Gene Therapy for Cardiovascular Disease: Basic Research and Clinical Prospects

Area of Science:

• Genetics
• Molecular Biology
• Cardiovascular Research

Background:

• Genetic factors significantly influence human diseases, driving research in life sciences.
• Gene-editing technologies have evolved from homologous recombination to zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and CRISPR/Cas9 systems.
• Cardiovascular disease (CVD) is a major cause of morbidity and mortality, with inherited forms often resistant to conventional therapies.

Purpose of the Study:

• To review the types and mechanisms of gene-editing techniques.
• To discuss the ethical considerations surrounding gene therapy.
• To explore the current applications of gene therapy in cardiovascular diseases, including atherosclerosis, inherited CVDs, and in-stent restenosis, along with delivery systems.

Main Methods:

• Review of scientific literature on gene-editing technologies.
• Analysis of the mechanisms and applications of ZFNs, TALENs, and CRISPR/Cas9.
• Examination of gene therapy strategies for cardiovascular conditions.

Main Results:

• Gene-editing technologies provide potential therapeutic avenues for genetic and refractory diseases.
• CRISPR/Cas9 system represents a significant advancement in gene-editing capabilities.
• Initial applications of gene therapy in cardiovascular disease models show promise but face obstacles.

Conclusions:

• Gene editing holds promise for treating cardiovascular diseases, particularly inherited forms.
• Clinical application of gene therapy for CVDs is hindered by incomplete disease understanding, technological limitations, and ethical concerns.
• Further research into gene editing mechanisms, delivery systems, and ethical frameworks is crucial for clinical translation.