Publication: Successful combination of sequential gene therapy and rescue Allo-HSCT in two children with X-CGD - importance of timing
Successful combination of sequential gene therapy and rescue Allo-HSCT in two children with X-CGD - importance of timing
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Siler, U., Paruzynski, A., Holtgreve-Grez, H., Kuzmenko, E., Koehl, U., Renner, E. D., Alhan, C., van de Loosdrecht, A. A., Schwäble, J., Pfluger, T., Tchinda, J., Schmugge, M., Jauch, A., Naundorf, S., Kühlcke, K., Notheis, G., Güngör, T., Kalle, C. V., Schmidt, M., … Reichenbach, J. (2015). Successful combination of sequential gene therapy and rescue Allo-HSCT in two children with X-CGD - importance of timing. Current Gene Therapy, 15(4), 416–427. https://doi.org/10.2174/1566523215666150515145255
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We report on a series of sequential events leading to long-term survival and cure of pediatric X-linked chronic granulomatous disease (X-CGD) patients after gamma-retroviral gene therapy (GT) and rescue HSCT. Due to therapyrefractory life-threatening infections requiring hematopoietic stem cell transplantation (HSCT) but absence of HLAidentical donors, we treated 2 boys with X-CGD by GT. Following GT both children completely resolved invasive Aspergillus nidulans infections. However, one child developed dual insertional activation of
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- Siler, Ulrich
- Paruzynski, Anna
- Holtgreve-Grez, Heidi
- Kuzmenko, Elena
- Koehl, Ulrike
- Renner, Eleonore D
- Alhan, Canan
- van de Loosdrecht, Arjan A
- Schwäble, Joachim
- Pfluger, Thomas
- Tchinda, Joelle
- Schmugge, Markus
- Jauch, Anna
- Naundorf, Sonja
- Kühlcke, Klaus
- Notheis, Gundula
- Güngör, Tayfun
- Kalle, Christof V
- Schmidt, Manfred
- Grez, Manuel
- Seger, Reinhard
- Reichenbach, Janine
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Siler, U., Paruzynski, A., Holtgreve-Grez, H., Kuzmenko, E., Koehl, U., Renner, E. D., Alhan, C., van de Loosdrecht, A. A., Schwäble, J., Pfluger, T., Tchinda, J., Schmugge, M., Jauch, A., Naundorf, S., Kühlcke, K., Notheis, G., Güngör, T., Kalle, C. V., Schmidt, M., … Reichenbach, J. (2015). Successful combination of sequential gene therapy and rescue Allo-HSCT in two children with X-CGD - importance of timing. Current Gene Therapy, 15(4), 416–427. https://doi.org/10.2174/1566523215666150515145255