Therapeutic approaches for neuronopathic lysosomal storage disorders.
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Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff diseaseImaging gene delivery in a mouse model of congenital neuronal ceroid lipofuscinosis.A Dysmorphometric Analysis to Investigate Facial Phenotypic Signatures as a Foundation for Non-invasive Monitoring of Lysosomal Storage Disorders.Newborn screening for lysosomal storage disorders and other neuronopathic conditions.Strategies for delivery of therapeutics into the central nervous system for treatment of lysosomal storage disorders.Lysosomal storage disorders: molecular basis and laboratory testing.Annexins as organizers of cholesterol- and sphingomyelin-enriched membrane microdomains in Niemann-Pick type C disease.Do annexins participate in lipid messenger mediated intracellular signaling? A question revisited.Blood-brain barrier structure and function and the challenges for CNS drug delivery.Lysosomal storage causes cellular dysfunction in mucolipidosis II skin fibroblasts.Chaperones as potential therapeutics for Krabbe disease.Impaired dynamics of the late endosome/lysosome compartment in human Niemann-Pick type C skin fibroblasts carrying mutation in NPC1 gene.A Basic ApoE-Based Peptide Mediator to Deliver Proteins across the Blood-Brain Barrier: Long-Term Efficacy, Toxicity, and Mechanism.Expanding the Boundaries of Biotherapeutics with Bispecific Antibodies
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Therapeutic approaches for neuronopathic lysosomal storage disorders.
description
article científic
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article scientifique
@fr
articolo scientifico
@it
artigo científico
@pt
bilimsel makale
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scientific article published on 17 February 2010
@en
vedecký článok
@sk
vetenskaplig artikel
@sv
videnskabelig artikel
@da
vědecký článek
@cs
name
Therapeutic approaches for neuronopathic lysosomal storage disorders.
@en
Therapeutic approaches for neuronopathic lysosomal storage disorders.
@nl
type
label
Therapeutic approaches for neuronopathic lysosomal storage disorders.
@en
Therapeutic approaches for neuronopathic lysosomal storage disorders.
@nl
prefLabel
Therapeutic approaches for neuronopathic lysosomal storage disorders.
@en
Therapeutic approaches for neuronopathic lysosomal storage disorders.
@nl
P2860
P1476
Therapeutic approaches for neuronopathic lysosomal storage disorders.
@en
P2093
Raphael Schiffmann
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P2888
P304
P356
10.1007/S10545-010-9047-0
P577
2010-02-17T00:00:00Z