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Update on gene therapy for adenosine deaminase-deficient severe combined immunodeficiency.
- Source :
-
Current opinion in allergy and clinical immunology [Curr Opin Allergy Clin Immunol] 2010 Dec; Vol. 10 (6), pp. 551-6. - Publication Year :
- 2010
-
Abstract
- Purpose of Review: The present review describes the recent progress in the treatment of adenosine deaminase-deficient severe combined immunodeficiency (ADA-SCID) using autologous gene-modified hematopoietic stem cells, comparing immune reconstitution with respect to allogeneic transplant and discussing differences with gene therapy for SCID-X1.<br />Recent Findings: Since 2000, more than 30 ADA-SCID patients have been treated with gene therapy worldwide, with successful outcome in most cases, consisting of progressive immune reconstitution, efficient systemic detoxification, and long-term multilineage engraftment. Gene therapy resulted in restoration of thymic activity and T-cell functions, although the kinetic of reconstitution was slower compared with standard bone marrow transplant. Unlike allogeneic transplant from alternative donors, survival after gene therapy is excellent. In comparison with SCID-X1, ADA-SCID gene therapy presents a better safety profile and engraftment of multilineage transduced stem/progenitor cells, thanks to the use of nonmyeloablative preconditioning. New approaches using safer integrating vectors are being developed, which may lead to safer and effective gene therapy for ADA-SCID and other genetic disorders.<br />Summary: In the last decade, gene therapy has been developed as a successful and safe alternative strategy for patients affected by ADA-SCID lacking a compatible sibling donor. The application of innovative vector technology might further improve its efficacy and safety profile.
- Subjects :
- Adenosine Deaminase deficiency
Adenosine Deaminase genetics
Adenosine Deaminase immunology
Agammaglobulinemia genetics
Agammaglobulinemia immunology
Agammaglobulinemia therapy
Animals
Cell Lineage genetics
Cell Survival genetics
Genetic Vectors genetics
Humans
Severe Combined Immunodeficiency genetics
Severe Combined Immunodeficiency immunology
Severe Combined Immunodeficiency therapy
T-Lymphocytes immunology
T-Lymphocytes metabolism
Thymus Gland growth & development
Thymus Gland immunology
Thymus Gland metabolism
Transplantation Conditioning
Adenosine Deaminase metabolism
Genetic Therapy trends
Hematopoietic Stem Cell Transplantation
Subjects
Details
- Language :
- English
- ISSN :
- 1473-6322
- Volume :
- 10
- Issue :
- 6
- Database :
- MEDLINE
- Journal :
- Current opinion in allergy and clinical immunology
- Publication Type :
- Academic Journal
- Accession number :
- 20966749
- Full Text :
- https://doi.org/10.1097/ACI.0b013e32833fea85