An experimental platform for systemic drug delivery to the retina

Proceedings of the National Academy of Sciences of the United States of America

Volumn 106, Issue 42, 2009, Pages 17817-17822

  Campbell, Matthew ^a   Nguyen, Anh T H ^a   Kiang, Anna Sophia ^a   Tam, Lawrence C S ^a   Gobbo, Oliviero L ^a   Kerskens, Christian ^a   Dhubhghaill, Sorcha Ni ^a   Humphries, Marian M ^a   Farrar, G Jane ^a   Kenna, Paul F ^a   Humphries, Peter ^a  

^a TRINITY COLLEGE DUBLIN   (Ireland)

Author keywords

Blood retina barrier; Claudin 5; Retinitis pigmentosa; RNAi; Tight junctions

Indexed keywords

CLAUDIN 5; CYSTEINE PROTEINASE INHIBITOR; N ACETYLLEUCYLLEUCYLMETHIONINE CARBOHYDRATE; SMALL INTERFERING RNA; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BLOOD RETINA BARRIER; BRAIN EDEMA; CELL DEATH; CONTROLLED STUDY; DRUG DELIVERY SYSTEM; HIGH THROUGHPUT SCREENING; LIGHT; MOLECULE; MOUSE; NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; PHOTORECEPTOR CELL; PRIORITY JOURNAL; RETINA; RETINA BLOOD VESSEL; RETINA DEGENERATION; RETINA EDEMA; RETINITIS PIGMENTOSA; RNA INTERFERENCE; TIGHT JUNCTION; TRANSCRIPTION INITIATION; VISION;

ANIMALS; BLOOD-RETINAL BARRIER; CALPAIN; CYSTEINE PROTEINASE INHIBITORS; DISEASE MODELS, ANIMAL; DRUG DELIVERY SYSTEMS; ELECTRORETINOGRAPHY; GUANOSINE TRIPHOSPHATE; HUMANS; IMP DEHYDROGENASE; MAGNETIC RESONANCE IMAGING; MEMBRANE PROTEINS; MICE; MICE, INBRED BALB C; MICE, INBRED C57BL; MICE, KNOCKOUT; OLIGOPEPTIDES; RETINA; RETINITIS PIGMENTOSA; RNA INTERFERENCE; RNA, SMALL INTERFERING;

MUS;

EID: 70449577160     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.0908561106     Document Type: Article

References (43)

1. Masland RH (2001) The fundamental plan of the retina. Nat Neurosci 4:877-886.
2. Gardiner TA, Archer DB, Curtis TM, Stitt AW (2007) Arteriolar involvement in the microvascular lesions of diabetic retinopathy: Implications for pathogenesis. Microcirculation 14:25-38. (Pubitemid 46321703)
3. Antonetti DA, Lieth E, Barber AJ, Gardner TW (1999) Molecular mechanisms of vascular permeability in diabetic retinopathy. Semin Ophthalmol 14:240-248. (Pubitemid 30061167)
4. Antonetti DA, et al. (1998) Vascular permeability in experimental diabetes is associated with reduced endothelial occludin content: Vascular endothelial growth factor decreases occludin in retinal endothelial cells. Diabetes 47:1953-1959.
5. Rizzolo LJ, Chen X, Weitzman M, Sun R, Zhang H (2007) Analysis of the RPE transcriptome reveals dynamic changes during the development of the outer blood-retinal barrier. Mol Vis 23:1259-1273. (Pubitemid 47128919)
6. Pardridge WM (2005) Molecular biology of the blood-brain barrier. Mol Biotechnol 30:57-70.
7. Campbell M, et al. (2006) Involvement of MAPKs in endostatin-mediated regulation of blood-retinal barrier function. Curr Eye Res 31:1033-1045. (Pubitemid 46005570)
8. Fanning AS, Anderson JM (1998) PDZ domains and the formation of protein networks at the plasma membrane. Curr Top Microbiol Immunol 228:209-233.
9. Kojima S, Rahner C, Peng S, Rizzolo LJ (2002) Claudin 5 is transiently expressed during the development of the retinal pigment epithelium. J Membr Biol 186:81-88. (Pubitemid 34252644)
10. Piontek J, et al. (2008) Formation of tight junction: Determinants of homophilic interaction between classic claudins. FASEB J 22:146-158.
11. Nitta T, et al. (2003) Size-selective loosening of the blood-brain barrier in claudin-5-deficient mice. J Cell Biol 161:653-660. (Pubitemid 36578587)
12. Campbell M, et al. (2008) RNAi-mediated reversible opening of the blood-brain barrier. J Gene Med 10:930-947.
13. Pardridge WM (2006) Molecular Trojan horses for blood-brain barrier drug delivery. Curr Opin Pharmacol 6:494-500. (Pubitemid 44348531)
14. Aherne A, et al. (2004) On the molecular pathology of neurodegeneration in IMPDH1-based retinitis pigmentosa. Hum Mol Genet 13:641-650. (Pubitemid 38379853)
15. Tam LC, et al. (2008) Therapeutic benefit derived from RNAi-mediated ablation of IMPDH1 transcripts in a murine model of autosomal dominant retinitis pigmentosa (RP10). Hum Mol Genet 17:2084-2100.
16. Joly S, Samardzija M, Wenzel A, Thiersch M, Grimm C (2009) Nonessential role of beta3 and beta5 integrin subunits for efficient clearance of cellular debris after light-induced photoreceptor degeneration. Invest Ophthalmol Vis Sci 50:1423-1432.
17. Sanges D, Comitato A, Tammaro R, Marigo V (2006) Apoptosis in retinal degeneration involves cross-talk between apoptosis-inducing factor (AIF) and caspase-12 and is blocked by calpain inhibitors. Proc Natl Acad Sci USA 103:17366-17371. (Pubitemid 44788975)
18. Perche O, Doly M, Ranchon-Cole I (2007) Caspase-dependent apoptosis in light-induced retinal degeneration. Invest Ophthalmol Vis Sci 48:2753-2759. (Pubitemid 351261230)
19. Paquet-Durand F, Johnson L, Ekström P (2007) Calpain activity in retinal degeneration. J Neurosci Res 85:693-702.
20. Sasaki T, et al. (1990) Inhibitory effect of di-and tripeptidyl aldehydes on calpains and cathepsins. J Enzyme Inhib 3:195-201. (Pubitemid 20080690)
21. Nussenblatt RB, Ferris F, III (2007) Age-related macular degeneration and the immune response: Implications for therapy. Am J Ophthalmol 144:618-626.
22. Bhatwadekar AD, et al. (2008) Advanced glycation of fibronectin impairs vascular repair by endothelial progenitor cells: Implications for vasodegeneration in diabetic retinopathy. Invest Ophthalmol Vis Sci 49:1232-1241.
23. Chadderton N, et al. (2009) Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy. Mol Ther 17:593-599.
24. O'Reilly M, et al. (2007) RNA interference-mediated suppression and replacement of human rhodopsin in vivo. Am J Hum Genet 81:127-135. (Pubitemid 47001162)
25. Montezuma SR, Sobrin L, Seddon JM (2007) Review of genetics in age related macular degeneration. Semin Ophthalmol 22:229-240.
26. Fong DS, Girach A, Boney A (2007) Visual side effects of successful scatter laser photocoagulation surgery for proliferative diabetic retinopathy: A literature review. Retina 27:816-824. (Pubitemid 47479374)
27. Humphries P, Kenna P, Farrar GJ (1992) On the molecular genetics of retinitis pigmentosa. Science 256:804-808.
28. De Boüard S, et al. (2007) Antiangiogenic and anti-invasive effects of sunitinib on experimental human glioblastoma. Neuro Oncol 9:412-423.
29. Sapieha P, et al. (2008) The succinate receptor GPR91 in neurons has a major role in retinal angiogenesis. Nat Med 14:1067-1076.
30. Edwards AO, Malek G (2007) Molecular genetics of AMD and current animal models. Angiogenesis 10:119-132. (Pubitemid 46440627)
31. Brankin B, Campbell M, Canning P, Gardiner TA, Stitt AW (2005) Endostatin modulates VEGF-mediated barrier dysfunction in the retinal microvascular endothelium. Exp Eye Res 81:22-31. (Pubitemid 40840542)
32. Matter K, Balda MS (2003) Holey barrier: Claudins and the regulation of brain endothelial permeability. J Cell Biol 161:459-460. (Pubitemid 36578567)
33. Koto T, et al. (2007) Hypoxia disrupts the barrier function of neural blood vessels through changes in the expression of claudin-5 in endothelial cells. Am J Pathol 170:1389-1397. (Pubitemid 47339345)
34. Haorah J, et al. (2005) Ethanol-induced activation of myosin light chain kinase leads to dysfunction of tight junctions and blood-brain barrier compromise. Alcohol Clin Exp Res 29:999-1009. (Pubitemid 40934204)
35. Nag S, Venugopalan R, Stewart DJ (2007) Increased caveolin-1 expression precedes decreased expression of occludin and claudin-5 during blood-brain barrier breakdown Acta Neuropathol 114:459-469. (Pubitemid 47521502)
36. McColl BW, Rothwell NJ, Allan SM (2008) Systemic inflammation alters the kinetics of cerebrovascular tight junction disruption after experimental stroke in mice. J Neurosci 28:9451-9462.
37. Donovan M, Cotter TG (2002) Caspase-independent photoreceptor apoptosis in vivo and differential expression of apoptotic protease activating factor-1 and caspase-3 during retinal development. Cell Death Differ 9:1220-1231. (Pubitemid 35331769)
38. Maguire AM, et al. (2008) Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 358:2240-2248. (Pubitemid 351724453)
39. Bainbridge JW, et al. (2008) Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med 358:2231-2239. (Pubitemid 351724452)
40. Hauswirth WW, et al. (2008) 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.
41. Foust KD, et al. (2009) Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol 27:59-65.
42. Reynolds A, et al. (2004) Rational siRNA design for RNA interference. Nat Biotechnol 22:326-330. (Pubitemid 38295766)
43. Kiang AS, et al. (2005) Toward a gene therapy for dominant disease: Validation of an RNA interference-based mutation-independent approach. Mol Ther 12:555-561.