LIPA and Hepatosplenomegaly: Intravenous injection of rscAAVrh74.miniCMV.LIPA largely corrected many of the phenotypes of LAL-D in a Lipa−/− mouse model, including hepatosplenomegaly, elevated serum transaminases, reduced LAL activity, and cholesterol and triglyceride accumulation in organs, demonstrating that AAV gene therapy may be a viable approach to treating LAL-D.