Non-Clinical Study Examining AAV8.TBG.hLDLR Vector-Associated Toxicity in Chow-Fed Wild-Type and LDLR<sup>+/-</sup> Rhesus Macaques.

Greig, Jenny A; Limberis, Maria P; Bell, Peter; Chen, Shu-Jen; Calcedo, Roberto; Rader, Daniel J; Wilson, James M

Non-Clinical Study Examining AAV8.TBG.hLDLR Vector-Associated Toxicity in Chow-Fed Wild-Type and LDLR^+/- Rhesus Macaques.

Greig, Jenny A; Limberis, Maria P; Bell, Peter; Chen, Shu-Jen; Calcedo, Roberto; Rader, Daniel J; Wilson, James M.

Afiliación

Greig JA; 1 Gene Therapy Program, Department of Medicine, University of Pennsylvania , Philadelphia, Pennsylvania.
Limberis MP; 2 Department of Pathology and Laboratory Medicine, University of Pennsylvania , Philadelphia, Pennsylvania.
Bell P; 1 Gene Therapy Program, Department of Medicine, University of Pennsylvania , Philadelphia, Pennsylvania.
Chen SJ; 1 Gene Therapy Program, Department of Medicine, University of Pennsylvania , Philadelphia, Pennsylvania.
Calcedo R; 1 Gene Therapy Program, Department of Medicine, University of Pennsylvania , Philadelphia, Pennsylvania.
Rader DJ; 3 Division of Translational Medicine and Human Genetics, Department of Medicine, University of Pennsylvania , Philadelphia, Pennsylvania.
Wilson JM; 4 Department of Genetics, University of Pennsylvania , Philadelphia, Pennsylvania.

Hum Gene Ther Clin Dev ; 28(1): 39-50, 2017 03.

Article en En | MEDLINE | ID: mdl-28319449

Asunto(s)

Desaminasas APOBEC-1/genética; Dependovirus/genética; Terapia Genética; Vectores Genéticos/administración & dosificación; Hiperlipoproteinemia Tipo II/terapia; Hígado/patología; Receptores de LDL/genética; Desaminasas APOBEC-1/deficiencia; Animales; Colesterol/sangre; Modelos Animales de Enfermedad; Femenino; Vectores Genéticos/toxicidad; Humanos; Hiperlipoproteinemia Tipo II/genética; Hígado/metabolismo; Macaca mulatta; Masculino; Mutación/genética; Receptores de LDL/deficiencia

Palabras clave

AAV; LDLR; familial hypercholesterolemia; gene therapy; low density lipoprotein receptor; toxicology

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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Receptores de LDL / Terapia Genética / Dependovirus / Desaminasas APOBEC-1 / Vectores Genéticos / Hiperlipoproteinemia Tipo II / Hígado Tipo de estudio: Risk_factors_studies Límite: Animals / Female / Humans / Male Idioma: En Revista: Hum Gene Ther Clin Dev Año: 2017 Tipo del documento: Article Pais de publicación: Estados Unidos

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