Abstract
Recent advances in the burgeoning field of genome engineering are accelerating the realization of personalized therapeutics for cardiovascular disease. In the postgenomic era, sequence-specific gene-editing tools enable the functional analysis of genetic alterations implicated in disease. In partnership with high-throughput model systems, efficient gene manipulation provides an increasingly powerful toolkit to study phenotypes associated with patient-specific genetic defects. Herein, this review emphasizes the latest developments in genome engineering and how applications within the field are transforming our understanding of personalized medicine with an emphasis on cardiovascular diseases.
Keywords:
TALENs; cardiovascular disease modeling; genome engineering; induced pluripotent stem cells; zebrafish.
Publication types
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Research Support, N.I.H., Extramural
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Research Support, Non-U.S. Gov't
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Review
MeSH terms
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Animals
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Bacterial Proteins / genetics
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Bacterial Proteins / physiology*
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Binding Sites
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Cardiovascular Diseases / genetics*
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Cardiovascular Diseases / therapy
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Cell Differentiation
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Cells, Cultured / cytology
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Cells, Cultured / transplantation
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DNA / genetics
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DNA / metabolism
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Deoxyribonucleases / genetics
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Deoxyribonucleases / physiology*
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Deoxyribonucleases, Type II Site-Specific / genetics
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Deoxyribonucleases, Type II Site-Specific / physiology*
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Forecasting
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Genes, Reporter
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Genetic Engineering / methods*
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Genetic Engineering / trends
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Genomics*
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Humans
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Induced Pluripotent Stem Cells / cytology*
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Induced Pluripotent Stem Cells / transplantation
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Models, Cardiovascular
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Models, Genetic
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Mutagenesis, Site-Directed / methods*
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Precision Medicine / trends*
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Protein Structure, Tertiary
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Recombinant Fusion Proteins / physiology
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Recombinational DNA Repair
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Substrate Specificity
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Xanthomonas / enzymology*
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Xanthomonas axonopodis / enzymology
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Zebrafish / genetics
Substances
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Bacterial Proteins
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Recombinant Fusion Proteins
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DNA
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Deoxyribonucleases
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endodeoxyribonuclease FokI
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Deoxyribonucleases, Type II Site-Specific