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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Hauptverfasser: 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.
Format: Artigo
Sprache:Inglês
Veröffentlicht: Mary Ann Liebert, Inc. 2012
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Research Articles
Online Zugang: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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