Gene Therapy

Further publications:   Vectorology / Biosafety     Regenerative Medicine     Hematopoiesis

2023

• CXCL1 confers a survival advantage in Kaposi's sarcoma-associated herpesvirus-infected human endothelial cells through STAT3 phosphorylation
  Lee M, Lee J, Kang S, Wirth D, Yoo S, Park C, Lee M.
  J Med Virol 2023;95(1)
  DOI: 10.1002/jmv.28020
  
   

2022

• Targeting transgenic proteins to alpha granules for platelet-directed gene therapy.
  Woods VMA, Latorre-Rey LJ, Schenk F, Rommel MGE, Moritz T, Modlich U.
  Mol Ther Nucleic Acids 2022;27:774-786
  DOI: 10.1016/j.omtn.2021.12.038
   
• Towards Novel Gene and Cell Therapy Approaches for Cervical Cancer.
  Polten R, Kutle I, Hachenberg J, Klapdor R, Morgan M, Schambach A.
  Cancers (Basel) 2022;15(1):263
  DOI: 10.3390/cancers15010263
   
• Ex Vivo Generation of CAR Macrophages from Hematopoietic Stem and Progenitor Cells for Use in Cancer Therapy.
  Paasch D, Meyer J, Stamopoulou A, Lenz D, Kuehle J, Kloos D, Buchegger T, Holzinger A, Falk CS, Kloth C, von Kaisenberg CS,
  Abken H, Schambach A, Lachmann N, Morgan M, Moritz T
  Cells 2022;11(6):994
  DOI: 10.3390/cells11060994
   
• Prime time for base editing in the mitochondria.
  Morgan MA, Lange L, Schambach A.
  Signal Transduct Target Ther 2022;7(1):213
  DOI: 10.1038/s41392-022-01068-x
   
• Hepatocellular Carcinoma Is a Natural Target for Adeno-Associated Virus (AAV) 2 Vectors.
  Meumann N, Schmithals C, Elenschneider L, Hansen T, Balakrishnan A, Hu Q, Hook S, Schmitz J, Bräsen JH, Franke A, Olarewaju O, Brandenberger C, Talbot SR, Fangmann J, Hacker UT, Odenthal M, Ott M, Piiper A, Büning H.
  Cancers (Basel) 2022;14(2):427
  DOI: 10.3390/cancers14020427
   
• Adeno-associated virus serotype 2 capsid variants for improved liver-directed gene therapy.
  Meumann N, Cabanes-Creus M, Ertelt M, Navarro RG, Lucifora J, Yuan Q, Nien-Huber K, Abdelrahman A, Vu X, Zhang L, Franke A, Schmithals C, Piiper A, Vogt A, Gonzalez-Carmona M, Frueh JT, Ullrich E, Meuleman P, Talbot SR, Odenthal M, Ott M, Seifried E, Schoeder CT, Schwäble J, Lisowski L, Büning H.
  Hepatology 2023;77(3):802-815
  DOI: 10.1002/hep.32733
   
• Editorial: Engineered immune cells in cancer immunotherapy (EICCI).
  Martin F, Schambach A, Maccalli C.
  Front Immunol 2022;13:1119363
  DOI: 10.3389/fimmu.2022.1119363
   
• Adeno-Associated Virus Vector Design-Moving the Adeno-Associated Virus to a Bioengineered Therapeutic Nanoparticle.
  Jäschke N, Büning H.
  Hematol Oncol Clin North Am 2022;36(4):667-685
  DOI: 10.1016/j.hoc.2022.04.002
   
• Selection and Validation of siRNAs Preventing Uptake and Replication of SARS-CoV-2.
  Friedrich M, Pfeifer G, Binder S, Aigner A, Vollmer Barbosa P, Makert GR, Fertey J, Ulbert S, Bodem J, König E, Geiger N, Schambach A, Schilling E, Buschmann T, Hauschildt S, Koehl U, Sewald K.
  Front Bioeng Biotechnol 2022;10:801870
  DOI: 10.3389/fbioe.2022.801870
   
• Development of an adeno-associated viral vector-based DNA vaccine as a novel strategy for cancer immunotherapy.
  Franke, Ann-Christin (Dr. rer. nat.)
  Medizinische Hochschule Hannover, 2022
   
• Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe disease.
  Dogan Y, Barese CN, Schindler JW, Yoon JK, Unnisa Z, Guda S, Jacobs ME, Oborski C, Maiwald T, Clarke DL, Schambach A, Pfeifer R, Harper C, Mason C, van Til NP.
  ​​​​​​​Mol Ther Methods Clin Dev 2022;27:464-487
  DOI: 10.1016/j.omtm.2022.10.017
   

2021

• Gene and Cell Therapy for Inherited and Acquired Immune Deficiency
  Hildegard Büning, Andrew H. Baker, Uta Griesenbach, Terence R. Flotte, and Adrian J. Thrasher
  HUMAN GENE THERAPY, VOLUME 32, NUMBERS 1 and 2 DOI: 10.1089/hum.2021.29147.hbu
   
• Generation of macrophages with enhanced functionality using chimeric antigen receptors.
  Paasch, Daniela (Dr. rer. nat. M.Sc.): Dissertation
  Medizinische Hochschule Hannover, 2021
   
• Generation of platelets with enhanced functionality by targeting transgenic proteins to alpha granules in megakaryocytes.
  Woods, Vanessa Mandeisa Alesia (Dr. rer. nat.)
  Medizinische Hochschule Hannover, 2021
  DOI: 10.26068/mhhrpm/20210719-002
   
• Low Energy Electron Irradiation Is a Potent Alternative to Gamma Irradiation for the Inactivation of (CAR-)NK-92 Cells in ATMP Manufacturing.
  Walcher L, Kistenmacher AK, Sommer C, Böhlen S, Ziemann C, Dehmel S, Braun A, Tretbar US, Klöß S, Schambach A, Morgan M, Löffler D, Kämpf C, Blumert C, Reiche K, Beckmann J, König U, Standfest B, Thoma M, Makert GR, Ulbert S, Kossatz-Böhlert U, Köhl U, Dünkel A, Fricke S.
  Front Immunol 2021;12:684052
  DOI: 10.3389/fimmu.2021.684052
   
• Synthetic Notch-Receptor-Mediated Transmission of a Transient Signal into Permanent Information via CRISPR/Cas9-Based Genome Editing.
  Sgodda M, Alfken S, Schambach A, Eggenschwiler R, Fidzinski P, Hummel M, Cantz T.
  Cells 2020;9(9)
  DOI: 10.3390/cells9091929
   
• Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning.
  Schwarzer A, Talbot SR, Selich A, Morgan M, Schott JW, Dittrich-Breiholz O, Bastone AL, Weigel B, Ha TC, Dziadek V, Gijsbers R, Thrasher AJ, Staal FJT, Gaspar HB, Modlich U, Schambach A, Rothe M.
  Mol Ther 2021;29(12):3383-3397
  DOI: 10.1016/j.ymthe.2021.06.017
   
• Optimierte Hämatopoetische Gentherapie für die Mendelian Susceptibility to Mycobacterial Diseases.
  Schiller, Jan Niklas (Dr. med.)
  Medizinische Hochschule Hannover, 2021
  DOI: 10.26068/mhhrpm/20211015-001
   
• Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47(phox)-Deficient Chronic Granulomatous Disease. 
  Schejtman A, Vetharoy W, Choi U, Rivat C, Theobald N, Piras G, Leon-Rico D, Buckland K, Armenteros-Monterroso E, Benedetti S, Ashworth MT, Rothe M, Schambach A, Gaspar HB, Kang EM, Malech HL, Thrasher AJ, Santilli G.
  Hum Gene Ther 2021;32(17-18):949-958
  DOI: 10.1089/hum.2020.276
   
• Two cases of T cell lymphoma following Piggybac-mediated CAR T cell therapy.
  Schambach A, Morgan M, Fehse B.
  Mol Ther 2021;29(9):2631-2633
  DOI: 10.1016/j.ymthe.2021.08.013
   
• Pyroptotic and Necroptotic Cell Death in the Tumor Microenvironment and Their Potential to Stimulate Anti-Tumor Immune Responses.
  Scarpitta A, Hacker UT, Büning H, Boyer O, Adriouch S.
  Front Oncol 2021;11:731598
  DOI: 10.3389/fonc.2021.731598
   
• Generation of an NFkappaB-Driven Alpharetroviral "All-in-One" Vector Construct as a Potent Tool for CAR NK Cell Therapy.
  Rudek LS, Zimmermann K, Galla M, Meyer J, Kuehle J, Stamopoulou A, Brand D, Sandalcioglu IE, Neyazi B, Moritz T, Rossig C, Altvater B, Falk CS, Abken H, Morgan MA, Schambach A.
  Front Immunol 2021;12:751138
  DOI: 10.3389/fimmu.2021.751138
   
• The Immunomodulatory CEA Cell Adhesion Molecule 6 (CEACAM6/CD66c) Is a Protein Receptor for the Influenza a Virus.
  Rahman SK, Ansari MA, Gaur P, Ahmad I, Chakravarty C, Verma DK, Sharma A, Chhibber S, Nehal N, Wirth D, Lal SK.
  Viruses 2021;13(5):726
  DOI: 10.3390/v13050726
   
• Human iPSC-derived macrophages for efficient Staphylococcus aureus clearance in a murine pulmonary infection model.
  Rafiei Hashtchin A, Fehlhaber B, Hetzel M, Manstein F, Stalp JL, Glage S, Abeln M, Zweigerdt R, Munder A, Viemann D, Ackermann M, Lachmann N.
  Blood Adv 2021;5(23):5190-5201
  DOI: 10.1182/bloodadvances.2021004853
   
• Novel AAV capsids for intravitreal gene therapy of photoreceptor disorders.
  Pavlou M, Schön C, Occelli LM, Rossi A, Meumann N, Boyd RF, Bartoe JT, Siedlecki J, Gerhardt MJ, Babutzka S, Bogedein J, Wagner JE, Priglinger SG, Biel M, Petersen-Jones SM, Büning H, Michalakis S.
  EMBO Mol Med 2021;13(4):e13392
  DOI: 10.15252/emmm.202013392
   
• Generation of macrophages with enhanced functionality using chimeric antigen receptors.
  Paasch, Daniela (Dr. rer. nat. M.Sc.)
  Medizinische Hochschule Hannover, 2021
   
• Impaired respiratory burst contributes to infections in PKCdelta-deficient patients.
  Neehus AL, Moriya K, Nieto-Patlán A, Le Voyer T, Lévy R, Özen A, Karakoc-Aydiner E, Baris S, Yildiran A, Altundag E, Roynard M, Haake K, Migaud M, Dorgham K, Gorochov G, Abel L, Lachmann N, Dogu F, Haskologlu S, İnce E, El-Benna J, Uzel G, Kiykim A, Boztug K, Roderick MR, Shahrooei M, Brogan PA, Abolhassani H, Hancioglu G, Parvaneh N, Belot A, Ikinciogullari A, Casanova JL, Puel A, Bustamante J.
  J Exp Med 2021;218(9):e20210501
  DOI: 10.1084/jem.20210501
   
• A universal protocol for isolating retinal ON bipolar cells across species via fluorescence-activated cell sorting.
  Murenu E, Pavlou M, Richter L, Rapti K, Just S, Cehajic-Kapetanovic J, Tafrishi N, Hayes A, Scholey R, Lucas R, Büning H, Grimm D, Michalakis S.
  Mol Ther Methods Clin Dev 2021;20:587-600
  DOI: 10.1016/j.omtm.2021.01.011
   
• Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal.
  Moscatelli I, Almarza E, Schambach A, Ricks D, Schulz A, Herzog CD, Henriksen K, Askmyr M, Schwartz JD, Richter J.
  Mol Ther Methods Clin Dev 2020;20:389-397
  DOI: 10.1016/j.omtm.2020.12.009
   
• Targeted cytokine delivery: cell therapy to remodel the pre-metastatic niche.
  Morgan MA, Lange L, Schambach A.
  Signal Transduct Target Ther 2021;6(1):282
  DOI: 10.1038/s41392-021-00694-1
   
• Improved Activity against Acute Myeloid Leukemia with Chimeric Antigen Receptor (CAR)-NK-92 Cells Designed to Target CD123.
  Morgan MA, Kloos A, Lenz D, Kattre N, Nowak J, Bentele M, Keisker M, Dahlke J, Zimmermann K, Sauer M, Heuser M, Schambach A.
  Viruses 2021;13(7):1365
  DOI: 10.3390/v13071365
   
• Retroviral gene therapy in Germany with a view on previous experience and future perspectives.
  Morgan MA, Galla M, Grez M, Fehse B, Schambach A.
  Gene Ther 2021;28(9):494-512
  DOI: 10.1038/s41434-021-00237-x
   
• Betibeglogene Autotemcel Gene Therapy for Non-beta(0)/beta(0) Genotype beta-Thalassemia.
  Locatelli F, Thompson AA, Kwiatkowski JL, Porter JB, Thrasher AJ, Hongeng S, Sauer MG, Thuret I, Lal A, Algeri M, Schneiderman J, Olson TS, Carpenter B, Amrolia PJ, Anurathapan U, Schambach A, Chabannon C, Schmidt M, Labik I, Elliot H, Guo R, Asmal M, Colvin RA, Walters MC.
  N Engl J Med 2022;386(5):415-427
  DOI: 10.1056/NEJMoa2113206
   
• Successful gene therapy of Diamond-Blackfan anemia in a mouse model and human CD34+ cord blood hematopoietic stem cells using a clinically applicable lentiviral vector.
  Liu Y, Dahl M, Debnath S, Rothe M, Smith EM, Grahn THM, Warsi S, Chen J, Flygare J, Schambach A, Karlsson S.
  Haematologica 2022;107(2):446-456
  DOI: 10.3324/haematol.2020.269142
   
• An Improved Lentiviral Fluorescent Genetic Barcoding Approach Distinguishes Hematopoietic Stem Cell Properties in Multiplexed In Vivo Experiments.
  Lieske A, Ha TC, Schambach A, Maetzig T.
  Hum Gene Ther 2021;32(19-20):1280-1294
  DOI: 10.1089/hum.2021.042
   
• Improved Functionality of Exhausted Intrahepatic CXCR5+ CD8+ T Cells Contributes to Chronic Antigen Clearance Upon Immunomodulation.
  Kumashie KG, Cebula M, Hagedorn C, Kreppel F, Pils MC, Koch-Nolte F, Rissiek B, Wirth D.
  Front Immunol 2021;11:592328
  DOI: 10.3389/fimmu.2020.592328
   
• In Vivo Lentiviral Gene Delivery of HLA-DR and Vaccination of Humanized Mice for Improving the Human T and B Cell Immune Reconstitution.
  Kumar S, Koenig J, Schneider A, Wermeling F, Boddul S, Theobald SJ, Vollmer M, Kloos D, Lachmann N, Klawonn F, Lienenklaus S, Talbot SR, Bleich A, Wenzel N, von Kaisenberg C, Keck J, Stripecke R.
  Biomedicines 2021;9(8):961
  DOI: 10.3390/biomedicines9080961
   
• Reprogramming enriches for somatic cell clones with small-scale mutations in cancer-associated genes.
  Kosanke M, Osetek K, Haase A, Wiehlmann L, Davenport C, Schwarzer A, Adams F, Kleppa MJ, Schambach A, Merkert S, Wunderlich S, Menke S, Dorda M, Martin U.
  Mol Ther 2021;29(8):2535-2553
  DOI: 10.1016/j.ymthe.2021.04.007
   
• Restored Macrophage Function Ameliorates Disease Pathophysiology in a Mouse Model for IL10 Receptor-deficient Very Early Onset Inflammatory Bowel Disease.
  Ackermann M, Mucci A, McCabe A, Frei S, Wright K, Snapper SB, Lachmann N, Williams DA, Brendel C.
  J Crohns Colitis 2021;15(9):1588-1595
  DOI: 10.1093/ecco-jcc/jjab031
   
• CARs and beyond: tailoring macrophage-based cell therapeutics to combat solid malignancies.
  Abdin SM, Paasch D, Morgan M, Lachmann N.
  J Immunother Cancer 2021;9(8):e002741
  DOI: 10.1136/jitc-2021-002741
   
• Correction: Effective drug treatment identified by in vivo screening in a transplantable patient-derived xenograft model of chronic myelomonocytic leukemia.
  Kloos A, Mintzas K, Winckler L, Gabdoulline R, Alwie Y, Jyotsana N, Kattre N, Schottmann R, Scherr M, Gupta C, Adams FF, Schwarzer A, Heckl D, Schambach A, Imren S, Humphries RK, Ganser A, Thol F, Heuser M.
  Leukemia 2021;35(12):3629
  DOI: 10.1038/s41375-021-01459-z
   
• NK Cell-Mediated Eradication of Ovarian Cancer Cells with a Novel Chimeric Antigen Receptor Directed against CD44.
  Klapdor R, Wang S, Morgan MA, Zimmermann K, Hachenberg J, Büning H, Dörk T, Hillemanns P, Schambach A.
  Biomedicines 2021;9(10):1339
  DOI: 10.3390/biomedicines9101339
   
• Prognostic role of docetaxel-induced suppression of free testosterone serum levels in metastatic prostate cancer patients.
  Kappler P, Morgan MA, Ivanyi P, Brunotte SJ, Ganser A, Reuter CWM.
  Sci Rep 2021;11(1):16457
  DOI: 10.1038/s41598-021-95874-y
   
• Pulmonary transplantation of alpha-1 antitrypsin (AAT)-transgenic macrophages provides a source of functional human AAT in vivo.
  Janosz E, Hetzel M, Spielmann H, Tumpara S, Rossdam C, Schwabbauer M, Kloos D, von Kaisenberg C, Schambach A, Buettner FFR, Janciauskiene S, Lachmann N, Moritz T.
  Gene Ther 2021;28(9):477-493
  DOI: 10.1038/s41434-021-00269-3
   
• Genetic Correction of IL-10RB Deficiency Reconstitutes Anti-Inflammatory Regulation in iPSC-Derived Macrophages.
  Hoffmann D, Sens J, Brennig S, Brand D, Philipp F, Vollmer Barbosa P, Kuehle J, Steinemann D, Lenz D, Buchegger T, Morgan M, Falk CS, Klein C, Lachmann N, Schambach A.
  J Pers Med 2021;11(3):221
  DOI: 10.3390/jpm11030221
   
• Beyond "Big Eaters": The Versatile Role of Alveolar Macrophages in Health and Disease.
  Hetzel M, Ackermann M, Lachmann N.
  Int J Mol Sci 2021;22(7):3308
  DOI: 10.3390/ijms22073308
   
• A Multiplex CRISPR-Screen Identifies PLA2G4A as Prognostic Marker and Druggable Target for HOXA9 and MEIS1 Dependent AML.
  Hassan JJ, Lieske A, Dörpmund N, Klatt D, Hoffmann D, Kleppa MJ, Kustikova OS, Stahlhut M, Schwarzer A, Schambach A, Maetzig T.
  Int J Mol Sci 2021;22(17):9411
  DOI: 10.3390/ijms22179411
   

• Proceedings From the First International Workshop at Sidra Medicine: “Engineered Immune Cells in Cancer Immunotherapy (EICCI): From Discovery to Off-the-Shelf Development”, 15th–16th February 2019, Doha, Qatar
  Guerrouahen B, Elnaggar M, Al-Mohannadi A, Kizhakayil D, Bonini C, Benjamin R, Brentjens R, Buchholz CJ, Casorati G, Ferrone S, Locke FL, Martin F, Schambach A, Turtle C, Veys P, van der Vliet HJ, Maccalli C
  Front Immunol 2021;11:589381
  DOI: 10.3389/fimmu.2020.589381
   

• DNA methylation changes during long-term in vitro cell culture are caused by epigenetic drift.
  Franzen J, Georgomanolis T, Selich A, Kuo CC, Stöger R, Brant L, Mulabdić MS, Fernandez-Rebollo E, Grezella C, Ostrowska A, Begemann M, Nikolić M, Rath B, Ho AD, Rothe M, Schambach A, Papantonis A, Wagner W.
  Commun Biol 2021;4(1):598
  DOI: 10.1038/s42003-021-02116-y
   

• A combination of cyclophosphamide and interleukin-2 allows CD4+ T cells converted to Tregs to control scurfy syndrome.
  Delville M, Bellier F, Leon J, Klifa R, Lizot S, Vinçon H, Sobrino S, Thouenon R, Marchal A, Garrigue A, Olivré J, Charbonnier S, Lagresle-Peyrou C, Amendola M, Schambach A, Gross D, Lamarthée B, Benoist C, Zuber J, André I, Cavazzana M, Six E.
  Blood 2021;137(17):2326-2336
  DOI: 10.1182/blood.2020009187
   

• Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector.
  Dahl M, Smith EMK, Warsi S, Rothe M, Ferraz MJ, Aerts JMFG, Golipour A, Harper C, Pfeifer R, Pizzurro D, Schambach A, Mason C, Karlsson S.
  Mol Ther Methods Clin Dev 2020;20:312-323
  DOI: 10.1016/j.omtm.2020.11.018
   

• Challenges and advances in translating gene therapy for hearing disorders.
  Büning H, Schambach A, Morgan M, Rossi A, Wichova H, Staecker H, Warnecke A, Lenarz T.
  Expert Rev Precis Med Drug Dev 2020;5(1):23-34
  DOI: 10.1080/23808993.2020.1707077
   

• The European Society of Gene and Cell Therapy: A Nearly 30-Year Endeavor to Make Gene Therapy a Clinical Reality.
  Büning H, Wilson E, Bueren J, Schambach A, Auricchio A.
  Ther 2021;32(19-20):979-982
  DOI: 10.1089/hum.2021.29183.hbu
   

• A first step toward in vivo gene editing in patients.
  Büning H, Schambach A.
  Nat Med 2021;27(9):1515-1517
  DOI: 10.1038/s41591-021-01476-6
   

• Gene Therapy "Made in Germany": A Historical Perspective, Analysis of the Status Quo, and Recommendations for Action by the German Society for Gene Therapy.
  Büning H, Fehse B, Ivics Z, Kochanek S, Koehl U, Kupatt C, Mussolino C, Nettelbeck DM, Schambach A, Uckert W, Wagner E, Cathomen T.
  Hum Gene Ther 2021;32(19-20):987-996
  DOI: 10.1089/hum.2021.29178.hbu
   

• Gene and Cell Therapy for Inherited and Acquired Immune Deficiency.
  Büning H, Baker AH, Griesenbach U, Flotte TR, Thrasher AJ.
  Hum Gene Ther 2021;32(1-2):1-3
  DOI: 10.1089/hum.2021.29147.hbu
   

• Die Inhibition von eIF4E als therapeutische Zielstruktur in akuten T-Zell-Leukämien im Mausmodell.
  Benn, Nomme-Katharina Susanne (Dr. med.):Dissertation
  Medizinische Hochschule Hannover, 2021
  
   

2020

• Synthetic Notch-Receptor-Mediated Transmission of a Transient Signal into Permanent Information via CRISPR/Cas9-Based Genome Editing
  Malte Sgodda, Susanne Alfken, Axel Schambach, Reto Eggenschwiler, Pawel Fidzinski, Michael Hummel and Tobias Cantz
  Cells 2020, 9, 1929; doi:10.3390/cells9091929
   
• Knockout-Induced Pluripotent Stem Cells for Disease and Therapy Modeling of IL-10-Associated Primary Immunodeficiencies
  Johanna Sens, Dirk Hoffmann, Lucas Lange, Philippe Vollmer Barbosa, Michael Morgan, Christine S. Falk and Axel Schambach
  Hum Gene Ther. 2021 Jan;32(1-2):77-95.doi: 10.1089/hum.2020.235. 
   

• High Cytotoxic Efficiency of Lentivirally and Alpharetrovirally Engineered CD19-Specific Chimeric Antigen Receptor Natural Killer Cells Against Acute Lymphoblastic Leukemia
  Stephan Müller, Tobias Bexte, Veronika Gebel, Franziska Kalensee, Eva Stolzenberg, Jessica Hartmann, Ulrike Koehl,
  Axel Schambach,Winfried S. Wels, Ute Modlich and Evelyn Ullrich
  Front. Immunol., 24 January 2020 https://doi.org/10.3389/fimmu.2019.03123
   

• Use of Cell and Genome Modification Technologies to Generate Improved “Off-the-Shelf” CAR T and CAR NK Cells
  Michael A. Morgan,Hildegard Büning, Martin Sauer and Axel Schambach
  Front. Immunol., 07 August 2020, Volume 11 - 2020 | https://doi.org/10.3389/fimmu.2020.01965
   

• Targeting Kaposi’s Sarcoma-Associated Herpesvirus ORF21 Tyrosine Kinase and Viral Lytic Reactivation by Tyrosine Kinase Inhibitors Approved for Clinical Use
  Guillaume Beauclair, Eleonora Naimo, Tatyana Dubich, Jessica Rückert, Sandra Koch, Akshay Dhingra, Dagmar Wirth, Thomas F. Schulz
  J Virol. 2020 Feb 14;94(5):e01791-19.doi: 10.1128/JVI.01791-19. Print 2020 Feb 14.
   

• The Immune-Modulatory Properties of iPSC-Derived Antigen-Presenting Cells
  Mania Ackermann, Anna Christina Dragon, Nico Lachmann
  Transfus Med Hemother. 2020 Dec;47(6):444-453.doi: 10.1159/000512721.
   

• Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy
  Christian Brendel, Olivier Negre, Michael Rothe, Swaroopa Guda, Geoff Parsons, Chad Harris, Meaghan McGuinness, Daniela Abriss, Alla Tsytsykova, Denise Klatt, Martin Bentler, Danilo Pellin,
  Lauryn Christiansen, Axel Schambach, John Manis, Helene Trebeden-Negre, Melissa Bonner, Erica Esrick,
  Gabor Veres, Myriam Armant and David A. Williams
  Molecular Therapy: Methods & Clinical Development Vol. 17 June 2020 https://doi.org/10.1016/j.omtm.2020.03.015
   

• Modeling MyD88 Deficiency In Vitro Provides New Insights in Its Function
  Nils Craig-Mueller, Ruba Hammad, Roland Elling, Jamal Alzubi, Barbara Timm, Julia Kolter, Nele Knelangen, Christien Bednarski, Birgitta Gläser, Sandra Ammann, Zoltán Ivics, Judith Fischer, Carsten Speckmann, Klaus Schwarz, Nico Lachmann, Stephan Ehl, Thomas Moritz, Philipp Henneke and Toni Cathomen
  Front. Immunol., 23 December 2020 Sec. Primary Immunodeficiencies Volume 11 - 2020 https://doi.org/10.3389/fimmu.2020.608802
   

• Generation of two hiPSC lines (MHHi016-A, MHHi016-B) from a primary ciliary dyskinesia patient carrying a homozygous 5 bp duplication (c.248_252dup (p.Gly85Cysfs*11)) in exon 1 of the CCNO gene.
  Julia Dahlmann, Anais Sahabian, Nora Drick, Alexandra Haase, Gudrun Göhring, Nico Lachmann, Felix C. Ringshausen, Tobias Welte, Ulrich Martin, Ruth Olmer
  Stem Cell Research, 18 May 2020, 46:101850 DOI: 10.1016/j.scr.2020.101850 PMID: 32464346 
   

• Growth differentiation factor 11 attenuates liver fibrosis via expansion of liver progenitor cells
  Zhen Dai, Guangqi Song, Asha Balakrishnan, Taihua Yang, Qinggong Yuan, Selina Möbus, Anna-Carina Weiss, Martin Bentler, Jimin Zhu, Xuemei Jiang, Xizhong Shen, Heike Bantel, Elmar Jaeckel, Andreas Kispert, Arndt Vogel, Anna Saborowski, Hildegard Büning, Michael Manns, Tobias Cantz, Michael Ott, Amar Deep Sharma
  Gut. 2020 Jun;69(6):1104-1115.doi: 10.1136/gutjnl-2019-318812. Epub 2019 Nov 25.
   

• CAR-T cells and TRUCKs that recognize an EBNA-3C-derived epitope presented on HLA-B*35 control Epstein-Barr virus-associated lymphoproliferation
  Anna Christina Dragon, Katharina Zimmermann, Thomas Nerreter, Deborah Sandfort, Julia Lahrberg, Stephan Klöß, Christina Kloth, Caroline Mangare, Agnes Bonifacius, Sabine Tischer-Zimmermann, Rainer Blasczyk, Britta Maecker-Kolhoff, Barbara Uchanska-Ziegler, Hinrich Abken, Axel Schambach, Michael Hudecek, Britta Eiz-Vesper
  J Immunother Cancer. 2020 Oct;8(2):e000736.doi: 10.1136/jitc-2020-000736.
   

• Generation of two human induced pluripotent stem cell lines (MHHi017-A, MHHi017-B) from a patient with primary ciliary dyskinesia carrying a homozygous mutation (c.7915C > T [p.Arg2639*]) in the DNAH5 gene
  Nora Drick, Julia Dahlmann, Anais Sahabian, Alexandra Haase, Gudrun Göhring, Nico Lachmann, Felix C Ringshausen, Tobias Welte, Ulrich Martin, Ruth Olmer
  Stem Cell Res. 2020 Jul;46:101848.doi: 10.1016/j.scr.2020.101848. Epub 2020 May 20.
   

• Fatal Cytomegalovirus Infection in an Adult with Inherited NOS2 Deficiency
  Scott B Drutman, Davood Mansouri, Seyed Alireza Mahdaviani, Anna-Lena Neehus, David Hum, Ruslana Bryk, Nicholas Hernandez, Serkan Belkaya, Franck Rapaport, Benedetta Bigio, Robert Fisch, Mahbuba Rahman, Taushif Khan, Fatima Al Ali, Majid Marjani, Nahal Mansouri, Lazaro Lorenzo-Diaz, Jean-François Emile, Nico Marr , Emmanuelle Jouanguy , Jacinta Bustamante , Laurent Abel , Stéphanie Boisson-Dupuis, Vivien Béziat, Carl Nathan, Jean-Laurent Casanova
  N Engl J Med. 2020 Jan 30;382(5):437-445.doi: 10.1056/NEJMoa1910640.
   

• Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID
  Laura Garcia-Perez, Marja van Eggermond, Lieke van Roon, Sandra A Vloemans, Martijn Cordes, Axel Schambach, Michael Rothe, Dagmar Berghuis, Chantal Lagresle-Peyrou, Marina Cavazzana, Fang Zhang, Adrian J Thrasher, Daniela Salvatori, Pauline Meij, Anna Villa, Jacques J M Van Dongen, Jaap-Jan Zwaginga, Mirjam van der Burg, H Bobby Gaspar,  Arjan Lankester, Frank J T Staal, Karin Pike-Overzet
  Mol Ther Methods Clin Dev. 2020 Mar 31;17:666-682.doi: 10.1016/j.omtm.2020.03.016. eCollection 2020 Jun 12.
   

• Towards Clinical Implementation of Adeno-Associated Virus (AAV) Vectors for Cancer Gene Therapy: Current Status and Future Perspectives
  Ulrich T Hacker, Martin Bentler, Dorota Kaniowska, Michael Morgan, Hildegard Büning
  Cancers (Basel). 2020 Jul 14;12(7):1889.doi: 10.3390/cancers12071889.
   

• Human Lentiviral Gene Therapy Restores the Cellular Phenotype of Autosomal Recessive Complete IFN-γR1 Deficiency
  Katharina Hahn, Liart Pollmann, Juliette Nowak, Ariane Hai Ha Nguyen, Kathrin Haake, Anna-Lena Neehus, Syed F. Hassnain Waqas, Frank Pessler, Ulrich Baumann, Miriam Hetzel, Jean-Laurent Casanova, Ansgar Schulz, Jacinta Bustamante, Mania Ackermann and Nico Lachmann
  Molecular Therapy: Methods & Clinical Development Vol. 17 June 2020 https://doi.org/10.1016/j.omtm.2020.04.002
   

• Effective hematopoietic stem cell-based gene therapy in a murine model of hereditary pulmonary alveolar proteinosis
  Miriam Hetzel, Elena Lopez-Rodriguez, Adele Mucci, Ariane Hai Ha Nguyen, Takuji Suzuki, Kenjiro Shima, Theresa Buchegger, Sabine Dettmer, Thomas Rodt, Jens P Bankstahl, Punam Malik, Lars Knudsen, Axel Schambach, Gesine Hansen, Bruce C Trapnell, Nico Lachmann, Thomas Moritz
  Haematologica. 2020 Apr;105(4):1147-1157.doi: 10.3324/haematol.2018.214866. Epub 2019 Jul 9.
   

• Competitive sgRNA Screen Identifies p38 MAPK as a Druggable Target to Improve HSPC Engraftment
  Denise Klatt, Teng-Cheong Ha, Maximilian Schinke, Anton Selich, Anna Lieske, Julia Dahlke, Michael Morgan, Tobias Maetzig, Axel Schambach
  Cells. 2020 Sep 29;9(10):2194.doi: 10.3390/cells9102194.

2019

• The stem cell–specific long noncoding RNA HOXA10-AS in the pathogenesis of KMT2A-rearranged leukemia
  Sina Al-Kershi, Raj Bhayadia, Michelle Ng, Lonneke Verboon, Stephan Emmrich, Lucie Gack, Adrian Schwarzer, Till Strowig,
  Dirk Heckl, and Jan-Henning Klusmann
  Blood Adv. 2019 Dec 23; 3(24): 4252-4263
   
• Characterization of a Novel Third-Generation Anti-CD24-CAR against Ovarian Cancer
  Rüdiger Klapdor, Shuo Wang, Michael Morgan, Thilo Dörk, Ulrich Hacker,
  Peter Hillemanns, Hildegard Büning and Axel Schambach
  Int. J. Mol. Sci. 2019, 20, 660; doi:10.3390/ijms20030660 
   
• Targeted Repair of p47-CGD in iPSCs by CRISPR/Cas9: Functional Correction without Cleavage in the Highly Homologous Pseudogenes
  Denise Klatt, Erica Cheng, Friederike Philipp, Anton Selich, Julia Dahlke, Reinhold E. Schmidt,
  Juliane W. Schott, Hildegard Büning, Dirk Hoffmann, Adrian J. Thrasher, and Axel Schambach
  StemCellReports|Vol. 13 |590–598 |October 8, 2019 https://doi.org/10.1016/j.stemcr.2019.08.008
   
• Development of Automated Separation, Expansion, and Quality Control Protocols for Clinical-Scale Manufacturing of Primary Human NK Cells and Alpharetroviral Chimeric Antigen Receptor Engineering
  Olaf Oberschmidt, Michael Morgan, Volker Huppert, Joerg Kessler, Tanja Gardlowski, Nadine Matthies, Krasimira Aleksandrova, Lubomir Arseniev,
  Axel Schambach, Ulrike Koehl and Stephan Kloess
  HUMAN GENE THERAPY METHODS, VOLUME 30 NUMBER 3 DOI: 10.1089/hgtb.2019.039
   
• Cytokine Selection of MSC Clones with Different Functionality
  Anton Selich, Teng-Cheong Ha, Michael Morgan, Christine S. Falk, Constantin von Kaisenberg,
  Axel Schambach and Michael Rothe
  StemCellReports|Vol. 13 |262–273 |August 13, 2019 https://doi.org/10.1016/j.stemcr.2019.06.001
   
• Umbilical cord as a long-term source of activatable mesenchymal stromal cells for immunomodulation
  Anton Selich, Katharina Zimmermann, Michel Tenspolde, Oliver Dittrich-Breiholz, Constantin von Kaisenberg,
  Axel Schambach and Michael Rothe
  Stem Cell Research & Therapy (2019) 10:285 doi.org/10.1186/s13287-019-1376-9
   
• Regulatory T cells engineered with a novel insulin-specific chimeric antigen receptor as a candidate immunotherapy for type 1diabetes
  Michel Tenspolde, Katharina Zimmermann, Leonie C. Weber, Martin Hapke,
  Maren Lieber, Janine Dywicki, Andre Frenzel,MichaelHust, Melanie Galla,
  Laura E. Buitrago-Molina, Michael P. Manns, Elmar Jaeckel, Matthias Hardtke-Wolenski
  J Autoimmun. 2019 Sep;103:102289.doi: 10.1016/j.jaut.2019.05.017. Epub 2019 Jun 5.

2018

• Chimeric Antigen Receptor T Cells: Extending Translation from Liquid to Solid Tumors.
  Morgan MA, Schambach A.
  Hum Gene Ther. 2018 Oct;29(10):1083-1097. doi: 10.1089/hum.2017.251.

• Engineering CAR-T Cells for Improved Function Against Solid Tumors.
  Morgan MA, Schambach A.
  Front Immunol. 2018 Oct 29;9:2493. doi: 10.3389/fimmu.2018.02493.

• Macrophage entrapped silica coated superparamagnetic iron oxide particles for controlled drug release in a 3D cancer model.
  Ullah S, Seidel K, Türkkan S, Warwas DP, Dubich T, Rohde M, Hauser H, Behrens P, Kirschning A, Köster M, Wirth D.
  J Control Release. 2019 Jan 28;294:327-336. doi: 10.1016/j.jconrel.2018.12.040.

• Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease.
  Brendel C, Rothe M, Santilli G, Charrier S, Stein S, Kunkel H, Abriss D, Müller-Kuller U, Gaspar B, Modlich U, Galy A, Schambach A, Thrasher AJ, Grez M.
  Hum Gene Ther Clin Dev. 2018 Jun;29(2):69-79. doi: 10.1089/humc.2017.245.

• Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease.
  Poletti V, Urbinati F, Charrier S, Corre G, Hollis RP, Campo Fernandez B, Martin S, Rothe M, Schambach A, Kohn DB, Mavilio F.
  Mol Ther Methods Clin Dev. 2018 Nov 1;11:167-179. doi: 10.1016/j.omtm.2018.10.014.

• Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency.
  Poletti V, Charrier S, Corre G, Gjata B, Vignaud A, Zhang F, Rothe M, Schambach A, Gaspar HB, Thrasher AJ, Mavilio F.
  Mol Ther Methods Clin Dev. 2018 Mar 10;9:257-269. doi: 10.1016/j.omtm.2018.03.002.

• Preclinical validation: LV/IL-12 transduction of patient leukemia cells for immunotherapy of AML.
  Huang J, Liu Y, Au BC, Barber DL, Arruda A, Schambach A, Rothe M, Minden MD, Paige CJ, Medin JA.
  Mol Ther Methods Clin Dev. 2016 Dec 7;3:16074.

• Pulmonary Transplantation of Human Induced Pluripotent Stem Cell-derived Macrophages Ameliorates Pulmonary Alveolar Proteinosis.
  Happle C, Lachmann N, Ackermann M, Mirenska A, Göhring G, Thomay K, Mucci A, Hetzel M, Glomb T, Suzuki T, Chalk C, Glage S, Dittrich-Breiholz O, Trapnell B, Moritz T, Hansen G.
  Am J Respir Crit Care Med. 2018 Aug 1;198(3):350-360. doi: 10.1164/rccm.201708-1562OC.

• T-cell gene therapy for perforin deficiency corrects cytotoxicity defects and prevents hemophagocytic lymphohistiocytosis manifestations.
  Ghosh S, Carmo M, Calero-Garcia M, Ricciardelli I, Bustamante Ogando JC, Blundell MP, Schambach A, Ashton-Rickardt PG, Booth C, Ehl S, Lehmberg K, Thrasher AJ, Gaspar HB.
  J Allergy Clin Immunol. 2018 Sep;142(3):904-913.e3. doi: 10.1016/j.jaci.2017.11.050.

2017

• Celebrating 25 Years of the European Society of Gene and Cell Therapy.
  Ali RR, Büning H.
  Hum Gene Ther 2017;28(11):939
   
• Twenty-Five Years of Gene Therapy: Prof. George Dickson on Past Progress and Future Promise.
  Büning H.
  Hum Gene Ther 2017;28(11):960-963
   
• Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia.
  Debnath S, Jaako P, Siva K, Rothe M, Chen J, Dahl M, Gaspar HB, Flygare J, Schambach A, Karlsson S.
  Mol Ther 2017;25(8):1805-1814
   
• Hematopoietic stem cell gene therapy for IFNgammaR1 deficiency protects mice from mycobacterial infections.
  Hetzel M, Mucci A, Blank P, Nguyen AHH, Schiller J, Halle O, Kühnel MP, Billig S, Meineke R, Brand D, Herder V, Baumgärtner W, Bange FC, Goethe R, Jonigk D, Förster R, Gentner B, Casanova JL, Bustamante J, Schambach A, Kalinke U, Lachmann N.
  Blood 2017:DOI: 10.1182/blood-2017-10-812859
   
• Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34(+) Cells for Correction of Fabry Disease.
  Huang J, Khan A, Au BC, Barber DL, Lopez-Vasquez L, Prokopishyn NL, Boutin M, Rothe M, Rip JW, Abaoui M, Nagree MS, Dworski S, Schambach A, Keating A, West ML, Klassen J, Turner PV, Sirrs S, Rupar CA, Auray-Blais C, Foley R, Medin JA.
  Mol Ther Methods Clin Dev 2017;5:241-258
   
• Improved killing of ovarian cancer stem cells by combining a novel CAR-based immunotherapy and chemotherapy.
  Klapdor R, Wang S, Hacker U, Büning H, Morgan M, Dörk-Bousset T, Hillemanns P, Schambach A.
  Hum Gene Ther 2017;28(10):886-896
   
• 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.
  Klöss S, Oberschmidt O, Morgan M, Dahlke J, Arseniev L, Huppert V, Granzin M, Gardlowski T, Matthies N, Soltenborn S, Schambach A, Koehl U.
  Hum Gene Ther 2017;28(10):897-913
   
• Targeting NSG Mice Engrafting Cells with a Clinically Applicable Lentiviral Vector Corrects Osteoclasts in Infantile Malignant Osteopetrosis.
  Moscatelli I, Löfvall H, Schneider Thudium C, Rothe M, Montano C, Kertesz Z, Sirin M, Schulz A, Schambach A, Henriksen K, Richter J.
  Hum Gene Ther 2017:DOI:10.1089/hum.2017.053
   
• Gene and Cell Therapy in Germany.
  von Kalle C, Fehse B, Büning H.
  Hum Gene Ther 2017;28(10):781
   
• Scavenger receptor class B member 1 (SCARB1) variants modulate hepatitis C virus replication cycle and viral load.
  Westhaus S, Deest M, Nguyen ATX, Stanke F, Heckl D, Costa R, Schambach A, Manns MP, Berg T, Vondran FWR, Sarrazin C, Ciesek S, von Hahn T.
  J Hepatol 2017;67(2):237-245
   
• Novel Mutant AAV2 Rep Proteins Support AAV2 Replication without Blocking HSV-1 Helpervirus Replication.
  Seyffert M, Glauser DL, Schraner EM, de Oliveira AP, Mansilla-Soto J, Vogt B, Büning H, Linden RM, Ackermann M, Fraefel C.
  PLoS One. 2017 Jan 26;12(1):e0170908. doi: 10.1371/journal.pone.0170908.
   
• Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency.
  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.
  Hum Gene Ther. 2017 Jan;28(1):112-124. doi: 10.1089/hum.2016.064.

2016

• Adeno-Associated Viral Vectors Transduce Mature Human Adipocytes in Three-Dimensional Slice Cultures.
  Kallendrusch S, Schopow N, Stadler SC, Büning H, Hacker UT.
  Hum Gene Ther Methods. 2016 Oct;27(5):171-173.

• Evaluation of Angiopoietin-2 as a biomarker in gastric cancer: results from the randomised phase III AVAGAST trial.
  Hacker UT, Escalona-Espinosa L, Consalvo N, Goede V, Schiffmann L, Scherer SJ, Hedge P, Van Cutsem E, Coutelle O, Büning H.
  Br J Cancer. 2016 Apr 12;114(8):855-62. doi: 10.1038/bjc.2016.30.

• Gene Insertion Into Genomic Safe Harbors for Human Gene Therapy.
  Papapetrou EP, Schambach A.
  Mol Ther. 2016 Apr;24(4):678-84. doi: 10.1038/mt.2016.38. Review.

• Retroviral Vectors for Cancer Gene Therapy.
  Schambach A, Morgan M.
  Recent Results Cancer Res. 2016;209:17-35. doi: 10.1007/978-3-319-42934-2_2.

• Reply to "Wild-type AAV Insertions in Hepatocellular Carcinoma Do Not Inform Debate Over Genotoxicity Risk of Vectorized AAV".
  Schmidt M, Gil-Farina I, Büning H.
  Mol Ther. 2016 Apr;24(4):661-2. doi: 10.1038/mt.2016.48.

• Inhibitors of Angiogenesis.
  Büning H, Hacker UT.
  Adv Exp Med Biol. 2016;917:261-85. doi: 10.1007/978-3-319-32805-8_12.

• Directed evolution of a recombinase that excises the provirus of most HIV-1 primary isolates with high specificity.
  Karpinski J, Hauber I, Chemnitz J, Schäfer 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.
  Nat Biotechnol. 2016 Apr;34(4):401-9. doi: 10.1038/nbt.3467.

• Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency.
  Garcia-Gomez M, Calabria A, Garcia-Bravo M, Benedicenti F, Kosinski P, López-Manzaneda S, Hill C, Del Mar Mañu-Pereira M, Martín MA, Orman I, Vives-Corrons JL, Kung C, Schambach A, Jin S, Bueren JA, Montini E, Navarro S, Segovia JC.
  Mol Ther. 2016 Aug;24(7):1187-98. doi: 10.1038/mt.2016.87.

• Gene Editing for the Efficient Correction of a Recurrent COL7A1 Mutation in Recessive Dystrophic Epidermolysis Bullosa Keratinocytes.
  Chamorro C, Mencía A, Almarza D, Duarte B, Büning H, Sallach J, Hausser I, Del Río M, Larcher F, Murillas R.
  Mol Ther Nucleic Acids. 2016 Apr 5;5:e307. doi: 10.1038/mtna.2016.19.

2015

• Adeno-associated Vector Toxicity-To Be or Not to Be?
  Büning H, Schmidt M.
  Mol Ther. 2015 Nov;23(11):1673-5. doi: 10.1038/mt.2015.182.

• TALEN-mediated functional correction of X-linked chronic granulomatous disease in patient-derived induced pluripotent stem cells.
  Dreyer AK, Hoffmann D, Lachmann N, Ackermann M, Steinemann D, Timm B, Siler U, Reichenbach J, Grez M, Moritz T, Schambach A, Cathomen T.
  Biomaterials. 2015 Nov;69:191-200. doi: 10.1016/j.biomaterials.2015.07.057. Epub 2015 Aug 3.

• Inherited DOCK2 Deficiency in Patients with Early-Onset Invasive Infections.
  Dobbs K, Domínguez 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, Çiftçi E, Felgentreff K, Ohsumi TK, Pedergnana V, Boisson B, Haskoloğlu Ş, 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, İkincioğulları A, Al-Herz W, Helminen M, Doğu F, Casanova JL, Boztuğ K, Notarangelo LD.
  N Engl J Med. 2015 Jun 18;372(25):2409-22. doi: 10.1056/NEJMoa1413462.

• Perforin gene transfer into hematopoietic stem cells improves immune dysregulation in murine models of perforin deficiency.
  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.
  Mol Ther. 2015 Apr;23(4):737-45. doi: 10.1038/mt.2014.242. Epub 2014 Dec 19.

2014

• A modified γ-retrovirus vector for X-linked severe combined immunodeficiency.
  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, Fernández 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.
  N Engl J Med. 2014 Oct 9;371(15):1407-17. doi: 10.1056/NEJMoa1404588.

• Tropism-modified AAV vectors overcome barriers to successful cutaneous therapy.
  Sallach J, Di Pasquale G, Larcher F, Niehoff N, Rübsam M, Huber A, Chiorini J, Almarza D, Eming SA, Ulus H, Nishimura S, Hacker UT, Hallek M, Niessen CM, Büning H.
  Mol Ther. 2014 May;22(5):929-39. doi: 10.1038/mt.2014.14.

• Hepatitis B virus infection enhances susceptibility toward adeno-associated viral vector transduction in vitro and in vivo.
  Hösel M, Lucifora J, Michler T, Holz G, Gruffaz M, Stahnke S, Zoulim F, Durantel D, Heikenwalder M, Nierhoff D, Millet R, Salvetti A, Protzer U, Büning H.
  Hepatology. 2014 Jun;59(6):2110-20. doi: 10.1002/hep.26990.

• Safety of gene therapy: new insights to a puzzling case.
  Rothe M, Schambach A, Biasco L.
  Curr Gene Ther. 2014;14(6):429-36. Review.

• Improved retroviral episome transfer of transcription factors enables sustained cell fate modification.
  Schott JW, Hoffmann D, Maetzig T, Müller FJ, Steinemann D, Zychlinski D, Cantz T, Baum C, Schambach A.
  Gene Ther. 2014 Nov;21(11):938-49. doi: 10.1038/gt.2014.69. Epub 2014 Aug 7.

• Expanding Flp-RMCE options: the potential of Recombinase Mediated Twin-Site Targeting (RMTT).
  Turan S, Qiao J, Madden S, Benham C, Kotz M, Schambach A, Bode J.
  Gene. 2014 Aug 10;546(2):135-44. doi: 10.1016/j.gene.2014.06.002. Epub 2014 Jun 4. Review.

• Non-integrating gamma-retroviral vectors as a versatile tool for transient zinc-finger nuclease delivery.
  Bobis-Wozowicz S, Galla M, Alzubi J, Kuehle J, Baum C, Schambach A, Cathomen T.
  Sci Rep. 2014 Apr 11;4:4656. doi: 10.1038/srep04656.

• Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.
  Braun CJ, Boztug K, Paruzynski A, Witzel M, Schwarzer A, Rothe M, Modlich U, Beier R, Göhring G, Steinemann D, Fronza R, Ball CR, Haemmerle R, Naundorf S, Kühlcke K, Rose M, Fraser C, Mathias L, Ferrari R, Abboud MR, Al-Herz W, Kondratenko I, Maródi L, Glimm H, Schlegelberger B, Schambach A, Albert MH, Schmidt M, von Kalle C, Klein C.
  Sci Transl Med. 2014 Mar 12;6(227):227ra33. doi: 10.1126/scitranslmed.3007280.

• Gene correction of human induced pluripotent stem cells repairs the cellular phenotype in pulmonary alveolar proteinosis.
  Lachmann N, Happle C, Ackermann M, Lüttge 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, Göhring G, Cantz T, Martin U, Schwerk N, Hansen G, Moritz T.
  Am J Respir Crit Care Med. 2014 Jan 15;189(2):167-82. doi: 10.1164/rccm.201306-1012OC.

2013

• Gene therapy on the move.
  Kaufmann KB, Büning H, Galy A, Schambach A, Grez M.
  EMBO Mol Med. 2013 Nov;5(11):1642-61. doi: 10.1002/emmm.201202287. Epub 2013 Sep 17. Review.

• From bench to bedside: preclinical evaluation of a self-inactivating gammaretroviral vector for the gene therapy of X-linked chronic granulomatous disease.
  Stein S, Scholz S, Schwäble J, Sadat MA, Modlich U, Schultze-Strasser S, Diaz M, Chen-Wichmann L, Müller-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, Kühlcke K, Baum C, Schambach A, Dinauer MC, Schmidt M, Grez M.
  Hum Gene Ther Clin Dev. 2013 Jun;24(2):86-98. doi: 10.1089/humc.2013.019.

• Alpharetroviral vector-mediated gene therapy for X-CGD: functional correction and lack of aberrant splicing.
  Kaufmann KB, Brendel C, Suerth JD, Mueller-Kuller U, Chen-Wichmann L, Schwäble J, Pahujani S, Kunkel H, Schambach A, Baum C, Grez M.
  Mol Ther. 2013 Mar;21(3):648-61. doi: 10.1038/mt.2012.249. Epub 2012 Dec 4.

2012

• MicroRNA-150-regulated vectors allow lymphocyte-sparing transgene expression in hematopoietic gene therapy.
  Lachmann N, Jagielska J, Heckl D, Brennig S, Pfaff N, Maetzig T, Modlich U, Cantz T, Gentner B, Schambach A, Moritz T.
  Gene Ther. 2012 Sep;19(9):915-24. doi: 10.1038/gt.2011.148. Epub 2011 Oct 6.

• Epidermal growth factor improves lentivirus vector gene transfer into primary mouse hepatocytes.
  Rothe M, Rittelmeyer I, Iken M, Rüdrich U, Schambach A, Glage S, Manns MP, Baum C, Bock M, Ott M, Modlich U.
  Gene Ther. 2012 Apr;19(4):425-34. doi: 10.1038/gt.2011.117. Epub 2011 Aug 18.

2011

• Promoter competition for gene therapy of SCID-X1.
  Baum C, Schambach A.
  Hum Gene Ther. 2011 Mar;22(3):255-6. doi: 10.1089/hum.2011.2280. No abstract available.

2002 - 2010

• Cell and virus genetics at the roots of gene therapy, retrovirology, and hematopoietic stem cell biology: Wolfram Ostertag (1937-2010).
  Stocking C, Grez M, Fehse B, von Laer D, Itoh K, Prassolov V, Nowock J, Kühlcke K, Just U, Schröder T, Klump H, Schiedlmeier B, Grassman E, Meyer J, Li Z, Schambach A, Modlich U, Kustikova O, Galla M, Bode J, Zander A, Baum C.
  Hum Gene Ther. 2010 Nov;21(11):1501-3. doi: 10.1089/hum.2010.1901. No abstract available.

• A new PG13-based packaging cell line for stable production of clinical-grade self-inactivating gamma-retroviral vectors using targeted integration.
  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.
  Gene Ther. 2010 Feb;17(2):272-80. doi: 10.1038/gt.2009.134. Epub 2009 Oct 29.

• The genomic risk of somatic gene therapy.
  Kustikova O, Brugman M, Baum C.
  Semin Cancer Biol. 2010 Aug;20(4):269-78. doi: 10.1016/j.semcancer.2010.06.003. Epub 2010 Jul 1. Review.

• Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency.
  Thornhill SI, Schambach A, Howe SJ, Ulaganathan M, Grassman E, Williams D, Schiedlmeier B, Sebire NJ, Gaspar HB, Kinnon C, Baum C, Thrasher AJ.
  Mol Ther. 2008 Mar;16(3):590-8. doi: 10.1038/sj.mt.6300393. Epub 2008 Jan 8.

• Gene therapy: X-SCID transgene leukaemogenicity.
  Thrasher AJ, Gaspar HB, Baum C, Modlich U, Schambach A, Candotti F, Otsu M, Sorrentino B, Scobie L, Cameron E, Blyth K, Neil J, Abina SH, Cavazzana-Calvo M, Fischer A.
  Nature. 2006 Sep 21;443(7109):E5-6; discussion E6-7.

• Towards hematopoietic stem cell-mediated protection against infection with human immunodeficiency virus.
  Schambach A, Schiedlmeier B, Kühlcke K, Verstegen M, Margison GP, Li Z, Kamino K, Bohne J, Alexandrov A, Hermann FG, von Laer D, Baum C.
  Gene Ther. 2006 Jul;13(13):1037-47. Epub 2006 Mar 16.

• Multidrug resistance 1 gene transfer can confer chemoprotection to human peripheral blood progenitor cells engrafted in immunodeficient mice.
  Schiedlmeier B, Schilz AJ, Kühlcke K, Laufs S, Baum C, Zeller WJ, Eckert HG, Fruehauf S.
  Hum Gene Ther. 2002 Jan 20;13(2):233-42.