Search Results - "Bloemendaal, Lysbeth Ten"
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The P4-ATPase ATP9A is a novel determinant of exosome release
Published in PloS one (04-04-2019)“…Extracellular vesicles (EVs) released by cells have a role in intercellular communication to regulate a wide range of biological processes. Two types of EVs…”
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2
Reduced spontaneous itch in mouse models of cholestasis
Published in Scientific reports (17-03-2021)“…Pruritus is one of the most distressing symptoms in cholestatic patients. Plasma autotaxin (ATX) activity correlates with the severity of pruritus in…”
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3
Low efficacy of recombinant SV40 in Ugt1a1-/- mice with severe inherited hyperbilirubinemia
Published in PloS one (01-01-2021)“…In contrast to AAV, Simian Virus 40 (rSV40) not inducing neutralizing antibodies (NAbs) allowing re-treatment seems a promising vector for neonatal treatment…”
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4
Adeno-associated viral vector serotype 5 poorly transduces liver in rat models
Published in PloS one (27-12-2013)“…Preclinical studies in mice and non-human primates showed that AAV serotype 5 provides efficient liver transduction and as such seems a promising vector for…”
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Long-Term Effects of Biliverdin Reductase a Deficiency in Ugt1 -/- Mice: Impact on Redox Status and Metabolism
Published in Antioxidants (20-12-2021)“…Accumulation of neurotoxic bilirubin due to a transient neonatal or persistent inherited deficiency of bilirubin glucuronidation activity can cause…”
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Liver-directed gene therapy results in long-term correction of progressive familial intrahepatic cholestasis type 3 in mice
Published in Journal of hepatology (01-07-2019)“…[Display omitted] •Adeno-associated virus (AAV)-mediated gene therapy can correct Abcb4 deficiency (PFIC3) in mice.•By restoring phospholipid transport to…”
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The Phospholipid Flippase ATP8B1 is Involved in the Pathogenesis of Ulcerative Colitis via Establishment of Intestinal Barrier Function
Published in Journal of Crohn's and colitis (06-08-2024)“…Patients with mutations in ATP8B1 develop progressive familial intrahepatic cholestasis type 1 [PFIC1], a severe liver disease that requires life-saving liver…”
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Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients
Published in Molecular therapy. Methods & clinical development (12-03-2021)“…A clinical trial using adeno-associated virus serotype 8 (AAV8)-human uridine diphosphate glucuronosyltransferase 1A1 (hUGT1A1) to treat inherited severe…”
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9
Mycophenolate mofetil impairs transduction of single-stranded adeno-associated viral vectors
Published in Human gene therapy (01-05-2011)“…Adeno-associated virus (AAV) liver-directed gene therapy seems a feasible treatment for Crigler-Najjar syndrome type I, an inherited liver disorder…”
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10
A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene
Published in Molecular therapy. Methods & clinical development (11-09-2020)“…Potency assessment of clinical-grade vector lots is crucial to support adeno-associated virus (AAV) vector release and is required for future marketing…”
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11
Insulin-like growth factor 1 enhances bile-duct proliferation and fibrosis in Abcb4−/− mice
Published in Biochimica et biophysica acta (01-06-2013)“…Adamant progression of chronic cholangiopathies towards cirrhosis and limited therapeutic options leave a liver transplantation the only effective treatment…”
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12
Cre Recombinase Mediates the Removal of Bacterial Backbone to Efficiently Generate rSV40
Published in Molecular therapy. Methods & clinical development (15-06-2018)“…Gene therapy has been shown to be a feasible approach to treat inherited disorders . Among the currently used viral vector systems, adeno-associated virus…”
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13
Novel immortalized human fetal liver cell line, cBAL111, has the potential to differentiate into functional hepatocytes
Published in BMC biotechnology (21-10-2009)“…A clonal cell line that combines both stable hepatic function and proliferation capacity is desirable for in vitro applications that depend on hepatic…”
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14
Increased reproducibility of quantitative reverse transcriptase–PCR
Published in Analytical biochemistry (15-05-2005)Get full text
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15
In vitro functionality of human fetal liver cells and clonal derivatives under proliferative conditions
Published in Cell transplantation (01-01-2006)“…Mature human hepatocytes are not suitable for large-scale in vitro applications that rely on hepatocyte function, due to their limited availability and…”
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16
Assessment of in vitro applicability of reversibly immortalized NKNT-3 cells and clonal derivatives
Published in Cell transplantation (01-05-2006)“…In vitro applications of human hepatocytes, such as bioartificial livers and toxicity assays, require thoroughly testing of human cell lines prior to using…”
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17
Low efficacy of recombinant SV40 in Ugt1a1.sup.-/- mice with severe inherited hyperbilirubinemia
Published in PloS one (23-04-2021)“…In contrast to AAV, Simian Virus 40 (rSV40) not inducing neutralizing antibodies (NAbs) allowing re-treatment seems a promising vector for neonatal treatment…”
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Journal Article -
18
Time-related analysis of metabolic liver functions, cellular morphology, and gene expression of hepatocytes cultured in the bioartificial liver of the Academic Medical Center in Amsterdam (AMC-BAL)
Published in Tissue engineering (01-06-2007)“…A comprehensive understanding of the mechanisms that underlie hepatic differentiation inside a bioartificial liver (BAL) device is obtained when functional,…”
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19
Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I
Published in Molecular therapy. Methods & clinical development (12-12-2024)“…Crigler-Najjar syndrome is an ultra-rare monogenic recessive liver disease caused by UGT1A1 gene mutations. Complete UGT1A1 deficiency results in severe…”
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20
Efficacy of AAV8-h UGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients
Published in Molecular therapy. Methods & clinical development (12-03-2021)“…A clinical trial using adeno-associated virus serotype 8 (AAV8)-human uridine diphosphate glucuronosyltransferase 1A1 (h ) to treat inherited severe…”
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