Ontology highlight
ABSTRACT:
SUBMITTER: Chiriaco M
PROVIDER: S-EPMC4435596 | biostudies-literature | 2014 Aug
REPOSITORIES: biostudies-literature
Chiriaco Maria M Farinelli Giada G Capo Valentina V Zonari Erika E Scaramuzza Samantha S Di Matteo Gigliola G Sergi Lucia Sergi LS Migliavacca Maddalena M Hernandez Raisa Jofra RJ Bombelli Ferdinando F Giorda Ezio E Kajaste-Rudnitski Anna A Trono Didier D Grez Manuel M Rossi Paolo P Finocchi Andrea A Naldini Luigi L Gentner Bernhard B Aiuti Alessandro A
Molecular therapy : the journal of the American Society of Gene Therapy 20140529 8
Regulated transgene expression may improve the safety and efficacy of hematopoietic stem cell (HSC) gene therapy. Clinical trials for X-linked chronic granulomatous disease (X-CGD) employing gammaretroviral vectors were limited by insertional oncogenesis or lack of persistent engraftment. Our novel strategy, based on regulated lentiviral vectors (LV), targets gp91(phox) expression to the differentiated myeloid compartment while sparing HSC, to reduce the risk of genotoxicity and potential pertur ...[more]