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Published in:

01-07-2009

Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID

Authors: Alessandro Aiuti, Immacolata Brigida, Francesca Ferrua, Barbara Cappelli, Robert Chiesa, Sarah Marktel, Maria-Grazia Roncarolo

Published in: Immunologic Research | Issue 1-3/2009

Abstract

Gene therapy is a highly attractive strategy for many types of inherited disorders of the immune system. Adenosine deaminase (ADA) deficient-severe combined immunodeficiency (SCID) has been the target of several clinical trials based on the use of hematopoietic stem/progenitor cells engineered with retroviral vectors. The introduction of a low intensity conditioning regimen has been a crucial factor in achieving stable engrafment of hematopoietic stem cells and therapeutic levels of ADA-expressing cells. Recent studies have demonstrated that gene therapy for ADA-SCID has favorable safety profile and is effective in restoring normal purine metabolism and immune functions. Stem cell gene therapy combined with appropriate conditioning regimens might be extended to other genetic disorders of the hematopoietic system.

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Title: Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID
Authors: Alessandro Aiuti
Immacolata Brigida
Francesca Ferrua
Barbara Cappelli
Robert Chiesa
Sarah Marktel
Maria-Grazia Roncarolo
Publication date: 01-07-2009
Publisher: Humana Press Inc
Published in: Immunologic Research / Issue 1-3/2009
Print ISSN: 0257-277X
Electronic ISSN: 1559-0755
DOI: https://doi.org/10.1007/s12026-009-8107-8

At a glance: The STEP trials

Springer Medicine

Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID

Abstract

At a glance: The STEP trials

Springer Medicine

Abstract

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