Genome editing abrogates angiogenesis in vivo

Nature Communications

Volumn 8, Issue 1, 2017, Pages

  Huang, Xionggao ^a,b   Zhou, Guohong ^a,c   Wu, Wenyi ^a,d   Duan, Yajian ^a,c   Ma, Gaoen ^a   Song, Jingyuan ^a,e   Xiao, Ru ^a   Vandenberghe, Luk ^a   Zhang, Feng ^f   D'Amore, Patricia A ^a   Lei, Hetian ^a  

^a HARVARD MEDICAL SCHOOL   (United States)

^b Hainan Eye Hospital   (China)

^c SHANXI EYE HOSPITAL   (China)

^d CENTRAL SOUTH UNIVERSITY   (China)

^e INSTITUTE OF MEDICINAL PLANT DEVELOPMENT   (China)

^f HARVARD UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ENDONUCLEASE; INTERCELLULAR ADHESION MOLECULE 2; PROMOTER OF INTERCELLULAR ADHESION MOLECULE 2; PROTEIN SPCAS9; UNCLASSIFIED DRUG; VASCULOTROPIN RECEPTOR 2;

ADHESION; CELLS AND CELL COMPONENTS; DIABETES; ENDOLITHON; GENE EXPRESSION; GENETIC ENGINEERING; LABORATORY METHOD; MOLECULAR ANALYSIS;

ADENO ASSOCIATED VIRUS; AGE RELATED MACULAR DEGENERATION; ANGIOGENESIS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT; CONTROLLED STUDY; FEMALE; GENE EDITING; IN VIVO STUDY; LASER-INDUCED CHOROIDAL NEOVASCULARIZATION; MALE; MOUSE; NONHUMAN; OXYGEN-INDUCED RETINOPATHY; PROLIFERATIVE DIABETIC RETINOPATHY; PROTEIN DEPLETION; PROTEIN EXPRESSION; STREPTOCOCCUS PYOGENES; VASCULAR ENDOTHELIAL CELL; ANIMAL; C57BL MOUSE; CELL CULTURE; CRISPR CAS SYSTEM; DEPENDOPARVOVIRUS; ENDOTHELIUM CELL; FLUORESCENCE MICROSCOPY; GENETICS; HUMAN; METABOLISM; NEOVASCULARIZATION (PATHOLOGY); PROCEDURES; RETINA NEOVASCULARIZATION; SUBRETINAL NEOVASCULARIZATION; WESTERN BLOTTING;

ADENO-ASSOCIATED VIRUS; STREPTOCOCCUS PYOGENES;

ANIMALS; BLOTTING, WESTERN; CELLS, CULTURED; CHOROIDAL NEOVASCULARIZATION; CRISPR-CAS SYSTEMS; DEPENDOVIRUS; ENDOTHELIAL CELLS; FEMALE; GENE EDITING; HUMANS; MALE; MICE, INBRED C57BL; MICROSCOPY, FLUORESCENCE; NEOVASCULARIZATION, PATHOLOGIC; RETINAL NEOVASCULARIZATION; VASCULAR ENDOTHELIAL GROWTH FACTOR RECEPTOR-2;

EID: 85026391149     PISSN: None     EISSN: 20411723     Source Type: Journal    
DOI: 10.1038/s41467-017-00140-3     Document Type: Article

References (40)

1. Senger, D. R. et al. Tumor cells secrete a vascular permeability factor that promotes accumulation of ascites fluid. Science 219, 983-985 (1983).
2. Shima, D. T. et al. Hypoxic induction of endothelial cell growth factors in retinal cells: identification and characterization of vascular endothelial growth factor (VEGF) as the mitogen. Mol. Med. 1, 182-193 (1995).
3. Muller, Y. A. et al. Vascular endothelial growth factor: crystal structure and functional mapping of the kinase domain receptor binding site. Proc. Natl Acad. Sci. USA 94, 7192-7197 (1997).
4. Holmes, K., Roberts, O. L., Thomas, A. M. & Cross, M. J. Vascular endothelial growth factor receptor-2: structure, function, intracellular signalling and therapeutic inhibition. Cell Signal. 19, 2003-2012 (2007).
5. Folkman, J. Angiogenesis in cancer, vascular, rheumatoid and other disease. Nat. Med. 1, 27-31 (1995).
6. Williams, R. et al. Epidemiology of diabetic retinopathy and macular oedema: a systematic review. Eye 18, 963-983 (2004).
7. Fraser-Bell, S., Kaines, A. & Hykin, P. G. Update on treatments for diabetic macular edema. Curr. Opin. Ophthalmol. 19, 185-189 (2008).
8. Mintz-Hittner, H. A., Kennedy, K. A., Chuang, A. Z. & Group, B.-R. C. Efficacy of intravitreal bevacizumab for stage 3 + retinopathy of prematurity. N. Engl. J. Med. 364, 603-615 (2011).
9. Stahl, A. et al. The mouse retina as an angiogenesis model. Invest. Ophthalmol. Vis. Sci. 51, 2813-2826 (2010).
10. Chakravarthy, U. et al. Alternative treatments to inhibit VEGF in age-related choroidal neovascularisation: 2-year findings of the IVAN randomised controlled trial. Lancet 382, 1258-1267 (2013).
11. Suzuki, M. et al. Predictive factors for non-response to intravitreal ranibizumab treatment in age-related macular degeneration. Br. J. Ophthalmol. 98, 1186-1191 (2014).
12. Deyle, D. R. & Russell, D. W. Adeno-associated virus vector integration. Curr. Opin. Mol. Ther. 11, 442-447 (2009).
13. Pillay, S. et al. An essential receptor for adeno-associated virus infection. Nature 530, 108-112 (2016).
14. Jinek, M. et al. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science 337, 816-821 (2012).
15. Duan, Y. et al. The clustered, regularly interspaced, short palindromic repeats-associated endonuclease 9 (CRISPR/Cas9)-created MDM2 T309G mutation enhances vitreous-induced expression of MDM2 and proliferation and survival of cells. J. Biol. Chem. 291, 16339-16347 (2016).
16. Swiech, L. et al. In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9. Nat. Biotechnol. 33, 102-106 (2015).
17. Tabebordbar, M. et al. In vivo gene editing in dystrophic mouse muscle and muscle stem cells. Science 351, 407-411 (2016).
18. Chitranshi, N., Gupta, V., Kumar, S. & Graham, S. L. Exploring the molecular interactions of 7,8-Dihydroxyflavone and Its derivatives with TrkB and VEGFR2 proteins. Int. J. Mol. Sci. 16, 21087-21108 (2015).
19. Dai, C., McAninch, R. E. & Sutton, R. E. Identification of synthetic endothelial cell-specific promoters by use of a high-throughput screen. J. Virol. 78, 6209-6221 (2004).
20. Ellis, B. L. et al. A survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with nine natural adeno-associated virus (AAV1-9) and one engineered adeno-associated virus serotype. Virol. J. 10, 74 (2013).
21. Grieger, J. C., Choi, V. W. & Samulski, R. J. Production and characterization of adeno-associated viral vectors. Nat. Protoc. 1, 1412-1428 (2006).
22. Huang, X. et al. Editing VEGFR2 blocks VEGF-induced activation of akt and tube formation. Invest. Ophthalmol. Vis. Sci. 58, 1228-1236 (2017).
23. Connor, K. M. et al. Quantification of oxygen-induced retinopathy in the mouse: a model of vessel loss, vessel regrowth and pathological angiogenesis. Nat. Protoc. 4, 1565-1573 (2009).
24. Lambert, V. et al. Laser-induced choroidal neovascularization model to study age-related macular degeneration in mice. Nat. Protoc. 8, 2197-2211 (2013).
25. Maguire, A. M. et al. Safety and efficacy of gene transfer for leber's congenital amaurosis. N. Engl. J. Med. 358, 2240-2248 (2008).
26. Bainbridge, J. W. et al. Effect of gene therapy on visual function in leber's congenital amaurosis. N. Engl. J. Med. 358, 2231-2239 (2008).
27. Hauswirth, W. W. et al. Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hum. Gene Ther. 19, 979-990 (2008).
28. Jessup, M. et al. Calcium upregulation by percutaneous administration of gene therapy in cardiac disease (CUPID): a phase 2 trial of intracoronary gene therapy of sarcoplasmic reticulum Ca2 + -ATPase in patients with advanced heart failure. Circulation 124, 304-313 (2011).
29. Gaudet, D., Methot, J. & Kastelein, J. Gene therapy for lipoprotein lipase deficiency. Curr. Opin. Lipidol. 23, 310-320 (2012).
30. Gaudet, D. et al. Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial. Gene Ther. 20, 361-369 (2013).
31. Bergers, G. et al. Matrix metalloproteinase-9 triggers the angiogenic switch during carcinogenesis. Nat. Cell Biol. 2, 737-744 (2000).
32. Giraudo, E., Inoue, M. & Hanahan, D. An amino-bisphosphonate targets MMP-9-expressing macrophages and angiogenesis to impair cervical carcinogenesis. J. Clin. Invest. 114, 623-633 (2004).
33. Zhang, F. CRISPR-Cas9: prospects and challenges. Hum. Gene Ther. 26, 409-410 (2015).
34. Cox, D. B., Platt, R. J. & Zhang, F. Therapeutic genome editing: prospects and challenges. Nat. Med. 21, 121-131 (2015).
35. Ruan, G. X. & Kazlauskas, A. Axl is essential for VEGF-A-dependent activation of PI3K/Akt. EMBO J. 31, 1692-1703 (2012).
36. Lei, H. et al. RasGAP promotes autophagy and thereby suppresses platelet-derived growth factor receptor-mediated signaling events, cellular responses, and pathology. Mol. Cell. Biol. 35, 1673-1685 (2015).
37. Saint-Geniez, M. et al. PGC-1alpha regulates normal and pathological angiogenesis in the retina. Am. J. Pathol. 182, 255-265 (2013).
38. Lei, H., Romeo, G. & Kazlauskas, A. Heat shock protein 90alpha-dependent translocation of annexin II to the surface of endothelial cells modulates plasmin activity in the diabetic rat aorta. Circ. Res. 94, 902-909 (2004).
39. Kawamoto, A. et al. Therapeutic potential of ex vivo expanded endothelial progenitor cells for myocardial ischemia. Circulation 103, 634-637 (2001).
40. Giani, A. et al. In vivo evaluation of laser-induced choroidal neovascularization using spectral-domain optical coherence tomography. Invest. Ophthalmol. Vis. Sci. 52, 3880-3887 (2011).