Gene therapy and genome editing for lipoprotein disorders

Area of Science:

• Genetics and Molecular Biology
• Cardiovascular Medicine
• Biotechnology

Background:

• Lipoprotein disorders are significantly influenced by genetic factors, contributing to atherosclerotic cardiovascular disease (ASCVD).
• Current management strategies, primarily lipid-lowering therapies, necessitate lifelong patient adherence.
• Genetic predispositions to dyslipidemias present a substantial challenge in cardiovascular disease etiology.

Purpose of the Study:

• To review major monogenic and polygenic lipoprotein metabolism disorders.
• To discuss emerging genetic-based therapies for managing these conditions.
• To highlight the potential of gene addition and editing technologies in treating lipoprotein disorders.

Main Methods:

• Review of current literature on genetic lipoprotein disorders and therapeutic advancements.
• Discussion of gene addition, gene editing (CRISPR, base editing), and RNA-based therapies.
• Exploration of machine learning and polygenic risk scores for personalized risk stratification.

Main Results:

• Gene addition and editing technologies offer novel, potentially curative approaches for lipoprotein disorders.
• These genetic strategies promise durable treatments, addressing the limitations of lifelong adherence to traditional therapies.
• Personalized medicine approaches, including machine learning and polygenic risk scores, enhance risk prediction and stratification.

Conclusions:

• Gene-based treatments hold transformative potential for managing genetic lipoprotein disorders.
• These advanced therapies could revolutionize ASCVD prevention and treatment paradigms.
• The integration of genetic insights and advanced technologies paves the way for personalized cardiovascular care.