A5089845548- Lysosomal Storage Disorders Research
- Trypanosoma species research and implications
- Glycogen Storage Diseases and Myoclonus
- Adipose Tissue and Metabolism
- Neurogenetic and Muscular Disorders Research
- Crystallization and Solubility Studies
- Child Nutrition and Feeding Issues
- X-ray Diffraction in Crystallography
- Adipokines, Inflammation, and Metabolic Diseases
- Carbohydrate Chemistry and Synthesis
- Pancreatitis Pathology and Treatment
- Hedgehog Signaling Pathway Studies
- Sirtuins and Resveratrol in Medicine
- Exercise and Physiological Responses
- Circadian rhythm and melatonin
- Congenital heart defects research
- Extracellular vesicles in disease
- Folate and B Vitamins Research
- Esophageal and GI Pathology
- Nanotechnology research and applications
- Biomedical Research and Pathophysiology
- Cellular transport and secretion
- Protein Tyrosine Phosphatases
- RNA regulation and disease
- Graphene and Nanomaterials Applications
University of Padua
2010-2022
Città della Speranza Foundation
2016-2017
Lysosomal Storage Disorders (LSDs) are a group of metabolic syndromes, each one due to the deficit lysosomal enzyme. Many LSDs affect most organ systems and overall about 75% patients present neurological impairment. Enzyme Replacement Therapy, although determining some systemic clinical improvements, is ineffective on CNS disease, enzymes' inability cross blood-brain barrier (BBB). With aim deliver therapeutic enzymes across BBB, we here assayed biodegradable biocompatible...
Mucopolysaccharidosis type II (MPSII) is a lysosomal storage disorder due to the deficit of enzyme iduronate 2-sulfatase (IDS), which leads accumulation glycosaminoglycans in most organ-systems, including brain, and resulting neurological involvement about two-thirds patients. The main treatment represented by weekly infusion functional enzyme, cannot cross blood-brain barrier reach central nervous system. In this study, tailored nanomedicine approach based on brain-targeted polymeric...
Background and purpose: Mucopolysaccharidoses (MPS) are lysosomal storage disorders resulting from a deficit of specific enzymes catalysing glycosaminoglycan (GAG) degradation. The typical pathology involves most the organ systems, including brain, in its severe forms. soy isoflavone genistein has recently attracted considerable attention as it can reduce GAG synthesis vitro . Furthermore, is able to cross blood–brain barrier rat. present study was undertaken assess ability urinary tissue...
Loss of lysosomal glucocerebrosidase (GBA1) function is responsible for several organ defects, including skeletal abnormalities in type 1 Gaucher disease (GD). Enhanced bone resorption by infiltrating macrophages has been proposed to lead major defects. However, while more recent evidences support the hypothesis that osteoblastic formation impaired, a clear pathogenetic mechanism not depicted yet. Here, combining different molecular approaches, we show Gba1 loss zebrafish associated with...
Morphogens release and activity can be negatively affected by an impaired glycosaminoglycans (GAGs) turnover proteoglycans assembly in the extracellular matrix, leading to altered tissue morphogenesis. In this work, we show that loss of Iduronate-2-sulfatase (IDS) activity, affecting GAGs catabolism responsible for a life-threatening valvulopathy mucopolysaccharidosis type II (MPSII), triggers early Sonic Hedgehog (Shh) Wnt/β-catenin signaling defects, aberrant heart development...
Antibody-drug conjugates (ADCs) are an established therapeutic entity in which potent cytotoxic drugs conjugated to a monoclonal antibody. In parallel with the great emphasis put on novel site-specific bioconjugation technologies, future advancements this field also rely exploring linker-drug architectures that improve efficacy and stability of ADCs. context, use hydrophilic linkers represents valid strategy mask or reduce inherent hydrophobicity most used positively impact physical vivo...
Lysosomal storage disorders (LSDs) are a group of about 50 genetic metabolic disorders, mainly affecting children, sharing the inability to degrade specific endolysosomal substrates. This results in failure cellular functions many organs, including brain that most patients may go through progressive neurodegeneration. In this study, we analyzed mouse model for Hunter syndrome, LSD mostly presenting with neurological involvement. Whole transcriptome analysis cerebral cortex and...
// Daniela Catanzaro 1, * , Silvia Nicolosi Veronica Cocetta 1 Marika Salvalaio Andrea Pagetta Eugenio Ragazzi Monica Montopoli 2 and Gianfranco Pasut 3 Department of Pharmaceutical Pharmacological Sciences, University Padua, Italy Venetian Institute Molecular Medicine, Padova, Veneto Oncology IOV, IRCCS, These authors have contributed equally to the work Correspondence to: Montopoli, email: monica.montopoli@unipd.it Pasut, gianfranco.pasut@unipd.it Keywords: Cisplatin; drug resistance;...
Skeletal abnormalities represent a major clinical burden in patients affected by the lysosomal storage disorder mucopolysaccharidosis type II (MPSII, OMIM #309900). While extensive research has emphasized detrimental role of stored glycosaminoglycans (GAGs) bone marrow (BM), limited understanding primary cellular mechanisms underlying defects MPSII hampered development bone-targeted therapeutic strategies beyond enzyme replacement therapy (ERT). We here investigated involvement key signaling...
Obesity and related comorbidities are a major health concern. The drugs used to treat these conditions largely inadequate or dangerous, well-researched approach based on nutraceuticals would be highly useful. Pterostilbene (Pt), i.e., 3,5-dimethylresveratrol, has been reported effective in animal models of obesity, acting different metabolic pathways. We investigate here its ability induce browning white adipose tissue. Pt (5 µM) was first tested 3T3-L1 mature adipocytes, then it...
Hunter syndrome (HS) is a lysosomal storage disease caused by iduronate-2-sulfatase (IDS) deficiency and loss of ability to break down recycle the glycosaminoglycans, heparan dermatan sulfate, leading impairment cellular processes cell death. Cell activities functioning intracellular organelles are controlled clock genes (CGs), driving rhythmic expression (CCGs). We aimed evaluate CGs downstream CCGs in HS, before after enzyme replacement treatment with IDS. The levels were evaluated whole...
Appropriate nutraceutical combinations may represent a valid approach to prevent vascular calcification associated with chronic kidney disease (CKD). In the present study, we tested effect of new combination named RenaTris®, containing MK-7, magnesium carbonate, and Sucrosomial® Iron, on in uremic rats. Rats were randomly divided into three groups, i.e., control (high-phosphate diet), diet 0.5% adenine), supplemented (0.5% adenine, Iron). After six weeks, sera examined. The increased...
Abstract Mucopolysaccharidosis type II (MPS II) is a neurometabolic disorder, due to the deficit of lysosomal hydrolase iduronate 2-sulfatase (IDS). This leads severe clinical condition caused by multi-organ accumulation glycosaminoglycans (GAGs/GAG) heparan- and dermatan-sulfate, whose elevated levels can be detected in body fluids. Since 2006, enzyme replacement therapy (ERT) has been clinically applied, showing efficacy some peripheral districts. In addition monitoring, GAG dosage...
Pterostilbene (Pt) is a natural phenol found in blueberries and grapes; it shows remarkable biomedical activities similar to those of resveratrol, but its higher bioavailability major advantage for possible applications. Our group has recently demonstrated that long-term (30 weeks) administration Pt mice maintained on high-fat diet counters weight gain promotes browning subcutaneous white adipose tissue (sWAT). By Real-time quantitative PCR Western Blot analysis the sWAT visceral (vWAT) from...