Induction of Sustained Hypercholesterolemia by Single Adeno-Associated Virus–Mediated Gene Transfer of Mutant hPCSK9 | Zendy

Marta Roche-Molina | Zendy; David SanzRosa | Zendy; Francisco M. Cruz | Zendy; Jaime GarcíaPrieto | Zendy; Sergio López | Zendy; Rocı́o Abia | Zendy; Francisco J.G. Muriana | Zendy; Valentı́n Fuster | Zendy; Borja Ibáñez | Zendy; Juan A. Bernal | Zendy

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Induction of Sustained Hypercholesterolemia by Single Adeno-Associated Virus–Mediated Gene Transfer of Mutant hPCSK9

Author(s) -

Marta Roche-Molina,

David SanzRosa,

Francisco M. Cruz,

Jaime GarcíaPrieto,

Sergio López,

Rocı́o Abia,

Francisco J.G. Muriana,

Valentı́n Fuster,

Borja Ibáñez,

Juan A. Bernal

Publication year - 2014

Publication title -

arteriosclerosis thrombosis and vascular biology

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 3.007

H-Index - 270

eISSN - 1524-4636

pISSN - 1079-5642

DOI - 10.1161/atvbaha.114.303617

Subject(s) - adeno associated virus , mutant , gene transfer , familial hypercholesterolemia , gene , genetic enhancement , biology , virology , virus , medicine , genetics , endocrinology , cholesterol , vector (molecular biology) , recombinant dna

Patients with mutations in the proprotein convertase subtilisin/kexin type 9 (PCSK9) gene have hypercholesterolemia and are at high risk of adverse cardiovascular events. We aimed to stably express the pathological human D374Y gain-of-function mutant form of PCSK9 (PCSK9(DY)) in adult wild-type mice to generate a hyperlipidemic and proatherogenic animal model, achieved with a single systemic injection with adeno-associated virus (AAV).

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