Starting Treatment with Agonist Replacement Therapy (START)
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ABSTRACT:
PROVIDER: PRJNA324306 | ENA |
REPOSITORIES: ENA
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Project description:<p>The Starting Treatment with Agonist Replacement Therapy (START) study was a 24-week, open-label, randomized trial of methadone <i>versus</i> Suboxone (buprenorphine/naloxone) for the treatment of opioid dependence. Patients were over the age of 18 and met DSM-IV qualifications for opioid dependence. The primary goal of the study was to determine if either medicine was associated with liver toxicity issues in this vulnerable patient population. Liver enzymes were measured at four time points. Urinalysis for opioids, cocaine, cannabis, benzodiazepines, and methamphetamine were performed on a weekly basis. A flexible dosing scheme was used during which physicians could alter medication dose based on withdrawal symptoms and urinalysis results. Neither medication was found to be associated with liver enzyme levels.</p>
| phs001135 | dbGaP
Project description:Starting Treatment with Agonist Replacement Therapy (START)
| PRJNA324307 | ENA
Project description:Enzyme Replacement Therapy is the only therapeutic option for Fabry patients with completely absent AGAL activity. However, it has many limitations, in terms of costs, high rh-protein re-quired, and side effects; thus, its optimization would be beneficial for patients. In this paper, we describe preliminary results paving the way for two possible approaches: i. combination of en-zyme replacement therapy with pharmacological chaperones; ii. identification of approved drugs that affect the AGAL interactors' expression. We show that galactose, a low-affinity phar-macological chaperone, can prolong rh-AGAL half-life in FD cells. We compared the wild-type AGAL and the rh-AGAL interactomes and identified a list of drugs known to affect them. This list represents a starting point to deeply screen approved drugs and identify those that can bene-fit the enzyme replacement therapy and those that can negatively interfere with it.
2023-05-10 | PXD039168 | Pride
Project description:Current approved anti-angiogenic drugs (AAD) for hepatocellular carcinoma (HCC) inhibit tumor angiogenesis, but affect the hepatic vasculature resulting in adverse effects. Tumor endothelial cells (TECs) differ from normal endothelial cells. In this study, we aimed to detect TEC-specific miRNAs and develop an anti-angiogenic treatment specific for TECs. We established HCC orthotopic mouse models. TEC-specific miRNAs were detected using a microRNA array. Finally, we evaluated the therapeutic effects of the TEC-specific miRNA agonist cocktail. In total, 260 TEC-specific genes were detected. Among the top ten downregulated TEC-specific miRNAs, miR-139-3p and 214-3p were important for the TEC phenotype. The TEC-specific microRNA agonist cocktail showed significant anti-tumor effects by inhibiting tumor angiogenesis without affecting hepatic vasculatures in HCC orthotopic mouse models. Moreover, it significantly downregulated tip-cell sprouting-related genes. We identified two downregulated TEC-specific miRNAs; microRNA replacement therapy, which targets the downregulated TEC-specific miRNAs, is an effective and promising treatment for HCC
2024-02-07 | GSE250147 | GEO
Project description:Automating iPSC generation to enable autologous photoreceptor cell replacement therapy
| PRJNA908874 | ENA
Project description:A tumor endothelial cell-specific microRNA replacement therapy for hepatocellular carcinoma
| PRJNA1052111 | ENA
Project description:Huntington’s disease (HD) is a monogenetic neurodegenerative disorder caused by the expansion of a polyglutamine (polyQ) stretch in huntingtin (htt). Here we show that mutant htt reduces the transcription of insulin-like growth factor 1 (IGF-1) and leads to loss of IGF-1 in HD brains, HD mouse models and mutant htt-transgenic microglial cells. IGF-1 replacement therapy by transplantation of genetically engineered mouse neuronal precursor cells (mNPCs) in a mouse model of HD reverted the motor phenotype and countered striatal neuronal loss.
2018-10-01 | GSE19291 | GEO
Project description:BACKGROUND:Studies of the timing of end-stage renal disease (ESRD) have primarily defined "early" versus "late" initiation of dialysis using estimated glomerular filtration rate (eGFR)-based criteria. Our objective was to determine the theoretical time that could be spent in chronic kidney disease (CKD) stage 5 prior to reaching a conservative eGFR threshold of 5 mL/min/1.73 m2 compared to the actual time spent in CKD stage 5 by risk factors of interest. METHODS:Eight-hundred and seventy Chronic Renal Insufficiency Cohort participants with CKD stage 5 who started renal replacement therapy (RRT) were included for retrospective study. We used mixed models to estimate the person-specific trajectory of renal function. We then used these individual trajectories to estimate the amount of time that would be spent in CKD stage 5 (between eGFR of 15 and 5 mL/min/1.73 m2) and compared this estimate to the actual time spent in CKD stage 5 prior to ESRD (between eGFR of 15 mL/min/1.73 m2 and ESRD). RESULTS:We found the median observed time between eGFR of 15 mL/min/1.73 m2 to RRT was 9.6 months, but the median predicted time between eGFR of 15 mL/min/1.73 m2 to eGFR of 5 mL/min/1.73 m2 was 17.7 months. Some of the largest differences between the predicted and actual amount of time spent in CKD stage 5 were noted among those with systolic blood pressure <140 mm Hg (9.7 months longer predicted compared to actual), proteinuria <1 g/g (9.1 months), and serum albumin ≥3.5 g/dL (9.0 months). CONCLUSION:We found marked differences between the actual and predicted time spent in CKD stage 5 based on risk factors of interest. We believe that placing timing of dialysis initiation in the perspective of time is novel and may identify subgroups of patients who may derive particular benefit from a more concerted effort to delay RRT.
| S-EPMC6667296 | biostudies-literature
Project description:The aim of the study was to investigate gene expression profiles of post-menopausal women receiving Premarin estrogen replacement therapy (ERT), compared to controls and to examine any correlations between the bacterial vaginosis (BV) status of the stubjects. This is the first study to use gene arrays to correlate changes in host expression to ERT and BV.
2018-04-28 | GSE113771 | GEO
Project description:DMXAA (STING agonist) efficacy on tumor microenvironment after CAR-T therapy
| PRJNA680502 | ENA