Manfred Schmidt, PhD
Phone: +49 6221 421600
Fax: +49 6221 421610




However beautiful the strategy,...


you should occasionally look at the results. (Winston Churchill)

Section Molecular and Gene Therapy


Our group/department has:

  • Invented linear amplification-mediated PCR (LAM-PCR), the global gold standard for comprehensive clonality, vector biosafety and integration site analyses after gene therapy (i.e. Schmidt et al. Nature Medicine 2003; Hacein-Bey-Abina Science and New England Journal of Medicine 2003; Gaspar et al. Lancet 2004; Hajitou et al. Cell 2006; Woods et al. Nature 2006; Ott et al. Nature Medicine 2006; Yanez et al. Nature Biotechnology 2006; Schmidt et al. Nature Methods 2007; Cartier et al. Science 2009; Stein et al. Nature Medicine 2010; Boztug et al. New England Journal of Medicine 2010; Aiuti et al. and Biffi et al. Science 2013).

  • Implemented a semi-automated (insertional) mutagenesis platform based on next generation sequencing for the dissection of molecular mechanisms leading to clonal imbalance and malignant transformation (Montini et al. Nature Biotechnology 2006; Gabriel et al.  Nature Medicine 2009; Paruzynski et al. Nature Protocols 2010; Ranzani et al. Nature Methods 2012; Dieter et al. Cell Stem Cell 2012).

  • Established quality assured analyte preparation for cancer diagnostics and biomarker discovery (Jones et al. Nature 2012; Weischenfeldt et al. Cancer Cell 2012). 

  • Establishment of a feeforservice laboratory for molecular analyses of vector biosafety and insertional mutagenesis.

2010 – 2013

  • Kaeppel, C., Beattie, S.G., Fronza, R., van Logtenstein, R., Salmon, F., Schmidt, S., Wolf, S., Nowrouzi, A., Glimm, H., von Kalle, C., Petry, H., Gaudet, D., Schmidt, M.: A largely random AAV integration profile after LPLD gene therapy. Nature Medicine. 19, 889-891, 2013.

  • Arens, A., Appelt, J.U., Bartholomae, C.C., Gabriel, R., Paruzynski, A., Gustafson, D., Cartier, N., Aubourg, P., Deichmann, A., Glimm, H., von Kalle, C., Schmidt, M.: Bioinformatic clonality analysis of next-generation sequencing-derived viral vector integration sites. Human Gene Therapy Methods. 23, 111-118, 2012.

  • Nowrouzi, A., Penaud-Budloo, M., Kaeppel, C., Appelt, J.U., Le Guiner, C., Moullier, P., von Kalle, C., Snyder R.O., Schmidt, M.: Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver. Molecular Therapy. 20, 1177-1786, 2012.

  • Gabriel, R., Lombardo, A., Arens, A., Miller, J.C., Genovese, P., Kaeppel, C., Nowrouzi, A., Bartholomae, C.C., Wang, J., Friedman, G., Holmes, M.C., Gregory, P.D., Glimm, H., Schmidt*, M. (*co-senior author), Naldini, L.*, von Kalle, C*.: An unbiased genome-wide analysis of zinc-finger nuclease specificity. Nature Biotechnology. 29, 816-823, 2011.

  • Bartholomae, C.C., Arens, A., Balaggan, K.S., Yáñez-Muñoz, R.J., Montini, E., Howe, S.J., Paruzynski, A., Korn, B., Appelt, U., MacNeil, A., Cesana, D., Abel, U., Glimm, H., Naldini, L., Ali, R.R., Thrasher, A.J., von Kalle, Schmidt, M.: Lentiviral Vector Integration Profiles Differ in Rodent Postmitotic Tissues. Molecular Therapy. 19, 703-7010, 2011.

  • The group is funded by grants from the DFG, BMBF and EU.

  • Since 2002 >50 group related awards: Travel Awards, Best Abstract Awards, Excellence of Research Awards and Young Investigator Awards (i.e. ASGCT, ESGCT)

  • 2005 Langen Research Award, Paul-Ehrlich-Institute, Langen, Germany