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UnknownSodium valproate, a potential repurposed treatment for the neurodegeneration in Wolfram syndrome (TREATWOLFRAM): trial protocol for a pivotal multicentre, randomised double-blind controlled trial.
Not available
S-EPMC11865774 | biostudies-literature
GenomicsMultidimensional analysis and therapeutic development using patient iPSC-derived disease models of Wolfram syndrome
Multidimensional analysis and therapeutic development using patient iPSC-derived disease models of Wolfram syndrome
PRJNA874718 | ENA
UnknownNeuroimaging evidence of deficient axon myelination in Wolfram syndrome.
Not available
S-EPMC4758056 | biostudies-literature
GenomicsEarly developmental alteration of neurite outgrowth occurs besides late-appearing neurodegenerative processes in Wolfram syndrome
Early developmental alteration of neurite outgrowth occurs besides late-appearing neurodegenerative processes in Wolfram syndrome
PRJNA659500 | ENA
TranscriptomicsMultidimensional analysis and therapeutic development using patient iPSC-derived disease models of Wolfram syndrome
Wolfram syndrome is a rare genetic disorder largely caused by pathogenic variants in the WFS1 gene and manifested by diabetes mellitus, optic nerve atrophy, and progressive neurodegeneration. Recent genetic and clinical findings have revealed Wolfram syndrome as a spectrum disorder. Therefore, a gen...
ORGANISM(S): Homo sapiens 
2022-09-01 | GSE212256 | GEO
GenomicsSingle Cell Sequencing Analysis for Wolfram Syndrome (WS4) Unedited and Corrected Stem Cell-Derived Beta Cells
Single Cell Sequencing Analysis for Wolfram Syndrome (WS4) Unedited and Corrected Stem Cell-Derived Beta Cells
PRJNA580207 | ENA
TranscriptomicsHypothalamic gene expression profile indicates a reduction in G-protein signaling in the wfs1 mutant mice
WFS1 gene is coding protein with unknown function but its functional deficiency causes different neuropsychiatric and neuroendocrine syndromes. In the present study we aimed to find the functional networks influenced by the Wfs1 deficiency in the hypothalamus. We performed gene expression profiling ...
ORGANISM(S): Mus musculus 
2011-11-02 | E-GEOD-33372 | biostudies-arrayexpress
GenomicsISR inhibition reverses pancreatic β-cell failure in Wolfram syndrome models
ISR inhibition reverses pancreatic β-cell failure in Wolfram syndrome models
PRJNA985707 | ENA
UnknownDiabetes and neurodegeneration in Wolfram syndrome: a multicenter study of phenotype and genotype.
Not available
S-EPMC3120194 | biostudies-literature
TranscriptomicsEarly developmental alteration of neurite outgrowth occurs besides late-appearing neurodegenerative processes in Wolfram syndrome
Developmental alteration in brain wiring that would make it more susceptible to later pathological processes has been suggested as a basis for the occurrence of neurodegenerative diseases, but mechanisms have remained elusive. A recent series of magnetic resonance imaging studies have demonstrated t...
ORGANISM(S): Homo sapiens 
2021-09-23 | GSE156911 | GEO
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