Issue Date | Title | Author(s) | Relation | scopus | WOS | Fulltext/Archive link |
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2003 | Packaging of an AAV vector encoding human acid alpha-glucosidase for gene therapy in glycogen storage disease type II with a modified hybrid adenovirus-AAV vector | Sun, B. D.; Chen, Y. T.; Bird, A.; Xu, F.; Hou, Y. X.; Amalfitano, A.; Koeberl, D. D. | Mol Ther 7, 467-477 | |||
2003 | Rare disorders of metabolism with elevated butyryl- and isobutyryl-carnitine detected by tandem mass spectrometry newborn screening | Koeberl, D. D.; Young, S. P.; Gregersen, N. S.; Vockley, J.; Smith, W. E.; Benjamin, D. K.; An, Y.; Weavil, S. D.; Chaing, S. H.; Bali, D.; McDonald, M. T.; Kishnani, P. S.; Chen, Y. T.; Millington, D. S. | Pediatr Res 54, 219-223 |