Direct gene transfer with compacted DNA nanoparticles in retinal pigment epithelial cells: Expression, repeat delivery and lack of toxicity

Nanomedicine

Volumn 7, Issue 4, 2012, Pages 521-539

  Han, Zongchao ^a   Koirala, Adarsha ^a   Makkia, Rasha ^a   Cooper, Mark J ^b   Naash, Muna I ^a  

^a UNIVERSITY OF OKLAHOMA COLLEGE OF MEDICINE   (United States)

^b Copernicus Therapeutics Inc ^*  (United States)

Author keywords

enhanced green fluorescent protein; gene transfer; nanoparticle; nonviral; repeat delivery; retinal pigment epithelial cell; subretinal injection; toxicity

Indexed keywords

BETA ACTIN; COMPACTED DNA NANOPARTICLE; DNA; ENHANCED GREEN FLUORESCENT PROTEIN; GAMMA INTERFERON; INTERLEUKIN 1BETA; INTERLEUKIN 6; MACROGOL; SODIUM CHLORIDE; TUMOR NECROSIS FACTOR ALPHA; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ARTICLE; COHORT ANALYSIS; CONTROLLED STUDY; DNA VECTOR; DRUG EFFICACY; DRUG SAFETY; ELECTRORETINOGRAPHY; IMMUNOHISTOCHEMISTRY; MACROPHAGE; MOUSE; NEUTROPHIL; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PIGMENT EPITHELIUM; PLASMID; PRIORITY JOURNAL; PROMOTER REGION; PROTEIN ANALYSIS; RETINA MACULA DEGENERATION; SUBSTITUTION REACTION; SYNTHESIS; VISUAL SYSTEM FUNCTION; VITELLOGENESIS; WILD TYPE;

ANIMALS; DNA; EPITHELIAL CELLS; GENE TRANSFER TECHNIQUES; GREEN FLUORESCENT PROTEINS; MICE; MICE, INBRED BALB C; NANOPARTICLES; RETINAL PIGMENT EPITHELIUM;

EID: 84859454384     PISSN: 17435889     EISSN: 17486963     Source Type: Journal    
DOI: 10.2217/nnm.11.158     Document Type: Article

References (42)

1. Ziady AG, Gedeon CR, Miller T et al. Transfection of airway epithelium by stable PEGylated poly-L-lysine DNA nanoparticles in vivo. Mol. Ther. 8(6), 936-947 (2003). (Pubitemid 38072306)
2. Liu G, Li D, Pasumarthy MK et al. Nanoparticles of compacted DNA transfect postmitotic cells. J. Biolog. Chem. 278 (35), 32578-32586 (2003). (Pubitemid 37055695)
3. Ziady AG, Gedeon CR, Muhammad O et al. Minimal toxicity of stabilized compacted DNA nanoparticles in the murine lung. Mol. Ther. 8(6), 948-956 (2003). (Pubitemid 38072307)
4. Cai X, Conley SM, Nash Z, Fliesler SJ, Cooper MJ, Naash MI. Gene delivery to mitotic and postmitotic photoreceptors via compacted DNA nanoparticles results in improved phenotype in a mouse model of retinitis pigmentosa. FASEB J. 24 (4), 1178-1191 (2010).
5. Farjo R, Skaggs J, Quiambao AB, Cooper MJ, Naash MI. Effcient non-viral ocular gene transfer with compacted DNA nanoparticles. PLoS ONE 1, e38 (2006).
6. Fink TL, Klepcyk PJ, Oette SM et al. Plasmid size up to 20 kbp does not limit effective in vivo lung gene transfer using compacted DNA nanoparticles. Gene Ther. 13(13), 1048-1051 (2006). (Pubitemid 43905857)
7. Yurek DM, Fletcher AM, Smith GM et al. Long-term transgene expression in the central nervous system using DNA nanoparticles. Mol. Ther. 17(4), 641-650 (2009).
8. Ziady AG, Davis PB, Konstan MW. Non-viral gene transfer therapy for cystic fbrosis. Exp. Opin Biol. Ther. 3 (3), 449-458 (2003). (Pubitemid 36712287)
9. Konstan M W, Davis PB, Wagener JS et al. Compacted DNA nanoparticles administered to the nasal mucosa of cystic fbrosis subjects are safe and demonstrate partial to complete cystic fbrosis transmembrane regulator reconstitution. Hum. Gene Ther. 15 (12), 1255-1269 (2004). (Pubitemid 40052437)
10. Ding XQ, Quiambao AB, Fitzgerald JB, Cooper MJ, Conley SM, Naash MI. Ocular delivery of compacted DNA-nanoparticles does not elicit toxicity in the mouse retina. PLoS ONE 4(10), e7410 (2009).
11. Reyes-Sandoval A, Ertl HC. CpG methylation of a plasmid vector results in extended transgene product expression by circumventing induction of immune responses. Mol. Ther. 9(2), 249-261 (2004). (Pubitemid 38256551)
12. Yew NS, Zhao H, Przybylska M et al. CpG-depleted plasmid DNA vectors with enhanced safety and long-term gene expression in vivo. Mol. Ther. 5(6), 731-738 (2002). (Pubitemid 34619066)
13. Yew NS, Zhao H, Wu IH et al. Reduced infammatory response to plasmid DNA vectors by elimination and inhibition of immunostimulatory CpG motifs. Mol. Ther. 1(3), 255-262 (2000).
14. Hyde SC, Pringle IA, Abdullah S et al. CpG-free plasmids confer reduced infammation and sustained pulmonary gene expression. Nat. Biotechnol. 26(5), 549-551 (2008). (Pubitemid 351668040)
15. Przybylska M, Wu IH, Zhao H et al. Partial correction of the a-galactosidase A defciency and reduction of glycolipid storage in Fabry mice using synthetic vectors. J. Gene. Med. 6(1), 85-92 (2004). (Pubitemid 40228006)
16. Koirala A, Makkia RS, Cooper MJ, Naash MI. Nanoparticle-mediated gene transfer specifc to retinal pigment epithelial cells. Biomaterials 32(35), 9483-9493 (2011).
17. Esumi N, Oshima Y, Li Y, Campochiaro PA, Zack DJ. Analysis of the VMD2 promoter and implication of E-box binding factors in its regulation. J. Biolog. Chem. 279 (18), 19064-19073 (2004). (Pubitemid 38623337)
18. Nour M, Quiambao AB, Peterson WM, Al-Ubaidi MR, Naash MI. P2Y (2) receptor agonist INS37217 enhances functional recovery after detachment caused by subretinal injection in normal and rds mice. Invest. Ophthalmol. Vis. Sci. 44 (10), 4505-4514 (2003). (Pubitemid 37186289)
19. Cai X, Conley SM, Naash MI. RPE65: role in the visual cycle, human retinal disease, and gene therapy. Ophthalmic Genet. 30(2), 57-62 (2009).
20. Hulse RE, Kunkler PE, Fedynyshyn JP, Kraig RP. Optimization of multiplexed bead-based cytokine immunoassays for rat serum and brain tissue. J. Neurosci. Methods 136(1), 87-98 (2004). (Pubitemid 38569875)
21. Chen ZY, He CY, Meuse L, Kay MA. Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo. Gene Ther. 11(10), 856-864 (2004). (Pubitemid 38714589)
22. Chen ZY, Yant SR, He CY, Meuse L, Shen S, Kay MA. Linear DNAs concatemerize in vivo and result in sustained transgene expression in mouse liver. Mol. Ther. 3(3), 403-410 (2001). (Pubitemid 32514657)
23. Butterfeld TA, Best TN, Merrick MA. The dual roles of neutrophils and macrophages in infammation: a critical balance between tissue damage and repair. J. Athl. Train. 41(4), 457-465 (2006).
24. Lu CH, Chao PC, Borel CO et al. Preincisional intravenous pentoxifylline attenuating perioperative cytokine response, reducing morphine consumption, and improving recovery of bowel function in patients undergoing colorectal cancer surgery. Anesth. Analg. 99 (5), 1465-1471 (2004). (Pubitemid 39407300)
25. Bryan D, Walker KB, Ferguson M, Thorpe R. Cytokine gene expression in a murine wound healing model. Cytokine 31(6), 429-438 (2005). (Pubitemid 41242767)
26. Sato Y, Ohshima T. The expression of mRNA of proinfammatory cytokines during skin wound healing in mice: a preliminary study for forensic wound age estimation (II). Int. J. Legal Med. 113 (3), 140-145 (2000). (Pubitemid 30339650)
27. Ahn DK, Chae JM, Choi HS et al. Central cyclooxygenase inhibitors reduced IL-1b-induced hyperalgesia in temporomandibular joint of freely moving rats. Pain 117(1-2), 204-213 (2005). (Pubitemid 41764788)
28. Inglis JJ, Nissim A, Lees DM, Hunt SP, Chernajovsky Y, Kidd BL. The differential contribution of tumour necrosis factor to thermal and mechanical hyperalgesia during chronic infammation. Arthr. Res. Ther. 7(4), R807-R816 (2005).
29. Vissers KC, De Jongh RF, Hoffmann VL, Meert TF. Exogenous interleukin-6 increases cold allodynia in rats with a mononeuropathy. Cytokine 30(4), 154-159 (2005). (Pubitemid 40615962)
30. Clark JD, Shi X, Li X et al. Morphine reduces local cytokine expression and neutrophil infltration after incision. Mol. Pain 3, 28 (2007).
31. Liang D, Shi X, Qiao Y, Angst MS, Yeomans DC, Clark JD. Chronic morphine administration enhances nociceptive sensitivity and local cytokine production after incision. Mol. Pain 4, 7 (2008).
32. Kawasaki T, Ogata M, Kawasaki C, Tomihisa T, Okamoto K, Shigematsu A. Surgical stress induces endotoxin hyporesponsiveness and an early decrease of monocyte mCD14 and HLA-DR expression during surgery. Anesth. Analg. 92(5), 1322-1326 (2001). (Pubitemid 32385966)
33. Ebrahimi M, Roudkenar MH, Imani Fooladi AA et al. Discrepancy between mRNA and protein expression of neutrophil gelatinase-associated lipocalin in bronchial epithelium induced by sulfur mustard. J. Biomed. Biotechnol. 2010, 823131 (2010).
34. Shebl FM, Pinto LA, Garcia-Pineres A et al. Comparison of mRNA and protein measures of cytokines following vaccination with human papillomavirus-16 L1 virus-like particles.Cancer Epidemiol. Biomarkers Prev. 19(4), 978-981 (2010).
35. Lichtinghagen R, Musholt PB, Lein M et al. Different mRNA and protein expression of matrix metalloproteinases 2 and 9 and tissue inhibitor of metalloproteinases 1 in benign and malignant prostate tissue. Eur. Urol. 42(4), 398-406 (2002). (Pubitemid 35175803)
36. Anderson L, Seilhamer J. A comparison of selected mRNA and protein abundances in human liver. Electrophoresis 18 (3-4), 533-537 (1997). (Pubitemid 27174443)
37. Nour M, Ding XQ, Stricker H, Fliesler SJ, Naash MI. Modulating expression of peripherin/rds in transgenic mice: critical levels and the effect of overexpression. Invest. Ophthalmol. Vis. Sci. 45(8), 2514-2521 (2004). (Pubitemid 38998844)
38. Li Q, Miller R, Han PY et al. Intraocular route of AAV2 vector administration defnes humoral immune response and therapeutic potential. Mol. Vis. 14, 1760-1769 (2008).
39. Li W, Kong F, Li X et al. Gene therapy following subretinal AAV5 vector delivery is not affected by a previous intravitreal AAV5 vector administration in the partner eye. Mol. Vis. 15, 267-275 (2009).
40. Amado D, Mingozzi F, Hui D et al. Safety and effcacy of subretinal readministration of a viral vector in large animals to treat congenital blindness. Sci. Transl. Med. 2(21), 21ra16 (2010).
41. Barker SE, Broderick CA, Robbie SJ et al. Subretinal delivery of adeno-associated virus serotype 2 results in minimal immune responses that allow repeat vector administration in immunocompetent mice. J. Gene. Med. 11(6), 486-497 (2009).
42. Cai X, Conley SM, Naash MI. Gene therapy in the retinal degeneration slow model of retinitis pigmentosa. Adv. Exp. Med. Biol. 664, 611-619 (2010).