- CAR-T cell therapy research
- Liver Disease Diagnosis and Treatment
- Ubiquitin and proteasome pathways
- Cell Adhesion Molecules Research
- Fibroblast Growth Factor Research
- Liver physiology and pathology
- Endoplasmic Reticulum Stress and Disease
- Glioma Diagnosis and Treatment
- Diet, Metabolism, and Disease
- Cancer-related gene regulation
- Liver Diseases and Immunity
- Diabetes and associated disorders
- Diet and metabolism studies
- Epigenetics and DNA Methylation
- Biomedical Research and Pathophysiology
- Bariatric Surgery and Outcomes
- MicroRNA in disease regulation
- Sarcoma Diagnosis and Treatment
- Peptidase Inhibition and Analysis
- Cancer-related Molecular Pathways
- Protein Degradation and Inhibitors
- Galectins and Cancer Biology
- RNA modifications and cancer
- Kruppel-like factors research
- Neuroblastoma Research and Treatments
Bambino Gesù Children's Hospital
2015-2024
Istituti di Ricovero e Cura a Carattere Scientifico
2014-2024
RELX Group (Netherlands)
2016
Boston Children's Hospital
2012-2015
There is evidence that nonalcoholic fatty liver disease (NAFLD) affected by gut microbiota. Therefore, we investigated its modifications in pediatric NAFLD patients using targeted metagenomics and metabolomics. Stools were collected from 61 consecutive diagnosed with (NAFL), steatohepatitis (NASH), or obesity 54 healthy controls (CTRLs), matched a case‐control fashion. Operational taxonomic units pyrosequenced targeting 16S ribosomal RNA volatile organic compounds determined solid‐phase...
// Sara Ceccarelli 1, * , Nadia Panera 2, Marco Mina 3 Daniela Gnani 1 Cristiano De Stefanis 2 Annalisa Crudele Chiara Rychlicki 4 Stefania Petrini 5 Giovannella Bruscalupi 6 Laura Agostinelli Stronati 7 Salvatore Cucchiara 8 Giovanni Musso 9 Cesare Furlanello Gianluca Svegliati-Baroni 4, 10 Valerio Nobili Anna Alisi Liver Research Unit, "Bambino Gesù" Children's Hospital-IRCCS, Rome, Italy Hepato-Metabolic Disease Predictive Models for Biomedicine and Environment Fondazione Bruno Kessler,...
Background There are no licensed treatments for non alcoholic fatty liver disease (NAFLD) in adults or children. In NAFLD, several studies have shown a benefit of omega-3 acid treatment on lipid profile, insulin-sensitivity and hepatic steatosis it has also been suggested that Vitamin D potential antifibrotic properties disease. Trial Design To date, however, there tested the combination Docosahexanoic (DHA) vitamin which may whole spectrum NAFLD. Our aim therefore, was to test effect daily...
Abstract Purpose: Medulloblastoma (MB), the most common childhood malignant brain tumor, has a poor prognosis in about 30% of patients. The current standard care, which includes surgery, radiation, and chemotherapy, is often responsible for cognitive, neurologic, endocrine side effects. We investigated whether chimeric antigen receptor (CAR) T cells directed toward disialoganglioside GD2 can represent potentially more effective treatment with reduced long-term Experimental Design: expression...
Atypical fibroblast growth factors (FGF) 21 and 19 play a central role in energy metabolism through the mediation of Klotho coreceptor. Contradictory findings are available about association FGF21 FGF19 with nonalcoholic fatty liver disease (NAFLD) humans. We investigated serum FGF21, coreceptor non-alcoholic steatohepatitis (NASH) fibrosis children NAFLD. Serum were measured 84 biopsy-proven NAFLD 23 controls (CTRL). The hepatic expression was 7 CTRL, 9 patients NASH (NASH+) 11 without...
•The KLB rs17618244 variant increases the risk of ballooning and lobular inflammation in children with NAFLD.•KLB plasma levels are lower carriers minor A allele.•KLB associated inflammation, fibrosis.•KLB mutant induces intracellular lipid accumulation HepG2 Huh7.•KLB causes upregulation lipotoxic proinflammatory genes. Background & AimNon-alcoholic fatty liver disease (NAFLD) is leading cause chronic adults children. Along obesity, diabetes insulin resistance, genetic factors strongly...
Abstract Background Diffuse midline gliomas (DMG) H3K27M-mutant, including diffuse intrinsic pontine glioma (DIPG), are pediatric brain tumors associated with grim prognosis. Although GD2-CAR T-cells demonstrated significant anti-tumor activity against DMG H3K27M-mutant in vivo, a multimodal approach may be needed to more effectively treat patients. We investigated GD2 expression DMG/DIPG and other high-grade (pHGG) sought identify chemical compounds that would enhance T-cell efficacy....
The prognosis of many patients with chemotherapy-refractory or multiply relapsed CD30+ non-Hodgkin Lymphoma (NHL) Hodgkin lymphoma (HL) still remains poor, and novel therapeutic approaches are warranted to address this unmet clinical need. In light consideration, we designed pre-clinically validated a Chimeric Antigen Receptor (CAR) construct characterized by anti-CD30 single-chain variable-fragment cassette, linked CD3ζ the signaling domains two costimulatory molecules, namely either...
Rhabdomyosarcoma (RMS) is an aggressive pediatric malignancy of the muscle, that includes Fusion Positive (FP)-RMS harboring PAX3/7-FOXO1 and Negative (FN)-RMS commonly with RAS pathway mutations. RMS express myogenic master transcription factors MYOD MYOG yet are unable to terminally differentiate. Here, we report SNAI2 highly expressed in FN-RMS, oncogenic, blocks differentiation, promotes growth. activates via super enhancers striped 3D contact architecture. Genome wide chromatin binding...
Hepatocellular carcinoma (HCC) is the most common type of liver cancer in humans. The focal adhesion tyrosine kinase (FAK) often over-expressed human HCC and FAK inhibition may reduce cell invasiveness. However, anti-oncogenic effect knockdown cells remains to be clarified. We found that depletion reduced vitro vivo tumorigenicity, by inducing G2/M arrest apoptosis, decreasing anchorage-independent growth, modulating expression several cancer-related genes. Among these genes, we showed...
Non-alcoholic fatty liver disease is one of the most important causes liver-related morbidity in children. In non-alcoholic disease, activation resident macrophage pool a central event progression injury. The aims present study were to evaluate polarization macrophages and possible role Wnt3a production by hepatic progenitor cell response pediatric disease. 32 children with biopsy-proven included. 20 out patients treated docosahexaenoic acid for 18 months biopsies at baseline after Hepatic...
Background Mechanism(s) connecting obstructive sleep apnoea syndrome (OSAS) to liver injury in paediatric non-alcoholic fatty disease (NAFLD) are unknown. We hypothesised alterations gut–liver axis and the pool phenotype of hepatic progenitor cells (HPCs) may be involved OSAS-associated NAFLD. Methods Eighty biopsy-proven NAFLD children (age, mean±SD, 11.4±2.0 years, 56% males, body mass index z -score 1.95±0.57) underwent a clinical–biochemical assessment, with measurement insulin...
Non-alcoholic fatty liver disease (NAFLD)-related fibrosis results in the encapsulation of injured parenchyma by a collagenous scar mainly imputable to hepatic stellate cells' activation. Approved pharmacological treatments against NAFLD-related are still lacking, but natural compounds such as hydroxytyrosol (HXT) and vitamin E (VitE), emerging promising therapeutic opportunities. In this study, potential anti-fibrotic effect HXT + VitE combination therapy was investigated vitro vivo....
Non-alcoholic fatty liver disease (NAFLD) is one of the most prevalent, chronic diseases, worldwide. It a multifactorial caused by complex interactions between genetic, epigenetic and environmental factors. Recently, several microRNAs, some which epigenetically regulated, have been found to be up- and/or down-regulated during NAFLD development. However, in NAFLD, essential role Polycomb Group protein Enhancer Zeste Homolog 2 (EZH2), controls silencing specific genes microRNAs trimethylating...
Abstract Background & Aims Treatment with the farnesoid X receptor ( FXR ) agonist obeticholic acid is ineffective in some patients non‐alcoholic steatohepatitis NASH but explanation uncertain. We investigated hepatic expression, and measurements of fibroblast growth factor 19 FGF 19) bile acids BA s) children NAFLD to investigate relationships . Methods 33 who underwent diagnostic liver biopsy were studied. Hepatic protein levels circulating concentrations compared those analysed five...
ABSTRACT Bone marrow (BM) is the major target organ for neuroblastoma (NB) metastasis and its involvement associated with poor outcome. Yet, mechanism by which NB cells invade BM largely unknown. Tumour microenvironment represents a key element in tumour progression mesenchymal stromal (MSCs) have been recognized as fundamental part of stroma. Here, we show that BM‐MSCs isolated from patients exhibit greater osteogenic potential than MSCs non‐infiltrated BM. We metastasis‐derived NB‐cell...
Background Pediatric high-grade gliomas (pHGGs) are among the most common and incurable malignant neoplasms of childhood. Despite aggressive, multimodal treatment, outcome children with has not significantly improved over past decades, prompting development innovative approaches. Methods To develop an effective we aimed at improving suboptimal antitumor efficacy oncolytic adenoviruses (OAs) by testing combination a gene-therapy approach using bispecific T-cell engager (BiTE) directed towards...
Hepatocellular carcinoma (HCC) is one of the most common and lethal malignant tumours worldwide. Sorafenib (SOR) effective single-drug systemic therapy against advanced HCC, but identification novel combination regimens for a continued improvement in overall survival big challenge. Recent studies highlighted crucial role focal adhesion kinase (FAK) HCC growth. The aim this study was to investigate antitumor effects three different FAK inhibitors (FAKi), alone or with SOR, using vitro vivo...
Non-alcoholic fatty liver disease (NAFLD) affects 3–12% of the general pediatric population. HMGB1 protein is presently considered a potent inflammatory mediator in several diseases, even if its role NAFLD still unknown clinical studies. Here we investigated relationships between circulating HMGB1, TGF-β and MCP-1 damage NAFLD. plasma levels were measured 110 obese children with biopsy-proven 40 age-matched controls. MCP-1, ALT, AST cholesterol significantly higher than control children. A...
Non-alcoholic fatty liver disease (NAFLD) is a complex ranging from steatosis to non-alcoholic steatohepatitis (NASH). Galectin-3 (Gal-3), which β-galactoside binding protein, has been associated with fibrosis, but its role in NAFLD remains elusive. We investigated the expression of Gal-3 resident cells and potential association damage 40 children biopsy-proven NAFLD. found that several expressed Gal-3. The number total positive decreased severity were correlated presence diagnosis NASH....