Recent advances in genome editing for cardiovascular disease

Area of Science:

• Genetics
• Cardiovascular Medicine
• Biotechnology

Background:

• Cardiovascular disease (CVD) remains a leading cause of mortality worldwide.
• Genetic factors play a significant role in the development of various CVDs.
• Understanding the pathogenicity of genetic variants is crucial for diagnosis and treatment.

Purpose of the Study:

• To review recent advancements in applying genome editing technologies to cardiovascular disease.
• To highlight the use of genome editing in studying disease mechanisms and developing therapeutic strategies.
• To discuss the potential of genome editing for personalized medicine in CVD.

Main Methods:

• Utilizing patient-derived induced pluripotent stem cells (iPSCs) to model CVD.
• Employing genome editing techniques to assess variant pathogenicity in iPSCs.
• Developing and testing somatic genome editing strategies in preclinical models (animal models).

Main Results:

• Genome editing in patient-derived iPSCs allows for functional assessment of genetic variants.
• Personalized therapeutic genome editing approaches can be tested in patient-specific iPSCs.
• Proof-of-concept studies in animal models demonstrate the potential of somatic genome editing for CVD treatment, particularly targeting the liver and heart.

Conclusions:

• Genome editing offers promising avenues for both understanding and treating cardiovascular diseases.
• Further technological development is needed for clinical translation, focusing on precision, delivery, and safety (off-target effects, immune response).
• Successful translation could lead to improved diagnostics and permanent correction of severe CVDs.