Intrathecal administration of AAV/GALC vectors in 10–11-day-old twitcher mice improves survival and is enhanced by bone marrow transplant

Subha Karumuthil-Melethil, Michael S. Marshall, Clifford Heindel, Benas Jakubauskas, Ernesto R. Bongarzone, Steven J. Gray

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34 Scopus citations

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Medicine & Life Sciences