A5087533290- Multiple Myeloma Research and Treatments
- Virus-based gene therapy research
- Advanced MRI Techniques and Applications
- Banking stability, regulation, efficiency
- Genomic variations and chromosomal abnormalities
- Cancer Genomics and Diagnostics
- Neuroscience and Neuropharmacology Research
- Carcinogens and Genotoxicity Assessment
- Chromosomal and Genetic Variations
- Protein Degradation and Inhibitors
- RNA Interference and Gene Delivery
- Cancer-related Molecular Pathways
- Chronic Lymphocytic Leukemia Research
- Mitochondrial Function and Pathology
- Neurotransmitter Receptor Influence on Behavior
- Chronic Myeloid Leukemia Treatments
- Forensic and Genetic Research
- Peptidase Inhibition and Analysis
- Ocular Oncology and Treatments
- Molecular Biology Techniques and Applications
- Cystic Fibrosis Research Advances
- Folate and B Vitamins Research
- Metabolism and Genetic Disorders
- Neuroblastoma Research and Treatments
- RNA regulation and disease
Bambino Gesù Children's Hospital
2019-2025
Istituti di Ricovero e Cura a Carattere Scientifico
2021-2025
Rowan University
2011-2024
Hospital Vozandes
2022
Universidad UTE
2017-2021
University of Exeter
2021
Sapienza University of Rome
2012-2019
Hospital Español
2019
National Center for Healthy Housing
2018
Pontificia Universidad Católica del Ecuador
1995-2017
In vivo microdialysis was used to assess the involvement of ventral tegmental area (VTA) mu, delta, and kappa opioid receptors in modulation basal extracellular striatal dopamine (DA) DA-metabolite concentrations. Independent groups chloral hydrate-anesthetized rats were given VTA microinjections selective agonists, DA concentrations assayed using HPLC. [D-Ala2, N-Me-Phe4-Gly5-ol]-enkephalin (DAMGO; a mu agonist) [D-Pen2, D-Pen5]-enkephalin (DDDPE; delta each caused dose-orderly increases...
Gene therapy for Canavan disease results in a decrease pathologically elevated N -acetyl-aspartate concentrations the brain and long-term clinical stabilization.
This clinical protocol describes virus-based gene transfer for Canavan disease, a childhood leukodystrophy. also known as Van Bogaert-Bertrand is monogeneic, autosomal recessive disease in which the coding enzyme aspartoacylase (ASPA) defective. The lack of functional leads to an increase central nervous system substrate molecule, N-acetyl-aspartate (NAA), impairs normal myelination and results spongiform degeneration brain. No effective treatment currently exists; however, has potential...
Canavan disease is a rare leukodystrophy with no current treatment. rAAV-ASPA has been developed for gene delivery to the central nervous system (CNS) disease. This study represents first use of viral vector in an attempt ameliorate neurodegenerative disorder.Subjects received intracranial infusions via six cranial burr holes. Adeno-associated virus, serotype 2 (AAV2), mediated intraparenchymal human aspartoacylase cDNA at maximum dose 1 x 10(12) genomes per subject. The immune response and...
The brain is generally considered immunoprivileged, although increasing examples of immunological responses to antigens, neuronal expression major histocompatibility class I genes, and neurological autoimmunity have been recognized. An adeno-associated virus (AAV) vaccine generated autoantibodies that targeted a specific protein, the NR1 subunit N -methyl- d -aspartate (NMDA) receptor. After peroral administration AAV vaccine, transgene persisted for at least 5 months was associated with...
Brain levels of glucose and lactate in the extracellular fluid (ECF), which reflects environment to neurons are exposed, have never been studied humans under conditions varying glycemia. The authors used intracerebral microdialysis conscious human subjects undergoing electro-physiologic evaluation for medically intractable epilepsy measured ECF basal during a hyperglycemia–hypoglycemia clamp study. Only measurements from nonepileptogenic areas were included. Under conditions, found metabolic...
Abstract Extracellular dopamine and DOPAC (3,4‐dihydroxyphenylacetic acid) levels in nucleus accumbens were sampled by microdialysis quantified with high‐ performance liquid chromatography during intravenous heroin self‐administration sessions rats. Dopamine 10 20 min samples elevated following the first injection of each session, reaching a plateau elevation within two or three injections falling back toward baseline only when drug access was terminated. Elevations range 150–300% unit...
The transcription factor Gata6 regulates proliferation and differentiation of epithelial endocrine cells cancers. Among hematopoietic cells, is expressed selectively in resident peritoneal macrophages. We thus examined whether the loss macrophage compartment affected macrophages, using Lyz2-Cre x Gata6flox/flox mice to tackle this issue. In mice, compartment, but not macrophages other organs, was contracted, with only a third normal number remaining. Heightened rates death explained marked...
Symptoms of Parkinson's disease (PD) progress despite optimized medical treatment. The present study investigated the effects a flexibility and relaxation programme, walking, Nordic walking (NW) on speed, stride length, length variability, Parkinson-specific disability (UPDRS), health-related quality life (PDQ 39). 90 PD patients were randomly allocated to 3 treatment groups. Patients participated in 6-month with exercise sessions per week, each lasting 70 min. Assessment after completion...
Abstract Background The use of human embryonic stem cells (hESCs) in research is increasing and hESCs hold the promise for many biological, clinical toxicological studies. Human ESCs are expected to be chromosomally stable since karyotypic changes represent a pitfall potential future applications. Recently, several studies have analysed genomic stability hESC lines maintained after prolonged vitro culture but controversial data has been reported. Here, we prompted compare chromosomal three...
Abstract Over the past decades, consistent studies have shown that race/ethnicity a great impact on cancer incidence, survival, drug response, molecular pathways and epigenetics. Despite influence of in outcomes its health care quality, comprehensive understanding racial/ethnic inclusion oncological research has never been addressed. We therefore explored composition samples/individuals included fundamental (patient-derived models, biobanks genomics) applied (clinical trials). Regarding...
With the ultimate goal of developing safe and effective in vivo gene therapy for treatment Canavan disease other neurological disorders, we developed a non–viral lipid-entrapped, polycation-condensed delivery system (LPD) central nervous transfer, conjunction with adeno-associated virus (AAV)–based plasmids containing recombinant aspartoacylase (ASPA). The was tested healthy rodents primates, before proceeding to preliminary studies 2 children disease. Toxicity expression testing first...
To determine the relationship between circulating metabolic fuels and their local concentrations in peripheral tissues we measured glycerol, glucose, amino acids by microdialysis muscle adipose interstitium of 10 fasted, nonobese human subjects during (a) baseline, (b) euglycemic hyperinsulinemia (3 mU/kg per min for 3 h) and, (c) norepinephrine reuptake blockade (NOR). At interstitial glycerol was strikingly higher (P < 0.0001) (3710 microM) tissue (2760 compared with plasma (87 microM),...
Abstract The molecular mechanisms underlying the genesis and progression of oligodendroglial tumors are poorly understood, since only restricted information on loss heterozygosity from isolated cases is available. commonest alterations appear to involve deletion 1p 19q, while for 9p, chromosome 10 or epidermal growth factor receptor gene amplification have been described in single tumors. We applied restriction fragment length polymorphism analysis 14 loci covering 1 7 19 a series 25 with an...