Lysosomal storage diseases (LSDs) are a group of 70 monogenic disorders characterized
by the lysosomal accumulation of a substrate. As a group, LSDs affect~ 1 in 5000 live births; …

Inflammatory brain diseases can affect brain parenchyma (encephalitis), meninges
(meningitis), or both (meningoencephalitis). 1 Depending on the underlying causes …

Abstract Niemann-Pick type C (NP-C) disease is a neurovisceral lysosomal storage disease
characterized by neurologic dysfunction, hepatosplenomegaly, and early death. Natural …

Sandhoff disease (SD) is a glycosphingolipid (GSL) storage disease that arises from an
autosomal recessive mutation in the gene for the β-subunit of β-Hexosaminidase A (Hexb …

Intravascular injection of certain adeno-associated virus vector serotypes can cross the
blood–brain barrier to deliver a gene into the CNS. However, gene distribution has been …

Background Feline models of neurologic diseases, such as lysosomal storage diseases,
leukodystrophies, Parkinson's disease, stroke and NeuroAIDS, accurately recreate many …

Lysosomal storage disorders (LSDs) are inherited diseases that result from the intracellular
accumulation of incompletely degraded macromolecules. The majority of LSDs affect both …

The histologic characteristics that are the basis for diagnosis of central nervous system
conditions cannot be visualized directly using magnetic resonance (MR) methods, but …

Objective Despite profound neurological symptomatology there are only few MRI studies
focused on the brain abnormalities in alpha-mannosidosis (AM). Our aim was to …

Background Grey matter (GM) abnormalities are robust features of schizophrenia and of
people at ultra high-risk for psychosis. However the extent to which neuroanatomical …