Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with...
Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy
About this item
Full title
Author / Creator
Aiuti, Alessandro , Cassani, Barbara , Andolfi, Grazia , Mirolo, Massimiliano , Biasco, Luca , Recchia, Alessandra , Urbinati, Fabrizia , Valacca, Cristina , Scaramuzza, Samantha , Aker, Memet , Slavin, Shimon , Cazzola, Matteo , Sartori, Daniela , Ambrosi, Alessandro , Di Serio, Clelia , Roncarolo, Maria Grazia , Mavilio, Fulvio and Bordignon, Claudio
Publisher
United States: American Society for Clinical Investigation
Journal title
Language
English
Formats
Publication information
Publisher
United States: American Society for Clinical Investigation
Subjects
More information
Scope and Contents
Contents
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-...
Alternative Titles
Full title
Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy
Authors, Artists and Contributors
Author / Creator
Cassani, Barbara
Andolfi, Grazia
Mirolo, Massimiliano
Biasco, Luca
Recchia, Alessandra
Urbinati, Fabrizia
Valacca, Cristina
Scaramuzza, Samantha
Aker, Memet
Slavin, Shimon
Cazzola, Matteo
Sartori, Daniela
Ambrosi, Alessandro
Di Serio, Clelia
Roncarolo, Maria Grazia
Mavilio, Fulvio
Bordignon, Claudio
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_1934603
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_1934603
Other Identifiers
ISSN
0021-9738
E-ISSN
1558-8238
DOI
10.1172/JCI31666
