Gene Therapy

Further publications:   Vectorology / Biosafety     Regenerative Medicine     Hematopoiesis

2019

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.