Related references
Note: Only part of the references are listed.Adeno-associated virus serotypes for gene therapeutics
Leszek Lisowski et al.
CURRENT OPINION IN PHARMACOLOGY (2015)
Engineering the AAV capsid to optimize vector-host-interactions
Hildegard Buening et al.
CURRENT OPINION IN PHARMACOLOGY (2015)
Concise Review: Cell Therapies for Hereditary Metabolic Liver Diseases-Concepts, Clinical Results, and Future Developments
Tobias Cantz et al.
STEM CELLS (2015)
Restoration of Physiologically Responsive Low-Density Lipoprotein Receptor-Mediated Endocytosis in Genetically Deficient Induced Pluripotent Stem Cells
Venkat M. Ramakrishnan et al.
SCIENTIFIC REPORTS (2015)
Homozygous autosomal dominant hypercholesterolaemia in the Netherlands: prevalence, genotype-phenotype relationship, and clinical outcome
Barbara Sjouke et al.
EUROPEAN HEART JOURNAL (2015)
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
Marina Cuchel et al.
TURK KARDIYOLOJI DERNEGI ARSIVI-ARCHIVES OF THE TURKISH SOCIETY OF CARDIOLOGY (2015)
AAV Vectors Expressing LDLR Gain-of-Function Variants Demonstrate Increased Efficacy in Mouse Models of Familial Hypercholesterolemia
Suryanarayan Somanathan et al.
CIRCULATION RESEARCH (2014)
Neutralizing antibodies against AAV2, AAV5 and AAV8 in healthy and HIV-1-infected subjects in China: implications for gene therapy using AAV vectors
Q. Liu et al.
GENE THERAPY (2014)
Selection and evaluation of clinically relevant AAV variants in a xenograft liver model
Leszek Lisowski et al.
NATURE (2014)
Engineering adeno-associated viruses for clinical gene therapy
Melissa A. Kotterman et al.
NATURE REVIEWS GENETICS (2014)
Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
A. C. Nathwani et al.
NEW ENGLAND JOURNAL OF MEDICINE (2014)
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
Marina Cuchel et al.
EUROPEAN HEART JOURNAL (2014)
Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells
Ashley T. Martino et al.
BLOOD (2013)
Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial
D. Gaudet et al.
GENE THERAPY (2013)
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
Sadik H. Kassim et al.
HUMAN GENE THERAPY (2013)
Disease-Corrected Hepatocyte-Like Cells from Familial Hypercholesterolemia-Induced Pluripotent Stem Cells
Faranak Fattahi et al.
MOLECULAR BIOTECHNOLOGY (2013)
Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes
Daniel J. Hui et al.
MOLECULAR THERAPY (2013)
The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy
Jonathan D. Finn et al.
BLOOD (2012)
Gene therapy for lipoprotein lipase deficiency
Daniel Gaudet et al.
CURRENT OPINION IN LIPIDOLOGY (2012)
RNAi-mediated knockdown of HMG CoA reductase enhances gene expression from physiologically regulated low-density lipoprotein receptor therapeutic vectors in vivo
O. Hibbitt et al.
GENE THERAPY (2012)
JD Induced Pluripotent Stem Cell-Derived Hepatocytes Faithfully Recapitulate the Pathophysiology of Familial Hypercholesterolemia
Max A. Cayo et al.
HEPATOLOGY (2012)
Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector
Dawn E. Bowles et al.
MOLECULAR THERAPY (2012)
Translational Lessons from a Case of Combined Heart and Liver Transplantation for Familial Hypercholesterolemia 20 Years Post-operatively
Michael Ibrahim et al.
JOURNAL OF CARDIOVASCULAR TRANSLATIONAL RESEARCH (2012)
Reduction in Mortality in Subjects With Homozygous Familial Hypercholesterolemia Associated With Advances in Lipid-Lowering Therapy
Frederick J. Raal et al.
CIRCULATION (2011)
Gene Therapy for Familial Hypercholesterolemia
Eline Van Craeyveld et al.
CURRENT PHARMACEUTICAL DESIGN (2011)
Adenovirus-Associated Virus Vector-Mediated Gene Transfer in Hemophilia B
Amit C. Nathwani et al.
NEW ENGLAND JOURNAL OF MEDICINE (2011)
Liver transplantation as a treatment option for three siblings with homozygous familial hypercholesterolemia
Tevfik Kucukkartallar et al.
PEDIATRIC TRANSPLANTATION (2011)
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
Sylvie Boutin et al.
HUMAN GENE THERAPY (2010)
Induced pluripotent stem cell-derived hepatocytes have the functional and proliferative capabilities needed for liver regeneration in mice
Silvia Espejel et al.
JOURNAL OF CLINICAL INVESTIGATION (2010)
Modeling inherited metabolic disorders of the liver using human induced pluripotent stem cells
S. Tamir Rashid et al.
JOURNAL OF CLINICAL INVESTIGATION (2010)
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
Frederick J. Raal et al.
LANCET (2010)
Brief Report: Dystrophin Immunity in Duchenne's Muscular Dystrophy.
Jerry R. Mendell et al.
NEW ENGLAND JOURNAL OF MEDICINE (2010)
Gene Therapy in a Humanized Mouse Model of Familial Hypercholesterolemia Leads to Marked Regression of Atherosclerosis
Sadik H. Kassim et al.
PLOS ONE (2010)
Worldwide Epidemiology of Neutralizing Antibodies to Adeno-Associated Viruses
Roberto Calcedo et al.
JOURNAL OF INFECTIOUS DISEASES (2009)
Undetectable Transcription of cap in a Clinical AAV Vector: Implications for Preformed Capsid in Immune Responses
Bernd Hauck et al.
MOLECULAR THERAPY (2009)
Cellular immune response to cryptic epitopes during therapeutic gene transfer
Chengwen Li et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2009)
Longitudinal Evaluation and Assessment of Cardiovascular Disease in Patients With Homozygous Familial Hypercholesterolemia
Daniel M. Kolansky et al.
AMERICAN JOURNAL OF CARDIOLOGY (2008)
CD8+ T-cell responses to adeno-associated virus capsid in humans
Federico Mingozzi et al.
NATURE MEDICINE (2007)
Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid
Luk H. Vandenberghe et al.
NATURE MEDICINE (2006)
Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver
AC Nathwani et al.
BLOOD (2006)
Directed evolution of adeno-associated virus yields enhanced gene delivery vectors
N Maheshri et al.
NATURE BIOTECHNOLOGY (2006)
Gene therapy with novel adeno-associated virus vectors substantially diminishes atherosclerosis in a murine model of familial hypercholesterolemia
C Lebherz et al.
JOURNAL OF GENE MEDICINE (2004)
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
GP Gao et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2002)
Efficacy and safety of ezetimibe coadministered with atorvastatin or simvastatin in patients with homozygous familial hypercholesterolemia
C Gagné et al.
CIRCULATION (2002)
Inhibition of cholesterol synthesis by atorvastatin in homozygous familial hypercholesterolaemia
FJ Raal et al.
ATHEROSCLEROSIS (2000)