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Big Data and Genome Editing Technology: A New Paradigm of Cardiovascular Genomics
Chayakrit Krittanawong1, Tao Sun2, Eyal Herzog3
1Department of Internal Medicine, Icahn School of Medicine at Mount Sinai, 1000 10th Ave, New York, NY 10019. United States.
Insights
Genome-editing technologies offer new therapeutic strategies for cardiovascular diseases (CVDs) by precisely modifying DNA. These advanced techniques hold promise for correcting genetic defects and developing novel CVD treatments.
Area of Science:
- Cardiovascular Medicine
- Genetics
- Molecular Biology
Background:
- Cardiovascular diseases (CVDs) are a diverse group of conditions with complex and often poorly understood genetic underpinnings.
- While genetic factors play a role in various CVDs, their precise mechanisms remain a significant challenge in clinical care.
Purpose of the Study:
- To explore the impact of genome-editing technologies on understanding and treating cardiovascular diseases.
- To discuss the potential of gene-editing tools in addressing the genetic basis of CVDs.
Main Methods:
- Review of current literature on genome-editing technologies, including zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and CRISPR/Cas9 systems.
- Analysis of genetic polymorphisms and mechanistic insights relevant to cardiovascular disease pathogenesis.
- Discussion of the application of site-specific genome engineering in a therapeutic context.
Main Results:
- Genome-editing technologies enable precise modification of DNA sequences for therapeutic purposes, such as correcting disease-causing variants or adding therapeutic genes.
- These technologies offer novel opportunities for developing targeted therapies for cardiovascular diseases.
- The potential applications range from correcting inherited cardiac conditions to addressing acquired forms of CVD.
Conclusions:
- Genome editing represents a transformative approach in cardiovascular medicine, offering new avenues for therapeutic intervention.
- Further research and development are crucial to overcome current challenges and fully realize the future prospects of genome editing in cardiovascular clinical care.
Abstract:
Opinion Statements: Cardiovascular diseases (CVDs) encompass a range of conditions extending from congenital heart disease to acute coronary syndrome most of which are heterogenous in nature and some of them are multiple genetic loci. However, the pathogenesis of most CVDs remains incompletely understood. The advance in genome-editing technologies, an engineering process of DNA sequences at precise genomic locations, has enabled a new paradigm that human genome can be precisely modified to achieve a therapeutic effect. Genome-editing includes the correction of genetic variants that cause disease, the addition of therapeutic genes to specific sites in the genomic locations, and the removal of deleterious genes or genome sequences. Site-specific genome engineering can be used as nucleases (known as molecular scissors) including zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated 9 (Cas9) systems to provide remarkable opportunities for developing novel therapies in cardiovascular clinical care. Here we discuss genetic polymorphisms and mechanistic insights in CVDs with an emphasis on the impact of genome-editing technologies. The current challenges and future prospects for genomeediting technologies in cardiovascular medicine are also discussed.
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