Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID
- 18 February 2009
- journal article
- Published by Springer Nature in Immunologic Research
- Vol. 44 (1), 150-159
- https://doi.org/10.1007/s12026-009-8107-8
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.Keywords
This publication has 47 references indexed in Scilit:
- Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patientsJournal of Clinical Investigation, 2008
- Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1Journal of Clinical Investigation, 2008
- Altered intracellular and extracellular signaling leads to impaired T-cell functions in ADA-SCID patientsBlood, 2008
- Development of lentiviral gene therapy for Wiskott Aldrich syndromeExpert Opinion on Biological Therapy, 2008
- Progress and Prospects: Gene Therapy Clinical Trials (Part 2)Gene Therapy, 2007
- Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapyJournal of Clinical Investigation, 2007
- 1088. Stem Cell Gene Therapy with No Pre-Conditioning for the ADA-Deficiency Patients Leads to Generalized Detoxification and Delayed, but Steady Hematological ReconstitutionMolecular Therapy, 2006
- Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case reportBlood, 2006
- Unique risk factors for insertional mutagenesis in a mouse model of XSCID gene therapyProceedings of the National Academy of Sciences, 2006
- Gene Therapy Insertional Mutagenesis InsightsScience, 2004