Gene therapy for cardiovascular disease: advances in vector development, targeting, and delivery for clinical translation

Efficient Transduction of Primary Vascular Cells by the Rare Adenovirus Serotype 49 Vector

(2015) Rachel S. Dakin et al.   HUMAN GENE THERAPY

Genome-wide Computational Analysis Reveals Cardiomyocyte-specific Transcriptional Cis-regulatory Motifs That Enable Efficient Cardiac Gene Therapy

(2015) Melvin Y Rincon et al.   MOLECULAR THERAPY

Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy

(2014) N. Nair et al.   BLOOD

Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing

(2014) Qiurong Ding et al.   CIRCULATION RESEARCH

Cardiac Calcium Handling on Trial

(2014) Sven T. Pleger et al.   CIRCULATION RESEARCH

Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates

(2014) Marinee K Chuah et al.   MOLECULAR THERAPY

MicroRNAs as Therapeutic Targets and Biomarkers of Cardiovascular Disease

(2014) E. N. Olson   Science Translational Medicine

Design of a Phase 2b Trial of Intracoronary Administration of AAV1/SERCA2a in Patients With Advanced Heart Failure

(2014) Barry Greenberg et al.   JACC-Heart Failure

Targeting endothelial cells by gene therapy

(2013) T. VandenDriessche et al.   BLOOD

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

(2013) A. T. Martino et al.   BLOOD

Specific gene delivery to liver sinusoidal and artery endothelial cells

(2013) T. Abel et al.   BLOOD

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

(2013) F. Mingozzi et al.   BLOOD

Long-Term Effects of AAV1/SERCA2a Gene Transfer in Patients With Severe Heart Failure

(2013) Krisztina Zsebo et al.   CIRCULATION RESEARCH

Cardiac SERCA2A/B: Therapeutic targets for heart failure

(2013) Mohammad Abrar Shareef et al.   EUROPEAN JOURNAL OF PHARMACOLOGY

Evaluation of ASPIRE trial: a Phase III pivotal registration trial, using intracoronary administration of Generx (Ad5FGF4) to treat patients with recurrent angina pectoris

(2013) Juan Carlos Kaski et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Cardiovascular gene therapy with vascular endothelial growth factors

(2013) Seppo Ylä-Herttuala   GENE

Characterization of a third generation lentiviral vector pseudotyped with Nipah virus envelope proteins for endothelial cell transduction

(2013) S R Witting et al.   GENE THERAPY

Therapeutic safety of high myocardial expression levels of the molecular inotrope S100A1 in a preclinical heart failure model

(2013) C Weber et al.   GENE THERAPY

Targeting Sarcoplasmic Reticulum Calcium ATPase by Gene Therapy

(2013) Judith K. Gwathmey et al.   HUMAN GENE THERAPY

Intravenous Adeno-Associated Virus Serotype 8 Encoding Urocortin-2 Provides Sustained Augmentation of Left Ventricular Function in Mice

(2013) Mei Hua Gao et al.   HUMAN GENE THERAPY

Progress in Gene Therapy for Heart Failure

(2013) Armen Yerevanian et al.   JOURNAL OF CARDIOVASCULAR PHARMACOLOGY

Potential of gene therapy as a treatment for heart failure

(2013) Roger J. Hajjar   JOURNAL OF CLINICAL INVESTIGATION

Calcium cycling proteins and heart failure: mechanisms and therapeutics

(2013) Andrew R. Marks   JOURNAL OF CLINICAL INVESTIGATION

Application of Mutated miR-206 Target Sites Enables Skeletal Muscle-specific Silencing of Transgene Expression of Cardiotropic AAV9 Vectors

(2013) Anja Geisler et al.   MOLECULAR THERAPY

Vascular endothelial growth factor in heart failure

(2013) Ziad Taimeh et al.   Nature Reviews Cardiology

Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

(2013) A. Aiuti et al.   SCIENCE

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

(2013) A. Biffi et al.   SCIENCE

Myosin light chain 2-based selection of human iPSC-derived early ventricular cardiac myocytes

(2013) Alexandra Bizy et al.   Stem Cell Research

PlGF: A Multitasking Cytokine with Disease-Restricted Activity

(2013) M. Dewerchin et al.   Cold Spring Harbor Perspectives in Medicine

VEGF promotes cardiac stem cells differentiation into vascular endothelial cells via the PI3K/Akt signaling pathway

(2013) Nuan Xiao et al.   Artificial Cells Nanomedicine and Biotechnology

Pathophysiological roles of FGF signaling in the heart

(2013) Nobuyuki Itoh et al.   Frontiers in Physiology

Nonviral gene therapy targeting cardiovascular system

(2012) Cheng-Huang Su et al.   AMERICAN JOURNAL OF PHYSIOLOGY-HEART AND CIRCULATORY PHYSIOLOGY

Myocardial β2-adrenoceptor gene delivery promotes coordinated cardiac adaptive remodelling and angiogenesis in heart failure

(2012) G Rengo et al.   BRITISH JOURNAL OF PHARMACOLOGY

High-dose plasmid-mediated VEGF gene transfer is safe in patients with severe ischemic heart disease (Genesis-I). A phase I, open-label, two-year follow-up trial

(2012) Liliana Favaloro et al.   CATHETERIZATION AND CARDIOVASCULAR INTERVENTIONS

Gene Therapy for Heart Failure

(2012) Lisa Tilemann et al.   CIRCULATION RESEARCH

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

Understanding lentiviral vector chromatin targeting: working to reduce insertional mutagenic potential for gene therapy

(2012) C Papayannakos et al.   GENE THERAPY

VEGF gene therapy: therapeutic angiogenesis in the clinic and beyond

(2012) M Giacca et al.   GENE THERAPY

European Society of Gene and Cell TherapyFrench Society of Cell and Gene TherapyCollaborative Congress 2012October 25–29, 2012Palais des Congrès de VersaillesVersailles, France

(2012)   HUMAN GENE THERAPY

Concomitant Intravenous Nitroglycerin With Intracoronary Delivery of AAV1.SERCA2a Enhances Gene Transfer in Porcine Hearts

(2012) Ioannis Karakikes et al.   MOLECULAR THERAPY

Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID)

(2011) Mariell Jessup et al.   CIRCULATION

G i -Biased β 2 AR Signaling Links GRK2 Upregulation to Heart Failure

(2011) Weizhong Zhu et al.   CIRCULATION RESEARCH

Repeated and targeted transfer of angiogenic plasmids into the infarcted rat heart via ultrasound targeted microbubble destruction enhances cardiac repair

(2011) Hiroko Fujii et al.   EUROPEAN HEART JOURNAL

Progress and prospects: hurdles to cardiovascular gene therapy clinical trials

(2011) M Hedman et al.   GENE THERAPY

10-year safety follow-up in patients with local VEGF gene transfer to ischemic lower limb

(2011) K Muona et al.   GENE THERAPY

Helper-Dependent Adenoviral Vector Achieves Prolonged, Stable Expression of Interleukin-10 in Rabbit Carotid Arteries but Does Not Limit Early Atherogenesis

(2011) Liang Du et al.   HUMAN GENE THERAPY

Transendocardial Delivery of AAV6 Results in Highly Efficient and Global Cardiac Gene Transfer in Rhesus Macaques

(2011) Guangping Gao et al.   HUMAN GENE THERAPY

Improved Function of the Failing Rat Heart by Regulated Expression of Insulin-Like Growth Factor I via Intramuscular Gene Transfer

(2011) N. Chin Lai et al.   HUMAN GENE THERAPY

The evolution of heart gene delivery vectors

(2011) Nalinda B. Wasala et al.   JOURNAL OF GENE MEDICINE

A single direct injection into the left ventricular wall of an adeno-associated virus 9 (AAV9) vector expressing extracellular superoxide dismutase from the cardiac troponin-T promoter protects mice against myocardial infarction

(2011) Konkal-Matt R. Prasad et al.   JOURNAL OF GENE MEDICINE

Effect of vascular endothelial growth factor gene transfer on infarct size, left ventricular function and myocardial perfusion in sheep after 2 months of coronary artery occlusion

(2011) Gustavo L. Vera Janavel et al.   JOURNAL OF GENE MEDICINE

S100A1 Genetically Targeted Therapy Reverses Dysfunction of Human Failing Cardiomyocytes

(2011) Henriette Brinks et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Engineering Liver-detargeted AAV9 Vectors for Cardiac and Musculoskeletal Gene Transfer

(2011) Nagesh Pulicherla et al.   MOLECULAR THERAPY

Molecular mechanisms and clinical applications of angiogenesis

(2011) Peter Carmeliet et al.   NATURE

Can CUPID rescue the broken hearted?

(2011) Bryony M. Mearns   Nature Reviews Cardiology

Principles and mechanisms of vessel normalization for cancer and other angiogenic diseases

(2011) Peter Carmeliet et al.   NATURE REVIEWS DRUG DISCOVERY

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

(2011) Federico Mingozzi et al.   NATURE REVIEWS GENETICS

State-of-the-art gene-based therapies: the road ahead

(2011) Mark A. Kay   NATURE REVIEWS GENETICS

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

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

PlGF Repairs Myocardial Ischemia through Mechanisms of Angiogenesis, Cardioprotection and Recruitment of Myo-Angiogenic Competent Marrow Progenitors

(2011) Hiroto Iwasaki et al.   PLoS One

Coexpression of VEGF and angiopoietin-1 promotes angiogenesis and cardiomyocyte proliferation reduces apoptosis in porcine myocardial infarction (MI) heart

(2011) Zhengxian Tao et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

Effects of cardiac overexpression of type 6 adenylyl cyclase affects on the response to chronic pressure overload

(2010) Aziz Guellich et al.   AMERICAN JOURNAL OF PHYSIOLOGY-HEART AND CIRCULATORY PHYSIOLOGY

Gene Delivery of Sarcoplasmic Reticulum Calcium ATPase Inhibits Ventricular Remodeling in Ischemic Mitral Regurgitation

(2010) Ronen Beeri et al.   Circulation-Heart Failure

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

microRNA122-regulated transgene expression increases specificity of cardiac gene transfer upon intravenous delivery of AAV9 vectors

(2010) A Geisler et al.   GENE THERAPY

Heart-targeted adeno-associated viral vectors selected by in vivo biopanning of a random viral display peptide library

(2010) Y Ying et al.   GENE THERAPY

Therapeutic angiogenesis for cardiovascular disease: biological context, challenges, prospects

(2010) I. Zachary et al.   HEART

Gene therapy strategies for hemophilia: benefits versus risks

(2010) Inge Petrus et al.   JOURNAL OF GENE MEDICINE

The next step in gene delivery: Molecular engineering of adeno-associated virus serotypes

(2010) Jinhui Wang et al.   JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY

SERCA2a Gene Transfer Enhances eNOS Expression and Activity in Endothelial Cells

(2010) Lahouaria Hadri et al.   MOLECULAR THERAPY

Recent Advances in Lentiviral Vector Development and Applications

(2010) Janka Mátrai et al.   MOLECULAR THERAPY

Challenges with advanced therapy medicinal products and how to meet them

(2010)   NATURE REVIEWS DRUG DISCOVERY

Development of Viral Vectors for Use in Cardiovascular Gene Therapy

(2010) Paul Williams et al.   Viruses-Basel

Systemic Trans-Splicing Adeno-Associated Viral Delivery Efficiently Transduces the Heart of Adult mdx Mouse, a Model for Duchenne Muscular Dystrophy

(2009) Arkasubhra Ghosh 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

Acute Heart Failure Syndromes

(2009) Mihai Gheorghiade et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

The Sympathetic Nervous System in Heart Failure

(2009) Filippos Triposkiadis et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

VEGF Gene Therapy Fails to Improve Perfusion of Ischemic Myocardium in Patients With Advanced Coronary Disease: Results of the NORTHERN Trial

(2009) Duncan J Stewart et al.   MOLECULAR THERAPY

Effect of Genome Size on AAV Vector Packaging

(2009) Zhijian Wu et al.   MOLECULAR THERAPY

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

Ultrasound-Targeted Gene Delivery Induces Angiogenesis After a Myocardial Infarction in Mice

(2009) Hiroko Fujii et al.   JACC-Cardiovascular Imaging

Specific monitoring of cardiomyogenic and endothelial differentiation by dual promoter-driven reporter systems in bone marrow mesenchymal stem cells

(2008) Seung-Cheol Choi et al.   BIOTECHNOLOGY LETTERS

G Protein–Coupled Receptor Kinase 2 Ablation in Cardiac Myocytes Before or After Myocardial Infarction Prevents Heart Failure

(2008) Philip W. Raake et al.   CIRCULATION RESEARCH

Construction and analysis of compact muscle-specific promoters for AAV vectors

(2008) B Wang et al.   GENE THERAPY

Design of a Phase 1/2 Trial of Intracoronary Administration of AAV1/SERCA2a in Patients With Heart Failure

(2008) Roger J. Hajjar et al.   JOURNAL OF CARDIAC FAILURE

Reversal of Cardiac Dysfunction After Long-Term Expression of SERCA2a by Gene Transfer in a Pre-Clinical Model of Heart Failure

(2008) Yoshiaki Kawase et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

The effect of electroporation pulses on functioning of the heart

(2008) Barbara Mali et al.   MEDICAL & BIOLOGICAL ENGINEERING & COMPUTING

Lentiviral Vector–mediated SERCA2 Gene Transfer Protects Against Heart Failure and Left Ventricular Remodeling After Myocardial Infarction in Rats

(2008) Kazuo Niwano et al.   MOLECULAR THERAPY

A Single Intravenous Injection of Adeno-associated Virus Serotype-9 Leads to Whole Body Skeletal Muscle Transduction in Dogs

(2008) Yongping Yue et al.   MOLECULAR THERAPY

Cardiac Expression of a Mini-dystrophin That Normalizes Skeletal Muscle Force Only Partially Restores Heart Function in Aged Mdx Mice

(2008) Brian Bostick et al.   MOLECULAR THERAPY

Fibroblast Growth Factor 4 Gene Therapy for Chronic Ischemic Heart Disease

(2008) Navin K. Kapur et al.   TRENDS IN CARDIOVASCULAR MEDICINE