Gene therapy for lysosomal acid lipase deficiency (lal-d)

Abstract

The disclosure provides gene therapy vectors, such as adeno-associated virus (AAV), designed for treatment of Lysosomal Acid Lipase Deficiency (LAL-D) disorders, such as Wolman disease and cholesteryl ester storage disease (CESD), nonalcoholic fatty liver disease (NAFLD), or nonalcoholic steatohepatitis (NASH). The disclosed rAAV provide a wild type lipase A (LIPA) cDNA to a subject in need which results in expression of the wild type protein.

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