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

Chronic administration of an HDAC inhibitor treats both neurological and systemic Niemann-Pick type C disease in a mouse model.

Science translational medicine - 17 Feb 2016

Alam Md Suhail, Getz Michelle, Haldar Kasturi

Abstract excerpt

Histone deacetylase inhibitors (HDACi) are approved for treating rare cancers and are of interest as potential therapies for neurodegenerative disorders. We evaluated a triple combination formulation (TCF) comprising the pan-HDACi vorinostat, the caging agent 2-hydroxypropyl-β-cyclodextrin (HPBCD), and polyethylene glycol (PEG) for treating a mouse model (the Npc1(nmf164) mouse) of Niemann-Pick type C (NPC)...

Read the complete abstract on PubMed

Topics

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

Share to Blood-Brain Barrier
Share to Histone Deacetylase Inhibitors

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Hydroxamic Acids

Share to Nerve Degeneration

Share to Niemann-Pick Disease, Type C

Share to Polyethylene Glycols

Share to Purkinje Cells

Share to Vorinostat

Source

Identifiers

PMID: 26888431
DOI: 10.1126/scitranslmed.aad9407

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Chronic administration of an HDAC inhibitor treats both neurological and systemic Niemann-Pick type C disease in a mouse model.PMID 26888431Read ↗

Gene Therapy in a Mouse Model of Niemann–Pick Disease Type C1Semantic proximity Read ↗

Histone deacetylase inhibitors correct the cholesterol storage defect in most Niemann-Pick C1 mutant cells.Semantic proximity Read ↗

Histone deacetylase inhibitors correct the cholesterol storage defect in most NPC1 mutant cellsSemantic proximity Read ↗

Optimization of systemic AAV9 gene therapy in Niemann-Pick disease type C1 miceSemantic proximity Read ↗

Treatment of Niemann–Pick Type C Disease by Histone Deacetylase InhibitorsSemantic proximity Read ↗

A murine Niemann-Pick C1 I1061T knock-in model recapitulates the pathological features of the most prevalent human disease allele.Semantic proximity Read ↗

Histone deacetylase inhibitor treatment dramatically reduces cholesterol accumulation in Niemann-Pick type C1 mutant human fibroblasts.Semantic proximity Read ↗

AAV9-NPC1 significantly ameliorates Purkinje cell death and behavioral abnormalities in mouse NPC diseaseSemantic proximity Read ↗

Acetyl-acetyl-L-leucine for Niemann-Pick type C: a multinational double-blind randomized placebo-controlled crossover studySemantic proximity Read ↗

AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease.Semantic proximity Read ↗

Quantitative comparison of the efficacy of various compounds in lowering intracellular cholesterol levels in Niemann-Pick type C fibroblasts.Semantic proximity Read ↗

Attenuation of the Niemann-Pick type C2 disease phenotype by intracisternal administration of an AAVrh.10 vector expressing Npc2.Semantic proximity Read ↗

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