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- W1595590526 abstract "Hematopoietic stem cells (HSCs) present arguably the best entry point for gene therapy of hematopoietic and immune systems since genetically modified HSCs are long-lived and would eventually transfer the therapeutic constructs to all their descendants. However, gene therapy via HSCs, although conceptually simple, has proven to be a technically formidable problem that has yet to be solved successfully. Despite overtly positive results obtained in gene therapy experiments performed with mouse and larger animal models, these achievements did not translate into clinically acceptable outcomes for non-human primates and human patients, with exception of a few specific disease instances where a therapeutic gene brought about significant survival advantages to transduced cells (Cavazzana-Calvo et al., 2000, Schmidt et al, 2003). Major differences between outcomes of conceptually similar experiments in mice and primates underscore the notion that the fundamental principles governing functioning of hematopoietic system in small short-lived vs. larger long-lived animals differ significantly. Low degree of chimerism obtained in experiments with primates and humans is likely a result of intrinsically low efficiency of viral transduction of long-term repopulating (LTR) HSCs coupled with subsequent massive silencing of integrated constructs (Ellis, 2005; Horn et al, 2002). One may hypothesize that this situation reflects a better protection of hematopoietic system from external influences, in particular invasion of foreign genetic material, in longer-living animals." @default.
- W1595590526 created "2016-06-24" @default.
- W1595590526 creator A5003524826 @default.
- W1595590526 creator A5028599195 @default.
- W1595590526 creator A5032201823 @default.
- W1595590526 date "2012-01-27" @default.
- W1595590526 modified "2023-10-16" @default.
- W1595590526 title "Gene Therapy of Hematopoietic and Immune Systems: Current State and Perspectives" @default.
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