Publication: Successful combination of sequential gene therapy and rescue Allo-HSCT in two children with X-CGD - importance of timing
Date
Date
Date
2015
Journal Article
Published version
Abstract
Abstract
Abstract
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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Views
101 since deposited on 2015-12-23
100last week
Acq. date: 2025-11-12
Additional indexing
Creators (Authors)
- 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
Volume
Volume
Volume
15
Number
Number
Number
4
Page range/Item number
Page range/Item number
Page range/Item number
416
Page end
Page end
Page end
427
Item Type
Item Type
Item Type
Journal Article
In collections
Dewey Decimal Classifikation
Dewey Decimal Classifikation
Dewey Decimal Classifikation
Keywords
Allo-HSCT, CGD, EVI1, Gene therapy, Primary immunodeficiency, Retroviral vector, STAT3, Transactivation
Language
Language
Language
English
Publication date
Publication date
Publication date
2015
Date available
Date available
Date available
2015-12-23
ISSN or e-ISSN
ISSN or e-ISSN
ISSN or e-ISSN
1566-5232
OA Status
OA Status
OA Status
Closed
Publisher DOI
Metrics
Views
101 since deposited on 2015-12-23
100last week
Acq. date: 2025-11-12
Closed
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