Injury to the central nervous system (CNS) results in oligodendrocyte cell death and
progressive demyelination. Demyelinated axons undergo considerable physiological …

Lysosomes and lysosomal enzymes play a central role in numerous cellular processes,
including cellular nutrition, recycling, signaling, defense, and cell death. Genetic deficiencies …

Common forms of Parkinson's disease have long been described as idiopathic, with no
single penetrant genetic factor capable of influencing disease aetiology. Recent genetic …

Krabbe disease (KD) is a neurodegenerative disorder caused by the lack of β-
galactosylceramidase enzymatic activity and by widespread accumulation of the cytotoxic …

Remyelination is a major therapeutic goal in human myelin disorders, serving to restore
function to demyelinated axons and providing neuroprotection. The target disorders that …

Neurodegenerative lysosomal storage disorders (LSDs) are severe and untreatable, and
mechanisms underlying cellular dysfunction are poorly understood. We found that toxic …

Abstract Treatment of neurological lysosomal storage disorders (LSDs) are limited because
of impermeability of the blood-brain barrier (BBB) to macromolecules. Nanoformulations …

Globoid cell leukodystrophy (GLD), also known as Krabbe disease, is a lysosomal storage
disorder causing extensive demyelination in the central and peripheral nervous systems …

Globoid cell leukodystrophy (GLD) is a metabolic disease caused by mutations in the
galactocerebrosidase (GALC) gene. GALC is a lysosomal enzyme whose function is to …

We report a global adeno-associated virus (AAV) 9-based gene therapy protocol to deliver
therapeutic galactosylceramidase (GALC), a lysosomal enzyme that is deficient in Krabbe's …