Stem cells immunologic system

Since the OTC trial, there has been rapid advancement in vector development with marked improvements in the safety (and efficacy) of new viral delivery systems. There is also a better understanding of the immunological responses to gene therapy vectors. The adverse events in the SCID trial are more recent. They appear to be a likely result of the choice of vector (retrovirus) and the ex vivo selection strategy used to modify the affected stem cells of the SCID patients. The risk benefit ratio for gene therapy treatment of these children is still deemed favorable in light of the fact that without treatment, they would not be alive. 

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