Comparative Efficacy and Safety of Multiple Routes of Direct CNS Administration of Adeno-Associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human Arylsulfatase A cDNA to Nonhuman Primates

Metachromatic leukodystrophy (MLD), a fatal disorder caused by deficiency of the lysosomal enzyme arylsulfatase A (ARSA), is associated with an accumulation of sulfatides, causing widespread demyelination in both central and peripheral nervous systems. On the basis of prior studies demonstrating tha...

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Vydáno v:Hum Gene Ther Clin Dev
Hlavní autoři: Rosenberg, Jonathan B., Sondhi, Dolan, Rubin, David G., Monette, Sébastien, Chen, Alvin, Cram, Sara, De, Bishnu P., Kaminsky, Stephen M., Sevin, Caroline, Aubourg, Patrick, Crystal, Ronald G.
Médium: Artigo
Jazyk:Inglês
Vydáno: Mary Ann Liebert, Inc. 2014
Témata:
Research Articles
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4227442/
https://ncbi.nlm.nih.gov/pubmed/25144894
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/humc.2013.239
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