Promise of adeno-associated virus as a gene therapy vector for cardiovascular diseases

Regulating the regulator: Insights into the cardiac protein phosphatase 1 interactome

(2016) David Y. Chiang et al.   JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY

Calcium upregulation by percutaneous administration of gene therapy in patients with cardiac disease (CUPID 2): a randomised, multinational, double-blind, placebo-controlled, phase 2b trial

(2016) Barry Greenberg et al.   LANCET

CUPID 2: A Phase 2b Trial Investigating the Efficacy and Safety of the Intracoronary Administration of AAV1/SERCA2a in Patients with Advanced Heart Failure

(2015) Barry Greenberg et al.   JOURNAL OF CARDIAC FAILURE

The war against heart failure: the Lancet lecture

(2015) Eugene Braunwald   LANCET

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

A split-Cas9 architecture for inducible genome editing and transcription modulation

(2015) Bernd Zetsche et al.   NATURE BIOTECHNOLOGY

A mouse model for adult cardiac-specific gene deletion with CRISPR/Cas9

(2015) Kelli J. Carroll et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

(2015) C. E. Nelson et al.   SCIENCE

Small-molecule activation of SERCA2a SUMOylation for the treatment of heart failure

(2015) Changwon Kho et al.   Nature Communications

The Role of SUMO-1 in Cardiac Oxidative Stress and Hypertrophy

(2014) Ahyoung Lee et al.   ANTIOXIDANTS & REDOX SIGNALING

The Global Burden of Ischemic Heart Disease in 1990 and 2010

(2014) Andrew E. Moran et al.   CIRCULATION

Mineralocorticoid and AT1receptors in the paraventricular nucleus contribute to sympathetic hyperactivity and cardiac dysfunction in rats post myocardial infarct

(2014) Bing S. Huang et al.   JOURNAL OF PHYSIOLOGY-LONDON

Inhibition of miR-25 improves cardiac contractility in the failing heart

(2014) Christine Wahlquist et al.   NATURE

In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9

(2014) Lukasz Swiech et al.   NATURE BIOTECHNOLOGY

Biological pacemaker created by minimally invasive somatic reprogramming in pigs with complete heart block

(2014) Y.-F. Hu et al.   Science Translational Medicine

A Needleless Liquid Jet Injection Delivery Method for Cardiac Gene Therapy: a Comparative Evaluation Versus Standard Routes of Delivery Reveals Enhanced Therapeutic Retention and Cardiac Specific Gene Expression

(2014) A. S. Fargnoli et al.   Journal of Cardiovascular Translational Research

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

(2013) F. Mingozzi et al.   BLOOD

One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering

(2013) Haoyi Wang et al.   CELL

One-Step Generation of Mice Carrying Reporter and Conditional Alleles by CRISPR/Cas-Mediated Genome Engineering

(2013) Hui Yang et al.   CELL

Signaling effectors underlying pathologic growth and remodeling of the heart

(2013) Jop H. van Berlo et al.   JOURNAL OF CLINICAL INVESTIGATION

Global and regional burden of stroke during 1990–2010: findings from the Global Burden of Disease Study 2010

(2013) Valery L Feigin et al.   LANCET

A Novel Chimeric Adenoassociated Virus 2/Human Bocavirus 1 Parvovirus Vector Efficiently Transduces Human Airway Epithelia

(2013) Ziying Yan et al.   MOLECULAR THERAPY

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

(2013) Daniel J Hui et al.   MOLECULAR THERAPY

Systemic Delivery of shRNA by AAV9 Provides Highly Efficient Knockdown of Ubiquitously Expressed GFP in Mouse Heart, but Not Liver

(2013) Bryan A. Piras et al.   PLoS One

Adeno-associated virus (AAV) vectors in gene therapy: immune challenges and strategies to circumvent them

(2013) Sangeetha Hareendran et al.   REVIEWS IN MEDICAL VIROLOGY

Genome-Scale CRISPR-Cas9 Knockout Screening in Human Cells

(2013) Ophir Shalem et al.   SCIENCE

Genetic Screens in Human Cells Using the CRISPR-Cas9 System

(2013) Tim Wang et al.   SCIENCE

Improved adeno-associated virus (AAV) serotype 1 and 5 vectors for gene therapy

(2013) Dwaipayan Sen et al.   Scientific Reports

Targeted Modifications in Adeno-Associated Virus Serotype 8 Capsid Improves Its Hepatic Gene Transfer Efficiency In Vivo

(2013) Dwaipayan Sen et al.   Human Gene Therapy Methods

Bioengineering of AAV2 Capsid at Specific Serine, Threonine, or Lysine Residues Improves Its Transduction Efficiency in Vitro and in Vivo

(2013) Nishanth Gabriel et al.   Human Gene Therapy Methods

Therapeutic Cardiac‐Targeted Delivery of miR‐1 Reverses Pressure Overload–Induced Cardiac Hypertrophy and Attenuates Pathological Remodeling

(2013) Ioannis Karakikes et al.   Journal of the American Heart Association

Global and regional burden of first-ever ischaemic and haemorrhagic stroke during 1990–2010: findings from the Global Burden of Disease Study 2010

(2013) Rita V Krishnamurthi et al.   Lancet Global Health

Regulation of sarco(endo)plasmic reticulum Ca2+-ATPase and calsequestrin gene expression in the heart

(2012) Angel Zarain-Herzberg et al.   CANADIAN JOURNAL OF PHYSIOLOGY AND PHARMACOLOGY

Modulation of Cardiac Contractility by the Phopholamban/SERCA2a Regulatome

(2012) Evangelia G. Kranias et al.   CIRCULATION RESEARCH

Targeting the β-Adrenergic Receptor System Through G-Protein–Coupled Receptor Kinase 2: A New Paradigm for Therapy and Prognostic Evaluation in Heart Failure

(2012) Giuseppe Rengo et al.   Circulation-Heart Failure

AAV9.I-1c Delivered via Direct Coronary Infusion in a Porcine Model of Heart Failure Improves Contractility and Mitigates Adverse Remodeling

(2012) Kenneth M. Fish et al.   Circulation-Heart Failure

AAV6. ARKct cardiac gene therapy ameliorates cardiac function and normalizes the catecholaminergic axis in a clinically relevant large animal heart failure model

(2012) P. W. J. Raake et al.   EUROPEAN HEART JOURNAL

Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue

(2012) F Mingozzi et al.   GENE THERAPY

Ongoing myocardial damage in patients with heart failure and preserved ejection fraction

(2012) Daisuke Kutsuzawa et al.   Journal of Cardiology

Single Amino Acid Modification of Adeno-Associated Virus Capsid Changes Transduction and Humoral Immune Profiles

(2012) C. Li et al.   JOURNAL OF VIROLOGY

Heart Failure-Inducible Gene Therapy Targeting Protein Phosphatase 1 Prevents Progressive Left Ventricular Remodeling

(2012) Yosuke Miyazaki et al.   PLoS One

Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia

(2011) C Li et al.   GENE THERAPY

SUMO1-dependent modulation of SERCA2a in heart failure

(2011) Changwon Kho et al.   NATURE

Cardiac AAV9-S100A1 Gene Therapy Rescues Post-Ischemic Heart Failure in a Preclinical Large Animal Model

(2011) S. T. Pleger et al.   Science Translational Medicine

Role of Cardiac Myocyte CXCR4 Expression in Development and Left Ventricular Remodeling After Acute Myocardial Infarction

(2010) Udit Agarwal et al.   CIRCULATION RESEARCH

Comparative Cardiac Gene Delivery of Adeno-Associated Virus Serotypes 1-9 reveals that AAV6 Mediates the Most Efficient Transduction in Mouse Heart

(2010) Carmela Zincarelli et al.   CTS-Clinical and Translational Science

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

SERCA2a Gene Transfer Enhances eNOS Expression and Activity in Endothelial Cells

(2010) Lahouaria Hadri et al.   MOLECULAR THERAPY

Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

(2010) Salima Hacein-Bey-Abina et al.   NEW ENGLAND JOURNAL OF MEDICINE

Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency

(2009) B. Neven et al.   BLOOD

Long-Term Cardiac-Targeted RNA Interference for the Treatment of Heart Failure Restores Cardiac Function and Reduces Pathological Hypertrophy

(2009) Lennart Suckau et al.   CIRCULATION

Inducible Expression of Active Protein Phosphatase-1 Inhibitor-1 Enhances Basal Cardiac Function and Protects Against Ischemia/Reperfusion Injury

(2009) Persoulla Nicolaou et al.   CIRCULATION RESEARCH

Adeno-Associated Virus-Mediated Gene Transfer to Nonhuman Primate Liver Can Elicit Destructive Transgene-Specific T Cell Responses

(2009) Guangping Gao et al.   HUMAN GENE THERAPY

Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID Trial), a First-in-Human Phase 1/2 Clinical Trial

(2009) Brian E. Jaski et al.   JOURNAL OF CARDIAC FAILURE

Adeno-associated virus serotype 2 induces cell-mediated immune responses directed against multiple epitopes of the capsid protein VP1

(2009) D. Madsen et al.   JOURNAL OF GENERAL VIROLOGY

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

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

S100A1 in cardiovascular health and disease: Closing the gap between basic science and clinical therapy

(2009) Carolin Kraus et al.   JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY

Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle

(2009) Aravind Asokan et al.   NATURE BIOTECHNOLOGY

A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection

(2009) L. Yang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

(2009) M. L. Brantly et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Effect of Invasive Coronary Revascularization in Acute Myocardial Infarction on Subsequent Death Rate and Frequency of Chronic Heart Failure

(2008) Finlay A. McAlister et al.   AMERICAN JOURNAL OF CARDIOLOGY

Heart Failure After Acute Myocardial Infarction

(2008) Pardeep S. Jhund et al.   CIRCULATION

Long-Term Trends in the Incidence of Heart Failure After Myocardial Infarction

(2008) Raghava S. Velagaleti et al.   CIRCULATION

Myocardial Adeno-Associated Virus Serotype 6–βARKct Gene Therapy Improves Cardiac Function and Normalizes the Neurohormonal Axis in Chronic Heart Failure

(2008) Giuseppe Rengo et al.   CIRCULATION

The Emerging Role of MicroRNAs in Cardiac Remodeling and Heart Failure

(2008) Vijay Divakaran et al.   CIRCULATION RESEARCH

Gene Therapy Using Adeno-Associated Virus Vectors

(2008) S. Daya et al.   CLINICAL MICROBIOLOGY REVIEWS

Risk factors for myocardial infarction in women and men: insights from the INTERHEART study

(2008) Sonia S. Anand et al.   EUROPEAN HEART JOURNAL

Augmentation of AAV-mediated cardiac gene transfer after systemic administration in adult rats

(2008) O J Müller et al.   GENE THERAPY

Adeno-Associated Virus (AAV) Serotype 9 Provides Global Cardiac Gene Transfer Superior to AAV1, AAV6, AAV7, and AAV8 in the Mouse and Rat

(2008) Lawrence T. Bish et al.   HUMAN GENE THERAPY

Efficient and Durable Gene Transfer to Transplanted Heart Using Adeno-associated Virus 9 Vector

(2008) Naoto Miyagi et al.   JOURNAL OF HEART AND LUNG TRANSPLANTATION

Biodistribution and Safety Profile of Recombinant Adeno-Associated Virus Serotype 6 Vectors following Intravenous Delivery

(2008) D. Stone et al.   JOURNAL OF VIROLOGY

In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses

(2008) D. Grimm et al.   JOURNAL OF VIROLOGY

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

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY

TNFR gene-modified mesenchymal stem cells attenuate inflammation and cardiac dysfunction following MI

(2008) Cuiyu Bao et al.   SCANDINAVIAN CARDIOVASCULAR JOURNAL