New and TALENted genome engineering toolbox.
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.
Center for Translational Science Activities, Mayo Clinic, Rochester, MN 55905, USA. ekker.stephen@mayo.edu
Jarryd M
JM
Campbell
Katherine A
KA
Hartjes
Timothy J
TJ
Nelson
Xiaolei
X
Xu
Stephen C
SC
Ekker
D000818:Animals; D001426:Bacterial Proteins / Q000235:genetics / Q000502:physiology*; D001665:Binding Sites; D002318:Cardiovascular Diseases / Q000235:genetics* / Q000628:therapy; D002454:Cell Differentiation; D002478:Cells, Cultured / Q000166:cytology / Q000637:transplantation; D004247:DNA / Q000235:genetics / Q000378:metabolism; D003851:Deoxyribonucleases / Q000235:genetics / Q000502:physiology*; D015252:Deoxyribonucleases, Type II Site-Specific / Q000235:genetics / Q000502:physiology*; D005544:Forecasting; D017930:Genes, Reporter; D005818:Genetic Engineering / Q000379:methods* / Q000639:trends; D023281:Genomics; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000166:cytology* / Q000637:transplantation; D008955:Models, Cardiovascular; D008957:Models, Genetic; D016297:Mutagenesis, Site-Directed / Q000379:methods*; D057285:Precision Medicine / Q000639:trends*; D017434:Protein Structure, Tertiary; D011993:Recombinant Fusion Proteins / Q000502:physiology; D059767:Recombinational DNA Repair; D013379:Substrate Specificity; D014974:Xanthomonas / Q000201:enzymology*; D053532:Xanthomonas axonopodis / Q000201:enzymology; D015027:Zebrafish / Q000235:genetics
D001426:Bacterial Proteins; D011993:Recombinant Fusion Proteins; D004247:DNA; D003851:Deoxyribonucleases; C035042:endodeoxyribonuclease FokI; D015252:Deoxyribonucleases, Type II Site-Specific
TALENs; cardiovascular disease modeling; genome engineering; induced pluripotent stem cells; zebrafish