CRISPR–Cas genome-editing nucleases hold substantial promise for developing human
therapeutic applications 1 , 2 , 3 , 4 , 5 – 6 but identifying unwanted off-target mutations is …

Osteogenesis imperfecta (OI) is a hereditary disease occurring in humans and dogs. It is
characterized by extremely fragile bones and teeth. Most human and some canine OI cases are …

Porcine circovirus type 2 (PCV 2) represents a widespread, globally occurring pathogen
with an increasing number of associated entities. To further elucidate the origin, spread and …

Lipid nanoparticles (LNPs) are the most clinically advanced delivery system for RNA-based
drugs but have predominantly been investigated for intravenous and intramuscular …

Background Plasma concentration of low-density lipoprotein (LDL) cholesterol is a well-established
risk factor for cardiovascular disease. Inhibition of proprotein convertase subtilisin/…

Theiler murine encephalomyelitis (TME) represents a highly relevant viral model for multiple
sclerosis. Matrix metalloproteinases (MMPs) degrade extracellular matrix molecules and …

The CRISPR-Cas9 system has increased the speed and precision of genetic editing in cells
and animals. However, model generation for drug development is still expensive and time-…

This article provides guidelines for organ and tissue sampling adapted to porcine animal
models in translational medical research. Detailed protocols for the determination of sampling …

α1-antitrypsin (AAT) is a circulating serine protease inhibitor secreted from the liver and
important in preventing proteolytic neutrophil elastase associated tissue damage, primarily in …

Despite enormous advances in translational biomedical research, there remains a growing
demand for improved animal models of human disease. This is particularly true for diseases …