Journal
MOLECULAR THERAPY
Volume 13, Issue 3, Pages 528-537Publisher
CELL PRESS
DOI: 10.1016/j.ymthe.2005.11.015
Keywords
vector transport; capsid; serotype; MPS VII; lysosomal storage disease; beta-glucuronidase
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Funding
- NIDDK NIH HHS [DK 63973, DK 46637, DK47757, R01 DK063973] Funding Source: Medline
- NINDS NIH HHS [R01 NS038690, NS 38690] Funding Source: Medline
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Recombinant adeno-associated viral (AAV) vectors can transduce cells of the CNS, resulting in longterm expression. AAV vector transduction varies depending on the serotype used and the region of the brain injected. AAV serotypes 7, 8, 9, and RHO have recently become available, but the transduction capabilities of these serotypes within the CNS have not been determined. We show that AAV 7, 8, 9, and Rh 10 vectors expressing cDNA for a lysosomal enzyme transcluce neurons, but not astrocytes or oligodendrocytes, in the cortex, striatum, hippocampus, and thalamus. Although all of the vectors contained the same genome, there were markedly different transduction patterns that could be due only to the differences in capsid proteins. The AAV 9 vector was found to undergo vector genome transport to distal neuronal cell bodies via known axonal pathways. This facilitated the distribution of enzyme, resulting in correction of lysosomal storage lesions in regions of a diseased brain that would not be corrected if the genome were not transported.
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