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- Title
Current progress on gene therapy for primary immunodeficiencies.
- Authors
Zhang, L; Thrasher, A J; Gaspar, H B
- Abstract
Primary immunodeficiencies have played a major role in the development of gene therapy for monogenic diseases of the bone marrow. The last decade has seen convincing evidence of long-term disease correction as a result of ex vivo viral vector-mediated gene transfer into autologous haematopoietic stem cells. The success of these early studies has been balanced by the development of vector-related insertional mutagenic events. More recently the use of alternative vector designs with self-inactivating designs, which have an improved safety profile has led to the initiation of a wave of new studies that are showing early signs of efficacy. The ongoing development of safer vector platforms and gene-correction technologies together with improvements in cell-transduction techniques and optimised conditioning regimes is likely to make gene therapy amenable for a greater number of PIDs. If long-term efficacy and safety are shown, gene therapy will become a standard treatment option for specific forms of PID.
- Subjects
IMMUNODEFICIENCY; BONE marrow diseases; GENETIC transformation; GENETIC vectors; STEM cells; GENETIC disorders; GENETIC mutation; GENE therapy
- Publication
Gene Therapy, 2013, Vol 20, Issue 10, p963
- ISSN
0969-7128
- Publication type
Article
- DOI
10.1038/gt.2013.21