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Molecular Therapy, ISSN 1525-0016, 03/2018, Volume 26, Issue 3, pp. 801 - 813
We describe a novel, two-nanoparticle mRNA delivery system and show that it is highly effective as a means of intracellular enzyme replacement therapy (i-ERT)... 
ornithine transcarbamylase deficiency | inherited metabolic disorder | lipid nanoparticle | OTCD | intracellular enzyme replacement therapy | hybrid mRNA technology delivery system | Otcspf -ash mice | mRNA delivery | i-ERT | LNP | mice | Otc | OTC DEFICIENCY | MEDICINE, RESEARCH & EXPERIMENTAL | EFFICACY | SIRNA DELIVERY | SPF(ASH) MICE | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | IMMUNE-RESPONSES | HEMOPHILIA-B | IN-VIVO | FACTOR-IX | GENETICS & HEREDITY | SUCCESSFUL GENE-THERAPY | VECTORS | Ornithine Carbamoyltransferase Deficiency Disease - therapy | RNA, Small Interfering - genetics | RNA, Messenger - genetics | Male | Ornithine Carbamoyltransferase Deficiency Disease - metabolism | Ornithine Carbamoyltransferase - genetics | Mice, Knockout | Ornithine Carbamoyltransferase Deficiency Disease - genetics | Nanoparticles | Animals | Metabolic Networks and Pathways | Micelles | RNA, Messenger - administration & dosage | Ornithine Carbamoyltransferase - metabolism | Urea - metabolism | Mice | Polymers | Nanotechnology | Disease Models, Animal | Genetic Therapy - methods | Enzymes | Animal models | Particle size | Disease | Polymer solubility | Liver | Lipids | Protein biosynthesis | mRNA | Vectors (Biology) | Proteins | Studies | Orotic acid | Ammonia | Histopathology | Hepatocytes | Polyethylene glycol | Software | Ornithine | Deoxyribonucleic acid--DNA | Original
Journal Article
Medicine, ISSN 0025-7974, 12/2018, Volume 97, Issue 51, p. e13843
Rationale: Graft-derived-cell-free DNA (Gcf-DNA) in plasma was a promising biomarker to monitor graft-rejection transplantation. However, little is known about... 
MEDICINE, GENERAL & INTERNAL | ASSAY | graft injury | QUANTIFICATION | REJECTION | Y-chromosome method | graft-derived cell-free DNA | METABOLIC-DISORDERS | living donor liver transplantation | UNIVERSAL BIOMARKER | Case studies | Complications and side effects | Care and treatment | DNA | Liver | Ornithine transcarbamylase deficiency | Transplantation | Diagnosis | Research | Risk factors
Journal Article
Biochemical and Biophysical Research Communications, ISSN 0006-291X, 09/2015, Volume 465, Issue 2, pp. 174 - 179
Journal Article
CASE REPORT - Ornithine transcarbamylase deficiency in a girl with hyperkinetic behaviour, 04/2004
Congenital ornithine transcarbamylase deficiency (OTCD, OMIM 311250, Xp21.1) in humans results in hyperammonaemia with subsequent neurological symptoms... 
Neurology | Pediatrics | pn04019 ornithine transcarbamylase deficiency, hyperammonaemia, encephalopathy, glutamine, MR-spectroscopy
Journal
Molecular Genetics and Metabolism, ISSN 1096-7192, 03/2018, Volume 123, Issue 3, pp. 297 - 300
Journal Article
PLoS ONE, ISSN 1932-6203, 04/2016, Volume 11, Issue 4, p. e0153358
Journal Article
Molecular Genetics and Metabolism, ISSN 1096-7192, 2003, Volume 80, Issue 1, pp. 148 - 158
Journal Article