Ontology highlight
ABSTRACT:
SUBMITTER: Marangoni F
PROVIDER: S-EPMC2835187 | biostudies-other | 2009 Jun
REPOSITORIES: biostudies-other
Marangoni Francesco F Bosticardo Marita M Charrier Sabine S Draghici Elena E Locci Michela M Scaramuzza Samantha S Panaroni Cristina C Ponzoni Maurilio M Sanvito Francesca F Doglioni Claudio C Liabeuf Marie M Gjata Bernard B Montus Marie M Siminovitch Katherine K Aiuti Alessandro A Naldini Luigi L Dupré Loïc L Roncarolo Maria Grazia MG Galy Anne A Villa Anna A
Molecular therapy : the journal of the American Society of Gene Therapy 20090303 6
Wiskott-Aldrich Syndrome (WAS) is a life-threatening X-linked disease characterized by immunodeficiency, thrombocytopenia, autoimmunity, and malignancies. Gene therapy could represent a therapeutic option for patients lacking a suitable bone marrow (BM) donor. In this study, we analyzed the long-term outcome of WAS gene therapy mediated by a clinically compatible lentiviral vector (LV) in a large cohort of was(null) mice. We demonstrated stable and full donor engraftment and Wiskott-Aldrich Synd ...[more]