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Nephropathic cystinosis is a severe monogenic kidney disorder caused by mutations in CTNS, encoding the lysosomal transporter cystinosin, resulting in lysosomal cystine accumulation. The sole treatment, cysteamine, slows down the disease progression, but does not correct the established renal proxim...
2021-06-24 | MTBLS2538 | MetaboLights
Increased morbidity and fetal growth restriction are reported in uninfected children born to human immunodeficiency virus type 1 (HIV-1)–infected women treated with antiretroviral (ARV) therapy. Viruses and/or pharmacological interventions such as ARVs can induce metabolic stress, skewing the cell’s...
2018-06-06 | MTBLS449 | MetaboLights

Hyperbilirubinemia is one of the most common diseases in neonates, and phototherapy is currently the most commonly used treatment in medicine, but previous studies have found that phototherapy can lead to changes in the intestinal flora of jaundice neonates, especially bifidobacteria. The focus o...

2026-03-26 | MTBLS14123 | MetaboLights
It is the ambition of the team formed by members of the Netherlands Cancer Institute (NKI) and the Cancer Genome Project at the Wellcome Trust Sanger Institute (WTSI) to unravel the genomic and phenotypic complexity of human cancers in order to identify optimal drug combinations for personalized can...
Huntington's disease is a genetic disease caused by a single mutation. It is characterised by progressive movement, emotional and cognitive deficits. R6/2 transgenic mice carrying the Huntington's disease mutation have a progressive neurological phenotype, including deterioration in cognitive functi...
ORGANISM(S): Mus musculus 
The COVID-19 pandemic highlighted the urgent need to develop effective and broad-spectrum antiviral therapies against coronaviruses. One strategy to address this concern is combination therapy using repurposed drugs against zoonotic viruses with pandemic potential. We previously demonstrated that th...
ORGANISM(S): Mus musculus (Mouse) Betacoronavirus 
2026-09-07 | PXD073706 | Pride
Identification of clinical combination therapies to induce durable responses in kidney cancers
Combination therapies of porcupine inhibition with ruxolitinib, ibrutinib or belumosudil in murine sclerodermatous GvHD
Synergistic combination therapies involving regorafenib in experimental glioma based on genome-wide CRISPR-Cas9 functional screens
Gain-of-function mutations in NOTCH1 are among the most frequent genetic alterations in T cell acute lymphoblastic leukemia (T-ALL), making the Notch signaling pathway a promising therapeutic target for personalized medicine. Yet, a major limitation for long-term success of targeted therapy is relap...
ORGANISM(S): Homo sapiens (Human) 
2024-01-26 | PXD038908 | Pride
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