Targeted delivery of therapeutic agents to the heart

Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy

(2020) A. Moretti et al.   NATURE MEDICINE

Engineering adeno-associated virus vectors for gene therapy

(2020) Chengwen Li et al.   NATURE REVIEWS GENETICS

Preclinical development of a miR-132 inhibitor for heart failure treatment

(2020) Ariana Foinquinos et al.   Nature Communications

Gene therapy beyond luxturna

(2020) Dominic A. Prado et al.   CURRENT OPINION IN OPHTHALMOLOGY

AntimiR-21 Prevents Myocardial Dysfunction in a Pig Model of Ischemia/Reperfusion Injury

(2020) Rabea Hinkel et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Native and bioengineered extracellular vesicles for cardiovascular therapeutics

(2020) Ricardo Cerqueira de Abreu et al.   Nature Reviews Cardiology

Intracoronary ALLogeneic heart STem cells to Achieve myocardial Regeneration (ALLSTAR): a randomized, placebo-controlled, double-blinded trial

(2020) Raj R Makkar et al.   EUROPEAN HEART JOURNAL

Synthetic mRNA Encoding VEGF-A in Patients Undergoing Coronary Artery Bypass Grafting: Design of a Phase 2a Clinical Trial

(2020) Vesa Anttila et al.   Molecular Therapy-Methods & Clinical Development

ESC Working Group on Cellular Biology of the Heart

(2019) Rosalinda Madonna et al.   CARDIOVASCULAR RESEARCH

Intradermal delivery of modified mRNA encoding VEGF-A in patients with type 2 diabetes

(2019) Li-Ming Gan et al.   Nature Communications

Intramyocardial Injection of Mesenchymal Precursor Cells and Successful Temporary Weaning From Left Ventricular Assist Device Support in Patients With Advanced Heart Failure

(2019) Terrence M. Yau et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Efficient exosome delivery in refractory tissues assisted by ultrasound-targeted microbubble destruction

(2019) Wenqi Sun et al.   DRUG DELIVERY

Heart Disease and Stroke Statistics—2019 Update: A Report From the American Heart Association

(2019) Emelia J. Benjamin et al.   CIRCULATION

MicroRNA therapy stimulates uncontrolled cardiac repair after myocardial infarction in pigs

(2019) Khatia Gabisonia et al.   NATURE

Impact of age and motor function in a phase 1/2A study of infants with SMA Type 1 receiving single-dose gene replacement therapy

(2019) Linda P. Lowes et al.   PEDIATRIC NEUROLOGY

Gene Therapy Leaves a Vicious Cycle

(2019) Reena Goswami et al.   Frontiers in Oncology

Exosomes Engineered to Express a Cardiomyocyte Binding Peptide Demonstrate Improved Cardiac Retention in Vivo

(2019) Kyle I. Mentkowski et al.   Scientific Reports

The biology of extracellular vesicles: The known unknowns

(2019) Leonid Margolis et al.   PLOS BIOLOGY

Predicting Functional Responses of Progenitor Cell Exosome Potential with Computational Modeling

(2019) David Trac et al.   Stem Cells Translational Medicine

The Global Burden of Cardiovascular Diseases and Risk Factors

(2019) George A. Mensah et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

An acute immune response underlies the benefit of cardiac stem-cell therapy

(2019) Ronald J. Vagnozzi et al.   NATURE

Mechanism of recipient cell-dependent differences in exosome uptake

(2018) Sayo Horibe et al.   BMC CANCER

Autologous CD34+ cell therapy improves exercise capacity, angina frequency and reduces mortality in no-option refractory angina: a patient-level pooled analysis of randomized double-blinded trials

(2018) Timothy D Henry et al.   EUROPEAN HEART JOURNAL

Exosomes in Myocardial Repair: Advances and Challenges in the Development of Next-Generation Therapeutics

(2018) Marta Adamiak et al.   MOLECULAR THERAPY

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

(2018) David Adams et al.   NEW ENGLAND JOURNAL OF MEDICINE

Conjugating Existing Clinical Drugs With Gold Nanoparticles for Better Treatment of Heart Diseases

(2018) Jingwen Zhang et al.   Frontiers in Physiology

Biocompatible, Purified VEGF-A mRNA Improves Cardiac Function after Intracardiac Injection 1 Week Post-myocardial Infarction in Swine

(2018) Leif Carlsson et al.   Molecular Therapy-Methods & Clinical Development

Targeting protein and peptide therapeutics to the heart via tannic acid modification

(2018) Mikyung Shin et al.   Nature Biomedical Engineering

Differential m6A, m6Am, and m1A Demethylation Mediated by FTO in the Cell Nucleus and Cytoplasm

(2018) Jiangbo Wei et al.   MOLECULAR CELL

Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

(2018) Leonela Amoasii et al.   SCIENCE

Polymers and hydrogels for local nucleic acid delivery

(2018) Lies A. L. Fliervoet et al.   Journal of Materials Chemistry B

Lentiviral Vector Gene Transfer of Endostatin/Angiostatin for Macular Degeneration (GEM) Study

(2017) Peter A. Campochiaro et al.   HUMAN GENE THERAPY

Nanoparticle-mediated drug delivery system for atherosclerotic cardiovascular disease

(2017) Tetsuya Matoba et al.   Journal of Cardiology

Pharmacokinetics of Exosomes—An Important Factor for Elucidating the Biological Roles of Exosomes and for the Development of Exosome-Based Therapeutics

(2017) Masaki Morishita et al.   JOURNAL OF PHARMACEUTICAL SCIENCES

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

(2017) Stephen Russell et al.   LANCET

Rescue of Hearing by Gene Delivery to Inner-Ear Hair Cells Using Exosome-Associated AAV

(2017) Bence György et al.   MOLECULAR THERAPY

Human Pericardial Fluid Contains Exosomes Enriched with Cardiovascular-Expressed MicroRNAs and Promotes Therapeutic Angiogenesis

(2017) Cristina Beltrami et al.   MOLECULAR THERAPY

Cardiovascular Gene Therapy: Past, Present, and Future

(2017) Seppo Ylä-Herttuala et al.   MOLECULAR THERAPY

Exosome-associated AAV2 vector mediates robust gene delivery into the murine retina upon intravitreal injection

(2017) Sarah J. Wassmer et al.   Scientific Reports

Extracellular vesicles for drug delivery

(2016) Pieter Vader et al.   ADVANCED DRUG DELIVERY REVIEWS

Route of Delivery Modulates the Efficacy of Mesenchymal Stem Cell Therapy for Myocardial InfarctionNovelty and Significance

(2016) Anthony J. Kanelidis et al.   CIRCULATION RESEARCH

Gene therapy for the treatment of heart failure: promise postponed

(2016) Jean-Sebastien Hulot et al.   EUROPEAN HEART JOURNAL

Exosome-associated AAV vector as a robust and convenient neuroscience tool

(2016) E Hudry et al.   GENE THERAPY

The RENEW Trial

(2016) Thomas J. Povsic et al.   JACC-Cardiovascular Interventions

Pim1 Kinase Overexpression Enhances ckit + Cardiac Stem Cell Cardiac Repair Following Myocardial Infarction in Swine

(2016) Shathiyah Kulandavelu et al.   JOURNAL OF THE AMERICAN COLLEGE OF 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

Driving CAR T-cells forward

(2016) Hollie J. Jackson et al.   Nature Reviews Clinical Oncology

Extracellular vesicles and viruses: Are they close relatives?

(2016) Esther Nolte-‘t Hoen et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Cardiac repair in guinea pigs with human engineered heart tissue from induced pluripotent stem cells

(2016) F. Weinberger et al.   Science Translational Medicine

Intracoronary Gene Transfer of Adenylyl Cyclase 6 in Patients With Heart Failure

(2016) H. Kirk Hammond et al.   JAMA Cardiology

Effect of the stop-flow technique on cardiac retention of c-kit positive human cardiac stem cells after intracoronary infusion in a porcine model of chronic ischemic cardiomyopathy

(2015) Matthew C. L. Keith et al.   BASIC RESEARCH IN CARDIOLOGY

Changes in ventricular remodelling and clinical status during the year following a single administration of stromal cell-derived factor-1 non-viral gene therapy in chronic ischaemic heart failure patients: the STOP-HF randomized Phase II trial

(2015) Eugene S. Chung et al.   EUROPEAN HEART JOURNAL

Bone marrow-derived mesenchymal stromal cell treatment in patients with severe ischaemic heart failure: a randomized placebo-controlled trial (MSC-HF trial)

(2015) Anders Bruun Mathiasen et al.   EUROPEAN HEART JOURNAL

Fibroblasts Rendered Antifibrotic, Antiapoptotic, and Angiogenic by Priming With Cardiosphere-Derived Extracellular Membrane Vesicles

(2015) Eleni Tseliou et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Plasma Exosomes Protect the Myocardium From Ischemia-Reperfusion Injury

(2015) Jose M. Vicencio et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Epicardial FSTL1 reconstitution regenerates the adult mammalian heart

(2015) Ke Wei et al.   NATURE

Glioma-derived extracellular vesicles selectively suppress immune responses

(2015) Justin E. Hellwinkel et al.   NEURO-ONCOLOGY

Physiology of pericardial fluid production and drainage

(2015) Konstantinos Vogiatzidis et al.   Frontiers in Physiology

Injectable Graphene Oxide/Hydrogel-Based Angiogenic Gene Delivery System for Vasculogenesis and Cardiac Repair

(2014) Arghya Paul et al.   ACS Nano

Naturally enveloped AAV vectors for shielding neutralizing antibodies and robust gene delivery in vivo

(2014) Bence György et al.   BIOMATERIALS

Exosomes and Cardiac Repair After Myocardial Infarction

(2014) Susmita Sahoo et al.   CIRCULATION RESEARCH

Autologous Mesenchymal Stem Cells Produce Concordant Improvements in Regional Function, Tissue Perfusion, and Fibrotic Burden When Administered to Patients Undergoing Coronary Artery Bypass Grafting

(2014) Vasileios Karantalis et al.   CIRCULATION RESEARCH

Development of microRNA therapeutics is coming of age

(2014) E. van Rooij et al.   EMBO Molecular Medicine

Nanoparticle-Mediated Drug Delivery System for Cardiovascular Disease

(2014) Tetsuya Matoba et al.   International Heart Journal

Magnetic resonance imaging of melanoma exosomes in lymph nodes

(2014) Lingzhi Hu et al.   MAGNETIC RESONANCE IN MEDICINE

Cardiac I-1c Overexpression With Reengineered AAV Improves Cardiac Function in Swine Ischemic Heart Failure

(2014) Kiyotake Ishikawa et al.   MOLECULAR THERAPY

Human embryonic-stem-cell-derived cardiomyocytes regenerate non-human primate hearts

(2014) James J. H. Chong et al.   NATURE

c-kit+ cells minimally contribute cardiomyocytes to the heart

(2014) Jop H. van Berlo et al.   NATURE

Non-viral vectors for gene-based therapy

(2014) Hao Yin et al.   NATURE REVIEWS GENETICS

Magnetic antibody-linked nanomatchmakers for therapeutic cell targeting

(2014) Ke Cheng et al.   Nature Communications

Human iPS cell-engineered cardiac tissue sheets with cardiomyocytes and vascular cells for cardiac regeneration

(2014) Hidetoshi Masumoto et al.   Scientific Reports

Bioinspired Exosome-Mimetic Nanovesicles for Targeted Delivery of Chemotherapeutics to Malignant Tumors

(2013) Su Chul Jang et al.   ACS Nano

The effect of bioengineered acellular collagen patch on cardiac remodeling and ventricular function post myocardial infarction

(2013) Vahid Serpooshan et al.   BIOMATERIALS

An Open-Label Dose Escalation Study to Evaluate the Safety of Administration of Nonviral Stromal Cell-Derived Factor-1 Plasmid to Treat Symptomatic Ischemic Heart Failure

(2013) Marc S. Penn et al.   CIRCULATION RESEARCH

A pathogenic picornavirus acquires an envelope by hijacking cellular membranes

(2013) Zongdi Feng et al.   NATURE

Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction

(2013) Lior Zangi et al.   NATURE BIOTECHNOLOGY

Mesenchymal stem cell-derived exosomes increase ATP levels, decrease oxidative stress and activate PI3K/Akt pathway to enhance myocardial viability and prevent adverse remodeling after myocardial ischemia/reperfusion injury

(2013) Fatih Arslan et al.   Stem Cell Research

Intracoronary and retrograde coronary venous myocardial delivery of adipose-derived stem cells in swine infarction lead to transient myocardial trapping with predominant pulmonary redistribution

(2012) Soon Jun Hong et al.   CATHETERIZATION AND CARDIOVASCULAR INTERVENTIONS

Cardiac gene therapy in large animals: bridge from bench to bedside

(2012) K Ishikawa et al.   GENE THERAPY

Lentiviral vectors and cardiovascular diseases: a genetic tool for manipulating cardiomyocyte differentiation and function

(2012) E Di Pasquale et al.   GENE THERAPY

Microvesicles derived from mesenchymal stem cells: Potent organelles for induction of tolerogenic signaling

(2012) Aram Mokarizadeh et al.   IMMUNOLOGY LETTERS

Comparison of Allogeneic vs Autologous Bone Marrow–Derived Mesenchymal Stem Cells Delivered by Transendocardial Injection in Patients With Ischemic Cardiomyopathy

(2012) Joshua M. Hare et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Therapeutic inhibition of the miR-34 family attenuates pathological cardiac remodeling and improves heart function

(2012) B. C. Bernardo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Human Pericytes for Ischemic Heart Repair

(2012) Chien-Wen Chen et al.   STEM CELLS

Intramyocardial, Autologous CD34+ Cell Therapy for Refractory Angina

(2011) Douglas W. Losordo et al.   CIRCULATION RESEARCH

Exosomes From Human CD34 + Stem Cells Mediate Their Proangiogenic Paracrine Activity

(2011) Susmita Sahoo et al.   CIRCULATION RESEARCH

Delivery of gelfoam-enabled cells and vectors into the pericardial space using a percutaneous approach in a porcine model

(2011) D Ladage et al.   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

A Pharmacokinetic Analysis of Molecular Cardiac Surgery With Recirculation Mediated Delivery of βARKct Gene Therapy: Developing a Quantitative Definition of the Therapeutic Window

(2011) Anthony S. Fargnoli et al.   JOURNAL OF CARDIAC FAILURE

Safety and feasibility of percutaneous retrograde coronary sinus delivery of autologous bone marrow mononuclear cell transplantation in patients with chronic refractory angina

(2011) Jorge Tuma et al.   Journal of Translational Medicine

Magnetic targeting strategies in gene delivery

(2011) Evgenya Delyagina et al.   Nanomedicine

SUMO1-dependent modulation of SERCA2a in heart failure

(2011) Changwon Kho et al.   NATURE

Randomized transcoronary delivery of CD34+ cells with perfusion versus stop-flow method in patients with recent myocardial infarction: Early cardiac retention of 99mTc-labeled cells activity

(2010) Piotr Musialek et al.   JOURNAL OF NUCLEAR CARDIOLOGY

In vivo myocardial distribution of multipotent progenitor cells following intracoronary delivery in a swine model of myocardial infarction

(2009) Hung Q. Ly et al.   EUROPEAN HEART JOURNAL

Long-Term Safety and Efficacy of Drug-Eluting versus Bare-Metal Stents in Sweden

(2009) Stefan K. James et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

Adeno-Associated Virus Vector Genomes Persist as Episomal Chromatin in Primate Muscle

(2008) M. Penaud-Budloo et al.   JOURNAL OF VIROLOGY

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

Phase I Clinical Trial of Autologous Ascites-derived Exosomes Combined With GM-CSF for Colorectal Cancer

(2008) Shengming Dai et al.   MOLECULAR THERAPY

Percutaneous Transendocardial Delivery of Self-complementary Adeno-associated Virus 6 Achieves Global Cardiac Gene Transfer in Canines

(2008) Lawrence T Bish et al.   MOLECULAR THERAPY

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

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY