Development and rescue of human familial hypercholesterolaemia in a xenograft mouse model

Lipoprotein(a) Concentrations, Rosuvastatin Therapy, and Residual Vascular Risk

(2013) Amit V. Khera et al.   CIRCULATION

JCL Roundtable: Diagnosis of severe familial hypercholesterolemia

(2013) W. Virgil Brown et al.   Journal of Clinical Lipidology

Lipoprotein(a): resurrected by genetics

(2013) F. Kronenberg et al.   JOURNAL OF INTERNAL MEDICINE

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

(2013) Leszek Lisowski et al.   NATURE

New potential cell source for hepatocyte transplantation: Discarded livers from metabolic disease liver transplants

(2013) Roberto Gramignoli et al.   Stem Cell Research

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

(2012) Sadik H. Kassim et al.   HUMAN GENE THERAPY

Evaluation of Adeno-Associated Viral Vectors for Liver-Directed Gene Transfer in Dogs

(2011) Peter Bell et al.   HUMAN GENE THERAPY

Familial Hypercholesterolemias: Prevalence, genetics, diagnosis and screening recommendations from the National Lipid Association Expert Panel on Familial Hypercholesterolemia

(2011) Paul N. Hopkins et al.   Journal of Clinical Lipidology

Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells

(2011) Kosuke Yusa et al.   NATURE

Natural killer T cells in lipoprotein metabolism and atherosclerosis

(2011) Godfrey Getz et al.   THROMBOSIS AND HAEMOSTASIS

Rapid, Simple, and Versatile Manufacturing of Recombinant Adeno-Associated Viral Vectors at Scale

(2010) Martin Lock et al.   HUMAN GENE THERAPY

Induced pluripotent stem cell–derived hepatocytes have the functional and proliferative capabilities needed for liver regeneration in mice

(2010) Silvia Espejel et al.   JOURNAL OF CLINICAL INVESTIGATION

Modeling inherited metabolic disorders of the liver using human induced pluripotent stem cells

(2010) S. Tamir Rashid et al.   JOURNAL OF CLINICAL INVESTIGATION

Human liver chimeric mice provide a model for hepatitis B and C virus infection and treatment

(2010) Karl-Dimiter Bissig et al.   JOURNAL OF CLINICAL INVESTIGATION

Biological, clinical and population relevance of 95 loci for blood lipids

(2010) Tanya M. Teslovich et al.   NATURE

Gene Therapy in a Humanized Mouse Model of Familial Hypercholesterolemia Leads to Marked Regression of Atherosclerosis

(2010) Sadik H. Kassim et al.   PLoS One

Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints

(2009) L H Vandenberghe et al.   GENE THERAPY

The Pleiotropic Effects of Natural AAV Infections on Liver-directed Gene Transfer in Macaques

(2009) Lili Wang et al.   MOLECULAR THERAPY

Systematic Evaluation of AAV Vectors for Liver directed Gene Transfer in Murine Models

(2009) Lili Wang et al.   MOLECULAR THERAPY

Vascular Calcifications in Homozygote Familial Hypercholesterolemia

(2008) Z. Awan et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY