Browse
Submit Data
Databases
API
Help

Dataset Information

0 Views

0 Connections

0 Citations

0 Reanalyses

0 Downloads

Omics score: 0

Gene expression data from DMD patient hiPSC-derived myoblasts treated with fenofibrate, ginsenoside Rd or DMSO

ABSTRACT: Gene expression data from DMD patient hiPSC-derived myoblasts treated with fenofibrate, ginsenoside Rd or DMSO

PROVIDER: PRJNA495367 | ENA |

REPOSITORIES: ENA

ACCESS DATA

Json Xml

Similar Datasets

Gene expression data from DMD patient hiPSC-derived myoblasts treated with fenofibrate, ginsenoside Rd or DMSO

Project description:Drug development costs a significant amount of time and resources for new pharmaceutical drugs. However, the progress has been limited for orphan diseases such as Duchenne muscular dystrophy (DMD). By using human induced pluripotent stem cells (hiPSCs), here we show an exemplary drug screening campaign and the identification of two potential drugs effective in a DMD mouse model. We developed a DMD-hiPSC screening platform utilizing high-content imaging to identify hit compounds that enhance myogenic fusion abilities of patient-specific myoblasts. Among 1524 compounds (Johns Hopkins Clinical Compound library), two hit compounds restored in vitro fusion defects of DMD patient hiPSC-derived myoblasts. Transcriptional profiling revealed that the function of two hit compounds, ginsenoside Rd (natural product, ginseng extract) and fenofibrate (FDA-approved drug), are associated with FLT3 signaling and TGF-β signaling, respectively. The preclinical tests in mdx mice show that the treatment of the two hit compounds can ameliorate the skeletal muscle and behavioral phenotypes caused by DYSTROPHIN deficiency, suggesting therapeutic potential of these two compounds. Our study demonstrates the feasibility of early-stage drug development for rare diseases using symptom-relevant cells derived from patient-specific hiPSCs.

2020-05-09 | GSE121023 | GEO

The effect of utrophin on the electrophysiological properties of DMD hiPSC-derived cardiomyocytes

Project description:DMD is a genetic disease, which leads to muscle weakness and cardiomyopathy. The latter remains incurable, being the main cause of death in DMD, therefore new therapeutic strategies are being sought to provide effective treatment. One of them considers upregulation of utrophin, a protein structurally and functionally homologous to dystrophin. In this study proteomic analysis of dystrophin-deficient and both dystrophin- and utrophin-deficient hiPSC-CM indicated on considerable differences in terms of contraction-related mechanisms. We thus investigated the role of utrophin in the maintenance of electrophysiological properties of DMD hiPSC-CM using the cells with additional utrophin deficiency and with utrophin upregulation. Obtained results indicated on disturbance of calcium handling in DMD hiPSC-CM, even more pronounced in DMD/UTRN KO hiPSC-CM and increased values of AHP in DMD hiPSC-CM. Utrophin upregulation improved both calcium oscillations and AHP values. Our findings highlight utrophin as important in the maintenance of the electrophysiological properties of DMD hiPSC-CM.

2024-06-20 | PXD044407 | Pride

Ginsenoside Rd monomer alleviates ulcerative colitis by regulating miRNAs and target genes in macrophages

Project description:Ginsenoside Rd monomer alleviates ulcerative colitis by regulating miRNAs and target genes in macrophages

| PRJNA970369 | ENA

Ginsenoside Rd monomer alleviates ulcerative colitis by regulating miRNAs and target genes in macrophages

Project description:to discover the miRNAs involved in the production of cytokines by RAW264.7 cells treated with LPS and ginsinoside Rd monomer We then performed gene expression profiling analysis using data obtained from RNA-seq of RAW264.7 cells.

2024-04-01 | GSE231990 | GEO

The effect of fenofibrate on endothelial cells

2009-08-24 | GSE15483 | GEO

Haemophilus influenzae Rd KW20 strain:Rd

Project description:Haemophilus influenzae Rd KW20 strain:Rd Genome sequencing

| PRJNA237703 | ENA

Outer membrane vesicle and outer membrane of Haemophilus influenzae Rd KW20, Rd ∆yrbE and Rd ∆vacJ

Project description:Bacterial outer membrane vesicles (OMVs) have important biological roles in pathogenesis and intercellular interactions, but a general mechanism of OMV formation is lacking. Here we show that the VacJ/Yrb ABC transport system, a proposed phospholipid (PL) transporter, is involved in OMV formation. Deletion or repression of VacJ/Yrb increases OMV production in two distantly related Gram-negative bacteria, Haemophilus influenzae and Vibrio cholerae. Within our studies we also analyzed the proteome of the OMV and outer membrane (OM) and found no massive alteration in Haemophilus influenzae Rd KW20, Rd ∆yrbE and Rd ∆vacJ. Lipidome analyses demonstrate that OMVs from VacJ/Yrb-defective mutants in H. influenzae are enriched in PLs and certain fatty acids. Furthermore, we demonstrate that OMV production and regulation of the VacJ/Yrb ABC transport system respond to iron starvation. Our results suggest a new general mechanism of OMV biogenesis based on PL accumulation in the outer leaflet of the outer membrane. This mechanism is highly conserved among Gram-negative bacteria, provides a means for regulation, can account for OMV formation under all growth conditions, and might have important pathophysiological roles in vivo.

2016-01-27 | PXD003249 | Pride

The effect of fenofibrate on endothelial cells

Project description:Fenofibrate is a synthetic ligand for the nuclear receptor peroxisome proliferator-activated receptor (PPAR) alpha, but there are reports that fenofibrate affects endothelial cells in PPARa-independent manner. In order to identify PPARa-dependently and PPARa-independently regulated transcripts we generated microarray data from human endothelial cells treated with fenofibrate with and without siRNA-mediated knock-down of PPARa. In this study, we generated microarray data from human umbilical vein endothelial cells (HUVECs) treated with fenofibrate. Time 0 indicates the start point of observation immediately prior to exposure to the 25umol fenofibrate. There are five time points (2, 4, 6, 8, and 18hours) (n=3 at each time point). The control is untreatment HUVEC (n=4).

2010-05-06 | E-GEOD-15483 | biostudies-arrayexpress

Changes of the gene expression profiling induced by fenofibrate

Project description:To make investigation of the apoptosis-inducing effect of fenofibrate, the Gene expression profile chip was used to compare the changes between the control group (0μM for 24h) and fenofibrate treatment group (50μM for 24h) in MDA-MB-231 cell lines.

2014-03-13 | GSE49965 | GEO

The effect of PPARa siRNA on endothelial cells treated with fenofibrate

2009-08-24 | GSE15482 | GEO