Gene therapy study for the treatment of Wiskott-Aldrich syndrome
On gene therapy of Wiskott-Aldrich syndrome (WAS):
Dewey, R. A., Avedillo Díez, I., Ballmaier, M., Filipovich, A., Greil, J., Güngör, T., Happel, C., Maschan, A., Noyan, F., Pannicke, U., Schwarz, K., Snapper, S., Welte, K., & Klein, C. (2006). Retroviral WASP gene transfer into human hematopoietic stem cells reconstitutes the actin cytoskeleton in myeloid progeny cells differentiated in vitro. Experimental hematology, 34(9), 1161–1169. https://doi.org/10.1016/j.exphem.2006.04.021
A comprehensive presentation of the WAS gene therapy study from 2004-2014. Online Version (German)
In April 2016, an article in the supplement of the Süddeutsche Zeitung triggered a new discussion about the study. The serious criticism voiced in the article prompted both Ludwig-Maximilians-Universität München and the Hannover Medical School to set up commissions to review the case. The results of the Munich commission can be viewed on the university's website. A response to the article in the Süddeutsche Zeitung is also provided by the article by Ulrich Schnabel and Jan Schweitzer, which was published a little later in the newspaper Die Zeit.
Boie, J. (2016, April 22). Arzt ohne Grenzen. Süddeutsche Zeitung Magazin. https://sz-magazin.sueddeutsche.de/gesundheit/wiskott-aldrich-syndrom-studie-82660
Schnabel, U., & Schweitzer, J. (2016, May 25). Hat dieser Arzt …alles versucht? ...zu viel gewollt? Die Zeit. https://www.zeit.de/2016/23/krebs-gentherapie-kinderonkologe-christoph-klein-vorwuerfe
A Final Report of the Commission on Self-Regulation in Science of the Medical Faculty of LMU Munich on the gene therapy study in Wiskott-Aldrich syndrome (published on July 18, 2017). Online Version (German)