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

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

Lysosomal acid lipase deficiency (LAL-D) presents as one of two rare autosomal recessive diseases: Wolman disease (WD), a severe disorder presenting in infancy characterized by absent or very low LAL activity, and cholesteryl ester storage disease (CESD), a less severe, later onset disease form. Rec...

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Bibliographic Details
Main Authors: Patricia Lam, Anna Ashbrook, Deborah A. Zygmunt, Cong Yan, Hong Du, Paul T. Martin
Format: Article
Language:English
Published: Elsevier 2022-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
liver disease
lysosomal storage disorder
lysosomal acid lipase
cholesterol
triglyceride
gene therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050122001115

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http://www.sciencedirect.com/science/article/pii/S2329050122001115

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