Search

Your search keyword '"Sternbach S"' showing total 2 results
Start Over Author "Sternbach S" Journal annals of neurology
2 results on '"Sternbach S"'

1. Ablating the Transporter Sodium-Dependent Dicarboxylate Transporter 3 Prevents Leukodystrophy in Canavan Disease Mice.

Author

Wang Y, Hull V, Sternbach S, Popovich B, Burns T, McDonough J, Guo F, and Pleasure D

Subjects
Animals, Astrocytes metabolism, Brain metabolism, Dicarboxylic Acid Transporters genetics, Disease Models, Animal, Mice, Transgenic, Neurodegenerative Diseases genetics, Symporters metabolism, Brain drug effects, Canavan Disease metabolism, Dicarboxylic Acid Transporters metabolism, Symporters genetics
Abstract

Canavan disease is caused by ASPA mutations that diminish brain aspartoacylase activity, and it is characterized by excessive brain storage of the aspartoacylase substrate, N-acetyl-l-aspartate (NAA), and by astroglial and intramyelinic vacuolation. Astroglia and the arachnoid mater express sodium-dependent dicarboxylate transporter (NaDC3), encoded by SLC13A3, a sodium-coupled transporter for NAA and other dicarboxylates. Constitutive Slc13a3 deletion in aspartoacylase-deficient Canavan disease mice prevents brain NAA overaccumulation, ataxia, and brain vacuolation. ANN NEUROL 2021;90:845-850., (© 2021 American Neurological Association.)

Published
2021
Full Text
View/download PDF

2. Antisense Oligonucleotide Reverses Leukodystrophy in Canavan Disease Mice.

Author

Hull V, Wang Y, Burns T, Zhang S, Sternbach S, McDonough J, Guo F, and Pleasure D

Subjects
Acetyltransferases antagonists & inhibitors, Amidohydrolases deficiency, Amidohydrolases genetics, Animals, Aspartic Acid biosynthesis, Ataxia complications, Ataxia drug therapy, Atrophy complications, Atrophy drug therapy, Canavan Disease complications, Canavan Disease pathology, Cerebellum pathology, Female, Gene Knockdown Techniques, Infusions, Intraventricular, Male, Mice, Mutation, Oligonucleotides, Antisense administration & dosage, Purkinje Cells pathology, Rotarod Performance Test, Thalamus pathology, Vacuoles drug effects, Vacuoles pathology, Aspartic Acid analogs & derivatives, Canavan Disease drug therapy, Oligonucleotides, Antisense therapeutic use
Abstract

Marked elevation in the brain concentration of N-acetyl-L-aspartate (NAA) is a characteristic feature of Canavan disease, a vacuolar leukodystrophy resulting from deficiency of the oligodendroglial NAA-cleaving enzyme aspartoacylase. We now demonstrate that inhibiting NAA synthesis by intracisternal administration of a locked nucleic acid antisense oligonucleotide to young-adult aspartoacylase-deficient mice reverses their pre-existing ataxia and diminishes cerebellar and thalamic vacuolation and Purkinje cell dendritic atrophy. Ann Neurol 2020;87:480-485., (© 2020 American Neurological Association.)

Published
2020
Full Text
View/download PDF

Catalog

Books, media, physical & digital resources
See catalog results