Gene therapy is a potentially promising approach to the management of heart failure. Several strategies have been proposed. Some include administering a useful transgene via a viral vector. These transgenes can either be used to improve cardiac contractility in failing myocardium such as augmenting SERCA2A function or by correcting a mutated gene that is interfering in cardiac function. Another approach is to excise mutated genes using CRISPR Cas9 gene editing. This approach may be particularly helpful in patients with autosomal dominant genetic mutations that interfere in cardiac function such as hypertrophic cardiomyopathies and removing mutated transthyretin genes which cause amyloid. mRNA Technology which was so useful in creating Covid vaccines may offer promise in the future. To date, only small clinical trials have been conducted using either approach to gene therapy with limited success have been conducted.This will likely change in the near future as larger, more expansive clinical trials are planned.

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Gene Therapy for Heart Failure

  • Thomas Thoonkuzhy,
  • Howard J. Eisen

摘要

Gene therapy is a potentially promising approach to the management of heart failure. Several strategies have been proposed. Some include administering a useful transgene via a viral vector. These transgenes can either be used to improve cardiac contractility in failing myocardium such as augmenting SERCA2A function or by correcting a mutated gene that is interfering in cardiac function. Another approach is to excise mutated genes using CRISPR Cas9 gene editing. This approach may be particularly helpful in patients with autosomal dominant genetic mutations that interfere in cardiac function such as hypertrophic cardiomyopathies and removing mutated transthyretin genes which cause amyloid. mRNA Technology which was so useful in creating Covid vaccines may offer promise in the future. To date, only small clinical trials have been conducted using either approach to gene therapy with limited success have been conducted.This will likely change in the near future as larger, more expansive clinical trials are planned.