Systemic Gene Transfer of a Hexosaminidase Variant Using an scAAV9.47 Vector Corrects GM2 Gangliosidosis in Sandhoff Mice.
| Autor: | Osmon, Karlaina J. L., Woodley, Evan, Thompson, Patrick, Ong, Katalina, Karumuthil-Melethil, Subha, Keimel, John G., Mark, Brian L., Mahuran, Don, Gray, Steven J., Walia, Jagdeep S. |
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| Zdroj: | Human Gene Therapy; Jul2016, Vol. 27 Issue 7, p497-508, 12p |
| Databáze: | Complementary Index |
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