Prime editing is a highly versatile CRISPR-based genome editing technology that works
without DNA double-strand break formation. Despite rapid technological advances, in vivo …

Resolvin D1 (RvD1) was shown to be a potent anti-inflammatory and proresolution lipid
mediator in several animal models of inflammation, but its mechanism of action in humans is not …

Ornithine transcarbamylase deficiency (OTCD) is a monogenic disease of ammonia metabolism
in hepatocytes. Severe disease is frequently treated by orthotopic liver transplantation. …

Hepatic gene therapy by delivering non-integrating therapeutic vectors in newborns remains
challenging due to the risk of dilution and loss of efficacy in the growing liver. Previously we …

Obesity leads to chronic inflammation of the adipose tissue which is tightly associated with
the metabolic syndrome, type 2 diabetes and cardiovascular disease. Inflammation of the …

Hydrodynamic tail vein injection (HTV) is the "gold standard" for delivering naked DNA vectors
to mouse liver, thereby transfecting predominately perivenous hepatocytes. While HTV …

Phenylalanine hydroxylase (PAH) deficiency, colloquially known as phenylketonuria (PKU),
is among the most common inborn errors of metabolism and in the past decade has become …

The most common ureagenesis defect is X‐linked ornithine transcarbamylase (OTC)
deficiency which is a main target for novel therapeutic interventions. The spf ash mouse model …

… Nicole Rimann, Hiu Man Grisch-Chan and Beat Thöny designed the animal experiments.
Nicole Rimann … Nicole Rimann performed amino acids and neurotransmitter analyses. Yvonne …

Background Tyrosinemia type 1 (HT1) is a rare metabolic disorder caused by a defect in the
tyrosine catabolic pathway. Since HT1 patients are treated with NTBC, outcome improved …