Artificial Chromosomes
Recent studies by R.L. Katona and co-authors add new data to artificial chromosomes findings
Published in Gene Therapy Weekly, February 5th, 2009
"Mammalian artificial chromosomes (MACs) are safe, stable, non-integrating genetic vectors with almost unlimited therapeutic transgene-carrying capacity. The combination of MAC and stem cell technologies offers a new strategy for stem cell-based therapy, the efficacy of which was confirmed and validated by using a mouse model of a devastating monogenic disease, galactocerebrosidase deficiency (Krabbe's disease)," scientists in Szeged, Hungary report.
"Therapeutic MACs were generated by sequence-specific loading of galactocerebrosidase transgenes into a platform MAC, and stable, pluripotent mouse embryonic stem cell lines were established with these chromosomes. The...
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Source: Gene Therapy Weekly (2009-02-05)
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