LIPA and lipodystrophy: Similar to humans, LAL knockout (Lal−/−) mice exhibit lipodystrophy, ectopic lipid accumulation, progressive hepatosplenomegaly, and dyslipidemia.10, 11, 12 The phenotype of Lal−/− mice resembles late-onset LAL-D, despite having no residual LAL activity.10, 11, 12 Lal−/− mice were used to test various treatment approaches for LAL-D, including enzyme replacement therapy (ERT) and gene therapy.13