Lentiviral-Mediated Gene Transfer to the Sheep Brain: Implications for Gene Therapy in Batten Disease

The neuronal ceroid lipofuscinoses (NCLs; Batten disease) are inherited neurodegenerative lysosomal storage diseases with common clinical features of blindness and seizures culminating in premature death. Gene-therapy strategies for these diseases depend on whether the missing activity is a secreted...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Päätekijät: Linterman, Kathryn S., Palmer, David N., Kay, Graham W., Barry, Lucy A., Mitchell, Nadia L., McFarlane, Robin G., Black, Michael A., Sands, Mark S., Hughes, Stephanie M.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: Mary Ann Liebert, Inc. 2011
Aiheet:
Research Articles
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC3159522/
https://ncbi.nlm.nih.gov/pubmed/21595499
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2011.026
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