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

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

Abstract 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 gen...

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Main Authors: Vasco Meneghini, Annalisa Lattanzi, Luigi Tiradani, Gabriele Bravo, Francesco Morena, Francesca Sanvito, Andrea Calabria, John Bringas, Jeanne M Fisher‐Perkins, Jason P Dufour, Kate C Baker, Claudio Doglioni, Eugenio Montini, Bruce A Bunnell, Krystof Bankiewicz, Sabata Martino, Luigi Naldini, Angela Gritti
Format: Article
Language:English
Published: Springer Nature 2016-03-01
Series:EMBO Molecular Medicine
Subjects:
brain
gene therapy
lentiviral vectors
leukodystrophy
non‐human primates
Online Access:https://doi.org/10.15252/emmm.201505850
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