Long-Term Expression and Safety of Administration of AAVrh.10hCLN2 to the Brain of Rats and Nonhuman Primates for the Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis

Late infantile neuronal ceroid lipofuscinosis (LINCL), a fatal, lysosomal storage disorder caused by mutations in the CLN2 gene, results in a deficiency of tripeptidyl-peptidase I (TPP-I) activity in neurons. Our prior studies showed that delivery of the human CLN2 cDNA directly to the CNS, using an...

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Hlavní autoři: Sondhi, Dolan, Johnson, Linda, Purpura, Keith, Monette, Sebastien, Souweidane, Mark M., Kaplitt, Michael G., Kosofsky, Barry, Yohay, Kaleb, Ballon, Douglas, Dyke, Jonathan, Kaminksy, Stephen M., Hackett, Neil R., Crystal, Ronald G.
Médium: Artigo
Jazyk:Inglês
Vydáno: Mary Ann Liebert, Inc. 2012
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Research Articles
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3847998/
https://ncbi.nlm.nih.gov/pubmed/23131032
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hgtb.2012.120
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