Recent Developments in Gene Therapy for Homozygous Familial Hypercholesterolemia

Adeno-associated virus serotypes for gene therapeutics

(2015) Leszek Lisowski et al.   CURRENT OPINION IN PHARMACOLOGY

Engineering the AAV capsid to optimize vector–host-interactions

(2015) Hildegard Büning et al.   CURRENT OPINION IN PHARMACOLOGY

Concise Review: Cell Therapies for Hereditary Metabolic Liver Diseases-Concepts, Clinical Results, and Future Developments

(2015) Tobias Cantz et al.   STEM CELLS

Restoration of Physiologically Responsive Low-Density Lipoprotein Receptor-Mediated Endocytosis in Genetically Deficient Induced Pluripotent Stem Cells

(2015) Venkat M. Ramakrishnan et al.   Scientific Reports

AAV Vectors Expressing LDLR Gain-of-Function Variants Demonstrate Increased Efficacy in Mouse Models of Familial Hypercholesterolemia

(2014) Suryanarayan Somanathan et al.   CIRCULATION RESEARCH

Homozygous autosomal dominant hypercholesterolaemia in the Netherlands: prevalence, genotype–phenotype relationship, and clinical outcome

(2014) Barbara Sjouke et al.   EUROPEAN HEART JOURNAL

Homozygous familial hypercholesterolaemia: new insights and guidance for clinicians to improve detection and clinical management. A position paper from the Consensus Panel on Familial Hypercholesterolaemia of the European Atherosclerosis Society

(2014) M. Cuchel et al.   EUROPEAN HEART JOURNAL

Neutralizing antibodies against AAV2, AAV5 and AAV8 in healthy and HIV-1-infected subjects in China: implications for gene therapy using AAV vectors

(2014) Q Liu et al.   GENE THERAPY

Engineering adeno-associated viruses for clinical gene therapy

(2014) Melissa A. Kotterman et al.   NATURE REVIEWS GENETICS

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells

(2013) A. T. Martino et al.   BLOOD

Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes

(2013) Daniel J Hui et al.   MOLECULAR THERAPY

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

(2013) Leszek Lisowski et al.   NATURE

The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy

(2012) J. D. Finn et al.   BLOOD

Gene therapy for lipoprotein lipase deficiency

(2012) Daniel Gaudet et al.   CURRENT OPINION IN LIPIDOLOGY

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

(2012) D Gaudet et al.   GENE THERAPY

JD induced pluripotent stem cell-derived hepatocytes faithfully recapitulate the pathophysiology of familial hypercholesterolemia

(2012) Max A. Cayo et al.   HEPATOLOGY

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

Disease-Corrected Hepatocyte-Like Cells from Familial Hypercholesterolemia-Induced Pluripotent Stem Cells

(2012) Faranak Fattahi et al.   MOLECULAR BIOTECHNOLOGY

Reduction in Mortality in Subjects With Homozygous Familial Hypercholesterolemia Associated With Advances in Lipid-Lowering Therapy

(2011) Frederick J. Raal et al.   CIRCULATION

Gene Therapy for Familial Hypercholesterolemia

(2011) Eline Van Craeyveld et al.   CURRENT PHARMACEUTICAL DESIGN

RNAi-mediated knockdown of HMG CoA reductase enhances gene expression from physiologically regulated low-density lipoprotein receptor therapeutic vectors in vivo

(2011) O Hibbitt et al.   GENE THERAPY

Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

(2011) Dawn E Bowles et al.   MOLECULAR THERAPY

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Liver transplantation as a treatment option for three siblings with homozygous familial hypercholesterolemia

(2011) Küçükkartallar Tevfik et al.   PEDIATRIC TRANSPLANTATION

Translational Lessons from a Case of Combined Heart and Liver Transplantation for Familial Hypercholesterolemia 20 Years Post-operatively

(2011) Michael Ibrahim et al.   Journal of Cardiovascular Translational Research

Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors

(2010) Sylvie Boutin 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

Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial

(2010) Frederick J Raal et al.   LANCET

Dystrophin Immunity in Duchenne's Muscular Dystrophy

(2010) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

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

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

(2009) Roberto Calcedo et al.   JOURNAL OF INFECTIOUS DISEASES

Cellular immune response to cryptic epitopes during therapeutic gene transfer

(2009) C. Li et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Longitudinal Evaluation and Assessment of Cardiovascular Disease in Patients With Homozygous Familial Hypercholesterolemia

(2008) Daniel M. Kolansky et al.   AMERICAN JOURNAL OF CARDIOLOGY

Undetectable Transcription of cap in a Clinical AAV Vector: Implications for Preformed Capsid in Immune Responses

(2008) Bernd Hauck et al.   MOLECULAR THERAPY