Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy

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; Grazia Roncarolo, Maria; Mavilio, Fulvio; Bordignon, Claudio

doi:10.1172/jci31666

Public

Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy

Published in Journal of Clinical Investigation • Aug 1, 2007

Authors:

Alessandro Aiuti

Barbara Cassani

Grazia Andolfi

Abstract

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 transpl...

Subject

Biology

Genetic enhancement

Insertional mutagenesis

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