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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Xehetasun bibliografikoak
Egile Nagusiak: 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.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: Mary Ann Liebert, Inc. 2012
Gaiak:
Research Articles
Sarrera elektronikoa: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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