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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Detalhes bibliográficos
Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2012
Assuntos:
Research Articles
Acesso em linha: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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