Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Modeling Niemann-Pick disease type C1 in zebrafish: a robust platform for in vivo screening of candidate therapeutic compounds.

Disease models & mechanisms - 15 Aug 2018

Tseng Wei-Chia, Loeb Hannah E, Pei Wuhong, Tsai-Morris Chon-Hwa, Xu Lisha, Cluzeau Celine V, Wassif Christopher A, Feldman Benjamin, Burgess Shawn M, Pavan William J, Porter Forbes D

Abstract excerpt

Niemann-Pick disease type C1 (NPC1) is a rare autosomal recessive lysosomal storage disease primarily caused by mutations in NPC1 NPC1 is characterized by abnormal accumulation of unesterified cholesterol and glycolipids in late endosomes and lysosomes. Common signs include neonatal jaundice, hepatosplenomegaly, cerebellar ataxia, seizures and cognitive decline. Both mouse and feline models of NPC1 mimic the...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Cholesterol
Share to Drug Evaluation, Preclinical

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Liver Diseases

Share to Niemann-Pick C1 Protein

Share to Niemann-Pick Disease, Type C

Share to Zebrafish

Share to Zebrafish Proteins

Source

Open in PubMed Open full text

Identifiers

PMID: 30135069
DOI: 10.1242/dmm.034165
PMCID: PMC6176986

Genes

NPC1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Modeling Niemann-Pick disease type C1 in zebrafish: a robust platform for in vivo screening of candidate therapeutic compounds.PMID 30135069Read ↗

Model construction of Niemann-Pick type C disease in zebrafishSemantic proximity Read ↗

Severe neurometabolic phenotype in <i>npc1</i> <sup>-/-</sup> zebrafish with a C-terminal mutationSemantic proximity Read ↗

npc2-Deficient Zebrafish Reproduce Neurological and Inflammatory Symptoms of Niemann-Pick Type C DiseaseSemantic proximity Read ↗

The role of Niemann-Pick type C2 in zebrafish embryonic developmentSemantic proximity Read ↗

Mutant induced neurons and humanized mice enable identification of Niemann-Pick type C1 proteostatic therapiesSemantic proximity Read ↗

A human iPSC-derived inducible neuronal model of Niemann-Pick disease, type C1Semantic proximity Read ↗

Niemann-Pick type C disease: molecular mechanisms and potential therapeutic approaches.Semantic proximity Read ↗

Different Niemann-Pick C1 Genotypes Generate Protein Phenotypes that Vary in their Intracellular Processing, Trafficking and LocalizationSemantic proximity Read ↗

Niemann-Pick Disease Type C: from molecule to clinic.Semantic proximity Read ↗

Whole-organism screening in a zebrafish model of CLN2 disease identifies pregnenolone as a modulator of lysosomal functions with anti-epileptic propertiesSemantic proximity Read ↗

Transcriptome of HPβCD-treated Niemann-Pick disease type C1 cells highlights GPNMB as a biomarker for therapeuticsSemantic proximity Read ↗

Genetic dissection of a cell-autonomous neurodegenerative disorder: lessons learned from mouse models of Niemann-Pick disease type CSemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.