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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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2016
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5128736/
https://www.ncbi.nlm.nih.gov/pubmed/27025653
http://dx.doi.org/10.15252/emmm.201505850

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