Effects of transgenic overexpression of polysialyltransferase (PST) on myelination and impacts of increased sulfatide accumulation in arylsulfatase A deficient mice and its relevance to human metachromatic leukodystrophy
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Sulfatide storage in neurons causes hyperexcitability and axonal degeneration in a mouse model of metachromatic leukodystrophy.
Metachromatic leukodystrophy is a lysosomal storage disorder caused by deficiency in the sulfolipid degrading enzyme arylsulfatase A (ASA). In the absence of a functional ASA gene, 3-O-sulfogalactosylceramide (sulfatide; SGalCer) and other sulfolipids accumulate. The storage is associated with progressive demyelination and various finally lethal neurological symptoms. Lipid storage, however, is...
متن کاملOligodendroglial progenitor cell therapy limits central neurological deficits in mice with metachromatic leukodystrophy.
This work describes the first successful oligodendrocyte-based cell therapy for presymptomatic arylsulfatase A (ARSA) null neonate mice, a murine model for human metachromatic leukodystrophy (MLD). We found that oligodendrocyte progenitors (OLPs) engrafted and survived into adulthood when transplanted in the neonatal MLD brain. Transplanted cells integrated nondisruptively, did not produce tumo...
متن کاملNeurobiology of Disease Increasing Sulfatide Synthesis in Myelin-Forming Cells of Arylsulfatase A-Deficient Mice Causes Demyelination and Neurological Symptoms Reminiscent of Human Metachromatic Leukodystrophy
Hariharasubramanian Ramakrishnan,1* Kerstin Khalaj Hedayati,2* Renate Lüllmann-Rauch,2 Carsten Wessig,3 Simon Ngamli Fewou,1 Helena Maier,1 Hans-Hilmar Goebel,4 Volkmar Gieselmann,1 and Matthias Eckhardt1 1Institute of Physiological Chemistry, Rheinische Friedrich-Wilhelms University of Bonn, 53115 Bonn, Germany, 2Institute of Anatomy, Christian-Albrechts University of Kiel, 24098 Kiel, Germany...
متن کاملSulfatide levels correlate with severity of neuropathy in metachromatic leukodystrophy
OBJECTIVE Metachromatic leukodystrophy (MLD) is an autosomal recessive lysosomal storage disorder due to deficient activity of arylsulfatase A (ASA) that causes accumulation of sulfatide and lysosulfatide. The disorder is associated with demyelination and axonal loss in the central and peripheral nervous systems. The late infantile form has an early-onset, rapidly progressive course with severe...
متن کاملIntracerebroventricular enzyme infusion corrects central nervous system pathology and dysfunction in a mouse model of metachromatic leukodystrophy.
Arylsulfatase A (ASA) catalyzes the desulfation of sulfatide, a major lipid component of myelin. Inherited functional deficiencies of ASA cause the lysosomal storage disease (LSD) metachromatic leukodystrophy (MLD), which is characterized by intralysosomal accumulation of sulfatide, progressive neurological symptoms and early death. Enzyme replacement therapy (ERT) using intravenous injection o...
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تاریخ انتشار 2007