Adeno-Associated Virus Serotype 8 Gene Therapy Leads to Significant Lowering of Plasma Cholesterol Levels in Humanized Mouse Models of Homozygous and Heterozygous Familial Hypercholesterolemia

Author:

Kassim Sadik H.1,Li Hui2,Bell Peter1,Somanathan Suryanarayan1,Lagor William2,Jacobs Frank1,Billheimer Jeffrey2,Wilson James M.1,Rader Daniel J.2

Affiliation:

1. Gene Therapy Program, Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104.

2. Cardiovascular Institute and Institute for Translational Medicine and Therapeutics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

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