Therapeutic efficacy of rscAAVrh74.miniCMV.LIPA gene therapy in a mouse model of lysosomal acid lipase deficiency

Author:

Lam Patricia,Ashbrook Anna,Zygmunt Deborah A.,Yan Cong,Du Hong,Martin Paul T.ORCID

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference56 articles.

1. Lysosomal acid lipase: from cellular lipid handler to immunometabolic target;Gomaraschi;Trends Pharmacol. Sci.,2019

2. Lysosomal acid lipase in lipid metabolism and beyond;Li;Arterioscler. Thromb. Vasc. Biol.,2019

3. Targeting wolman disease and cholesteryl ester storage disease: disease pathogenesis and therapeutic development;Aguisanda;Curr. Chem. Genom. Transl. Med.,2017

4. Lysosomal acid lipase deficiency: therapeutic options;Pastores;Drug Des. Dev. Ther.,2020

5. Generalized xanthomatosis with calcified adrenals;Abramov;AMA J. Dis. Child.,1956

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