A5045790555- Inflammatory Bowel Disease
- Microscopic Colitis
- Celiac Disease Research and Management
- Cardiac Ischemia and Reperfusion
- Eosinophilic Esophagitis
- Virus-based gene therapy research
- Immunodeficiency and Autoimmune Disorders
- Pancreatitis Pathology and Treatment
- Helicobacter pylori-related gastroenterology studies
- Tryptophan and brain disorders
- Cardiac and Coronary Surgery Techniques
- Digestive system and related health
- Cardiac Valve Diseases and Treatments
- Transplantation: Methods and Outcomes
- Organ Transplantation Techniques and Outcomes
- Adolescent and Pediatric Healthcare
- RNA Interference and Gene Delivery
- CRISPR and Genetic Engineering
- Viral gastroenteritis research and epidemiology
- Genetic Neurodegenerative Diseases
- Clinical Nutrition and Gastroenterology
- Aortic Disease and Treatment Approaches
- Pancreatic and Hepatic Oncology Research
- Viral Infectious Diseases and Gene Expression in Insects
- Mitochondrial Function and Pathology
University Hospital in Halle
2021-2025
Semmelweis University
2011-2024
University of Debrecen
2009-2024
University of Pecs
2017-2024
Martin Luther University Halle-Wittenberg
2020-2024
Heidelberg University
2014-2024
University Hospital Heidelberg
2014-2024
University of Szeged
2014-2023
Deutsches Herzzentrum München
2022-2023
Bluebird Bio (United States)
2016-2022
Slowing Brain Disease with Gene Therapy X-linked adrenoleukodystrophy (ALD), the hereditary brain demyelinating disorder that was featured in movie “ Lorenzo's Oil ,” is typically treated by transplantation of bone marrow from matched donors. This treatment slows progression disease introducing cells differentiate into myelin-producing cells. Cartier et al. (p. 818 ; see Perspective Naldini ) tested an alternative gene therapy–based approach two young patients without A lentiviral vector...
The diagnosis of pediatric-onset inflammatory bowel disease (PIBD) can be challenging in choosing the most informative diagnostic tests and correctly classifying PIBD into its different subtypes. Recent advances our understanding natural history phenotype PIBD, increasing availability serological fecal biomarkers, emergence novel endoscopic imaging technologies taken together have made previous Porto criteria for obsolete.We aimed to revise original using an evidence-based approach consensus...
Children and adolescents with Crohn's disease (CD) present often a more complicated course compared to adult patients. In addition, the potential impact of CD on growth, pubertal emotional development patients underlines need for specific management strategy pediatric-onset CD. To develop first evidenced based consensus driven guidelines an expert panel 33 IBD specialists was formed after open call within European Colitis Organisation Society Pediatric Gastroenterolog, Hepatology Nutrition....
Sickle cell disease results from a homozygous missense mutation in the β-globin gene that causes polymerization of hemoglobin S. Gene therapy for patients with this disorder is complicated by complex cellular abnormalities and challenges achieving effective, persistent inhibition We describe our first patient treated lentiviral vector-mediated addition an antisickling into autologous hematopoietic stem cells. Adverse events were consistent busulfan conditioning. Fifteen months after...
Donor availability and transplantation-related risks limit the broad use of allogeneic hematopoietic-cell transplantation in patients with transfusion-dependent β-thalassemia. After previously establishing that lentiviral transfer a marked β-globin (βA-T87Q) gene could substitute for long-term red-cell transfusions patient β-thalassemia, we wanted to evaluate safety efficacy such therapy
The contemporary management of ambulatory ulcerative colitis (UC) continues to be challenging with ∼20% children needing a colectomy within childhood years. We thus aimed standardize daily treatment pediatric UC and inflammatory bowel diseases (IBD)-unclassified through detailed recommendations practice points.These guidelines are joint effort the European Crohn's Colitis Organization (ECCO) Paediatric IBD Porto group Society Gastroenterology, Hepatology Nutrition (ESPGHAN). An extensive...
ABSTRACT Background and Aims: Pediatric ulcerative colitis (UC) shares many features with adult‐onset disease but there are some unique considerations; therefore, therapeutic approaches have to be adapted these particular needs. We aimed formulate guidelines for managing UC in children based on a systematic review (SR) of the literature robust consensus process. The present article is product joint effort European Crohn's Colitis Organization (ECCO) Society Paediatric Gastroenterology,...
Progressive multiple sclerosis (MS) is a chronic disease with unique pattern, which histologically classified into the subpial type 3 lesions in autopsy. The lesion also homologous to that of cuprizone (CPZ) toxin-induced animal models demyelination. Aberration tryptophan (TRP)-kynurenine (KYN) metabolic system has been observed patients MS; nevertheless, KYN metabolite profile progressive MS remains inconclusive. In this study, C57Bl/6J male mice were treated 0.2% CPZ toxin for 5 weeks and...
Recent studies have opened the possibility that quiescent, G 0 /G 1 hematopoietic stem cells (HSC) can be gene transduced; lentiviruses (such as HIV type 1, HIV) encode proteins permit transport of viral genome into nucleus nondividing cells. We and others recently demonstrated efficient transduction by using an HIV-1-based vector delivery system various human cell types including CD34 + or terminally differentiated neurons. Here we compare efficiency two vectors, HIV-based murine leukemia...
The ornithine transcarbamylase-deficient sparse fur mouse is an excellent model to study the most common human urea cycle disorder. mutation has been well characterized by both biochemical and enzymological methods, but its exact nature not revealed. A single base substitution in complementary DNA for transcarbamylase from identified means of a combination two recently described techniques rapid mutational analysis. This strategy simpler than conventional library construction, screening,...
Medical therapy for Crohn's disease (CD) has changed significantly over the past 20 years with increasing use of immunosuppressives. In contrast, surgery rates are still high and there is little evidence that outcomes CD have decades. The objective this study was to analyze evolution surgical medical in population-based Veszprem province database.Data 506 incident patients were analyzed (age at diagnosis: 31.5 years, s.d. 13.8 years). Both hospital outpatient records collected...
Summary Inflammatory bowel disease (IBD) may result from exaggerated stimulation of the mucosal immune system by luminal bacterial flora. Bacterial products are recognized pattern recognition receptors such as Toll-like (TLRs), which key regulators innate system. Therefore, expression TLR2, TLR3 and TLR4 in colonic biopsy samples taken children with active IBD were studied compared to controls. Colonic collected macroscopically inflamed non-inflamed regions mucosa 12 freshly diagnosed...
AAV2-sFLT01 is a vector that expresses modified soluble Flt1 receptor designed to neutralize the proangiogenic activities of vascular endothelial growth factor (VEGF) for treatment age-related macular degeneration (AMD) via an intravitreal injection. Owing minimal data available route administration adeno-associated virus (AAV), we initiated 12-month safety study administered intravitreally at doses 2.4 × 10(9) genomes (vg) and 10(10) vg cynomolgus monkeys. Expression sFlt01 protein peaked...
Adalimumab is a fully human monoclonal antibody targeting tumour necrosis factor with proven efficacy in the treatment of Crohn's disease (CD).To investigate predictors medium-term clinical and mucosal healing during adalimumab therapy, patients CD, specialised centres approved for biological therapy Hungary.Data capture 201 CD was standardised prospective (male/female: 112/89, median age: 33.0 years, duration: 8 years). Previous infliximab had been administered 48% patients, concomitant...
A recombinant adeno-associated virus serotype 2 Reference Standard Material (rAAV2 RSM) has been produced and characterized with the purpose of providing a reference standard for particle titer, vector genome infectious titer AAV2 gene transfer vectors. Production purification material were carried out by helper virus–free transient transfection chromatographic purification. The purified bulk was vialed, confirmed negative microbial contamination, then distributed characterization along...
Acute severe colitis (ASC) is one of the few emergencies in pediatric gastroenterology. Tight monitoring and timely medical surgical interventions may improve outcomes minimize morbidity mortality. We aimed to standardize daily treatment ASC children through detailed recommendations practice points which are based on a systematic review literature consensus experts.These guidelines joint effort European Crohn's Colitis Organization (ECCO) Society Paediatric Gastroenterology, Hepatology...
The aim of the study was to evaluate incidence, baseline disease characteristics, and location based on Paris classification in pediatric inflammatory bowel (IBD) Hungarian nationwide inception cohort. In addition, 1-year follow-up with therapy analyzed.From January 1, 2007 December 31, 2009, newly diagnosed patients IBD were prospectively registered. Twenty-seven gastroenterology centers participated data collection ensuring from whole country. Newly younger than 18 years reported. Disease...
A previously published clinical trial demonstrated the benefit of autologous CD34+ cells transduced with a selfinactivating lentiviral vector (HPV569) containing an engineered β-globin gene (βA-T87Q-globin) in subject β thalassemia major. This has been modified to increase transduction efficacy without compromising safety. In vitro analyses indicated that changes resulted both increased titers (3 4 fold) and (2 3 fold). An vivo study which 58 β-thalassemic mice were transplanted vector- or...
Hematopoietic stem cell transplantation (HSCT) is a therapy used for multiple malignant and nonmalignant diseases, with chemotherapy pretransplantation myeloablation. The post-HSCT brain contains peripheral engrafted parenchymal macrophages, despite their absence in the normal brain, engraftment mechanism still undefined. Here we show that HSCT broadly disrupts mouse regenerative populations, including permanent loss of adult neurogenesis. Microglial density was halved, causing microglial...
In 2005, the Inflammatory Bowel Disease (IBD) Working Group of European Society for Pediatric Gastroenterology, Hepatology, and Nutrition published consensus guidelines on diagnostic workup paediatric IBD, Porto criteria. According to these guidelines, children suspected having IBD should undergo an oesophagogastroduodenoscopy (OGD), ileocolonoscopy, (except in cases definitive ulcerative colitis) adequate imaging small bowel. To audit evaluate patients with Europe, created EUROKIDS, a...
Background— The role of the nitric oxide/cGMP/cGMP–dependent protein kinase G pathway in myocardial protection and preconditioning has been object intensive investigations. novel soluble guanylate cyclase activator cinaciguat reported to elevate intracellular [cGMP] activate vivo. We investigated effects on infarction induced by isoproterenol rats. Methods Results— Rats were treated orally twice a day for 4 days with vehicle or (10 mg/kg). Isoproterenol (85 mg/kg) was injected subcutaneously...