Results 111 to 120 of about 11,019 (198)

Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: Canavan disease. [PDF]

, 1999
The neurogenetic prototypic disease on which we chose to test our gene therapy strategy is Canavan disease (CD). CD is an autosomal recessive leukodystrophy associated with spongiform degeneration of the brain.
During, Matthew J, Janson, Christopher G, Leone, Paola, McPhee, Scott J   +3 more
core   +1 more source

N-acetylaspartate mitigates pro-inflammatory responses in microglial cells by intersecting lipid metabolism and acetylation processes

Cell Communication and Signaling
Background Microglia play a crucial role in brain development and repair by facilitating processes such as synaptic pruning and debris clearance.
Federica Felice, Pamela De Falco, Martina Milani, Serena Castelli, Antonella Ragnini-Wilson, Giacomo Lazzarino, Nadia D’Ambrosi, Fabio Ciccarone, Maria Rosa Ciriolo   +8 more
doaj   +1 more source

High Throughput Screening Cascade To Identify Human Aspartate N-Acetyltransferase (ANAT) Inhibitors for Canavan Disease. [PDF]

ACS Chem Neurosci, 2021
Nešuta O, Thomas AG, Alt J, Hin N, Neužilová A, Long S, Tsukamoto T, Rojas C, Wei H, Slusher BS.   +9 more
europepmc   +1 more source

Alexander and Canavan Disease

Iranian Journal of Child Neurology, 2014
How to Cite This Article: Shalbafan B. Alexander and Canavan Disease. Iran J Child Neurol. Autumn 2014;8;4(Suppl.1):20-21.
openaire   +1 more source

Human iPSC-derived neural progenitor cells rescue motor function and brain pathology in symptomatic Canavan disease mice. [PDF]

Stem Cell Reports
Jackson N, Feng L, Chao J, Ye P, Luong Q, Galo K, Sun G, Hu W, Shi Y.   +8 more
europepmc   +1 more source

Deep Intronic SVA_E Insertion Identified as the Most Common Pathogenic Variant Associated With Canavan Disease: A Diagnostic Blind Spot. [PDF]

Neurol Genet
Dominguez Gonzalez CA, Bell KM, Rajagopalan R, de Silva MG, Lemes A, Zabala C, Pérez-Vidarte F, Cerisola A, Vossough A, Whitehead MT, Cunningham C, Brown NJ, Quin R, Simons C, Conway T, Uebergang E, Rius R, Kumaheri MA, Kotes ER, Vohra A, Zalusky MPG, Anderson ZB, Storz SHR, Ward SA, Goffena J, Gustafson JA, White SM, Vanderver A, Miller DE.   +28 more
europepmc   +1 more source

v. 68, issue 1, August 31, 1999 [PDF]

, 1999

core   +1 more source

Antisense Oligonucleotide Reverses Leukodystrophy in Canavan Disease Mice. [PDF]

Ann Neurol, 2020
Hull V, Wang Y, Burns T, Zhang S, Sternbach S, McDonough J, Guo F, Pleasure D.   +7 more
europepmc   +1 more source

Systematic review of the effects of exercise and nutrition interventions on body composition in women with metastatic breast cancer [PDF]

, 2018
Innerarity, Celia, Kelly, Jaimon T, Van der Meij, Barbara S   +2 more
core  

Deep Intronic SVA_E Retrotransposition as a Novel Factor in Canavan Disease Pathogenesis. [PDF]

Hum Gene Ther
Weiß M, Selig M, Friedrich J, Wierczeiko A, Diederich S, Sigel H, Bredow J, Eichler FS, Nagy A, Seyler D, Holthöfer L, Gerber S, Schweiger S, Linke M, Bley A.   +14 more
europepmc   +1 more source