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Lentiviral CRISPR/Cas9-Mediated Genome Editing for the Study of Hematopoietic Cells in Disease Models
Published on: October 3, 2019
Gene/Genome Editing in Cardiovascular Biology and Disease
Tushar Verma1, Ravi Pratap Singh2, Mahima Chaudhary1
1School of Pharmacy, Lingaya's Vidyapeeth, Faridabad, Haryana, India.
Current Gene Therapy
|July 20, 2026
Summary
Genome editing technologies like CRISPR/Cas9 offer a new way to treat cardiovascular diseases by correcting genetic defects. Further research is needed to overcome challenges for successful clinical application.
Area of Science:
- Cardiovascular Science
- Genetics
- Biotechnology
Background:
- Cardiovascular diseases remain a leading global cause of death.
- Traditional treatments manage symptoms but do not address root genetic causes.
- Genome editing tools, including CRISPR/Cas9, are revolutionizing cardiovascular research and therapy development.
Purpose of the Study:
- To review recent advancements in genome editing for cardiovascular diseases.
- To highlight the potential of CRISPR/Cas9, base editing, and prime editing in treating genetic cardiovascular conditions.
- To discuss the current status and future challenges of genome editing in cardiology.
Main Methods:
- Systematic literature review of peer-reviewed articles published between 2020 and 2025.
- Searched major scientific databases (PubMed, Web of Science, Scopus) using keywords related to genome editing and cardiovascular diseases.
- Included studies on CRISPR/Cas9, base editing, prime editing, cardiomyopathy, and atherosclerosis.
Main Results:
- CRISPR/Cas9 facilitates rapid creation of cardiac models for studying genetic variations.
- Base editing enables precise single-nucleotide corrections, such as PCSK9 targeting for LDL cholesterol reduction.
- Prime editing addresses complex mutations (e.g., RBM20 in dilated cardiomyopathy), and early trials show in vivo genome editing feasibility.
- Improved cardiac delivery systems (AAV vectors, lipid nanoparticles) enhance safety and efficacy.
Conclusions:
- Genome editing transforms cardiovascular research from association to causal intervention.
- Next-generation editors minimize risks associated with double-strand breaks, improving clinical potential.
- Key challenges include efficient tissue-specific delivery, off-target effects, immunogenicity, and ethical considerations for permanent genetic modifications.
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