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Items: 33

1.

Molecular Evidence of Genome Editing in a Mouse Model of Immunodeficiency.

Abdul-Razak HH, Rocca CJ, Howe SJ, Alonso-Ferrero ME, Wang J, Gabriel R, Bartholomae CC, Gan CHV, Garín MI, Roberts A, Blundell MP, Prakash V, Molina-Estevez FJ, Pantoglou J, Guenechea G, Holmes MC, Gregory PD, Kinnon C, von Kalle C, Schmidt M, Bueren JA, Thrasher AJ, Yáñez-Muñoz RJ.

Sci Rep. 2018 May 29;8(1):8214. doi: 10.1038/s41598-018-26439-9.

2.

The LAM-PCR Method to Sequence LV Integration Sites.

Wang W, Bartholomae CC, Gabriel R, Deichmann A, Schmidt M.

Methods Mol Biol. 2016;1448:107-20. doi: 10.1007/978-1-4939-3753-0_9.

PMID:
27317177
3.

Liver-directed lentiviral gene therapy in a dog model of hemophilia B.

Cantore A, Ranzani M, Bartholomae CC, Volpin M, Valle PD, Sanvito F, Sergi LS, Gallina P, Benedicenti F, Bellinger D, Raymer R, Merricks E, Bellintani F, Martin S, Doglioni C, D'Angelo A, VandenDriessche T, Chuah MK, Schmidt M, Nichols T, Montini E, Naldini L.

Sci Transl Med. 2015 Mar 4;7(277):277ra28. doi: 10.1126/scitranslmed.aaa1405.

4.

Enhancer hijacking activates GFI1 family oncogenes in medulloblastoma.

Northcott PA, Lee C, Zichner T, Stütz AM, Erkek S, Kawauchi D, Shih DJ, Hovestadt V, Zapatka M, Sturm D, Jones DT, Kool M, Remke M, Cavalli FM, Zuyderduyn S, Bader GD, VandenBerg S, Esparza LA, Ryzhova M, Wang W, Wittmann A, Stark S, Sieber L, Seker-Cin H, Linke L, Kratochwil F, Jäger N, Buchhalter I, Imbusch CD, Zipprich G, Raeder B, Schmidt S, Diessl N, Wolf S, Wiemann S, Brors B, Lawerenz C, Eils J, Warnatz HJ, Risch T, Yaspo ML, Weber UD, Bartholomae CC, von Kalle C, Turányi E, Hauser P, Sanden E, Darabi A, Siesjö P, Sterba J, Zitterbart K, Sumerauer D, van Sluis P, Versteeg R, Volckmann R, Koster J, Schuhmann MU, Ebinger M, Grimes HL, Robinson GW, Gajjar A, Mynarek M, von Hoff K, Rutkowski S, Pietsch T, Scheurlen W, Felsberg J, Reifenberger G, Kulozik AE, von Deimling A, Witt O, Eils R, Gilbertson RJ, Korshunov A, Taylor MD, Lichter P, Korbel JO, Wechsler-Reya RJ, Pfister SM.

Nature. 2014 Jul 24;511(7510):428-34. doi: 10.1038/nature13379. Epub 2014 Jun 22.

5.

Linear amplification mediated PCR--localization of genetic elements and characterization of unknown flanking DNA.

Gabriel R, Kutschera I, Bartholomae CC, von Kalle C, Schmidt M.

J Vis Exp. 2014 Jun 25;(88):e51543. doi: 10.3791/51543.

6.

Impact of raltegravir on HIV-1 RNA and DNA forms following initiation of antiretroviral therapy in treatment-naive patients.

Stephan C, Baldauf HM, Barry J, Giordano FA, Bartholomae CC, Haberl A, Bickel M, Schmidt M, Laufs S, Kaderali L, Keppler OT.

J Antimicrob Chemother. 2014 Oct;69(10):2809-18. doi: 10.1093/jac/dku213. Epub 2014 Jun 23.

PMID:
24962031
7.

Decoding the regulatory landscape of medulloblastoma using DNA methylation sequencing.

Hovestadt V, Jones DT, Picelli S, Wang W, Kool M, Northcott PA, Sultan M, Stachurski K, Ryzhova M, Warnatz HJ, Ralser M, Brun S, Bunt J, Jäger N, Kleinheinz K, Erkek S, Weber UD, Bartholomae CC, von Kalle C, Lawerenz C, Eils J, Koster J, Versteeg R, Milde T, Witt O, Schmidt S, Wolf S, Pietsch T, Rutkowski S, Scheurlen W, Taylor MD, Brors B, Felsberg J, Reifenberger G, Borkhardt A, Lehrach H, Wechsler-Reya RJ, Eils R, Yaspo ML, Landgraf P, Korshunov A, Zapatka M, Radlwimmer B, Pfister SM, Lichter P.

Nature. 2014 Jun 26;510(7506):537-41. doi: 10.1038/nature13268. Epub 2014 May 18.

PMID:
24847876
8.

Genome sequencing of SHH medulloblastoma predicts genotype-related response to smoothened inhibition.

Kool M, Jones DT, Jäger N, Northcott PA, Pugh TJ, Hovestadt V, Piro RM, Esparza LA, Markant SL, Remke M, Milde T, Bourdeaut F, Ryzhova M, Sturm D, Pfaff E, Stark S, Hutter S, Seker-Cin H, Johann P, Bender S, Schmidt C, Rausch T, Shih D, Reimand J, Sieber L, Wittmann A, Linke L, Witt H, Weber UD, Zapatka M, König R, Beroukhim R, Bergthold G, van Sluis P, Volckmann R, Koster J, Versteeg R, Schmidt S, Wolf S, Lawerenz C, Bartholomae CC, von Kalle C, Unterberg A, Herold-Mende C, Hofer S, Kulozik AE, von Deimling A, Scheurlen W, Felsberg J, Reifenberger G, Hasselblatt M, Crawford JR, Grant GA, Jabado N, Perry A, Cowdrey C, Croul S, Zadeh G, Korbel JO, Doz F, Delattre O, Bader GD, McCabe MG, Collins VP, Kieran MW, Cho YJ, Pomeroy SL, Witt O, Brors B, Taylor MD, Schüller U, Korshunov A, Eils R, Wechsler-Reya RJ, Lichter P, Pfister SM; ICGC PedBrain Tumor Project.

Cancer Cell. 2014 Mar 17;25(3):393-405. doi: 10.1016/j.ccr.2014.02.004.

9.

Transgenic expression of human glial cell line-derived neurotrophic factor from integration-deficient lentiviral vectors is neuroprotective in a rodent model of Parkinson's disease.

Lu-Nguyen NB, Broadstock M, Schliesser MG, Bartholomae CC, von Kalle C, Schmidt M, Yáñez-Muñoz RJ.

Hum Gene Ther. 2014 Jul;25(7):631-41. doi: 10.1089/hum.2014.003. Epub 2014 Apr 15.

10.

Integration-deficient lentiviral vectors expressing codon-optimized R338L human FIX restore normal hemostasis in Hemophilia B mice.

Suwanmanee T, Hu G, Gui T, Bartholomae CC, Kutschera I, von Kalle C, Schmidt M, Monahan PE, Kafri T.

Mol Ther. 2014 Mar;22(3):567-574. doi: 10.1038/mt.2013.188. Epub 2013 Aug 14.

11.

Recurrent somatic alterations of FGFR1 and NTRK2 in pilocytic astrocytoma.

Jones DT, Hutter B, Jäger N, Korshunov A, Kool M, Warnatz HJ, Zichner T, Lambert SR, Ryzhova M, Quang DA, Fontebasso AM, Stütz AM, Hutter S, Zuckermann M, Sturm D, Gronych J, Lasitschka B, Schmidt S, Seker-Cin H, Witt H, Sultan M, Ralser M, Northcott PA, Hovestadt V, Bender S, Pfaff E, Stark S, Faury D, Schwartzentruber J, Majewski J, Weber UD, Zapatka M, Raeder B, Schlesner M, Worth CL, Bartholomae CC, von Kalle C, Imbusch CD, Radomski S, Lawerenz C, van Sluis P, Koster J, Volckmann R, Versteeg R, Lehrach H, Monoranu C, Winkler B, Unterberg A, Herold-Mende C, Milde T, Kulozik AE, Ebinger M, Schuhmann MU, Cho YJ, Pomeroy SL, von Deimling A, Witt O, Taylor MD, Wolf S, Karajannis MA, Eberhart CG, Scheurlen W, Hasselblatt M, Ligon KL, Kieran MW, Korbel JO, Yaspo ML, Brors B, Felsberg J, Reifenberger G, Collins VP, Jabado N, Eils R, Lichter P, Pfister SM; International Cancer Genome Consortium PedBrain Tumor Project.

Nat Genet. 2013 Aug;45(8):927-32. doi: 10.1038/ng.2682. Epub 2013 Jun 30.

12.

Integrative genomic analyses reveal an androgen-driven somatic alteration landscape in early-onset prostate cancer.

Weischenfeldt J, Simon R, Feuerbach L, Schlangen K, Weichenhan D, Minner S, Wuttig D, Warnatz HJ, Stehr H, Rausch T, Jäger N, Gu L, Bogatyrova O, Stütz AM, Claus R, Eils J, Eils R, Gerhäuser C, Huang PH, Hutter B, Kabbe R, Lawerenz C, Radomski S, Bartholomae CC, Fälth M, Gade S, Schmidt M, Amschler N, Haß T, Galal R, Gjoni J, Kuner R, Baer C, Masser S, von Kalle C, Zichner T, Benes V, Raeder B, Mader M, Amstislavskiy V, Avci M, Lehrach H, Parkhomchuk D, Sultan M, Burkhardt L, Graefen M, Huland H, Kluth M, Krohn A, Sirma H, Stumm L, Steurer S, Grupp K, Sültmann H, Sauter G, Plass C, Brors B, Yaspo ML, Korbel JO, Schlomm T.

Cancer Cell. 2013 Feb 11;23(2):159-70. doi: 10.1016/j.ccr.2013.01.002.

13.

Lentiviral vector-based insertional mutagenesis identifies genes associated with liver cancer.

Ranzani M, Cesana D, Bartholomae CC, Sanvito F, Pala M, Benedicenti F, Gallina P, Sergi LS, Merella S, Bulfone A, Doglioni C, von Kalle C, Kim YJ, Schmidt M, Tonon G, Naldini L, Montini E.

Nat Methods. 2013 Feb;10(2):155-61. doi: 10.1038/nmeth.2331. Epub 2013 Jan 13.

14.

Comparative clonal analysis of reconstitution kinetics after transplantation of hematopoietic stem cells gene marked with a lentiviral SIN or a γ-retroviral LTR vector.

Cornils K, Bartholomae CC, Thielecke L, Lange C, Arens A, Glauche I, Mock U, Riecken K, Gerdes S, von Kalle C, Schmidt M, Roeder I, Fehse B.

Exp Hematol. 2013 Jan;41(1):28-38.e3. doi: 10.1016/j.exphem.2012.09.003. Epub 2012 Sep 16.

PMID:
22989760
15.

Dissecting the genomic complexity underlying medulloblastoma.

Jones DT, Jäger N, Kool M, Zichner T, Hutter B, Sultan M, Cho YJ, Pugh TJ, Hovestadt V, Stütz AM, Rausch T, Warnatz HJ, Ryzhova M, Bender S, Sturm D, Pleier S, Cin H, Pfaff E, Sieber L, Wittmann A, Remke M, Witt H, Hutter S, Tzaridis T, Weischenfeldt J, Raeder B, Avci M, Amstislavskiy V, Zapatka M, Weber UD, Wang Q, Lasitschka B, Bartholomae CC, Schmidt M, von Kalle C, Ast V, Lawerenz C, Eils J, Kabbe R, Benes V, van Sluis P, Koster J, Volckmann R, Shih D, Betts MJ, Russell RB, Coco S, Tonini GP, Schüller U, Hans V, Graf N, Kim YJ, Monoranu C, Roggendorf W, Unterberg A, Herold-Mende C, Milde T, Kulozik AE, von Deimling A, Witt O, Maass E, Rössler J, Ebinger M, Schuhmann MU, Frühwald MC, Hasselblatt M, Jabado N, Rutkowski S, von Bueren AO, Williamson D, Clifford SC, McCabe MG, Collins VP, Wolf S, Wiemann S, Lehrach H, Brors B, Scheurlen W, Felsberg J, Reifenberger G, Northcott PA, Taylor MD, Meyerson M, Pomeroy SL, Yaspo ML, Korbel JO, Korshunov A, Eils R, Pfister SM, Lichter P.

Nature. 2012 Aug 2;488(7409):100-5. doi: 10.1038/nature11284.

16.

Overexpression of the anti-apoptotic protein AVEN contributes to increased malignancy in hematopoietic neoplasms.

Eißmann M, Melzer IM, Fernández SB, Michel G, Hrabě de Angelis M, Hoefler G, Finkenwirth P, Jauch A, Schoell B, Grez M, Schmidt M, Bartholomae CC, Newrzela S, Haetscher N, Rieger MA, Zachskorn C, Mittelbronn M, Zörnig M.

Oncogene. 2013 May 16;32(20):2586-91. doi: 10.1038/onc.2012.263. Epub 2012 Jul 2.

PMID:
22751129
17.

Bioinformatic clonality analysis of next-generation sequencing-derived viral vector integration sites.

Arens A, Appelt JU, Bartholomae CC, Gabriel R, Paruzynski A, Gustafson D, Cartier N, Aubourg P, Deichmann A, Glimm H, von Kalle C, Schmidt M.

Hum Gene Ther Methods. 2012 Apr;23(2):111-8. doi: 10.1089/hgtb.2011.219. Epub 2012 May 4.

18.

Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome.

Scaramuzza S, Biasco L, Ripamonti A, Castiello MC, Loperfido M, Draghici E, Hernandez RJ, Benedicenti F, Radrizzani M, Salomoni M, Ranzani M, Bartholomae CC, Vicenzi E, Finocchi A, Bredius R, Bosticardo M, Schmidt M, von Kalle C, Montini E, Biffi A, Roncarolo MG, Naldini L, Villa A, Aiuti A.

Mol Ther. 2013 Jan;21(1):175-84. doi: 10.1038/mt.2012.23. Epub 2012 Feb 28. Erratum in: Mol Ther. 2016 Jun;24(6):1159.

19.

Insertion site pattern: global approach by linear amplification-mediated PCR and mass sequencing.

Bartholomae CC, Glimm H, von Kalle C, Schmidt M.

Methods Mol Biol. 2012;859:255-65. doi: 10.1007/978-1-61779-603-6_15.

PMID:
22367877
20.

Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy.

Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Bougnères P, Schmidt M, Kalle CV, Fischer A, Cavazzana-Calvo M, Aubourg P.

Methods Enzymol. 2012;507:187-98. doi: 10.1016/B978-0-12-386509-0.00010-7.

PMID:
22365775
21.

Analyzing the number of common integration sites of viral vectors--new methods and computer programs.

Abel U, Deichmann A, Nowrouzi A, Gabriel R, Bartholomae CC, Glimm H, von Kalle C, Schmidt M.

PLoS One. 2011;6(10):e24247. doi: 10.1371/journal.pone.0024247. Epub 2011 Oct 14.

22.

Retroviral gene therapy for X-linked chronic granulomatous disease: results from phase I/II trial.

Kang HJ, Bartholomae CC, Paruzynski A, Arens A, Kim S, Yu SS, Hong Y, Joo CW, Yoon NK, Rhim JW, Kim JG, Von Kalle C, Schmidt M, Kim S, Ahn HS.

Mol Ther. 2011 Nov;19(11):2092-101. doi: 10.1038/mt.2011.166. Epub 2011 Aug 30.

23.

Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy.

Deichmann A, Brugman MH, Bartholomae CC, Schwarzwaelder K, Verstegen MM, Howe SJ, Arens A, Ott MG, Hoelzer D, Seger R, Grez M, Hacein-Bey-Abina S, Cavazzana-Calvo M, Fischer A, Paruzynski A, Gabriel R, Glimm H, Abel U, Cattoglio C, Mavilio F, Cassani B, Aiuti A, Dunbar CE, Baum C, Gaspar HB, Thrasher AJ, von Kalle C, Schmidt M, Wagemaker G.

Mol Ther. 2011 Nov;19(11):2031-9. doi: 10.1038/mt.2011.178. Epub 2011 Aug 23.

24.

An unbiased genome-wide analysis of zinc-finger nuclease specificity.

Gabriel R, Lombardo A, Arens A, Miller JC, Genovese P, Kaeppel C, Nowrouzi A, Bartholomae CC, Wang J, Friedman G, Holmes MC, Gregory PD, Glimm H, Schmidt M, Naldini L, von Kalle C.

Nat Biotechnol. 2011 Aug 7;29(9):816-23. doi: 10.1038/nbt.1948.

PMID:
21822255
25.

Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.

Tolar J, Adair JE, Antoniou M, Bartholomae CC, Becker PS, Blazar BR, Bueren J, Carroll T, Cavazzana-Calvo M, Clapp DW, Dalgleish R, Galy A, Gaspar HB, Hanenberg H, Von Kalle C, Kiem HP, Lindeman D, Naldini L, Navarro S, Renella R, Rio P, Sevilla J, Schmidt M, Verhoeyen E, Wagner JE, Williams DA, Thrasher AJ.

Mol Ther. 2011 Jul;19(7):1193-8. doi: 10.1038/mt.2011.78. Epub 2011 May 3. Review.

26.

Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk.

Mátrai J, Cantore A, Bartholomae CC, Annoni A, Wang W, Acosta-Sanchez A, Samara-Kuko E, De Waele L, Ma L, Genovese P, Damo M, Arens A, Goudy K, Nichols TC, von Kalle C, L Chuah MK, Roncarolo MG, Schmidt M, Vandendriessche T, Naldini L.

Hepatology. 2011 May;53(5):1696-707. doi: 10.1002/hep.24230.

27.

Lentiviral vector integration profiles differ in rodent postmitotic tissues.

Bartholomae CC, Arens A, Balaggan KS, Yáñez-Muñoz RJ, Montini E, Howe SJ, Paruzynski A, Korn B, Appelt JU, Macneil A, Cesana D, Abel U, Glimm H, Naldini L, Ali RR, Thrasher AJ, von Kalle C, Schmidt M.

Mol Ther. 2011 Apr;19(4):703-10. doi: 10.1038/mt.2011.19. Epub 2011 Mar 1.

28.

Genome-wide high-throughput integrome analyses by nrLAM-PCR and next-generation sequencing.

Paruzynski A, Arens A, Gabriel R, Bartholomae CC, Scholz S, Wang W, Wolf S, Glimm H, Schmidt M, von Kalle C.

Nat Protoc. 2010 Aug;5(8):1379-95. doi: 10.1038/nprot.2010.87. Epub 2010 Jul 8.

PMID:
20671722
29.

Retroviral vector performance in defined chromosomal Loci of modular packaging cell lines.

Gama-Norton L, Herrmann S, Schucht R, Coroadinha AS, Löw R, Alves PM, Bartholomae CC, Schmidt M, Baum C, Schambach A, Hauser H, Wirth D.

Hum Gene Ther. 2010 Aug;21(8):979-91. doi: 10.1089/hum.2009.089.

PMID:
20222806
30.

Comprehensive genomic access to vector integration in clinical gene therapy.

Gabriel R, Eckenberg R, Paruzynski A, Bartholomae CC, Nowrouzi A, Arens A, Howe SJ, Recchia A, Cattoglio C, Wang W, Faber K, Schwarzwaelder K, Kirsten R, Deichmann A, Ball CR, Balaggan KS, Yáñez-Muñoz RJ, Ali RR, Gaspar HB, Biasco L, Aiuti A, Cesana D, Montini E, Naldini L, Cohen-Haguenauer O, Mavilio F, Thrasher AJ, Glimm H, von Kalle C, Saurin W, Schmidt M.

Nat Med. 2009 Dec;15(12):1431-6. doi: 10.1038/nm.2057. Epub 2009 Nov 22.

PMID:
19966782
31.

Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy.

Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Veres G, Schmidt M, Kutschera I, Vidaud M, Abel U, Dal-Cortivo L, Caccavelli L, Mahlaoui N, Kiermer V, Mittelstaedt D, Bellesme C, Lahlou N, Lefrère F, Blanche S, Audit M, Payen E, Leboulch P, l'Homme B, Bougnères P, Von Kalle C, Fischer A, Cavazzana-Calvo M, Aubourg P.

Science. 2009 Nov 6;326(5954):818-23. doi: 10.1126/science.1171242.

32.

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy.

Montini E, Cesana D, Schmidt M, Sanvito F, Bartholomae CC, Ranzani M, Benedicenti F, Sergi LS, Ambrosi A, Ponzoni M, Doglioni C, Di Serio C, von Kalle C, Naldini L.

J Clin Invest. 2009 Apr;119(4):964-75. doi: 10.1172/JCI37630. Epub 2009 Mar 23.

33.

Detection of retroviral integration sites by linear amplification-mediated PCR and tracking of individual integration clones in different samples.

Schmidt M, Schwarzwaelder K, Bartholomae CC, Glimm H, von Kalle C.

Methods Mol Biol. 2009;506:363-72. doi: 10.1007/978-1-59745-409-4_24.

PMID:
19110638

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