# Gene Therapy for Wiskott-Aldrich Syndrome—Long-Term Efficacy and Genotoxicity

**Type:** Papers  
**Canonical URL:** https://scholariq.org/papers/gene-therapy-for-wiskott-aldrich-syndrome-long-term-efficacy-and-genotoxicity/

## Facts

| Field | Value |
| --- | --- |
| Author Names | Christian Braun,Kaan Boztuǧ,Anna Paruzynski,Maximilian Witzel,Adrian Schwarzer,Michael Rothe,Ute Modlich,Rita Beier,Gudrun Göhring,Doris Steinemann,Raffaele Fronza,Claudia R. Ball,Reinhard Haemmerle,Sonja Naundorf,Klaus Kühlcke,Martina Rose,Chris Fraser,Liesl Mathias,Rudolf Ferrari,Miguel R. Abboud,Waleed Al–Herz,Irina Kondratenko,László Maródi,Hanno Glimm,Brigitte Schlegelberger,Axel Schambach,Michael H. Albert,Manfred Schmidt,Christof von Kalle,Christoph Klein |
| Citations | 558 |
| DOI | 10.1126/scitranslmed.3007280 |
| Fields | Biochemistry, Genetics and Molecular Biology,Medicine |
| Open Access | true |
| OA Status | green |
| OA URL | http://hdl.handle.net/2437/203130 |
| OpenAlex ID | https://openalex.org/W2130174662 |
| PMID | 24622513 |
| Type | article |
| Year | 2014 |

## Paper authors

- [Axel Schambach](https://scholariq.org/researchers/axel-schambach/)

## Paper primary topic

- [Virus-based gene therapy research](https://scholariq.org/topics/virus-based-gene-therapy-research/)

## Paper topics

- [Virus-based gene therapy research](https://scholariq.org/topics/virus-based-gene-therapy-research/)
- [CAR-T cell therapy research](https://scholariq.org/topics/car-t-cell-therapy-research/)
- [Viral Infectious Diseases and Gene Expression in Insects](https://scholariq.org/topics/viral-infectious-diseases-and-gene-expression-in-insects/)

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Source: ScholarIQ — public research metadata, principally OpenAlex. See https://scholariq.org/sources/ for provenance and https://scholariq.org/methodology/ for what these figures mean.