Table 1.
Summary of Disease Parameters in Preclinical Modelsa
Mouse | Cat | Human | |
---|---|---|---|
Genotype | LPL−/− | LPLG412R (catalytically inactive mutant) | Multiple |
Ad rescue at birth | Yes | No | No |
Immune response to hLPL | No | Yes | No |
Hypertriglyceridemia | Yes | Yes | Yes |
Pancreatitis | No | Yes | Yes |
Muscle damage | Yes | No | No |
The murine lipoprotein lipase deficiency (LPLD) model exhibits several characteristics distinct from the disease in humans, including a requirement for adenovirus-mediated rescue of LPL expression at birth, preexisting muscle damage and lipid accumulation, and absence of pancreatitis. The feline model more faithfully recapitulates the features of the human disease.
Ad, adenovirus; hLPL, human lipoprotein lipase.