini R, Arap W.

Proc Natl Acad Sci U S A. 2017 Jul 25;114(30):8065-8070. doi: 10.1073/pnas.1704173114. Epub 2017 Jul 11.


Intracellular targeting of annexin A2 inhibits tumor cell adhesion, migration, and in vivo grafting.

Staquicini DI, Rangel R, Guzman-Rojas L, Staquicini FI, Dobroff AS, Tarleton CA, Ozbun MA, Kolonin MG, Gelovani JG, Marchiò S, Sidman RL, Hajjar KA, Arap W, Pasqualini R.

Sci Rep. 2017 Jun 26;7(1):4243. doi: 10.1038/s41598-017-03470-w.


An AAVP-based solid-phase transducing matrix for transgene delivery: potential for translational applications.

Smith TL, Souza GR, Sidman RL, Arap W, Pasqualini R.

Cancer Gene Ther. 2017 Aug;24(8):358-360. doi: 10.1038/cgt.2017.19. Epub 2017 May 26.


Probing the lithium-response pathway in hiPSCs implicates the phosphoregulatory set-point for a cytoskeletal modulator in bipolar pathogenesis.

Tobe BTD, Crain AM, Winquist AM, Calabrese B, Makihara H, Zhao WN, Lalonde J, Nakamura H, Konopaske G, Sidor M, Pernia CD, Yamashita N, Wada M, Inoue Y, Nakamura F, Sheridan SD, Logan RW, Brandel M, Wu D, Hunsberger J, Dorsett L, Duerr C, Basa RCB, McCarthy MJ, Udeshi ND, Mertins P, Carr SA, Rouleau GA, Mastrangelo L, Li J, Gutierrez GJ, Brill LM, Venizelos N, Chen G, Nye JS, Manji H, Price JH, McClung CA, Akiskal HS, Alda M, Chuang DM, Coyle JT, Liu Y, Teng YD, Ohshima T, Mikoshiba K, Sidman RL, Halpain S, Haggarty SJ, Goshima Y, Snyder EY.

Proc Natl Acad Sci U S A. 2017 May 30;114(22):E4462-E4471. doi: 10.1073/pnas.1700111114. Epub 2017 May 12.


Interaction between Tumor Cell Surface Receptor RAGE and Proteinase 3 Mediates Prostate Cancer Metastasis to Bone.

Kolonin MG, Sergeeva A, Staquicini DI, Smith TL, Tarleton CA, Molldrem JJ, Sidman RL, Marchiò S, Pasqualini R, Arap W.

Cancer Res. 2017 Jun 15;77(12):3144-3150. doi: 10.1158/0008-5472.CAN-16-0708. Epub 2017 Apr 20.


Glucosylceramide synthase inhibition alleviates aberrations in synucleinopathy models.

Sardi SP, Viel C, Clarke J, Treleaven CM, Richards AM, Park H, Olszewski MA, Dodge JC, Marshall J, Makino E, Wang B, Sidman RL, Cheng SH, Shihabuddin LS.

Proc Natl Acad Sci U S A. 2017 Mar 7;114(10):2699-2704. doi: 10.1073/pnas.1616152114. Epub 2017 Feb 21.


Cerebellar Pathways in Mouse Model of Purkinje Cell Degeneration Detected by High-Angular Resolution Diffusion Imaging Tractography.

Kanamaru Y, Li J, Stewart N, Sidman RL, Takahashi E.

Cerebellum. 2017 Jun;16(3):648-655. doi: 10.1007/s12311-016-0842-5.


Defining recovery neurobiology of injured spinal cord by synthetic matrix-assisted hMSC implantation.

Ropper AE, Thakor DK, Han I, Yu D, Zeng X, Anderson JE, Aljuboori Z, Kim SW, Wang H, Sidman RL, Zafonte RD, Teng YD.

Proc Natl Acad Sci U S A. 2017 Jan 31;114(5):E820-E829. doi: 10.1073/pnas.1616340114. Epub 2017 Jan 17.


Targeted molecular-genetic imaging and ligand-directed therapy in aggressive variant prostate cancer.

Ferrara F, Staquicini DI, Driessen WHP, D'Angelo S, Dobroff AS, Barry M, Lomo LC, Staquicini FI, Cardó-Vila M, Soghomonyan S, Alauddin MM, Flores LG 2nd, Arap MA, Lauer RC, Mathew P, Efstathiou E, Aparicio AM, Troncoso P, Navone NM, Logothetis CJ, Marchiò S, Gelovani JG, Sidman RL, Pasqualini R, Arap W.

Proc Natl Acad Sci U S A. 2016 Nov 8;113(45):12786-12791. doi: 10.1073/pnas.1615400113. Epub 2016 Oct 24.


Towards a transcriptome-based theranostic platform for unfavorable breast cancer phenotypes.

Dobroff AS, D'Angelo S, Eckhardt BL, Ferrara F, Staquicini DI, Cardó-Vila M, Staquicini FI, Nunes DN, Kim K, Driessen WHP, Hajitou A, Lomo LC, Barry M, Krishnamurthy S, Sahin A, Woodward WA, Prossnitz ER, Anderson RL, Dias-Neto E, Brown-Glaberman UA, Royce ME, Ueno NT, Cristofanilli M, Hortobagyi GN, Marchiò S, Gelovani JG, Sidman RL, Arap W, Pasqualini R.

Proc Natl Acad Sci U S A. 2016 Nov 8;113(45):12780-12785. doi: 10.1073/pnas.1615288113. Epub 2016 Oct 24.


Interleukin-11 Receptor Is a Candidate Target for Ligand-Directed Therapy in Lung Cancer: Analysis of Clinical Samples and BMTP-11 Preclinical Activity.

Cardó-Vila M, Marchiò S, Sato M, Staquicini FI, Smith TL, Bronk JK, Yin G, Zurita AJ, Sun M, Behrens C, Sidman RL, Lee JJ, Hong WK, Wistuba II, Arap W, Pasqualini R.

Am J Pathol. 2016 Aug;186(8):2162-2170. doi: 10.1016/j.ajpath.2016.04.013. Epub 2016 Jun 16.


Brain endothelial cell-targeted gene therapy of neurovascular disorders.

Marchiò S, Sidman RL, Arap W, Pasqualini R.

EMBO Mol Med. 2016 Jun 1;8(6):592-4. doi: 10.15252/emmm.201606407. Print 2016 Jun. No abstract available.


AAVP displaying octreotide for ligand-directed therapeutic transgene delivery in neuroendocrine tumors of the pancreas.

Smith TL, Yuan Z, Cardó-Vila M, Sanchez Claros C, Adem A, Cui MH, Branch CA, Gelovani JG, Libutti SK, Sidman RL, Pasqualini R, Arap W.

Proc Natl Acad Sci U S A. 2016 Mar 1;113(9):2466-71. doi: 10.1073/pnas.1525709113. Epub 2016 Feb 16.


Self-targeting of TNF-releasing cancer cells in preclinical models of primary and metastatic tumors.

Dondossola E, Dobroff AS, Marchiò S, Cardó-Vila M, Hosoya H, Libutti SK, Corti A, Sidman RL, Arap W, Pasqualini R.

Proc Natl Acad Sci U S A. 2016 Feb 23;113(8):2223-8. doi: 10.1073/pnas.1525697113. Epub 2016 Feb 8.


Integrated nanotechnology platform for tumor-targeted multimodal imaging and therapeutic cargo release.

Hosoya H, Dobroff AS, Driessen WH, Cristini V, Brinker LM, Staquicini FI, Cardó-Vila M, D'Angelo S, Ferrara F, Proneth B, Lin YS, Dunphy DR, Dogra P, Melancon MP, Stafford RJ, Miyazono K, Gelovani JG, Kataoka K, Brinker CJ, Sidman RL, Arap W, Pasqualini R.

Proc Natl Acad Sci U S A. 2016 Feb 16;113(7):1877-82. doi: 10.1073/pnas.1525796113. Epub 2016 Feb 2.


Ligand-targeted theranostic nanomedicines against cancer.

Yao VJ, D'Angelo S, Butler KS, Theron C, Smith TL, Marchiò S, Gelovani JG, Sidman RL, Dobroff AS, Brinker CJ, Bradbury ARM, Arap W, Pasqualini R.

J Control Release. 2016 Oct 28;240:267-286. doi: 10.1016/j.jconrel.2016.01.002. Epub 2016 Jan 6. Review.


Targeted Treatment of Experimental Spinal Cord Glioma With Dual Gene-Engineered Human Neural Stem Cells.

Ropper AE, Zeng X, Haragopal H, Anderson JE, Aljuboori Z, Han I, Abd-El-Barr M, Lee HJ, Sidman RL, Snyder EY, Viapiano MS, Kim SU, Chi JH, Teng YD.

Neurosurgery. 2016 Sep;79(3):481-91. doi: 10.1227/NEU.0000000000001174.


The peptidomimetic Vasotide targets two retinal VEGF receptors and reduces pathological angiogenesis in murine and nonhuman primate models of retinal disease.

Sidman RL, Li J, Lawrence M, Hu W, Musso GF, Giordano RJ, Cardó-Vila M, Pasqualini R, Arap W.

Sci Transl Med. 2015 Oct 14;7(309):309ra165. doi: 10.1126/scitranslmed.aac4882.


PRUNE2 is a human prostate cancer suppressor regulated by the intronic long noncoding RNA PCA3.

Salameh A, Lee AK, Cardó-Vila M, Nunes DN, Efstathiou E, Staquicini FI, Dobroff AS, Marchiò S, Navone NM, Hosoya H, Lauer RC, Wen S, Salmeron CC, Hoang A, Newsham I, Lima LA, Carraro DM, Oliviero S, Kolonin MG, Sidman RL, Do KA, Troncoso P, Logothetis CJ, Brentani RR, Calin GA, Cavenee WK, Dias-Neto E, Pasqualini R, Arap W.

Proc Natl Acad Sci U S A. 2015 Jul 7;112(27):8403-8. doi: 10.1073/pnas.1507882112. Epub 2015 Jun 15.


Glycosphingolipids are modulators of disease pathogenesis in amyotrophic lateral sclerosis.

Dodge JC, Treleaven CM, Pacheco J, Cooper S, Bao C, Abraham M, Cromwell M, Sardi SP, Chuang WL, Sidman RL, Cheng SH, Shihabuddin LS.

Proc Natl Acad Sci U S A. 2015 Jun 30;112(26):8100-5. doi: 10.1073/pnas.1508767112. Epub 2015 Jun 8.


Synchronous down-modulation of miR-17 family members is an early causative event in the retinal angiogenic switch.

Nunes DN, Dias-Neto E, Cardó-Vila M, Edwards JK, Dobroff AS, Giordano RJ, Mandelin J, Brentani HP, Hasselgren C, Yao VJ, Marchiò S, Pereira CA, Passetti F, Calin GA, Sidman RL, Arap W, Pasqualini R.

Proc Natl Acad Sci U S A. 2015 Mar 24;112(12):3770-5. doi: 10.1073/pnas.1500008112. Epub 2015 Mar 9.


Selection and identification of ligand peptides targeting a model of castrate-resistant osteogenic prostate cancer and their receptors.

Mandelin J, Cardó-Vila M, Driessen WH, Mathew P, Navone NM, Lin SH, Logothetis CJ, Rietz AC, Dobroff AS, Proneth B, Sidman RL, Pasqualini R, Arap W.

Proc Natl Acad Sci U S A. 2015 Mar 24;112(12):3776-81. doi: 10.1073/pnas.1500128112. Epub 2015 Mar 11.


Discovery and horizontal follow-up of an autoantibody signature in human prostate cancer.

Mintz PJ, Rietz AC, Cardó-Vila M, Ozawa MG, Dondossola E, Do KA, Kim J, Troncoso P, Logothetis CJ, Sidman RL, Pasqualini R, Arap W.

Proc Natl Acad Sci U S A. 2015 Feb 24;112(8):2515-20. doi: 10.1073/pnas.1500097112. Epub 2015 Feb 9.


Ligand-directed targeting of lymphatic vessels uncovers mechanistic insights in melanoma metastasis.

Christianson DR, Dobroff AS, Proneth B, Zurita AJ, Salameh A, Dondossola E, Makino J, Bologa CG, Smith TL, Yao VJ, Calderone TL, O'Connell DJ, Oprea TI, Kataoka K, Cahill DJ, Gershenwald JE, Sidman RL, Arap W, Pasqualini R.

Proc Natl Acad Sci U S A. 2015 Feb 24;112(8):2521-6. doi: 10.1073/pnas.1424994112. Epub 2015 Feb 6.


Ligand-directed profiling of organelles with internalizing phage libraries.

Dobroff AS, Rangel R, Guzman-Roja L, Salmeron CC, Gelovani JG, Sidman RL, Bologa CG, Oprea TI, Brinker CJ, Pasqualini R, Arap W.

Curr Protoc Protein Sci. 2015 Feb 2;79:30.4.1-30. doi: 10.1002/0471140864.ps3004s79.


Receptor tyrosine kinase EphA5 is a functional molecular target in human lung cancer.

Staquicini FI, Qian MD, Salameh A, Dobroff AS, Edwards JK, Cimino DF, Moeller BJ, Kelly P, Nunez MI, Tang X, Liu DD, Lee JJ, Hong WK, Ferrara F, Bradbury AR, Lobb RR, Edelman MJ, Sidman RL, Wistuba II, Arap W, Pasqualini R.

J Biol Chem. 2015 Mar 20;290(12):7345-59. doi: 10.1074/jbc.M114.630525. Epub 2015 Jan 26.


Bone marrow-derived CD13+ cells sustain tumor progression: A potential non-malignant target for anticancer therapy.

Dondossola E, Corti A, Sidman RL, Arap W, Pasqualini R.

Oncoimmunology. 2014 Jan 16;3:e27716. eCollection 2014.


Design, development, and validation of a high-throughput drug-screening assay for targeting of human leukemia.

Karjalainen K, Pasqualini R, Cortes JE, Kornblau SM, Lichtiger B, O'Brien S, Kantarjian HM, Sidman RL, Arap W, Koivunen E.

Cancer. 2014 Feb 15;120(4):589-602. doi: 10.1002/cncr.28419. Epub 2013 Oct 25.


CD13-positive bone marrow-derived myeloid cells promote angiogenesis, tumor growth, and metastasis.

Dondossola E, Rangel R, Guzman-Rojas L, Barbu EM, Hosoya H, St John LS, Molldrem JJ, Corti A, Sidman RL, Arap W, Pasqualini R.

Proc Natl Acad Sci U S A. 2013 Dec 17;110(51):20717-22. doi: 10.1073/pnas.1321139110. Epub 2013 Dec 2.


An antimicrobial peptidomimetic induces Mucorales cell death through mitochondria-mediated apoptosis.

Barbu EM, Shirazi F, McGrath DM, Albert N, Sidman RL, Pasqualini R, Arap W, Kontoyiannis DP.

PLoS One. 2013 Oct 3;8(10):e76981. doi: 10.1371/journal.pone.0076981. eCollection 2013.


Targeting mammalian organelles with internalizing phage (iPhage) libraries.

Rangel R, Dobroff AS, Guzman-Rojas L, Salmeron CC, Gelovani JG, Sidman RL, Pasqualini R, Arap W.

Nat Protoc. 2013 Oct;8(10):1916-39. doi: 10.1038/nprot.2013.119. Epub 2013 Sep 12.


Metabolic signatures of amyotrophic lateral sclerosis reveal insights into disease pathogenesis.

Dodge JC, Treleaven CM, Fidler JA, Tamsett TJ, Bao C, Searles M, Taksir TV, Misra K, Sidman RL, Cheng SH, Shihabuddin LS.

Proc Natl Acad Sci U S A. 2013 Jun 25;110(26):10812-7. doi: 10.1073/pnas.1308421110. Epub 2013 Jun 10.


Tissue plasminogen activator regulates Purkinje neuron development and survival.

Li J, Yu L, Gu X, Ma Y, Pasqualini R, Arap W, Snyder EY, Sidman RL.

Proc Natl Acad Sci U S A. 2013 Jun 25;110(26):E2410-9. doi: 10.1073/pnas.1305010110. Epub 2013 May 14.


Alleviation of chronic pain following rat spinal cord compression injury with multimodal actions of huperzine A.

Yu D, Thakor DK, Han I, Ropper AE, Haragopal H, Sidman RL, Zafonte R, Schachter SC, Teng YD.

Proc Natl Acad Sci U S A. 2013 Feb 19;110(8):E746-55. doi: 10.1073/pnas.1300083110. Epub 2013 Feb 5.


Mechanism of action and initial evaluation of a membrane active all-D-enantiomer antimicrobial peptidomimetic.

McGrath DM, Barbu EM, Driessen WH, Lasco TM, Tarrand JJ, Okhuysen PC, Kontoyiannis DP, Sidman RL, Pasqualini R, Arap W.

Proc Natl Acad Sci U S A. 2013 Feb 26;110(9):3477-82. doi: 10.1073/pnas.1221924110. Epub 2013 Jan 23.


Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathies.

Sardi SP, Clarke J, Viel C, Chan M, Tamsett TJ, Treleaven CM, Bu J, Sweet L, Passini MA, Dodge JC, Yu WH, Sidman RL, Cheng SH, Shihabuddin LS.

Proc Natl Acad Sci U S A. 2013 Feb 26;110(9):3537-42. doi: 10.1073/pnas.1220464110. Epub 2013 Jan 7.


Multimodal actions of neural stem cells in a mouse model of ALS: a meta-analysis.

Teng YD, Benn SC, Kalkanis SN, Shefner JM, Onario RC, Cheng B, Lachyankar MB, Marconi M, Li J, Yu D, Han I, Maragakis NJ, Lládo J, Erkmen K, Redmond DE Jr, Sidman RL, Przedborski S, Rothstein JD, Brown RH Jr, Snyder EY.

Sci Transl Med. 2012 Dec 19;4(165):165ra164. doi: 10.1126/scitranslmed.3004579.


Functional multipotency of stem cells: a conceptual review of neurotrophic factor-based evidence and its role in translational research.

Teng YD, Yu D, Ropper AE, Li J, Kabatas S, Wakeman DR, Wang J, Sullivan MP, Redmond DE Jr, Langer R, Snyder EY, Sidman RL.

Curr Neuropharmacol. 2011 Dec;9(4):574-85. doi: 10.2174/157015911798376299.


Combinatorial targeting and discovery of ligand-receptors in organelles of mammalian cells.

Rangel R, Guzman-Rojas L, le Roux LG, Staquicini FI, Hosoya H, Barbu EM, Ozawa MG, Nie J, Dunner K Jr, Langley RR, Sage EH, Koivunen E, Gelovani JG, Lobb RR, Sidman RL, Pasqualini R, Arap W.

Nat Commun. 2012 Apr 17;3:788. doi: 10.1038/ncomms1773.


Cooperative effects of aminopeptidase N (CD13) expressed by nonmalignant and cancer cells within the tumor microenvironment.

Guzman-Rojas L, Rangel R, Salameh A, Edwards JK, Dondossola E, Kim YG, Saghatelian A, Giordano RJ, Kolonin MG, Staquicini FI, Koivunen E, Sidman RL, Arap W, Pasqualini R.

Proc Natl Acad Sci U S A. 2012 Jan 31;109(5):1637-42. doi: 10.1073/pnas.1120790109. Epub 2012 Jan 17.


Vascular ligand-receptor mapping by direct combinatorial selection in cancer patients.

Staquicini FI, Cardó-Vila M, Kolonin MG, Trepel M, Edwards JK, Nunes DN, Sergeeva A, Efstathiou E, Sun J, Almeida NF, Tu SM, Botz GH, Wallace MJ, O'Connell DJ, Krajewski S, Gershenwald JE, Molldrem JJ, Flamm AL, Koivunen E, Pentz RD, Dias-Neto E, Setubal JC, Cahill DJ, Troncoso P, Do KA, Logothetis CJ, Sidman RL, Pasqualini R, Arap W.

Proc Natl Acad Sci U S A. 2011 Nov 15;108(46):18637-42. doi: 10.1073/pnas.1114503108. Epub 2011 Nov 2.

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