Stable correction of a genetic deficiency in human cells by an episome carrying a 115 kb genomic transgene.
Persistent expression of a transgene at therapeutic levels is required for successful gene therapy, but many small vectors with heterologous promoters are prone to vector loss and transcriptional silencing. The delivery of genomic DNA would enable genes to be transferred as complete loci, including...
| Główni autorzy: | , , , , |
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| Format: | Journal article |
| Język: | English |
| Wydane: |
2000
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