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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Enregistré dans:
Détails bibliographiques
Publié dans:EMBO Mol Med
Auteurs principaux: 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
Format: Artigo
Langue:Inglês
Publié: John Wiley and Sons Inc. 2016
Sujets:
Research Articles
Accès en ligne: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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