Gene therapy articles within Nature Reviews Cardiology

Featured

  • Research Highlight |

    Gene therapy involving adenine base editing can correct a pathogenic variant in the Scn5a gene and alleviate arrhythmia phenotypes in a mouse model of long QT syndrome type 3.

    • Irene Fernández-Ruiz

  • Review Article |

    In this Review, the authors discuss current treatment regimens for lowering plasma LDL cholesterol levels to reduce the risk of cardiovascular disease, highlight treatment gaps and challenges, as well as describe opportunities raised by novel available therapies and potential future therapeutic approaches.

    • Julia Brandts
    •  & Kausik K. Ray

  • Research Highlight |

    Adenine base editing can be used to correct a genetic variant in preclinical models of hypertrophic cardiomyopathy, and this approach could be applied to other monogenic cardiac diseases.

    • Gregory B. Lim

  • Research Highlight |

    Gene therapy targeting the protein trafficking regulator MOG1 successfully reversed clinical features of Brugada syndrome in a knock-in mouse model.

    • Karina Huynh

  • Review Article |

    Numerous new CRISPR-based genome-editing tools have expanded the application of genome editing to include gene-expression control, epigenome editing, RNA editing and live-cell imaging. In this Review, Wu and colleagues describe how these novel CRISPR tools are used to investigate biological processes and disease pathophysiology for cardiovascular research and medicine.

    • Masataka Nishiga
    • , Chun Liu
    •  & Joseph C. Wu

  • Clinical Outlook |

    Human genetic studies combined with biotechnological advances have guided and accelerated the development of PCSK9-targeting therapies. In this Clinical Outlook, we highlight present and future approaches for PCSK9 inhibition to reduce LDL-cholesterol levels and the risk of atherosclerotic cardiovascular disease.

    • Mahmoud Al Rifai
    •  & Christie M. Ballantyne

  • Research Highlight |

    A form of adenine base editing has been used in mouse embryos to correct a genetic variant associated with hypertrophic cardiomyopathy. This approach is potentially safer than other forms of germline gene editing, which carry a risk of off-target editing and the introduction of indels.

    • Gregory B. Lim

  • Research Highlight |

    PCSK9 inhibitors are a class of cholesterol-lowering drugs that regulate LDL-receptor degradation. Three new studies show successful reduction of PCSK9 levels by different approaches that all resulted in the lowering of LDL-cholesterol levels.

    • Andrew Robson

  • Review Article |

    For therapeutic materials to be delivered to the heart, several barriers need to be overcome. In this Review, Ishikawa and colleagues discuss strategies for targeted delivery of therapeutic materials to the heart, including the use of adeno-associated viruses and exosomes, with a focus on agents directed at modifying gene expression.

    • Susmita Sahoo
    • , Taro Kariya
    •  & Kiyotake Ishikawa

  • Research Highlight |

    Gene therapy with H19, a highly conserved long non-coding RNA H19, prevents and reverses pathological cardiac hypertrophy in animal models, according to a study by Thomas Thum and colleagues.

    • Irene Fernández-Ruiz

  • Research Highlight |

    Cardiomyocyte-selective gene therapy to inhibit calcium/calmodulin-dependent protein kinase II effectively suppresses ventricular arrhythmias in mice with mutations known to cause catecholaminergic polymorphic ventricular tachycardia.

    • Gregory B. Lim

  • Review Article |

    With the increase in the availability of RNA screening tools, numerous non-coding RNAs with important functions in the heart have been identified. In this Review, Lu and Thum discuss the non-coding RNA candidates with diagnostic and therapeutic potential in cardiovascular disease.

    • Dongchao Lu
    •  & Thomas Thum

  • Review Article |

    Conduction system disorders lead to slow heart rates that are insufficient to support the circulation, necessitating implantation of electronic pacemakers. Current pacemakers, although effective, have limitations including lead malfunction, lack of autonomic responsiveness, and device-related infections. In this Review, Marbán and colleagues discuss next-generation electronic devices designed to address current limitations, as well as biological pacemakers as alternatives to implantable hardware.

    • Eugenio Cingolani
    • , Joshua I. Goldhaber
    •  & Eduardo Marbán

  • Review Article |

    Genome editing is being rapidly adopted into all fields of biomedical research, including the cardiovascular field. In this Review, Strong and Musunuru discuss the applications of genome-editing technology, including zinc finger nucleases, TALENs, and CRISPR/Cas9 systems, throughout cardiovascular disease research, their current limitations, and the prospect ofin vivogenome-editing therapies in the future.

    • Alanna Strong
    •  & Kiran Musunuru

  • Review Article |

    Gene therapy for cardiac arrhythmias is now undergoing clinical testing. In this Review, Bongianino and Priori discuss the principles of gene therapy and how this approach can be tailored and targeted to the heart. They then summarize the preclinical and clinical experience of gene therapy applied to acquired and inherited arrhythmias of the atria or ventricles.

    • Rossana Bongianino
    •  & Silvia G. Priori

  • Review Article |

    Dysfunctional blood vessel regulation is a key component in the pathophysiology of heart failure. Vascular endothelial growth factor (VEGF) has an essential role in the formation of new vessels. In this Review, Taimeh et al. outline the biological characterization of VEGF, and examine the evidence for its potential therapeutic application, including the novel concept of VEGF as adjuvant therapy to stem cell transplantation, in patients with heart failure.

    • Ziad Taimeh
    • , John Loughran
    •  & Roberto Bolli

Browse broader subjects

Browse narrower subjects

Browse Gene therapy across other nature.com journals