REBIRTH Mitglieder

Schambach, Axel

Schambach, Axel, Prof. Dr. med. PhD


REBIRTH Unit Regenerative Gene Therapy

Hannover Medical School
Carl-Neuberg-Str. 1
30625 Hannover



A. Schambach (*1974), MD, PhD, is W3 Professor for Gene Modification of Somatic Cells and Director of the Institute of Experimental Hematology at MHH. He studied medicine in Hamburg, San Diego, San Francisco, Dallas and Zürich received his PhD in Molecular Medicine. His main research interest is to understand the molecular pathophysiology of inborn and acquired diseases to develop tailor-made gene therapy and molecular medicine strategies. Here he focuses on novel integrating vectors as gene delivery tool, on designer nucleases, as well as on reprogramming and transdifferentiation strategies, and on stem cell-based disease modeling.

Biography / About

Date of Birth:August, 1974
Function:Full (W3) Professor, Acting Director of the Institute of Experimental Hematology, Hannover Medical School (MHH)


1994German A-levels (Abitur), Alexander-von-Humboldt Gymnasium Hamburg, Germany
1995 - 2001State Examination, Human Medicine, University of Hamburg, Germany
1998USMLE Licensing Examination Step I
2002 - 2005PhD, International PhD Program ‘Molecular Medicine’, MHH, thesis with excellent / highest distinction
2003Dr. med. thesis, summa cum laude, University of Hamburg
2012Post-doctoral lecturing qualification (Habilitation) and Venia legendi for Molecular Medicine, MHH

Academic appointments and Research posts:

2002 - 2003Pediatric Resident, Children’s Hospital, MHH
2002 - 2006Scientist, Experimental Hematology (Prof. Baum), MHH, gene therapy
2007 - 2012Group Leader, Junior Research Group ‘Hematopoietic Cell Therapy’, REBIRTH Cluster of Excellence, MHH
2009 - 2014Group Leader, German-Chinese Junior Research Group ‘New methods for gene therapy of inherited and acquired diseases’, MHH/Guangzhou, China
Since 2012Group Leader, Research Groups ‘Regenerative Gene Therapy’ and ‘Synthetic Cells for Regeneration’, REBIRTH, MHH
Since 2012Associate Faculty and Lecturer, Boston Children’s Hospital, Division of Hematology/Oncology, Harvard Medical School, Boston, MA, USA
Since 2013Full Professor (W3) for Gene Modification of Somatic Cells, MHH
Since 2013Acting Director, Institute of Experimental Hematology, MHH

Other professional activities:

Since 2005Reviewer for jounals (e.g. Blood, Gene Therapy, Human Gene Therapy, Molecular Therapy, Biomaterials, Nature Protocols, PNAS) and funding organisations (across Europe)
Since 2009Member of the Student Selection Committee of the PhD Programs ‘Regenerative Sciences’ and ‘Molecular Medicine’ and the Human Medicine Program as well as the German Academic Scholarship Foundation (‘Studienstiftung des deutschen Volkes’)

Awards and Prizes:

1996 - 2001Scholarship of the German Academic Scholarshop Foundation
2004Hans-Dietrich Hermann Prize for Molecular Medicine
2006 - 2008Else Kröner Memorial Stipend
2010Travel Award and Excellence in Research Award, ASGCT
2010Young Investigator Award, ESGCT
2010Certificate of Excellence, REBIRTH Cluster of Excellence
2016CRACK-IT Award, NC3R, London (100,000 GBP)

Major research interests:

  • Gene and cell therapy for treatment of acquired and inherited diseases
  • From the identification of genetic causes of diseases to tailored molecular therapies
  • Stem cell biology and new stem cell sources
  • Reprogramming / transdifferentiation
  • (retro)virology
  • Regenerative medicine

Selected Publications:

  1. 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.
  2. Song G, Pacher M, Balakrishnan A, Yuan Q, Tsay HC, Yang D, Reetz J, Brandes S, Dai Z, Pützer BM, Araúzo-Bravo MJ, Steinemann D, Luedde T, Schwabe RF, Manns MP, Schöler 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. 18:797-808.
  3. 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. *co-corresponding author
  4. 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* (*equal contribution). 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. *co-corresponding author
  5. 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. A modified γ-retrovirus vector for X-linked severe combined immunodeficiency. N Engl J Med. 2014. 371(15):1407-17.
  6. 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. 35:4335-56.
  7. 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. 22:919-28.
  8. Warlich E, Kuehle J, Cantz T, Brugman MH, Maetzig T, Galla M, Filipczyk AA, Halle S, Klump H, Schöler HR, Baum C, Schroeder T, Schambach A. Lentiviral vector design and imaging approaches to visualize the early stages of cellular reprogramming. Mol Ther. 2011. 19:782-9.
  9. 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, 3053-64. * co-corresponding author
  10. 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, 7805-10. *co-corresponding author.

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