Genome Editing in Cardiovascular Biology

Circulation Research

Volumn 120, Issue 5, 2017, Pages 778-780

  Seeger, Timon ^a,b   Porteus, Matthew ^c,d   Wu, Joseph C ^a,b,d  

^a STANFORD UNIVERSITY   (United States)

^b Department of Medicine   (United States)

^c Department of Pediatrics   (United States)

^d STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

cardiomyopathies; cardiovascular diseases; CRISPR; gene editing; induced pluripotent stem cells

Indexed keywords

CARDIOVASCULAR DISEASE; CARDIOVASCULAR SYSTEM; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT; EX VIVO STUDY; GENE EDITING; HEART FIBROBLAST; HUMAN; IN VIVO STUDY; NONHUMAN; PRIORITY JOURNAL; REVIEW; TRANSLATIONAL RESEARCH; CARDIAC MUSCLE; CARDIAC MUSCLE CELL; CARDIOLOGY; CELL CULTURE; CYTOLOGY; EMBRYONIC STEM CELL; FIBROBLAST; GENETICS; HUMAN GENOME; INDUCED PLURIPOTENT STEM CELL; PHYSIOLOGY; PROCEDURES; SMOOTH MUSCLE CELL; TRANSPLANTATION; TRENDS;

CARDIOLOGY; CARDIOVASCULAR DISEASES; CELLS, CULTURED; EMBRYONIC STEM CELLS; FIBROBLASTS; GENE EDITING; GENOME, HUMAN; HUMANS; INDUCED PLURIPOTENT STEM CELLS; MYOCARDIUM; MYOCYTES, CARDIAC; MYOCYTES, SMOOTH MUSCLE; TRANSLATIONAL MEDICAL RESEARCH;

EID: 85014647695     PISSN: 00097330     EISSN: 15244571     Source Type: Journal    
DOI: 10.1161/CIRCRESAHA.116.310197     Document Type: Review

References (17)

1. O'Connell MR, Oakes BL, Sternberg SH, East-Seletsky A, Kaplan M, Doudna JA, Programmable RNA recognition and cleavage by CRISPR/Cas9. Nature 2014 516 263 266. doi: 10.1038/nature13769
2. Gilbert LA, Horlbeck MA, Adamson B, Villalta JE, Chen Y, Whitehead EH, Guimaraes C, Panning B, Ploegh HL, Bassik MC, Qi LS, Kampmann M, Weissman JS, Genome-scale CRISPR-mediated control of gene repression and activation. Cell 2014 159 647 661. doi: 10.1016/j.cell.2014.09.029
3. Matsa E, Ahrens JH, Wu JC, Human induced pluripotent stem cells as a platform for personalized and precision cardiovascular medicine. Physiol Rev 2016 96 1093 1126. doi: 10.1152/physrev.00036.2015
4. Tzatzalos E, Abilez OJ, Shukla P, Wu JC, Engineered heart tissues and induced pluripotent stem cells: Macro-and microstructures for disease modeling, drug screening, and translational studies. Adv Drug Deliv Rev 2016 96 234 244. doi: 10.1016/j.addr.2015.09.010
5. Veres A, Gosis BS, Ding Q, Collins R, Ragavendran A, Brand H, Erdin S, Cowan CA, Talkowski ME, Musunuru K, Low incidence of off-target mutations in individual CRISPR-Cas9 and TALEN targeted human stem cell clones detected by whole-genome sequencing. Cell Stem Cell 2014 15 27 30. doi: 10.1016/j.stem.2014.04.020
6. Menasché P, Vanneaux V, Hagège A, Human embryonic stem cell-derived cardiac progenitors for severe heart failure treatment: first clinical case report. Eur Heart J 2015 36 2011 2017. doi: 10.1093/eurheartj/ehv189
7. Neofytou E, O'Brien CG, Couture LA, Wu JC, Hurdles to clinical translation of human induced pluripotent stem cells. J Clin Invest 2015 125 2551 2557. doi: 10.1172/JCI80575
8. Nguyen PK, Neofytou E, Rhee J-W, Wu JC, Potential strategies to address the major clinical barriers facing stem cell regenerative therapy for cardiovascular disease: areview. JAMA Cardiol 2016 1 953 962. doi: 10.1001/jamacardio.2016.2750
9. Wang Y, Zhang WY, Hu S, Genome editing of human embryonic stem cells and induced pluripotent stem cells with zinc finger nucleases for cellular imaging. Circ Res 2012 111 1494 1503. doi: 10.1161/CIRCRESAHA.112.274969
10. Ran FA, Cong L, Yan WX, Scott DA, Gootenberg JS, Kriz AJ, Zetsche B, Shalem O, Wu X, Makarova KS, Koonin EV, Sharp PA, Zhang F, In vivo genome editing using Staphylococcus aureus Cas9. Nature 2015 520 186 191. doi: 10.1038/nature14299
11. Mendell JR, Rodino-Klapac LR, Duchenne muscular dystrophy: CRISPR/Cas9 treatment. Cell Res 2016 26 513 514. doi: 10.1038/cr.2016.28
12. Yang Y, Wang L, Bell P, McMenamin D, He Z, White J, Yu H, Xu C, Morizono H, Musunuru K, Batshaw ML, Wilson JM, A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice. Nat Biotechnol 2016 34 334 338. doi: 10.1038/nbt.3469
13. Heyer WD, Ehmsen KT, Liu J, Regulation of homologous recombination in eukaryotes. Annu Rev Genet 2010 44 113 139. doi: 10.1146/annurev-genet-051710-150955
14. Gramlich M, Pane LS, Zhou Q, Antisense-mediated exon skipping: a therapeutic strategy for titin-based dilated cardiomyopathy. EMBO Mol Med 2015 7 562 576. doi: 10.15252/emmm.201505047
15. Gedicke-Hornung C, Behrens-Gawlik V, Reischmann S, Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in mice. EMBO Mol Med 2013 5 1128 1145. doi: 10.1002/emmm.201202168
16. Ehret GB, Ferreira T, Chasman DI, CHARGE-EchoGen Consortium; CHARGE-HF Consortium; Wellcome Trust Case Control Consortium The genetics of blood pressure regulation and its target organs from association studies in 342,415 individuals. Nat Genet 2016 48 1171 1184. doi: 10.1038/ng.3667
17. Nikpay M, Goel A, Won HH, CARDIoGRAMplusC4D Consortium A comprehensive 1,000 Genomes-based genome-wide association meta-analysis of coronary artery disease. Nat Genet 2015 47 1121 1130. doi: 10.1038/ng.3396