Nanoparticle-assisted targeted delivery of eye-specific genes to eyes significantly improves the vision of blind mice in vivo

Nano Letters

Volumn 14, Issue 9, 2014, Pages 5257-5263

  Rajala, Ammaji ^a,b   Wang, Yuhong ^a,b   Zhu, Ye ^c   Ranjo Bishop, Michelle ^a,b   Ma, Jian Xing ^a   Mao, Chuanbin ^c   Rajala, Raju V S ^a,b  

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

^b UNIVERSITY OF OKLAHOMA HEALTH SCIENCES CENTER   (United States)

^c UNIVERSITY OF OKLAHOMA   (United States)

Author keywords

eyes; gene therapy; Lipid nanoparticles; nonviral vector; retina; retinal pigment epithelium

Indexed keywords

ALDEHYDES; BIOCOMPATIBILITY; CELL SIGNALING; DISEASES; GENE THERAPY; GENE TRANSFER; MAMMALS; NANOPARTICLES; OPHTHALMOLOGY; PEPTIDES; VIRUSES;

EYES; LIPID NANOPARTICLES; NONVIRAL VECTORS; RETINA; RETINAL PIGMENT EPITHELIUM;

GENE EXPRESSION;

BIOMATERIAL; CIS TRANS ISOMERASE; DNA; GREEN FLUORESCENT PROTEIN; LIPID; LIPOSOME; NANOPARTICLE; NUCLEAR LOCALIZATION SIGNAL; PEPTIDE; PROTAMINE; RETINOID ISOMEROHYDROLASE;

ANIMAL; BAGG ALBINO MOUSE; BLINDNESS; CHEMISTRY; GENE THERAPY; GENE TRANSFER; GENETICS; KNOCKOUT MOUSE; METABOLISM; MOUSE; NANOMEDICINE; NUCLEAR LOCALIZATION SIGNAL; PHOTOCHEMISTRY; PHYSIOLOGY; PIGMENT EPITHELIUM; PROCEDURES; RETINA; VISION;

ANIMALS; BIOCOMPATIBLE MATERIALS; BLINDNESS; CIS-TRANS-ISOMERASES; DNA; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GREEN FLUORESCENT PROTEINS; LIPIDS; LIPOSOMES; MICE; MICE, INBRED BALB C; MICE, KNOCKOUT; NANOMEDICINE; NANOPARTICLES; NUCLEAR LOCALIZATION SIGNALS; PEPTIDES; PHOTOCHEMISTRY; PROTAMINES; RETINA; RETINAL PIGMENT EPITHELIUM; VISION, OCULAR;

EID: 84914162408     PISSN: 15306984     EISSN: 15306992     Source Type: Journal    
DOI: 10.1021/nl502275s     Document Type: Article

References (36)

1. Mulligan, R. C. Science 1993, 260, 926-932
2. Herz, J.; Gerard, R. D. Proc. Natl. Acad. Sci. U.S.A 1993, 90, 2812-2816
3. Simon, R. H.; Engelhardt, J. F.; Yang, Y.; Zepeda, M.; Weber-Pendleton, S.; Grossman, M.; Wilson, J. M. Hum. Gene Ther. 1993, 4, 771-780
4. Ali, M.; Lemoine, N. R.; Ring, C. J. Gene Ther. 1994, 1, 367-384
5. Wu, Z.; Yang, H.; Colosi, P. Mol. Ther. 2010, 18, 80-86
6. Bragonzi, A.; Dina, G.; Villa, A.; Calori, G.; Biffi, A.; Bordignon, C.; Assael, B. M.; Conese, M. Gene Ther. 2000, 7, 1753-1760
7. Kollen, W. J.; Mulberg, A. E.; Wei, X.; Sugita, M.; Raghuram, V.; Wang, J.; Foskett, J. K.; Glick, M. C.; Scanlin, T. F. Hum. Gene Ther. 1999, 10, 615-622
8. El-Aneed, A. J. Controlled Release 2004, 94, 1-14
9. Li, S.; Huang, L. Gene Ther. 1997, 4, 891-900
10. Li, S.; Rizzo, M. A.; Bhattacharya, S.; Huang, L. Gene Ther. 1998, 5, 930-937
11. Koirala, A.; Makkia, R. S.; Conley, S. M.; Cooper, M. J.; Naash, M. I. Hum. Mol. Genet. 2013, 22, 1632-1642
12. Han, Z.; Conley, S. M.; Makkia, R. S.; Cooper, M. J.; Naash, M. I. J. Clin. Invest 2012, 122, 3221-3226
13. Rowe-Rendleman, C. L.; Durazo, S. A.; Kompella, U. B.; Rittenhouse, K. D.; Di, P. A.; Weiner, A. L.; Grossniklaus, H. E.; Naash, M. I.; Lewin, A. S.; Horsager, A.; Edelhauser, H. F. Invest. Ophthalmol. Visual Sci. 2014, 55, 2714-2730
14. Koirala, A.; Conley, S. M.; Makkia, R.; Liu, Z.; Cooper, M. J.; Sparrow, J. R.; Naash, M. I. J. Controlled Release 2013, 172, 745-752
15. Cao, B.; Qiu, P.; Mao, C. Microsc. Res. Technol. 2013, 76, 936-941
16. Ma, K.; Wang, D. D.; Lin, Y.; Wang, J.; Petrenko, V.; Mao, C. Adv. Funct. Mater. 2013, 23, 1172-1181
17. Gandra, N.; Wang, D. D.; Zhu, Y.; Mao, C. Angew. Chem., Int. Ed. 2013, 52, 11278-11281
18. del Pozo-Rodriguez, A.; Delgado, D.; Gascon, A. R.; Solinis, M. A. J. Ocul. Pharmacol. Ther. 2013, 29, 173-188
19. Futaki, S.; Suzuki, T.; Ohashi, W.; Yagami, T.; Tanaka, S.; Ueda, K.; Sugiura, Y. J. Biol. Chem. 2001, 276, 5836-5840
20. Koirala, A.; Makkia, R. S.; Cooper, M. J.; Naash, M. I. Biomaterials 2011, 32, 9483-9493
21. Rajala, A.; Dighe, R.; Agbaga, M. P.; Anderson, R. E.; Rajala, R. V. J. Biol. Chem. 2013, 288, 19503-19515
22. Bemelmans, A. P.; Kostic, C.; Hornfeld, D.; Jaquet, M.; Crippa, S. V.; Hauswirth, W. W.; Lem, J.; Wang, Z.; Schorderet, D. E.; Munier, F. L.; Wenzel, A.; Arsenijevic, Y. Adv. Exp. Med. Biol. 2006, 572, 247-253
23. Lai, C. M.; Yu, M. J.; Brankov, M.; Barnett, N. L.; Zhou, X.; Redmond, T. M.; Narfstrom, K.; Rakoczy, P. E. Genet. Vaccines. Ther. 2004, 2, 3
24. Chen, Y.; Moiseyev, G.; Takahashi, Y.; Ma, J. X. Invest. Ophthalmol. Vis. Sci. 2006, 47, 1177-1184
25. Moiseyev, G.; Takahashi, Y.; Chen, Y.; Kim, S.; Ma, J. X. J. Biol. Chem. 2008, 283, 8110-8117
26. Redmond, T. M.; Yu, S.; Lee, E.; Bok, D.; Hamasaki, D.; Chen, N.; Goletz, P.; Ma, J. X.; Crouch, R. K.; Pfeifer, K. Nat. Genet. 1998, 20, 344-351
27. Ekesten, B.; Gouras, P.; Salchow, D. J. Vision Res. 2001, 41, 2425-2433
28. Bemelmans, A. P.; Kostic, C.; Crippa, S. V.; Hauswirth, W. W.; Lem, J.; Munier, F. L.; Seeliger, M. W.; Wenzel, A.; Arsenijevic, Y. PLoS. Med. 2006, 3, e347
29. Trevino, S. G.; Villazana-Espinoza, E. T.; Muniz, A.; Tsin, A. T. J. Exp. Biol. 2005, 208, 4151-4157
30. Perry, R. J.; McNaughton, P. A. J. Physiol 1991, 433, 561-587
31. Moiseyev, G.; Chen, Y.; Takahashi, Y.; Wu, B. X.; Ma, J. X. Proc. Natl. Acad. Sci. U.S.A 2005, 102, 12413-12418
32. Rando, R. R. Biochemistry 1991, 30, 595-602
33. Znoiko, S. L.; Rohrer, B.; Lu, K.; Lohr, H. R.; Crouch, R. K.; Ma, J. X. Invest Ophthalmol. Visual Sci. 2005, 46, 1473-1479
34. Blanks, J. C.; Johnson, L. V. J. Comp. Neurol. 1983, 221, 31-41
35. Alexandropoulos, K.; Baltimore, D. Genes Dev. 1996, 10, 1341-1355
36. Tang, P. H.; Buhusi, M. C.; Ma, J. X.; Crouch, R. K. J. Neurosci. 2011, 31, 18618-18626