Gene therapy study for the treatment of Wiskott-Aldrich syndrome

On gene therapy of Wiskott-Aldrich syndrome (WAS):

Dewey, Ricardo A. / Avedillo Diez, Ines / Ballmaier, Matthias / Filipovich, Alexandra / Greil, Johann / Güngör, Tayfun / Happel, Christoph / Maschan, Alexey / Noyan, Fatih / Pannicke, Ulrich / Schwarz, Klaus / Snapper, Scott / Welte, Karl / Klein, Christoph (2006): Retroviral WASP gene transfer into human hematopoietic stem cells reconstitutes the actin cystoskeleton in myeloid progeny cells differentiated in vitro. In: Experimental Hematology 34 (9), 1161–1169. doi: 10.1016/j.exphem.2006.04.021 Online Version

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, Johannes (2016): Arzt ohne Grenzen. In: Süddeutsche Zeitung Magazin (22. April 2016). Online Version (German)

Schnabel, Ulrich / Schweitzer, Jan (2016): Hat dieser Arzt …alles versucht? ...zu viel gewollt?. In: Die Zeit (2. Juni 2016). Online Version (German)

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)

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