Regenerative Gene Therapy

Objectives

The Research Group Regenerative Gene Therapy aims to reach the following goals:

  • Novel gene therapy approaches with improved efficacy and biosafety for use in otherwise difficult to treat diseases.
  • Clinical translation of gene therapy for at least one candidate disease.
  • Molecular therapeutics to fight immunodeficiencies, infections and cancer/leukemia.
  • Understand transcriptional networks governing (stem) cell identity to develop new tools for regenerative medicine, incl. reprogramming, transdifferentiation and disease modelling.
  • Support other groups with vector expertise for various genetic interventions.

Research Focus

In this Research Group we plan to

  • Develop efficient and more importantly safer systems for gene therapy.
  • Progress in preclinical validation of gene therapy strategies in appropriate mouse models.
  • Strengthen REBIRTH‘s contribution to (inter)national networks in advanced gene and cell manipulations.
  • Improve non-integrating retroviral vectors, incl. retroviral mRNA, episome and protein delivery.
  • Analyze retroviral vector-host interactions.

Achievements

What we have achieved in the running and previous funding periods of the Cluster of Excellence REBIRTH:

  • Participation in clinical translation of gene therapy trials, e.g. international multicenter trial for X-SCID.
  • Bridge function between basic science and clinical translation, embedded in various (inter)national networks (e.g. SFB738, PidNet, Carpud, ASGCT, ESGCT, TAGTC, CellPID).
  • Formal REBIRTH link to Harvard/Boston: Axel Schambach is Associate Faculty and Visiting Scientist in Pediatrics at Boston Children‘s Hospital / Harvard Medical School (in cooperation with David Williams).

Collaborations

Companies

  • Rentschler Biotec
  • Miltenyi
  • Apceth
  • Eufets
  • Plasmid Factory
  • F-Prime / Alerion

Further Research Projects

  • DFG, SFB738 project C4: Vectors for transient cell modification
  • DFG, SFB738 project C9: Designer nucleases for generation of innovative transplants
  • BMBF, PIDNET: German Network on Primary Immunodeficiency Diseases
  • BMBF, IFB-Tx

Awards

  • A. Schambach, M. Rothe, CRACK-IT Award, NC3R, UK (2016)
  • A. Schambach, Young Investigator Award ESGCT (2011)
  • A. Schambach, Duanqing Preis, German-Chinese Junior Research Group, DAAD (2009-2014)
  • C. Baum, Ursula M. Händel, Animal Welfare Award, DFG (2009)
  • A. Schambach, Else Kröner Memorial Scholarship (2006)

Teaching

Members of the group are lecturers in the Ph.D. programs of the Hannover Biomedical Research School (e.g. Ph.D. program Molecular Medicine, Ph.D. program Regenerative Sciences) and the Bachelor (e.g. Biology) and Master programs (e.g. Biomedicine). Furthermore, we participate in the training of medical students at MHH and are supervisors and co-supervisors of multiple students for internships, diploma theses and Ph.D. students.

 

 

Publications

2013 - ongoing

2017

Biasco L, Rothe M, Buning H, Schambach A. Analyzing the Genotoxicity of Retroviral Vectors in Hematopoietic Cell Gene Therapy. Mol Ther Methods Clin Dev. 2018;8:21-30. Epub 2017/11/22.

Schambach A, Schott JW, Morgan MA. Uncoupling the Oncogenic Engine. Cancer Res. 2017;77(22):6060-4. Epub 2017/11/04.

Geis FK, Galla M, Hoffmann D, Kuehle J, Zychlinski D, Maetzig T, Schott JW, Schwarzer A, Goffinet C, Goff SP, Schambach A. Correction to: Potent and reversible lentiviral vector restriction in murine induced pluripotent stem cells. Retrovirology. 2017;14(1):48. Epub 2017/10/20.

Stahlhut M, Ha TC, Morgan M, Schambach A, Kustikova OS. Comparison of Tetracycline-regulated Promoters in Lentiviral-based Vectors in Murine Transplantation Studies. Curr Gene Ther. 2016;16(4):242-8. Epub 2016/10/16.

Peking P, Koller U, Duarte B, Murillas R, Wolf S, Maetzig T, Rothe M, Kocher T, Garcia M, Brachtl G, Schambach A, Larcher F, Reichelt J, Bauer JW, Murauer EM. An RNA-targeted therapy for dystrophic epidermolysis bullosa. Nucleic Acids Res. 2017;45(17):10259-69. Epub 2017/10/04.

Biasco L, Rothe M, Schott JW, Schambach A. Integrating Vectors for Gene Therapy and Clonal Tracking of Engineered Hematopoiesis. Hematol Oncol Clin North Am. 2017;31(5):737-52. Epub 2017/09/13.

Hetzel M, Suzuki T, Hashtchin AR, Arumugam P, Carey B, Schwabbauer M, Kuhn A, Meyer J, Schambach A, Van Der Loo J, Moritz T, Trapnell BC, Lachmann N. Function and Safety of Lentivirus-Mediated Gene Transfer for CSF2RA-Deficiency. Hum Gene Ther Methods. 2017/09/01 ed2017.

Klapdor R, Wang S, Hacker U, Buning H, Morgan M, Dork T, Hillemanns P, Schambach A. Improved Killing of Ovarian Cancer Stem Cells by Combining a Novel Chimeric Antigen Receptor-Based Immunotherapy and Chemotherapy. Hum Gene Ther. 2017;28(10):886-96. Epub 2017/08/25.

Kloss S, Oberschmidt O, Morgan M, Dahlke J, Arseniev L, Huppert V, Granzin M, Gardlowski T, Matthies N, Soltenborn S, Schambach A, Koehl U. Optimization of Human NK Cell Manufacturing: Fully Automated Separation, Improved Ex Vivo Expansion Using IL-21 with Autologous Feeder Cells, and Generation of Anti-CD123-CAR-Expressing Effector Cells. Hum Gene Ther. 2017;28(10):897-913. Epub 2017/08/16.

Kunkiel J, Godecke N, Ackermann M, Hoffmann D, Schambach A, Lachmann N, Wirth D, Moritz T. The CpG-sites of the CBX3 ubiquitous chromatin opening element are critical structural determinants for the anti-silencing function. Sci Rep. 2017/08/13 ed2017. p. 7919.

Schwarzer A, Emmrich S, Schmidt F, Beck D, Ng M, Reimer C, Adams FF, Grasedieck S, Witte D, Kabler S, Wong JWH, Shah A, Huang Y, Jammal R, Maroz A, Jongen-Lavrencic M, Schambach A, Kuchenbauer F, Pimanda JE, Reinhardt D, Heckl D, Klusmann JH. The non-coding RNA landscape of human hematopoiesis and leukemia. Nat Commun. 2017/08/11 ed2017. p. 218.

Stahlhut M, Schambach A, Kustikova OS. Multimodal Lentiviral Vectors for Pharmacologically Controlled Switching Between Constitutive Single Gene Expression and Tetracycline-Regulated Multiple Gene Collaboration. Hum Gene Ther Methods. 2017;28(4):191-204. Epub 2017/07/08.

Adams FF, Heckl D, Hoffmann T, Talbot SR, Kloos A, Thol F, Heuser M, Zuber J, Schambach A, Schwarzer A. An optimized lentiviral vector system for conditional RNAi and efficient cloning of microRNA embedded short hairpin RNA libraries. Biomaterials. 2017;139:102-15. Epub 2017/06/10.

Vink CA, Counsell JR, Perocheau DP, Karda R, Buckley SMK, Brugman MH, Galla M, Schambach A, McKay TR, Waddington SN, Howe SJ. Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy. Mol Ther. 2017/05/30 ed2017. p. 1790-804.

Debnath S, Jaako P, Siva K, Rothe M, Chen J, Dahl M, Gaspar HB, Flygare J, Schambach A, Karlsson S. Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia. Mol Ther. 2017/04/25 ed2017. p. 1805-14.

Hoffmann D, Schott JW, Geis FK, Lange L, Muller FJ, Lenz D, Zychlinski D, Steinemann D, Morgan M, Moritz T, Schambach A. Detailed comparison of retroviral vectors and promoter configurations for stable and high transgene expression in human induced pluripotent stem cells. Gene Ther. 2017;24(5):298-307. Epub 2017/03/28.

Pfaff N, Liebhaber S, Möbus S, Beh-Pajooh A, Fiedler J, Pfanne A, Schambach A, Thum T, Cantz T, Moritz T. Inhibition of miRNA-212/132 improves the reprogramming of fibroblasts into induced pluripotent stem cells by de-repressing important epigenetic remodelling factors. Stem Cell Res. 2017/03/18 ed2017. p. 70-5.

Maetzig T, Ruschmann J, Lai CK, Ngom M, Imren S, Rosten P, Norddahl GL, von Krosigk N, Sanchez Milde L, May C, Selich A, Rothe M, Dhillon I, Schambach A, Humphries RK. A Lentiviral Fluorescent Genetic Barcoding System for Flow Cytometry-Based Multiplex Tracking. Mol Ther. 2017/03/03 ed2017. p. 606-20.

Schambach A, Morgan M. Retroviral Vectors for Cancer Gene Therapy. Recent Results Cancer Res. 2016;209:17-35. Epub 2017/01/20.

Punwani D, Kawahara M, Yu J, Sanford U, Roy S, Patel K, Carbonaro DA, Karlen AD, Khan S, Cornetta K, Rothe M, Schambach A, Kohn DB, Malech HL, McIvor RS, Puck JM, Cowan MJ. Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency. Hum Gene Ther. 2017;28(1):112-24.

2016

Viereck J, Kumarswamy R, Foinquinos A, Xiao K, Avramopoulos P, Kunz M, Dittrich M, Maetzig T, Zimmer K, Remke J, Just A, Fendrich J, Scherf K, Bolesani E, Schambach A, Weidemann F, Zweigerdt R, de Windt LJ, Engelhardt S, Dandekar T, Batkai S, Thum T. Long noncoding RNA Chast promotes cardiac remodeling. Science translational medicine. 2016;8(326):326ra22.

Thiesler CT, Cajic S, Hoffmann D, Thiel C, van Diepen L, Hennig R, Sgodda M, Weibetamann R, Reichl U, Steinemann D, Diekmann U, Huber NM, Oberbeck A, Cantz T, Kuss AW, Korner C, Schambach A, Rapp E, Buettner FF. Glycomic Characterization of Induced Pluripotent Stem Cells Derived from a Patient Suffering from Phosphomannomutase 2 Congenital Disorder of Glycosylation (PMM2-CDG). Molecular & cellular proteomics : MCP. 2016;15(4):1435-52.

Stahlhut M, Schambach A, Kustikova OS. Transduction of Murine Hematopoietic Stem Cells with Tetracycline-regulated Lentiviral Vectors. Methods in molecular biology. 2016;1448:65-76.

Stahlhut M, Ha TC, Morgan M, Schambach A, Kustikova OS. Comparison of tetracycline-regulated promoters in lentiviral-based vectors in murine transplantation studies. Curr Gene Ther. 2016.

Song G, Pacher M, Balakrishnan A, Yuan Q, Tsay HC, Yang D, Reetz J, Brandes S, Dai Z, Putzer BM, Arauzo-Bravo MJ, Steinemann D, Luedde T, Schwabe RF, Manns MP, Scholer HR, Schambach A, Cantz T, Ott M, Sharma AD. Direct Reprogramming of Hepatic Myofibroblasts into Hepatocytes In Vivo Attenuates Liver Fibrosis. Cell stem cell. 2016;18(6):797-808.

Selich A, Daudert J, Hass R, Philipp F, von Kaisenberg C, Paul G, Cornils K, Fehse B, Rittinghausen S, Schambach A, Rothe M. Massive Clonal Selection and Transiently Contributing Clones During Expansion of Mesenchymal Stem Cell Cultures Revealed by Lentiviral RGB-Barcode Technology. Stem cells translational medicine. 2016;5(5):591-601.

Schott JW, Morgan M, Galla M, Schambach A. Viral and Synthetic RNA Vector Technologies and Applications. Molecular therapy : the journal of the American Society of Gene Therapy. 2016;24(9):1513-27.

Schambach A, Morgan M. Retroviral Vectors for Cancer Gene Therapy. Recent Results Cancer Res. 2016;209:17-35.

Salari A, Thomay K, Himmler K, Vajen B, Schienke A, Hagedorn M, Ebersold J, Kreipe HH, Kruger A, Schambach A, Schlegelberger B, Gohring G. Establishing a murine xenograft-model for long-term analysis of factors inducing chromosomal instability in myelodysplastic syndrome: Pitfalls and successes. Cancer Genet. 2016;209(6):258-66.

Papapetrou EP, Schambach A. Gene Insertion Into Genomic Safe Harbors for Human Gene Therapy. Molecular therapy : the journal of the American Society of Gene Therapy. 2016;24(4):678-84.

Mucci A, Kunkiel J, Suzuki T, Brennig S, Glage S, Kuhnel MP, Ackermann M, Happle C, Kuhn A, Schambach A, Trapnell BC, Hansen G, Moritz T, Lachmann N. Murine iPSC-Derived Macrophages as a Tool for Disease Modeling of Hereditary Pulmonary Alveolar Proteinosis due to Csf2rb Deficiency. Stem cell reports. 2016;7(2):292-305.

Manukjan G, Ripperger T, Venturini L, Stadler M, Gohring G, Schambach A, Schlegelberger B, Steinemann D. GABP is necessary for stem/progenitor cell maintenance and myeloid differentiation in human hematopoiesis and chronic myeloid leukemia. Stem cell research. 2016;16(3):677-81.

Maetzig T, Schambach A. Development of Inducible Molecular Switches Based on All-in-One Lentiviral Vectors Equipped with Drug Controlled FLP Recombinase. Methods in molecular biology. 2016;1448:23-39.

Labenski V, Suerth JD, Barczak E, Heckl D, Levy C, Bernadin O, Charpentier E, Williams DA, Fehse B, Verhoeyen E, Schambach A. Alpharetroviral self-inactivating vectors produced by a superinfection-resistant stable packaging cell line allow genetic modification of primary human T lymphocytes. Biomaterials. 2016;97:97-109.

Karpinski J, Hauber I, Chemnitz J, Schafer C, Paszkowski-Rogacz M, Chakraborty D, Beschorner N, Hofmann-Sieber H, Lange UC, Grundhoff A, Hackmann K, Schrock E, Abi-Ghanem J, Pisabarro MT, Surendranath V, Schambach A, Lindner C, van Lunzen J, Hauber J, Buchholz F. Directed evolution of a recombinase that excises the provirus of most HIV-1 primary isolates with high specificity. Nat Biotechnol. 2016;34(4):401-9.

Hubner J, Hoseini SS, Suerth JD, Hoffmann D, Maluski M, Herbst J, Maul H, Ghosh A, Eiz-Vesper B, Yuan Q, Ott M, Heuser M, Schambach A, Sauer MG. Generation of Genetically Engineered Precursor T-Cells From Human Umbilical Cord Blood Using an Optimized Alpharetroviral Vector Platform. Molecular therapy : the journal of the American Society of Gene Therapy. 2016;24(7):1216-26.

Huang J, Liu Y, Au BC, Barber DL, Arruda A, Schambach A, Rothe M, Minden MD, Paige CJ, Medin JA. Preclinical validation: LV/IL-12 transduction of patient leukemia cells for immunotherapy of AML. Mol Ther Methods Clin Dev. 2016;3:16074.

Garcia-Gomez M, Calabria A, Garcia-Bravo M, Benedicenti F, Kosinski P, Lopez-Manzaneda S, Hill C, Del Mar Manu-Pereira M, Martin MA, Orman I, Vives-Corrons JL, Kung C, Schambach A, Jin S, Bueren JA, Montini E, Navarro S, Segovia JC. Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency. Molecular therapy : the journal of the American Society of Gene Therapy. 2016;24(7):1187-98.

Eggenschwiler R, Moslem M, Fraguas MS, Galla M, Papp O, Naujock M, Fonfara I, Gensch I, Wahner A, Beh-Pajooh A, Mussolino C, Tauscher M, Steinemann D, Wegner F, Petri S, Schambach A, Charpentier E, Cathomen T, Cantz T. Improved bi-allelic modification of a transcriptionally silent locus in patient-derived iPSC by Cas9 nickase. Scientific reports. 2016;6:38198.

Brendel C, Guda S, Renella R, Bauer DE, Canver MC, Kim YJ, Heeney MM, Klatt D, Fogel J, Milsom MD, Orkin SH, Gregory RI, Williams DA. Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype. J Clin Invest. 2016;126(10):3868-78.

Borger AK, Eicke D, Wolf C, Gras C, Aufderbeck S, Schulze K, Engels L, Eiz-Vesper B, Schambach A, Guzman CA, Lachmann N, Moritz T, Martin U, Blasczyk R, Figueiredo C. Generation of HLA-universal iPSCs-derived megakaryocytes and platelets for survival under refractoriness conditions. Molecular medicine. 2016;22.

Abbey M, Hakim C, Anand R, Lafera J, Schambach A, Kispert A, Taft MH, Kaever V, Kotlyarov A, Gaestel M, Menon MB. GTPase domain driven dimerization of SEPT7 is dispensable for the critical role of septins in fibroblast cytokinesis. Scientific reports. 2016;6:20007.

2015

Uhlig KM, Schulke S, Scheuplein VA, Malczyk AH, Reusch J, Kugelmann S, Muth A, Koch V, Hutzler S, Bodmer BS, Schambach A, Buchholz CJ, Waibler Z, Scheurer S, Muhlebach MD. Lentiviral Protein Transfer Vectors Are an Efficient Vaccine Platform and Induce a Strong Antigen-Specific Cytotoxic T Cell Response. J Virol. 2015;89(17):9044-60.

Suerth JD, Morgan MA, Kloess S, Heckl D, Neudorfl C, Falk CS, Koehl U, Schambach A. Efficient Generation of Gene-Modified Human Natural Killer Cells Via Alpharetroviral Vectors. J Mol Med (Berl). 2015. Epub 2015/08/25.

Stahlhut M, Schwarzer A, Eder M, Yang M, Li Z, Morgan M, Schambach A, Kustikova OS. Lentiviral Vector System for Coordinated Constitutive and Drug Controlled Tetracycline-Regulated Gene Co-Expression. Biomaterials. 2015;63:189-201.

Schwarzer A, Holtmann H, Brugman M, Meyer J, Schauerte C, Zuber J, Steinemann D, Schlegelberger B, Li Z, Baum C. Hyperactivation of Mtorc1 and Mtorc2 by Multiple Oncogenic Events Causes Addiction to Eif4e-Dependent Mrna Translation in T-Cell Leukemia. Oncogene. 2015;34(27):3593-604.

Schott JW, Jaeschke NM, Hoffmann D, Maetzig T, Ballmaier M, Godinho T, Cathomen T, Schambach A. Deciphering the Impact of Parameters Influencing Transgene Expression Kinetics after Repeated Cell Transduction with Integration-Deficient Retroviral Vectors. Cytometry A. 2015;87(5):405-18.

Schott JW, Hoffmann D, Schambach A. Retrovirus-Based Vectors for Transient and Permanent Cell Modification. Curr Opin Pharmacol. 2015;24:135-46.

Rissone A, Weinacht KG, la Marca G, Bishop K, Giocaliere E, Jagadeesh J, Felgentreff K, Dobbs K, Al-Herz W, Jones M, Chandrasekharappa S, Kirby M, Wincovitch S, Simon KL, Itan Y, DeVine A, Schlaeger T, Schambach A, Sood R, Notarangelo LD, Candotti F. Reticular Dysgenesis-Associated Ak2 Protects Hematopoietic Stem and Progenitor Cell Development from Oxidative Stress. J Exp Med. 2015;212(8):1185-202.

Ripperger T, Manukjan G, Meyer J, Wolter S, Schambach A, Bohne J, Modlich U, Li Z, Skawran B, Schlegelberger B, Steinemann D. The Heteromeric Transcription Factor Gabp Activates the Itgam/Cd11b Promoter and Induces Myeloid Differentiation. Biochim Biophys Acta. 2015;1849(9):1145-54.

Regelin M, Blume J, Pommerencke J, Vakilzadeh R, Witzlau K, Lyszkiewicz M, Zietara N, Saran N, Schambach A, Krueger A. Responsiveness of Developing T Cells to Il-7 Signals Is Sustained by Mir-17 Approximately 92. J Immunol. 2015;195(10):4832-40. Epub 2015/10/18.

Rahman SH, Kuehle J, Reimann C, Mlambo T, Alzubi J, Maeder ML, Riedel H, Fisch P, Cantz T, Rudolph C, Mussolino C, Joung JK, Schambach A, Cathomen T. Rescue of DNA-Pk Signaling and T-Cell Differentiation by Targeted Genome Editing in a Prkdc Deficient Ipsc Disease Model. PLoS Genet. 2015;11(5):e1005239.

Muller-Kuller U, Ackermann M, Kolodziej S, Brendel C, Fritsch J, Lachmann N, Kunkel H, Lausen J, Schambach A, Moritz T, Grez M. A Minimal Ubiquitous Chromatin Opening Element (Ucoe) Effectively Prevents Silencing of Juxtaposed Heterologous Promoters by Epigenetic Remodeling in Multipotent and Pluripotent Stem Cells. Nucleic Acids Res. 2015;43(3):1577-92.

Manukjan G, Ripperger T, Santer L, von Neuhoff N, Ganser A, Schambach A, Schlegelberger B, Steinemann D. Expression of the Ets Transcription Factor Gabpalpha Is Positively Correlated to the Bcr-Abl1/Abl1 Ratio in Cml Patients and Affects Imatinib Sensitivity in Vitro. Exp Hematol. 2015;43(10):880-90. Epub 2015/06/15.

Lyszkiewicz M, Zietara N, Fohse L, Puchalka J, Diestelhorst J, Witzlau K, Prinz I, Schambach A, Krueger A. Limited Niche Availability Suppresses Murine Intrathymic Dendritic-Cell Development from Noncommitted Progenitors. Blood. 2015;125(3):457-64.

Lachmann N, Czarnecki K, Brennig S, Phaltane R, Heise M, Heinz N, Kempf H, Dilloo D, Kaever V, Schambach A, Heuser M, Moritz T. Deoxycytidine-Kinase Knockdown as a Novel Myeloprotective Strategy in the Context of Fludarabine, Cytarabine or Cladribine Therapy. Leukemia. 2015;29(11):2266-9. Epub 2015/04/30.

Lachmann N, Ackermann M, Frenzel E, Liebhaber S, Brennig S, Happle C, Hoffmann D, Klimenkova O, Luttge D, Buchegger T, Kuhnel MP, Schambach A, Janciauskiene S, Figueiredo C, Hansen G, Skokowa J, Moritz T. Large-Scale Hematopoietic Differentiation of Human Induced Pluripotent Stem Cells Provides Granulocytes or Macrophages for Cell Replacement Therapies. Stem Cell Reports. 2015;4(2):282-96.

Hoseini SS, Hapke M, Herbst J, Wedekind D, Baumann R, Heinz N, Schiedlmeier B, Vignali DA, van den Brink MR, Schambach A, Blazar BR, Sauer MG. Inducible T-Cell Receptor Expression in Precursor T Cells for Leukemia Control. Leukemia. 2015;29(7):1530-42.

Hoffmann D, Gohring G, Heuser M, Ganser A, Schambach A, Morgan MA. Letter to the Editor: Production of Mature Healthy Hematopoietic Cells from Induced Pluripotent Stem Cells Derived from an Aml Diagnostic Sample Containing the T(8;21) Translocation. Stem Cells. 2015.

Heinz N, Ehrnstrom B, Schambach A, Schwarzer A, Modlich U, Schiedlmeier B. Comparison of Different Cytokine Conditions Reveals Resveratrol as a New Molecule for Ex Vivo Cultivation of Cord Blood-Derived Hematopoietic Stem Cells. Stem Cells Transl Med. 2015;4(9):1064-72. Epub 2015/07/15.

Glienke W, Esser R, Priesner C, Suerth JD, Schambach A, Wels WS, Grez M, Kloess S, Arseniev L, Koehl U. Advantages and Applications of Car-Expressing Natural Killer Cells. Front Pharmacol. 2015;6:21.

Dreyer AK, Hoffmann D, Lachmann N, Ackermann M, Steinemann D, Timm B, Siler U, Reichenbach J, Grez M, Moritz T, Schambach A, Cathomen T. Talen-Mediated Functional Correction of X-Linked Chronic Granulomatous Disease in Patient-Derived Induced Pluripotent Stem Cells. Biomaterials. 2015;69:191-200.

Dobbs K, Dominguez Conde C, Zhang SY, Parolini S, Audry M, Chou J, Haapaniemi E, Keles S, Bilic I, Okada S, Massaad MJ, Rounioja S, Alwahadneh AM, Serwas NK, Capuder K, Ciftci E, Felgentreff K, Ohsumi TK, Pedergnana V, Boisson B, Haskologlu S, Ensari A, Schuster M, Moretta A, Itan Y, Patrizi O, Rozenberg F, Lebon P, Saarela J, Knip M, Petrovski S, Goldstein DB, Parrott RE, Savas B, Schambach A, Tabellini G, Bock C, Chatila TA, Comeau AM, Geha RS, Abel L, Buckley RH, Ikinciogullari A, Al-Herz W, Helminen M, Dogu F, Casanova JL, Boztug K, Notarangelo LD. Inherited Dock2 Deficiency in Patients with Early-Onset Invasive Infections. N Engl J Med. 2015;372(25):2409-22.

Dahl M, Doyle A, Olsson K, Mansson JE, Marques AR, Mirzaian M, Aerts JM, Ehinger M, Rothe M, Modlich U, Schambach A, Karlsson S. Lentiviral Gene Therapy Using Cellular Promoters Cures Type 1 Gaucher Disease in Mice. Mol Ther. 2015;23(5):835-44. Epub 2015/02/07.

Carmo M, Risma KA, Arumugam P, Tiwari S, Hontz AE, Montiel-Equihua CA, Alonso-Ferrero ME, Blundell MP, Schambach A, Baum C, Malik P, Thrasher AJ, Jordan MB, Gaspar HB. Perforin Gene Transfer into Hematopoietic Stem Cells Improves Immune Dysregulation in Murine Models of Perforin Deficiency. Mol Ther. 2015;23(4):737-45. Epub 2014/12/20.

Zhang H, Goudeva L, Immenschuh S, Schambach A, Skokowa J, Eiz-Vesper B, Blasczyk R, Figueiredo C. Mir-155 Is Associated with the Leukemogenic Potential of the Class Iv Granulocyte Colony-Stimulating Factor Receptor in Cd34(+) Progenitor Cells. Mol Med. 2014;20:736-46. Epub 2015/03/03.

2014

Wunderlich S, Kircher M, Vieth B, Haase A, Merkert S, Beier J, Gohring G, Glage S, Schambach A, Curnow EC, Paabo S, Martin U, Enard W. Primate Ips Cells as Tools for Evolutionary Analyses. Stem Cell Res. 2014;12(3):622-9.

Suerth JD, Labenski V, Schambach A. Alpharetroviral Vectors: From a Cancer-Causing Agent to a Useful Tool for Human Gene Therapy. Viruses. 2014;6(12):4811-38.

Rothe M, Schambach A, Biasco L. Safety of Gene Therapy: New Insights to a Puzzling Case. Curr Gene Ther. 2014;14(6):429-36.

Pike-Overzet K, Baum C, Bredius RG, Cavazzana M, Driessen GJ, Fibbe WE, Gaspar HB, Hoeben RC, Lagresle-Peyrou C, Lankester A, Meij P, Schambach A, Thrasher A, Van Dongen JJ, Zwaginga JJ, Staal FJ. Successful Rag1-Scid Gene Therapy Depends on the Level of Rag1 Expression. J Allergy Clin Immunol. 2014;134(1):242-3.

Moiani A, Suerth JD, Gandolfi F, Rizzi E, Severgnini M, De Bellis G, Schambach A, Mavilio F. Genome-Wide Analysis of Alpharetroviral Integration in Human Hematopoietic Stem/Progenitor Cells. Genes (Basel). 2014;5(2):415-29.

Maetzig T, Kuehle J, Schwarzer A, Turan S, Rothe M, Chaturvedi A, Morgan M, Ha TC, Heuser M, Hammerschmidt W, Baum C, Schambach A. All-in-One Inducible Lentiviral Vector Systems Based on Drug Controlled Flp Recombinase. Biomaterials. 2014;35(14):4345-56.

Liu L, Xu Y, He M, Zhang M, Cui F, Lu L, Yao M, Tian W, Benda C, Zhuang Q, Huang Z, Li W, Li X, Zhao P, Fan W, Luo Z, Li Y, Wu Y, Hutchins AP, Wang D, Tse HF, Schambach A, Frampton J, Qin B, Bao X, Yao H, Zhang B, Sun H, Pei D, Wang H, Wang J, Esteban MA. Transcriptional Pause Release Is a Rate-Limiting Step for Somatic Cell Reprogramming. Cell Stem Cell. 2014;15(5):574-88.

Limbourg A, Schnabel S, Lozanovski VJ, Napp LC, Ha TC, Maetzig T, Bauersachs J, Naim HY, Schambach A, Limbourg FP. Genetic Reporter Analysis Reveals an Expandable Reservoir of Oct4+ Cells in Adult Skin. Cell Regen (Lond). 2014;3(1):9.

Lachmann N, Happle C, Ackermann M, Luttge D, Wetzke M, Merkert S, Hetzel M, Kensah G, Jara-Avaca M, Mucci A, Skuljec J, Dittrich AM, Pfaff N, Brennig S, Schambach A, Steinemann D, Gohring G, Cantz T, Martin U, Schwerk N, Hansen G, Moritz T. Gene Correction of Human Induced Pluripotent Stem Cells Repairs the Cellular Phenotype in Pulmonary Alveolar Proteinosis. Am J Respir Crit Care Med. 2014;189(2):167-82.

Kuehle J, Turan S, Cantz T, Hoffmann D, Suerth JD, Maetzig T, Zychlinski D, Klein C, Steinemann D, Baum C, Bode J, Schambach A. Modified Lentiviral Ltrs Allow Flp Recombinase-Mediated Cassette Exchange and in Vivo Tracing of "Factor-Free" Induced Pluripotent Stem Cells. Mol Ther. 2014;22(5):919-28.

Jaako P, Debnath S, Olsson K, Modlich U, Rothe M, Schambach A, Flygare J, Karlsson S. Gene Therapy Cures the Anemia and Lethal Bone Marrow Failure in a Mouse Model of Rps19-Deficient Diamond-Blackfan Anemia. Haematologica. 2014;99(12):1792-8.

Huang J, Guo X, Fan N, Song J, Zhao B, Ouyang Z, Liu Z, Zhao Y, Yan Q, Yi X, Schambach A, Frampton J, Esteban MA, Yang D, Yang H, Lai L. Rag1/2 Knockout Pigs with Severe Combined Immunodeficiency. J Immunol. 2014;193(3):1496-503.

Haemmerle R, Phaltane R, Rothe M, Schroder S, Schambach A, Moritz T, Modlich U. Clonal Dominance with Retroviral Vector Insertions near the Angpt1 and Angpt2 Genes in a Human Xenotransplant Mouse Model. Mol Ther Nucleic Acids. 2014;3:e200.

Hacein-Bey-Abina S, Pai SY, Gaspar HB, Armant M, Berry CC, Blanche S, Bleesing J, Blondeau J, de Boer H, Buckland KF, Caccavelli L, Cros G, De Oliveira S, Fernandez KS, Guo D, Harris CE, Hopkins G, Lehmann LE, Lim A, London WB, van der Loo JC, Malani N, Male F, Malik P, Marinovic MA, McNicol AM, Moshous D, Neven B, Oleastro M, Picard C, Ritz J, Rivat C, Schambach A, Shaw KL, Sherman EA, Silberstein LE, Six E, Touzot F, Tsytsykova A, Xu-Bayford J, Baum C, Bushman FD, Fischer A, Kohn DB, Filipovich AH, Notarangelo LD, Cavazzana M, Williams DA, Thrasher AJ. A Modified Gamma-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency. N Engl J Med. 2014;371(15):1407-17. Epub 2014/10/09.

Felgentreff K, Du L, Weinacht KG, Dobbs K, Bartish M, Giliani S, Schlaeger T, DeVine A, Schambach A, Woodbine LJ, Davies G, Baxi SN, van der Burg M, Bleesing J, Gennery A, Manis J, Pan-Hammarstrom Q, Notarangelo LD. Differential Role of Nonhomologous End Joining Factors in the Generation, DNA Damage Response, and Myeloid Differentiation of Human Induced Pluripotent Stem Cells. Proc Natl Acad Sci U S A. 2014;111(24):8889-94.

Braun CJ, Boztug K, Paruzynski A, Witzel M, Schwarzer A, Rothe M, Modlich U, Beier R, Gohring G, Steinemann D, Fronza R, Ball CR, Haemmerle R, Naundorf S, Kuhlcke K, Rose M, Fraser C, Mathias L, Ferrari R, Abboud MR, Al-Herz W, Kondratenko I, Marodi L, Glimm H, Schlegelberger B, Schambach A, Albert MH, Schmidt M, von Kalle C, Klein C. Gene Therapy for Wiskott-Aldrich Syndrome--Long-Term Efficacy and Genotoxicity. Sci Transl Med. 2014;6(227):227ra33.

Bobis-Wozowicz S, Galla M, Alzubi J, Kuehle J, Baum C, Schambach A, Cathomen T. Non-Integrating Gamma-Retroviral Vectors as a Versatile Tool for Transient Zinc-Finger Nuclease Delivery. Sci Rep. 2014;4:4656.

Ackermann M, Lachmann N, Hartung S, Eggenschwiler R, Pfaff N, Happle C, Mucci A, Gohring G, Niemann H, Hansen G, Schambach A, Cantz T, Zweigerdt R, Moritz T. Promoter and Lineage Independent Anti-Silencing Activity of the A2 Ubiquitous Chromatin Opening Element for Optimized Human Pluripotent Stem Cell-Based Gene Therapy. Biomaterials. 2014;35(5):1531-42.

2013

Sundarasetty BS, Singh VK, Salguero G, Geffers R, Rickmann M, Macke L, Borchers S, Figueiredo C, Schambach A, Gullberg U, Provasi E, Bonini C, Ganser A, Woelfel T, Stripecke R. Lentivirus-Induced Dendritic Cells for Immunization against High-Risk Wt1(+) Acute Myeloid Leukemia. Hum Gene Ther. 2013;24(2):220-37.

Stein S, Scholz S, Schwable J, Sadat MA, Modlich U, Schultze-Strasser S, Diaz M, Chen-Wichmann L, Muller-Kuller U, Brendel C, Fronza R, Kaufmann KB, Naundorf S, Pech NK, Travers JB, Matute JD, Presson RG, Jr., Sandusky GE, Kunkel H, Rudolf E, Dillmann A, von Kalle C, Kuhlcke K, Baum C, Schambach A, Dinauer MC, Schmidt M, Grez M. From Bench to Bedside: Preclinical Evaluation of a Self-Inactivating Gammaretroviral Vector for the Gene Therapy of X-Linked Chronic Granulomatous Disease. Hum Gene Ther Clin Dev. 2013;24(2):86-98.

Sgodda M, Mobus S, Hoepfner J, Sharma AD, Schambach A, Greber B, Ott M, Cantz T. Improved Hepatic Differentiation Strategies for Human Induced Pluripotent Stem Cells. Curr Mol Med. 2013;13(5):842-55.

Schambach A, Moritz T. Toward Position-Independent Retroviral Vector Expression in Pluripotent Stem Cells. Mol Ther. 2013;21(8):1474-7.

Rothe M, Modlich U, Schambach A. Biosafety Challenges for Use of Lentiviral Vectors in Gene Therapy. Curr Gene Ther. 2013;13(6):453-68.

Rittelmeyer I, Rothe M, Brugman MH, Iken M, Schambach A, Manns MP, Baum C, Modlich U, Ott M. Hepatic Lentiviral Gene Transfer Is Associated with Clonal Selection, but Not with Tumor Formation in Serially Transplanted Rodents. Hepatology. 2013;58(1):397-408.

Pfaff N, Lachmann N, Ackermann M, Kohlscheen S, Brendel C, Maetzig T, Niemann H, Antoniou MN, Grez M, Schambach A, Cantz T, Moritz T. A Ubiquitous Chromatin Opening Element Prevents Transgene Silencing in Pluripotent Stem Cells and Their Differentiated Progeny. Stem Cells. 2013;31(3):488-99.

Lachmann N, Brennig S, Pfaff N, Schermeier H, Dahlmann J, Phaltane R, Gruh I, Modlich U, Schambach A, Baum C, Moritz T. Efficient in Vivo Regulation of Cytidine Deaminase Expression in the Haematopoietic System Using a Doxycycline-Inducible Lentiviral Vector System. Gene Ther. 2013;20(3):298-307.

Kustikova OS, Schwarzer A, Stahlhut M, Brugman MH, Neumann T, Yang M, Li Z, Schambach A, Heinz N, Gerdes S, Roeder I, Ha TC, Steinemann D, Schlegelberger B, Baum C. Activation of Evi1 Inhibits Cell Cycle Progression and Differentiation of Hematopoietic Progenitor Cells. Leukemia. 2013;27(5):1127-38.

Kaufmann KB, Buning H, Galy A, Schambach A, Grez M. Gene Therapy on the Move. EMBO Mol Med. 2013;5(11):1642-61.

Kaufmann KB, Brendel C, Suerth JD, Mueller-Kuller U, Chen-Wichmann L, Schwable J, Pahujani S, Kunkel H, Schambach A, Baum C, Grez M. Alpharetroviral Vector-Mediated Gene Therapy for X-Cgd: Functional Correction and Lack of Aberrant Splicing. Mol Ther. 2013;21(3):648-61.

Hauber I, Hofmann-Sieber H, Chemnitz J, Dubrau D, Chusainow J, Stucka R, Hartjen P, Schambach A, Ziegler P, Hackmann K, Schrock E, Schumacher U, Lindner C, Grundhoff A, Baum C, Manz MG, Buchholz F, Hauber J. Highly Significant Antiviral Activity of Hiv-1 Ltr-Specific Tre-Recombinase in Humanized Mice. PLoS Pathog. 2013;9(9):e1003587.

Gupta SS, Maetzig T, Maertens GN, Sharif A, Rothe M, Weidner-Glunde M, Galla M, Schambach A, Cherepanov P, Schulz TF. Bromo- and Extraterminal Domain Chromatin Regulators Serve as Cofactors for Murine Leukemia Virus Integration. J Virol. 2013;87(23):12721-36.

Grabundzija I, Wang J, Sebe A, Erdei Z, Kajdi R, Devaraj A, Steinemann D, Szuhai K, Stein U, Cantz T, Schambach A, Baum C, Izsvak Z, Sarkadi B, Ivics Z. Sleeping Beauty Transposon-Based System for Cellular Reprogramming and Targeted Gene Insertion in Induced Pluripotent Stem Cells. Nucleic Acids Res. 2013;41(3):1829-47.

Galla M, Schambach A, Baum C. Retrovirus-Based Mrna Transfer for Transient Cell Manipulation. Methods Mol Biol. 2013;969:139-61.

Eggenschwiler R, Loya K, Wu G, Sharma AD, Sgodda M, Zychlinski D, Herr C, Steinemann D, Teckman J, Bals R, Ott M, Schambach A, Scholer HR, Cantz T. Sustained Knockdown of a Disease-Causing Gene in Patient-Specific Induced Pluripotent Stem Cells Using Lentiviral Vector-Based Gene Therapy. Stem Cells Transl Med. 2013;2(9):641-54.

Brugman MH, Suerth JD, Rothe M, Suerbaum S, Schambach A, Modlich U, Kustikova O, Baum C. Evaluating a Ligation-Mediated Pcr and Pyrosequencing Method for the Detection of Clonal Contribution in Polyclonal Retrovirally Transduced Samples. Hum Gene Ther Methods. 2013;24(2):68-79.

2006 - 2012

2012

Zhou Q, Schneider IC, Edes I, Honegger A, Bach P, Schonfeld K, Schambach A, Wels WS, Kneissl S, Uckert W, Buchholz CJ. T-Cell Receptor Gene Transfer Exclusively to Human Cd8(+) Cells Enhances Tumor Cell Killing. Blood. 2012;120(22):4334-42.

Wunderlich S, Haase A, Merkert S, Beier J, Schwanke K, Schambach A, Glage S, Gohring G, Curnow EC, Martin U. Induction of Pluripotent Stem Cells from a Cynomolgus Monkey Using a Polycistronic Simian Immunodeficiency Virus-Based Vector, Differentiation toward Functional Cardiomyocytes, and Generation of Stably Expressing Reporter Lines. Cell Reprogram. 2012;14(6):471-84.

Wiedemann A, Hemmer K, Bernemann I, Gohring G, Pogozhykh O, Figueiredo C, Glage S, Schambach A, Schwamborn JC, Blasczyk R, Muller T. Induced Pluripotent Stem Cells Generated from Adult Bone Marrow-Derived Cells of the Nonhuman Primate (Callithrix Jacchus) Using a Novel Quad-Cistronic and Excisable Lentiviral Vector. Cell Reprogram. 2012;14(6):485-96.

Weinacht KG, Brauer PM, Felgentreff K, Devine A, Gennery AR, Giliani S, Al-Herz W, Schambach A, Zuniga-Pflucker JC, Notarangelo LD. The Role of Induced Pluripotent Stem Cells in Research and Therapy of Primary Immunodeficiencies. Curr Opin Immunol. 2012;24(5):617-24.

Wang J, Sun Q, Morita Y, Jiang H, Gross A, Lechel A, Hildner K, Guachalla LM, Gompf A, Hartmann D, Schambach A, Wuestefeld T, Dauch D, Schrezenmeier H, Hofmann WK, Nakauchi H, Ju Z, Kestler HA, Zender L, Rudolph KL. A Differentiation Checkpoint Limits Hematopoietic Stem Cell Self-Renewal in Response to DNA Damage. Cell. 2012;148(5):1001-14.

Voelkel C, Galla M, Dannhauser PN, Maetzig T, Sodeik B, Schambach A, Baum C. Pseudotype-Independent Nonspecific Uptake of Gammaretroviral and Lentiviral Particles in Human Cells. Hum Gene Ther. 2012;23(3):274-86.

van der Loo JC, Swaney WP, Grassman E, Terwilliger A, Higashimoto T, Schambach A, Hacein-Bey-Abina S, Nordling DL, Cavazzana-Calvo M, Thrasher AJ, Williams DA, Reeves L, Malik P. Critical Variables Affecting Clinical-Grade Production of the Self-Inactivating Gamma-Retroviral Vector for the Treatment of X-Linked Severe Combined Immunodeficiency. Gene Ther. 2012;19(8):872-6.

van der Loo JC, Swaney WP, Grassman E, Terwilliger A, Higashimoto T, Schambach A, Baum C, Thrasher AJ, Williams DA, Nordling DL, Reeves L, Malik P. Scale-up and Manufacturing of Clinical-Grade Self-Inactivating Gamma-Retroviral Vectors by Transient Transfection. Gene Ther. 2012;19(3):246-54.

Thol F, Yun H, Sonntag AK, Damm F, Weissinger EM, Krauter J, Wagner K, Morgan M, Wichmann M, Gohring G, Bug G, Ottmann O, Hofmann WK, Schambach A, Schlegelberger B, Haferlach T, Bowen D, Mills K, Ganser A, Heuser M. Prognostic Significance of Combined Mn1, Erg, Baalc, and Evi1 (Mebe) Expression in Patients with Myelodysplastic Syndromes. Ann Hematol. 2012;91(8):1221-33.

Suerth JD, Schambach A, Baum C. Genetic Modification of Lymphocytes by Retrovirus-Based Vectors. Curr Opin Immunol. 2012;24(5):598-608.

Suerth JD, Maetzig T, Brugman MH, Heinz N, Appelt JU, Kaufmann KB, Schmidt M, Grez M, Modlich U, Baum C, Schambach A. Alpharetroviral Self-Inactivating Vectors: Long-Term Transgene Expression in Murine Hematopoietic Cells and Low Genotoxicity. Mol Ther. 2012;20(5):1022-32.

Schambach A, Baum C. Vectorizing Mrna and Proteins. Curr Gene Ther. 2012;12(5):345-6.

Rothe M, Rittelmeyer I, Iken M, Rudrich U, Schambach A, Glage S, Manns MP, Baum C, Bock M, Ott M, Modlich U. Epidermal Growth Factor Improves Lentivirus Vector Gene Transfer into Primary Mouse Hepatocytes. Gene Ther. 2012;19(4):425-34.

Pfaff N, Lachmann N, Kohlscheen S, Sgodda M, Arauzo-Bravo MJ, Greber B, Kues W, Glage S, Baum C, Niemann H, Schambach A, Cantz T, Moritz T. Efficient Hematopoietic Redifferentiation of Induced Pluripotent Stem Cells Derived from Primitive Murine Bone Marrow Cells. Stem Cells Dev. 2012;21(5):689-701.

Otte A, Gohring G, Steinemann D, Schlegelberger B, Groos S, Langer F, Kreipe HH, Schambach A, Neumann T, Hillemanns P, Park-Simon TW, Hass R. A Tumor-Derived Population (Sccoht-1) as Cellular Model for a Small Cell Ovarian Carcinoma of the Hypercalcemic Type. Int J Oncol. 2012;41(2):765-75.

Muller LU, Milsom MD, Harris CE, Vyas R, Brumme KM, Parmar K, Moreau LA, Schambach A, Park IH, London WB, Strait K, Schlaeger T, Devine AL, Grassman E, D'Andrea A, Daley GQ, Williams DA. Overcoming Reprogramming Resistance of Fanconi Anemia Cells. Blood. 2012;119(23):5449-57.

Maetzig T, Baum C, Schambach A. Retroviral Protein Transfer: Falling Apart to Make an Impact. Curr Gene Ther. 2012;12(5):389-409.

Li W, Wang X, Fan W, Zhao P, Chan YC, Chen S, Zhang S, Guo X, Zhang Y, Li Y, Cai J, Qin D, Li X, Yang J, Peng T, Zychlinski D, Hoffmann D, Zhang R, Deng K, Ng KM, Menten B, Zhong M, Wu J, Li Z, Chen Y, Schambach A, Tse HF, Pei D, Esteban MA. Modeling Abnormal Early Development with Induced Pluripotent Stem Cells from Aneuploid Syndromes. Hum Mol Genet. 2012;21(1):32-45.

Lachmann N, Jagielska J, Heckl D, Brennig S, Pfaff N, Maetzig T, Modlich U, Cantz T, Gentner B, Schambach A, Moritz T. Microrna-150-Regulated Vectors Allow Lymphocyte-Sparing Transgene Expression in Hematopoietic Gene Therapy. Gene Ther. 2012;19(9):915-24.

Koch L, Deiwick A, Schlie S, Michael S, Gruene M, Coger V, Zychlinski D, Schambach A, Reimers K, Vogt PM, Chichkov B. Skin Tissue Generation by Laser Cell Printing. Biotechnol Bioeng. 2012;109(7):1855-63.

Knippenberg S, Thau N, Schwabe K, Dengler R, Schambach A, Hass R, Petri S. Intraspinal Injection of Human Umbilical Cord Blood-Derived Cells Is Neuroprotective in a Transgenic Mouse Model of Amyotrophic Lateral Sclerosis. Neurodegener Dis. 2012;9(3):107-20.

Geiger H, Pawar SA, Kerschen EJ, Nattamai KJ, Hernandez I, Liang HP, Fernandez JA, Cancelas JA, Ryan MA, Kustikova O, Schambach A, Fu Q, Wang J, Fink LM, Petersen KU, Zhou D, Griffin JH, Baum C, Weiler H, Hauer-Jensen M. Pharmacological Targeting of the Thrombomodulin-Activated Protein C Pathway Mitigates Radiation Toxicity. Nat Med. 2012;18(7):1123-9.

Gaussin A, Modlich U, Bauche C, Niederlander NJ, Schambach A, Duros C, Artus A, Baum C, Cohen-Haguenauer O, Mermod N. Ctf/Nf1 Transcription Factors Act as Potent Genetic Insulators for Integrating Gene Transfer Vectors. Gene Ther. 2012;19(1):15-24.

Foll D, Hinrichs J, Tischer S, Battermann A, Schambach A, Figueiredo C, Immenschuh S, Blasczyk R, Eiz-Vesper B. Closing the Gap: Discrimination of the Expression Profile of Hla Questionable Alleles by a Cytokine-Induced Secretion Approach Using Hla-a*32:11q. Tissue Antigens. 2012;79(5):340-50.

Daenthanasanmak A, Salguero G, Borchers S, Figueiredo C, Jacobs R, Sundarasetty BS, Schneider A, Schambach A, Eiz-Vesper B, Blasczyk R, Weissinger EM, Ganser A, Stripecke R. Integrase-Defective Lentiviral Vectors Encoding Cytokines Induce Differentiation of Human Dendritic Cells and Stimulate Multivalent Immune Responses in Vitro and in Vivo. Vaccine. 2012;30(34):5118-31.

Brennig S, Rattmann I, Lachmann N, Schambach A, Williams DA, Moritz T. In Vivo Enrichment of Cytidine Deaminase Gene-Modified Hematopoietic Cells by Prolonged Cytosine-Arabinoside Application. Cytotherapy. 2012;14(4):451-60.

Baum C. Conditional Negative Selection of Gene-Modified Hematopoietic Stem Cells. Mol Ther. 2012;20(10):1841-2.

Abdollahpour H, Appaswamy G, Kotlarz D, Diestelhorst J, Beier R, Schaffer AA, Gertz EM, Schambach A, Kreipe HH, Pfeifer D, Engelhardt KR, Rezaei N, Grimbacher B, Lohrmann S, Sherkat R, Klein C. The Phenotype of Human Stk4 Deficiency. Blood. 2012;119(15):3450-7.

2011

Zhang S, Chen S, Li W, Guo X, Zhao P, Xu J, Chen Y, Pan Q, Liu X, Zychlinski D, Lu H, Tortorella MD, Schambach A, Wang Y, Pei D, Esteban MA. Rescue of Atp7b Function in Hepatocyte-Like Cells from Wilson's Disease Induced Pluripotent Stem Cells Using Gene Therapy or the Chaperone Drug Curcumin. Hum Mol Genet. 2011;20(16):3176-87.

Wu G, Liu N, Rittelmeyer I, Sharma AD, Sgodda M, Zaehres H, Bleidissel M, Greber B, Gentile L, Han DW, Rudolph C, Steinemann D, Schambach A, Ott M, Scholer HR, Cantz T. Generation of Healthy Mice from Gene-Corrected Disease-Specific Induced Pluripotent Stem Cells. PLoS Biol. 2011;9(7):e1001099.

Warlich E, Kuehle J, Cantz T, Brugman MH, Maetzig T, Galla M, Filipczyk AA, Halle S, Klump H, Scholer HR, Baum C, Schroeder T, Schambach A. Lentiviral Vector Design and Imaging Approaches to Visualize the Early Stages of Cellular Reprogramming. Mol Ther. 2011;19(4):782-9.

Tiemann U, Sgodda M, Warlich E, Ballmaier M, Scholer HR, Schambach A, Cantz T. Optimal Reprogramming Factor Stoichiometry Increases Colony Numbers and Affects Molecular Characteristics of Murine Induced Pluripotent Stem Cells. Cytometry A. 2011;79(6):426-35.

Schott JW, Galla M, Godinho T, Baum C, Schambach A. Viral and Non-Viral Approaches for Transient Delivery of Mrna and Proteins. Curr Gene Ther. 2011;11(5):382-98.

Pincha M, Salguero G, Wedekind D, Sundarasetty BS, Lin A, Kasahara N, Brugman MH, Jirmo AC, Modlich U, Gutzmer R, Busche G, Ganser A, Stripecke R. Lentiviral Vectors for Induction of Self-Differentiation and Conditional Ablation of Dendritic Cells. Gene Ther. 2011;18(8):750-64.

Pike-Overzet K, Rodijk M, Ng YY, Baert MR, Lagresle-Peyrou C, Schambach A, Zhang F, Hoeben RC, Hacein-Bey-Abina S, Lankester AC, Bredius RG, Driessen GJ, Thrasher AJ, Baum C, Cavazzana-Calvo M, van Dongen JJ, Staal FJ. Correction of Murine Rag1 Deficiency by Self-Inactivating Lentiviral Vector-Mediated Gene Transfer. Leukemia. 2011;25(9):1471-83.

Pfaff N, Fiedler J, Holzmann A, Schambach A, Moritz T, Cantz T, Thum T. Mirna Screening Reveals a New Mirna Family Stimulating Ips Cell Generation Via Regulation of Meox2. EMBO Rep. 2011;12(11):1153-9.

Maetzig T, Galla M, Baum C, Schambach A. Gammaretroviral Vectors: Biology, Technology and Application. Viruses. 2011;3(6):677-713.

Maetzig T, Brugman MH, Bartels S, Heinz N, Kustikova OS, Modlich U, Li Z, Galla M, Schiedlmeier B, Schambach A, Baum C. Polyclonal Fluctuation of Lentiviral Vector-Transduced and Expanded Murine Hematopoietic Stem Cells. Blood. 2011;117(11):3053-64.

Jaako P, Flygare J, Olsson K, Quere R, Ehinger M, Henson A, Ellis S, Schambach A, Baum C, Richter J, Larsson J, Bryder D, Karlsson S. Mice with Ribosomal Protein S19 Deficiency Develop Bone Marrow Failure and Symptoms Like Patients with Diamond-Blackfan Anemia. Blood. 2011;118(23):6087-96.

Iacob R, Rudrich U, Rothe M, Kirsch S, Maasoumy B, Narain N, Verfaillie CM, Sancho-Bru P, Iken M, Popescu I, Schambach A, Manns MP, Bock M. Induction of a Mature Hepatocyte Phenotype in Adult Liver Derived Progenitor Cells by Ectopic Expression of Transcription Factors. Stem Cell Res. 2011;6(3):251-61.

Huston MW, van Til NP, Visser TP, Arshad S, Brugman MH, Cattoglio C, Nowrouzi A, Li Y, Schambach A, Schmidt M, Baum C, von Kalle C, Mavilio F, Zhang F, Blundell MP, Thrasher AJ, Verstegen MM, Wagemaker G. Correction of Murine Scid-X1 by Lentiviral Gene Therapy Using a Codon-Optimized Il2rg Gene and Minimal Pretransplant Conditioning. Mol Ther. 2011;19(10):1867-77.

Huelsmann PM, Hofmann AD, Knoepfel SA, Popp J, Rauch P, Di Giallonardo F, Danke C, Gueckel E, Schambach A, Wolff H, Metzner KJ, Berens C. A Suicide Gene Approach Using the Human Pro-Apoptotic Protein Tbid Inhibits Hiv-1 Replication. BMC Biotechnol. 2011;11:4.

Heinz N, Schambach A, Galla M, Maetzig T, Baum C, Loew R, Schiedlmeier B. Retroviral and Transposon-Based Tet-Regulated All-in-One Vectors with Reduced Background Expression and Improved Dynamic Range. Hum Gene Ther. 2011;22(2):166-76.

Heckl D, Wicke DC, Brugman MH, Meyer J, Schambach A, Busche G, Ballmaier M, Baum C, Modlich U. Lentiviral Gene Transfer Regenerates Hematopoietic Stem Cells in a Mouse Model for Mpl-Deficient Aplastic Anemia. Blood. 2011;117(14):3737-47.

Galla M, Schambach A, Falk CS, Maetzig T, Kuehle J, Lange K, Zychlinski D, Heinz N, Brugman MH, Gohring G, Izsvak Z, Ivics Z, Baum C. Avoiding Cytotoxicity of Transposases by Dose-Controlled Mrna Delivery. Nucleic Acids Res. 2011;39(16):7147-60.

Baum C, Schambach A. Promoter Competition for Gene Therapy of Scid-X1. Hum Gene Ther. 2011;22(3):255-6.

Baum C, Modlich U, Gohring G, Schlegelberger B. Concise Review: Managing Genotoxicity in the Therapeutic Modification of Stem Cells. Stem Cells. 2011;29(10):1479-84.

Baum C. Gene Therapy for Scid-X1: Focus on Clinical Data. Mol Ther. 2011;19(12):2103-4.

Bals C, Schambach A, Meyer J, Scheper T, Rinas U. Expression and Purification of Bioactive Soluble Murine Stem Cell Factor from Recombinant Escherichia Coli Using Thioredoxin as Fusion Partner. J Biotechnol. 2011;152(1-2):1-8.

Avedillo Diez I, Zychlinski D, Coci EG, Galla M, Modlich U, Dewey RA, Schwarzer A, Maetzig T, Mpofu N, Jaeckel E, Boztug K, Baum C, Klein C, Schambach A. Development of Novel Efficient Sin Vectors with Improved Safety Features for Wiskott-Aldrich Syndrome Stem Cell Based Gene Therapy. Mol Pharm. 2011;8(5):1525-37.

2010

Wurm M, Schambach A, Lindemann D, Hanenberg H, Standker L, Forssmann WG, Blasczyk R, Horn PA. The Influence of Semen-Derived Enhancer of Virus Infection on the Efficiency of Retroviral Gene Transfer. J Gene Med. 2010;12(2):137-46.

Voelkel C, Galla M, Maetzig T, Warlich E, Kuehle J, Zychlinski D, Bode J, Cantz T, Schambach A, Baum C. Protein Transduction from Retroviral Gag Precursors. Proc Natl Acad Sci U S A. 2010;107(17):7805-10.

Turan S, Kuehle J, Schambach A, Baum C, Bode J. Multiplexing Rmce: Versatile Extensions of the Flp-Recombinase-Mediated Cassette-Exchange Technology. J Mol Biol. 2010;402(1):52-69.

Tomala M, Lavrentieva A, Moretti P, Rinas U, Kasper C, Stahl F, Schambach A, Warlich E, Martin U, Cantz T, Scheper T. Preparation of Bioactive Soluble Human Leukemia Inhibitory Factor from Recombinant Escherichia Coli Using Thioredoxin as Fusion Partner. Protein Expr Purif. 2010;73(1):51-7.

Suerth JD, Maetzig T, Galla M, Baum C, Schambach A. Self-Inactivating Alpharetroviral Vectors with a Split-Packaging Design. J Virol. 2010;84(13):6626-35.

Schambach A, Cantz T, Baum C, Cathomen T. Generation and Genetic Modification of Induced Pluripotent Stem Cells. Expert Opin Biol Ther. 2010;10(7):1089-103.

Ng YY, Baert MR, Pike-Overzet K, Rodijk M, Brugman MH, Schambach A, Baum C, Hendriks RW, van Dongen JJ, Staal FJ. Correction of B-Cell Development in Btk-Deficient Mice Using Lentiviral Vectors with Codon-Optimized Human Btk. Leukemia. 2010;24(9):1617-30.

Metcalf JA, Ma X, Linders B, Wu S, Schambach A, Ohlemiller KK, Kovacs A, Bigg M, He L, Tollefsen DM, Ponder KP. A Self-Inactivating Gamma-Retroviral Vector Reduces Manifestations of Mucopolysaccharidosis I in Mice. Mol Ther. 2010;18(2):334-42.

Maetzig T, Galla M, Brugman MH, Loew R, Baum C, Schambach A. Mechanisms Controlling Titer and Expression of Bidirectional Lentiviral and Gammaretroviral Vectors. Gene Ther. 2010;17(3):400-11.

Loew R, Meyer Y, Kuehlcke K, Gama-Norton L, Wirth D, Hauser H, Stein S, Grez M, Thornhill S, Thrasher A, Baum C, Schambach A. A New Pg13-Based Packaging Cell Line for Stable Production of Clinical-Grade Self-Inactivating Gamma-Retroviral Vectors Using Targeted Integration. Gene Therapy. 2010;17(2):272-80.

Gama-Norton L, Herrmann S, Schucht R, Coroadinha AS, Low R, Alves PM, Bartholomae CC, Schmidt M, Baum C, Schambach A, Hauser H, Wirth D. Retroviral Vector Performance in Defined Chromosomal Loci of Modular Packaging Cell Lines. Hum Gene Ther. 2010;21(8):979-91.

Cathomen T, Schambach A. Zinc-Finger Nucleases Meet Ips Cells: Zinc Positive: Tailored Genome Engineering Meets Reprogramming. Gene Ther. 2010;17(1):1-3.

Broll S, Oumard A, Hahn K, Schambach A, Bode J. Minicircle Performance Depending on S/Mar-Nuclear Matrix Interactions. J Mol Biol. 2010;395(5):950-65.

2009

Skokowa J, Lan D, Thakur BK, Wang F, Gupta K, Cario G, Brechlin AM, Schambach A, Hinrichsen L, Meyer G, Gaestel M, Stanulla M, Tong Q, Welte K. Nampt Is Essential for the G-Csf-Induced Myeloid Differentiation Via a Nad(+)-Sirtuin-1-Dependent Pathway. Nat Med. 2009;15(2):151-8.

Schambach A, Swaney WP, van der Loo JC. Design and Production of Retro- and Lentiviral Vectors for Gene Expression in Hematopoietic Cells. Methods Mol Biol. 2009;506:191-205.

Pauwels K, Gijsbers R, Toelen J, Schambach A, Willard-Gallo K, Verheust C, Debyser Z, Herman P. State-of-the-Art Lentiviral Vectors for Research Use: Risk Assessment and Biosafety Recommendations. Curr Gene Ther. 2009;9(6):459-74.

Moreno-Carranza B, Gentsch M, Stein S, Schambach A, Santilli G, Rudolf E, Ryser MF, Haria S, Thrasher AJ, Baum C, Brenner S, Grez M. Transgene Optimization Significantly Improves Sin Vector Titers, Gp91phox Expression and Reconstitution of Superoxide Production in X-Cgd Cells. Gene Ther. 2009;16(1):111-8.

Modlich U, Schambach A, Li Z, Schiedlmeier B. Murine Hematopoietic Stem Cell Transduction Using Retroviral Vectors. Methods Mol Biol. 2009;506:23-31.

Modlich U, Navarro S, Zychlinski D, Maetzig T, Knoess S, Brugman MH, Schambach A, Charrier S, Galy A, Thrasher AJ, Bueren J, Baum C. Insertional Transformation of Hematopoietic Cells by Self-Inactivating Lentiviral and Gammaretroviral Vectors. Mol Ther. 2009;17(11):1919-28.

Maruggi G, Porcellini S, Facchini G, Perna SK, Cattoglio C, Sartori D, Ambrosi A, Schambach A, Baum C, Bonini C, Bovolenta C, Mavilio F, Recchia A. Transcriptional Enhancers Induce Insertional Gene Deregulation Independently from the Vector Type and Design. Mol Ther. 2009;17(5):851-6.

Cornils K, Lange C, Schambach A, Brugman MH, Nowak R, Lioznov M, Baum C, Fehse B. Stem Cell Marking with Promotor-Deprived Self-Inactivating Retroviral Vectors Does Not Lead to Induced Clonal Imbalance. Mol Ther. 2009;17(1):131-43.

2008

Zychlinski D, Schambach A, Modlich U, Maetzig T, Meyer J, Grassman E, Mishra A, Baum C. Physiological Promoters Reduce the Genotoxic Risk of Integrating Gene Vectors. Mol Ther. 2008;16(4):718-25.

Thornhill SI, Schambach A, Howe SJ, Ulaganathan M, Grassman E, Williams D, Schiedlmeier B, Sebire NJ, Gaspar HB, Kinnon C, Baum C, Thrasher AJ. Self-Inactivating Gammaretroviral Vectors for Gene Therapy of X-Linked Severe Combined Immunodeficiency. Mol Ther. 2008;16(3):590-8.

Sharma AD, Cantz T, Vogel A, Schambach A, Haridass D, Iken M, Bleidissel M, Manns MP, Scholer HR, Ott M. Murine Embryonic Stem Cell-Derived Hepatic Progenitor Cells Engraft in Recipient Livers with Limited Capacity of Liver Tissue Formation. Cell Transplant. 2008;17(3):313-23.

Schambach A, Baum C. Clinical Application of Lentiviral Vectors - Concepts and Practice. Curr Gene Ther. 2008;8(6):474-82.

Muller LU, Milsom MD, Kim MO, Schambach A, Schuesler T, Williams DA. Rapid Lentiviral Transduction Preserves the Engraftment Potential of Fanca(-/-) Hematopoietic Stem Cells. Mol Ther. 2008;16(6):1154-60.

Modlich U, Schambach A, Brugman MH, Wicke DC, Knoess S, Li Z, Maetzig T, Rudolph C, Schlegelberger B, Baum C. Leukemia Induction after a Single Retroviral Vector Insertion in Evi1 or Prdm16. Leukemia. 2008;22(8):1519-28.

Milsom MD, Jerabek-Willemsen M, Harris CE, Schambach A, Broun E, Bailey J, Jansen M, Schleimer D, Nattamai K, Wilhelm J, Watson A, Geiger H, Margison GP, Moritz T, Baum C, Thomale J, Williams DA. Reciprocal Relationship between O6-Methylguanine-DNA Methyltransferase P140k Expression Level and Chemoprotection of Hematopoietic Stem Cells. Cancer Res. 2008;68(15):6171-80.

2007

Zhang F, Thornhill SI, Howe SJ, Ulaganathan M, Schambach A, Sinclair J, Kinnon C, Gaspar HB, Antoniou M, Thrasher AJ. Lentiviral Vectors Containing an Enhancer-Less Ubiquitously Acting Chromatin Opening Element (Ucoe) Provide Highly Reproducible and Stable Transgene Expression in Hematopoietic Cells. Blood. 2007;110(5):1448-57.

Schambach A, Galla M, Maetzig T, Loew R, Baum C. Improving Transcriptional Termination of Self-Inactivating Gamma-Retroviral and Lentiviral Vectors. Mol Ther. 2007;15(6):1167-73.

Schambach A, Baum C. Vector Design for Expression of O6-Methylguanine-DNA Methyltransferase in Hematopoietic Cells. DNA Repair (Amst). 2007;6(8):1187-96.

Heuser M, Argiropoulos B, Kuchenbauer F, Yung E, Piper J, Fung S, Schlenk RF, Dohner K, Hinrichsen T, Rudolph C, Schambach A, Baum C, Schlegelberger B, Dohner H, Ganser A, Humphries RK. Mn1 Overexpression Induces Acute Myeloid Leukemia in Mice and Predicts Atra Resistance in Patients with Aml. Blood. 2007;110(5):1639-47.

Baum C, Schambach A, Modlich U, Thrasher A. [Gene Therapy of Scid-X1]. Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz. 2007;50(12):1507-17. Gentherapie der SCID-X1.

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