Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe‐affected non‐human primates by intracerebral lentiviral gene therapy

Metachromatic leukodystrophy (MLD) and globoid cell leukodystrophy (GLD or Krabbe disease) are severe neurodegenerative lysosomal storage diseases (LSD) caused by arylsulfatase A (ARSA) and galactosylceramidase (GALC) deficiency, respectively. Our previous studies established lentiviral gene therapy...

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Vydáno v:EMBO Mol Med
Hlavní autoři: Meneghini, Vasco, Lattanzi, Annalisa, Tiradani, Luigi, Bravo, Gabriele, Morena, Francesco, Sanvito, Francesca, Calabria, Andrea, Bringas, John, Fisher‐Perkins, Jeanne M, Dufour, Jason P, Baker, Kate C, Doglioni, Claudio, Montini, Eugenio, Bunnell, Bruce A, Bankiewicz, Krystof, Martino, Sabata, Naldini, Luigi, Gritti, Angela
Médium: Artigo
Jazyk:Inglês
Vydáno: John Wiley and Sons Inc. 2016
Témata:
Research Articles
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5128736/
https://ncbi.nlm.nih.gov/pubmed/27025653
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.15252/emmm.201505850
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