Ontology highlight
ABSTRACT:
SUBMITTER: Aiuti A
PROVIDER: S-EPMC1934603 | biostudies-literature | 2007 Aug
REPOSITORIES: biostudies-literature
Aiuti Alessandro A Cassani Barbara B Andolfi Grazia G Mirolo Massimiliano M Biasco Luca L Recchia Alessandra A Urbinati Fabrizia F Valacca Cristina C Scaramuzza Samantha S Aker Memet M Slavin Shimon S Cazzola Matteo M Sartori Daniela D Ambrosi Alessandro A Di Serio Clelia C Roncarolo Maria Grazia MG Mavilio Fulvio F Bordignon Claudio C
The Journal of clinical investigation 20070801 8
Gene transfer into HSCs is an effective treatment for SCID, although potentially limited by the risk of insertional mutagenesis. We performed a genome-wide analysis of retroviral vector integrations in genetically corrected HSCs and their multilineage progeny before and up to 47 months after transplantation into 5 patients with adenosine deaminase-deficient SCID. Gene-dense regions, promoters, and transcriptionally active genes were preferred retroviral integrations sites (RISs) both in preinfus ...[more]