Somatic Gene Therapy

We develop new treatment methods for currently incurable disorders. We use and develop techniques for viral and non-viral gene transfer (vectors), which allow to efficiently introduce nucleic acids (DNA, RNA) into body cells. Vector-mediated gene transfer should either help to treat specific diseases (somatic gene therapy) or to prevent them as in the case of infectious diseases (genetic vaccination).

In recent years gene therapy has been very successful in clinical studies not only for several genetic but also for malignant diseases. It can be assumed that these new methods will become very important for the treatment of numerous diseases in the future.

Our current research projects include:

  • Oncolytic viruses for tumor therapy
  • Studying the interaction of viral vectors with cellular and extracellular barriers
  • Production of viral vectors: Adenovirus, Adeno-Associated Virus (AAV)
  • Genetic vaccination


  1. Nilson, R., Lübbers, O., Weiß, L., Singh, K., Scharffetter-Kochanek, K., Rojewski, M., Schrezenmeier, H., Zeplin, P.H., Funk, W., Krutzke, L., Kochanek, S., Kritzinger, A., 2021. Transduction Enhancers Enable Efficient Human Adenovirus Type 5-Mediated Gene Transfer into Human Multipotent Mesenchymal Stromal Cells. Viruses 13, 1136.
  2. Seefelder, M., Kochanek, S., 2021. A meta-analysis of transcriptomic profiles of Huntington’s disease patients. PloS One 16, e0253037.

  3. Huang, B., Guo, Q., Niedermeier, M.L., Cheng, J., Engler, T., Maurer, M., Pautsch, A., Baumeister, W., Stengel, F., Kochanek, S., Fernández-Busnadiego, R., 2021. Pathological polyQ expansion does not alter the conformation of the Huntingtin-HAP40 complex. Struct. Lond. Engl. 1993 S0969-2126(21)00119–2.

  4. Krutzke, L., Allmendinger, E., Hirt, K., Kochanek, S., 2020. Chorioallantoic Membrane Tumor Model for Evaluating Oncolytic Viruses. Hum. Gene Ther. 31, 1100–1113.

  5. Seefelder, M., Alva, V., Huang, B., Engler, T., Baumeister, W., Guo, Q., Fernández-Busnadiego, R., Lupas, A.N., Kochanek, S., 2020. The evolution of the huntingtin-associated protein 40 (HAP40) in conjunction with huntingtin. BMC Evol. Biol. 20, 162.

  6. Krüger-Haag A, Lehmann C, Schmidt E, Sonntag F, Hörer M, Kochanek S. 2019. Evaluation of life cycle defective adenovirus mutants for production of adeno-associated virus vectors. The Journal of Gene Medicine, DOI: 10.1002/jgm.3094
  7. Kritzinger A, Ferger B, Gillardon F, Stierstorfer B, Birk G, Kochanek S, Ciossek T. 2018. Age-related pathology after adenoviral overexpression of the leucine-rich repeat kinase 2 in the mouse striatum. Neurobiology of Aging, DOI: 10.1016/j.neurobiolaging.2018.02.008
  8. Kratzer RF, Espenlaub S, Hoffmeister A, Kron MW, Kreppel F. 2017. Covalent decoration of adenovirus vector capsids with the carbohydrate epitope alphaGal does not improve vector immunogenicity, but allows to study the in vivo fate of adenovirus immunocomplexes. PLoS One 12:e0176852.
  9. Krutzke L, Prill JM, Engler T, Schmidt CQ, Xu Z, Byrnes AP, Simmet T, Kreppel F. 2016. Substitution of blood coagulation factor X-binding to Ad5 by position-specific PEGylation: Preventing vector clearance and preserving infectivity. J Control Release 235:379-392
  10. Emmerling VV, Pegel A, Milian EG, Venereo-Sanchez A, Kunz M, Wegele J, Kamen AA, Kochanek S, Hoerer M. 2016. Rational plasmid design and bioprocess optimization to enhance recombinant adeno-associated virus (AAV) productivity in mammalian cells. Biotechnol J 11:290-297
  11. Emmerling VV, Fischer S, Stiefel F, Holzmann K, Handrick R, Hesse F, Horer M, Kochanek S, Otte K. 2016. Temperature-sensitive miR-483 is a conserved regulator of recombinant protein and viral vector production in mammalian cells. Biotechnol Bioeng 113:830-841
  12. Emmerling VV, Fischer S, Kleemann M, Handrick R, Kochanek S, Otte K. 2016. miR-483 is a self-regulating microRNA and can activate its own expression via USF1 in HeLa cells. Int J Biochem Cell Biol 80:81-86.
  13. Silva AC, Simao D, Kuppers C, Lucas T, Sousa MF, Cruz P, Carrondo MJ, Kochanek S, Alves PM. 2015. Human amniocyte-derived cells are a promising cell host for adenoviral vector production under serum-free conditions. Biotechnol J 10:760-771.
  14. Lucas T, Benihoud K, Vigant F, Schmidt CQ, Bachem MG, Simmet T, Kochanek S. 2015. Hexon modification to improve the activity of oncolytic adenovirus vectors against neoplastic and stromal cells in pancreatic cancer. PLoS One 10:e0117254
  15. Bernhard CA, Ried C, Kochanek S, Brocker T. 2015. CD169+ macrophages are sufficient for priming of CTLs with specificities left out by cross-priming dendritic cells. Proc Natl Acad Sci U S A 112:5461-5466.
  16. Genzel Y, Behrendt I, Rodig J, Rapp E, Kueppers C, Kochanek S, Schiedner G, Reichl U. 2013. CAP, a new human suspension cell line for influenza virus production. Appl Microbiol Biotechnol 97:111-122.
  17. Laakkonen JP, Engler T, Romero IA, Weksler B, Couraud PO, Kreppel F, Kochanek S. 2012. Transcellular targeting of fiber- and hexon-modified adenovirus vectors across the brain microvascular endothelial cells in vitro. PLoS One 7:e45977.
  18. Zong S, Kron MW, Epp C, Engler T, Bujard H, Kochanek S, Kreppel F. 2011. DeltaE1 and high-capacity adenoviral vectors expressing full-length codon-optimized merozoite surface protein 1 for vaccination against Plasmodium falciparum. J Gene Med 13:670-679.
  19. Prill JM, Espenlaub S, Samen U, Engler T, Schmidt E, Vetrini F, Rosewell A, Grove N, Palmer D, Ng P, Kochanek S, Kreppel F. 2011. Modifications of adenovirus hexon allow for either hepatocyte detargeting or targeting with potential evasion from Kupffer cells. Mol Ther 19:83-92.
  20. Kron MW, Espenlaub S, Engler T, Schirmbeck R, Kochanek S, Kreppel F. 2011. miRNA-mediated silencing in hepatocytes can increase adaptive immune responses to adenovirus vector-delivered transgenic antigens. Mol Ther 19:1547-1557.
  21. Stephen SL, Montini E, Sivanandam VG, Al-Dhalimy M, Kestler HA, Finegold M, Grompe M, Kochanek S. 2010. Chromosomal integration of adenoviral vector DNA in vivo. J Virol 84:9987-9994.
  22. Kreppel F, Gackowski J, Schmidt E, Kochanek S. 2005. Combined genetic and chemical capsid modifications enable flexible and efficient de- and retargeting of adenovirus vectors. Mol Ther 12:107-117.
  23. Chuah MK, Schiedner G, Thorrez L, Brown B, Johnston M, Gillijns V, Hertel S, Van Rooijen N, Lillicrap D, Collen D, VandenDriessche T, Kochanek S. 2003. Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors. Blood 101:1734-1743.