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

Authors :: 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
Serio, Clelia Di
Roncarolo, Maria Grazia
Mavilio, Fulvio
Bordignon, Claudio
Source :: Journal of Clinical Investigation. August, 2007, Vol. 117 Issue 8, p2233, 8 p.
Publication Year :: 2007
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 [...]

Subjects :: Genetic transformation -- Research
Adenosine deaminase deficiency -- Research
Adenosine deaminase deficiency -- Care and treatment
Adenosine deaminase deficiency -- Genetic aspects
T cells -- Research

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