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Volumn 24, Issue 4, 2010, Pages 1178-1191

Gene delivery to mitotic and postmitotic photoreceptors via compacted DNA nanoparticles results in improved phenotype in a mouse model of retinitis pigmentosa

Author keywords

Gene replacement therapy; Mouse opsin promoter; Retinal degeneration slow protein

Indexed keywords

INTERLEUKIN 6; NANOPARTICLE; OPSIN; TUMOR NECROSIS FACTOR ALPHA; DNA; INTERMEDIATE FILAMENT PROTEIN; MEMBRANE PROTEIN; NERVE PROTEIN; PERIPHERIN;

EID: 77951632478     PISSN: 08926638     EISSN: 15306860     Source Type: Journal    
DOI: 10.1096/fj.09-139147     Document Type: Article
Times cited : (113)

References (48)
  • 1
    • 33750957652 scopus 로고    scopus 로고
    • Phenotypic variation in patients with mutation in the peripherin/RDS gene
    • Online
    • Weleber, R. (1999) Phenotypic variation in patients with mutation in the peripherin/RDS gene. [Online] Digit. J. Opthalmol. 5(2)
    • (1999) Digit. J. Opthalmol. , vol.5 , Issue.2
    • Weleber, R.1
  • 2
    • 0026078839 scopus 로고
    • Photoreceptor peripherin is the normal product of the gene responsible for retinal degeneration in the rds mouse
    • Connell, G., Bascom, R., Molday, L., Reid, D., McInnes, R. R., and Molday, R. S. (1991) Photoreceptor peripherin is the normal product of the gene responsible for retinal degeneration in the rds mouse. Proc. Natl. Acad. Sci. U. S. A. 88, 723-726
    • (1991) Proc. Natl. Acad. Sci. U. S. A. , vol.88 , pp. 723-726
    • Connell, G.1    Bascom, R.2    Molday, L.3    Reid, D.4    McInnes, R.R.5    Molday, R.S.6
  • 3
    • 0026053969 scopus 로고
    • The retinal degeneration slow (rds) gene product is a photoreceptor disc membrane-associated glycoprotein
    • Travis, G. H., Sutcliffe, J. G., and Bok, D. (1991) The retinal degeneration slow (rds) gene product is a photoreceptor disc membrane-associated glycoprotein. Neuron 6, 61-70
    • (1991) Neuron , vol.6 , pp. 61-70
    • Travis, G.H.1    Sutcliffe, J.G.2    Bok, D.3
  • 4
    • 0029842023 scopus 로고    scopus 로고
    • Mutations and polymorphisms in the human peripherin-RDS gene and their involvement in inherited retinal degeneration
    • Keen, T. J., and Inglehearn, C. F. (1996) Mutations and polymorphisms in the human peripherin-RDS gene and their involvement in inherited retinal degeneration. Hum. Mutat. 8, 297-303
    • (1996) Hum. Mutat. , vol.8 , pp. 297-303
    • Keen, T.J.1    Inglehearn, C.F.2
  • 5
    • 0031772105 scopus 로고    scopus 로고
    • The role of the peripherin/RDS gene in retinal dystrophies
    • Kohl, S., Giddings, I., Besch, D., Apfelstedt-Sylla, E., Zrenner, E., and Wissinger, B. (1998) The role of the peripherin/RDS gene in retinal dystrophies. Acta Anat. 162, 75-84 (Pubitemid 28550590)
    • (1998) Acta Anatomica , vol.162 , Issue.2-3 , pp. 75-84
    • Wissinger, B.1
  • 6
    • 0030831115 scopus 로고    scopus 로고
    • The effect of peripherin/rds haploinsufficiency on rod and cone photoreceptors
    • Cheng, T., Peachey, N. S., Li, S., Goto, Y., Cao, Y., and Naash, M. I. (1997) The effect of peripherin/rds haploinsufficiency on rod and cone photoreceptors. J. Neurosci. 17, 8118-8128 (Pubitemid 27461036)
    • (1997) Journal of Neuroscience , vol.17 , Issue.21 , pp. 8118-8128
    • Cheng, T.1    Peachey, N.S.2    Li, S.3    Goto, Y.4    Cao, Y.5    Naash, M.I.6
  • 7
    • 38949131913 scopus 로고    scopus 로고
    • Genetic supplementation of RDS alleviates a loss-of-function phenotype in C214S model of retinitis pigmentosa
    • Nour, M., Fliesler, S. J., and Naash, M. I. (2008) Genetic supplementation of RDS alleviates a loss-of-function phenotype in C214S model of retinitis pigmentosa. Adv. Exp. Med. Biol. 613, 129-138
    • (2008) Adv. Exp. Med. Biol. , vol.613 , pp. 129-138
    • Nour, M.1    Fliesler, S.J.2    Naash, M.I.3
  • 8
    • 2942685882 scopus 로고    scopus 로고
    • Retinitis pigmentosa: Understanding the clinical presentation, mechanisms and treatment options
    • Kalloniatis, M., and Fletcher, E. L. (2004) Retinitis pigmentosa: understanding the clinical presentation, mechanisms and treatment options. Clin. Exp. Optom. 87, 65-80
    • (2004) Clin. Exp. Optom. , vol.87 , pp. 65-80
    • Kalloniatis, M.1    Fletcher, E.L.2
  • 10
    • 0027521914 scopus 로고
    • Apoptosis: Final common pathway of photoreceptor death in rd, rds, and rhodopsin mutant mice
    • Chang, G. Q., Hao, Y., and Wong, F. (1993) Apoptosis: final common pathway of photoreceptor death in rd, rds, and rhodopsin mutant mice. Neuron 11, 595-605
    • (1993) Neuron , vol.11 , pp. 595-605
    • Chang, G.Q.1    Hao, Y.2    Wong, F.3
  • 11
    • 0030972482 scopus 로고    scopus 로고
    • Correlation of DNA fragmentation and chromatin condensation in apoptotic nuclei of the Ser 6 mouse retina
    • Chang, G. Q., Gaitan, A., Hao, Y., and Wong, F. (1997) Correlation of DNA fragmentation and chromatin condensation in apoptotic nuclei of the Ser 6 mouse retina. Microsc. Res. Tech. 36, 123-129
    • (1997) Microsc. Res. Tech. , vol.36 , pp. 123-129
    • Chang, G.Q.1    Gaitan, A.2    Hao, Y.3    Wong, F.4
  • 12
    • 3242880192 scopus 로고    scopus 로고
    • Modulating expression of peripherin/rds in transgenic mice: Critical levels and the effect of overexpression
    • Nour, M., Ding, X. Q., Stricker, H., Fliesler, S. J., and Naash, M. I. (2004) Modulating expression of peripherin/rds in transgenic mice: critical levels and the effect of overexpression. Invest. Ophthalmol. Vis. Sci. 45, 2514-2521
    • (2004) Invest. Ophthalmol. Vis. Sci. , vol.45 , pp. 2514-2521
    • Nour, M.1    Ding, X.Q.2    Stricker, H.3    Fliesler, S.J.4    Naash, M.I.5
  • 15
    • 0022295906 scopus 로고
    • Development and degeneration of retina in rds mutant mice: Photoreceptor abnormalities in the heterozygotes
    • Hawkins, R. K., Jansen, H. G., and Sanyal, S. (1985) Development and degeneration of retina in rds mutant mice: photoreceptor abnormalities in the heterozygotes. Exp. Eye Res. 41, 701-720
    • (1985) Exp. Eye Res. , vol.41 , pp. 701-720
    • Hawkins, R.K.1    Jansen, H.G.2    Sanyal, S.3
  • 16
    • 0029094912 scopus 로고
    • Retinal degeneration slow (rds) in mouse results from simple insertion of a t haplotype-specific element into protein-coding exon II
    • Ma, J., Norton, J. C., Allen, A. C., Burns, J. B., Hasel, K. W., Burns, J. L., Sutcliffe, J. G., and Travis, G. H. (1995) Retinal degeneration slow (rds) in mouse results from simple insertion of a t haplotype-specific element into protein-coding exon II. Genomics 28, 212-219
    • (1995) Genomics , vol.28 , pp. 212-219
    • Ma, J.1    Norton, J.C.2    Allen, A.C.3    Burns, J.B.4    Hasel, K.W.5    Burns, J.L.6    Sutcliffe, J.G.7    Travis, G.H.8
  • 17
    • 38749097725 scopus 로고    scopus 로고
    • Nanoparticle applications in ocular gene therapy
    • Cai, X., Conley, S., and Naash, M. (2008) Nanoparticle applications in ocular gene therapy. Vis. Res. 48, 319-324
    • (2008) Vis. Res. , vol.48 , pp. 319-324
    • Cai, X.1    Conley, S.2    Naash, M.3
  • 19
    • 65449178420 scopus 로고    scopus 로고
    • A partial structural and functional rescue of a retinitis pigmentosa model with compacted DNA nanoparticles
    • Cai, X., Nash, Z., Conley, S. M., Fliesler, S. J., Cooper, M. J., and Naash, M. I. (2009) A partial structural and functional rescue of a retinitis pigmentosa model with compacted DNA nanoparticles. PLoS ONE 4, e5290
    • (2009) PLoS ONE , vol.4
    • Cai, X.1    Nash, Z.2    Conley, S.M.3    Fliesler, S.J.4    Cooper, M.J.5    Naash, M.I.6
  • 20
    • 34248570405 scopus 로고    scopus 로고
    • Efficient non-viral ocular gene transfer with compacted DNA nanoparticles
    • Farjo, R., Skaggs, J., Quiambao, A. B., Cooper, M. J., and Naash, M. I. (2006) Efficient non-viral ocular gene transfer with compacted DNA nanoparticles. PLoS ONE 1, e38
    • (2006) PLoS ONE , vol.1
    • Farjo, R.1    Skaggs, J.2    Quiambao, A.B.3    Cooper, M.J.4    Naash, M.I.5
  • 21
    • 34247876190 scopus 로고    scopus 로고
    • Vectors for airway gene delivery
    • Davis, P. B., and Cooper, M. J. (2007) Vectors for airway gene delivery. AAPS J. 9, E11-17
    • (2007) AAPS J. , vol.9
    • Davis, P.B.1    Cooper, M.J.2
  • 24
    • 33745224788 scopus 로고    scopus 로고
    • Plasmid size up to 20 kbp does not limit effective in vivo lung gene transfer using compacted DNA nanoparticles
    • Fink, T. L., Klepcyk, P. J., Oette, S. M., Gedeon, C. R., Hyatt, S. L., Kowalczyk, T. H., Moen, R. C., and Cooper, M. J. (2006) Plasmid size up to 20 kbp does not limit effective in vivo lung gene transfer using compacted DNA nanoparticles. Gene Ther. 13, 1048-1051
    • (2006) Gene Ther. , vol.13 , pp. 1048-1051
    • Fink, T.L.1    Klepcyk, P.J.2    Oette, S.M.3    Gedeon, C.R.4    Hyatt, S.L.5    Kowalczyk, T.H.6    Moen, R.C.7    Cooper, M.J.8
  • 25
    • 70350733741 scopus 로고    scopus 로고
    • Ocular delivery of compacted DNA nanoparticles does not elicit toxicity in the mouse retina
    • Ding, X. Q., Quiambao, A. B., Fitzgerald, J. B., Cooper, M. J., Conley, S. M., and Naash, M. I. (2009) Ocular delivery of compacted DNA nanoparticles does not elicit toxicity in the mouse retina. PLoS ONE 4, e7410
    • (2009) PLoS ONE , vol.4
    • Ding, X.Q.1    Quiambao, A.B.2    Fitzgerald, J.B.3    Cooper, M.J.4    Conley, S.M.5    Naash, M.I.6
  • 26
    • 33645720940 scopus 로고    scopus 로고
    • Does recombinant adeno-associated virus-vectored proximal region of mouse rhodopsin promoter support only rod-type specific expression in vivo?
    • Glushakova, L. G., Timmers, A. M., Issa, T. M., Cortez, N. G., Pang, J., Teusner, J. T., and Hauswirth, W. W. (2006) Does recombinant adeno-associated virus-vectored proximal region of mouse rhodopsin promoter support only rod-type specific expression in vivo? Mol. Vis. 12, 298-309
    • (2006) Mol. Vis. , vol.12 , pp. 298-309
    • Glushakova, L.G.1    Timmers, A.M.2    Issa, T.M.3    Cortez, N.G.4    Pang, J.5    Teusner, J.T.6    Hauswirth, W.W.7
  • 28
    • 0035504663 scopus 로고    scopus 로고
    • Gene replacement therapy in the retinal degeneration slow (rds) mouse: The effect on retinal degeneration following partial transduction of the retina
    • Sarra, G. M., Stephens, C., de Alwis, M., Bainbridge, J. W., Smith, A. J., Thrasher, A. J., and Ali, R. R. (2001) Gene replacement therapy in the retinal degeneration slow (rds) mouse: the effect on retinal degeneration following partial transduction of the retina. Hum. Mol. Genet. 10, 2353-2361 (Pubitemid 33069541)
    • (2001) Human Molecular Genetics , vol.10 , Issue.21 , pp. 2353-2361
    • Sarra, G.-M.1    Stephens, C.2    De Alwis, M.3    Bainbridge, J.W.B.4    Smith, A.J.5    Thrasher, A.J.6    Ali, R.R.7
  • 30
    • 0031193714 scopus 로고    scopus 로고
    • A 221-bp fragment of the mouse opsin promoter directs expression specifically to the rod photoreceptors of transgenic mice
    • Quiambao, A. B., Peachey, N. S., Mangini, N. J., Rohlich, P., Hollyfield, J. G., and al-Ubaidi, M. R. (1997) A 221-bp fragment of the mouse opsin promoter directs expression specifically to the rod photoreceptors of transgenic mice. Vis. Neurosci. 14, 617-625
    • (1997) Vis. Neurosci. , vol.14 , pp. 617-625
    • Quiambao, A.B.1    Peachey, N.S.2    Mangini, N.J.3    Rohlich, P.4    Hollyfield, J.G.5    Al-Ubaidi, M.R.6
  • 31
    • 0025371311 scopus 로고
    • Molecular cloning, primary structure, and orientation of the vertebrate photoreceptor cell protein peripherin in the rod outer segment disk membrane
    • Connell, G. J., and Molday, R. S. (1990) Molecular cloning, primary structure, and orientation of the vertebrate photoreceptor cell protein peripherin in the rod outer segment disk membrane. Biochemistry 29, 4691-4698
    • (1990) Biochemistry , vol.29 , pp. 4691-4698
    • Connell, G.J.1    Molday, R.S.2
  • 32
    • 0141430039 scopus 로고    scopus 로고
    • P2Y(2) receptor agonist INS37217 enhances functional recovery after detachment caused by subretinal injection in normal and rds mice
    • Nour, M., Quiambao, A. B., Peterson, W. M., Al-Ubaidi, M. R., and Naash, M. I. (2003) P2Y(2) receptor agonist INS37217 enhances functional recovery after detachment caused by subretinal injection in normal and rds mice. Invest. Ophthalmol. Vis. Sci. 44, 4505-4514
    • (2003) Invest. Ophthalmol. Vis. Sci. , vol.44 , pp. 4505-4514
    • Nour, M.1    Quiambao, A.B.2    Peterson, W.M.3    Al-Ubaidi, M.R.4    Naash, M.I.5
  • 33
    • 33645748724 scopus 로고    scopus 로고
    • Retention of function without normal disc morphogenesis occurs in cone but not rod photoreceptors
    • Farjo, R., Skaggs, J. S., Nagel, B. A., Quiambao, A. B., Nash, Z. A., Fliesler, S. J., and Naash, M. I. (2006) Retention of function without normal disc morphogenesis occurs in cone but not rod photoreceptors. J. Cell. Biol. 173, 59-68
    • (2006) J. Cell. Biol. , vol.173 , pp. 59-68
    • Farjo, R.1    Skaggs, J.S.2    Nagel, B.A.3    Quiambao, A.B.4    Nash, Z.A.5    Fliesler, S.J.6    Naash, M.I.7
  • 34
    • 5744251093 scopus 로고    scopus 로고
    • The R172W mutation in peripherin/rds causes a cone-rod dystrophy in transgenic mice
    • Ding, X. Q., Nour, M., Ritter, L. M., Goldberg, A. F., Fliesler, S. J., and Naash, M. I. (2004) The R172W mutation in peripherin/rds causes a cone-rod dystrophy in transgenic mice. Hum. Mol. Genet. 13, 2075-2087
    • (2004) Hum. Mol. Genet. , vol.13 , pp. 2075-2087
    • Ding, X.Q.1    Nour, M.2    Ritter, L.M.3    Goldberg, A.F.4    Fliesler, S.J.5    Naash, M.I.6
  • 36
    • 38549148792 scopus 로고    scopus 로고
    • Late-onset cone photoreceptor degeneration induced by R172W mutation in Rds and partial rescue by gene supplementation
    • Conley, S., Nour, M., Fliesler, S. J., and Naash, M. I. (2007) Late-onset cone photoreceptor degeneration induced by R172W mutation in Rds and partial rescue by gene supplementation. Invest. Ophthalmol. Vis. Sci. 48, 5397-5407
    • (2007) Invest. Ophthalmol. Vis. Sci. , vol.48 , pp. 5397-5407
    • Conley, S.1    Nour, M.2    Fliesler, S.J.3    Naash, M.I.4
  • 37
    • 65449178420 scopus 로고    scopus 로고
    • A partial structural and functional rescue of a retinitis pigmentosa model with compacted DNA nanoparticles
    • Cai, X., Nash, Z. A., Conley, S. M., Fliesler, S. J., Cooper, M. J., and Naash, M. (2009) A partial structural and functional rescue of a retinitis pigmentosa model with compacted DNA nanoparticles. PLoS ONE 4, e5290
    • (2009) PLoS ONE , vol.4
    • Cai, X.1    Nash, Z.A.2    Conley, S.M.3    Fliesler, S.J.4    Cooper, M.J.5    Naash, M.6
  • 41
    • 33646573375 scopus 로고    scopus 로고
    • Microglia and macrophages are increased in response to ischemia-induced retinopathy in the mouse retina
    • Davies, M. H., Eubanks, J. P., and Powers, M. R. (2006) Microglia and macrophages are increased in response to ischemia-induced retinopathy in the mouse retina. Mol. Vis. 12, 467-477
    • (2006) Mol. Vis. , vol.12 , pp. 467-477
    • Davies, M.H.1    Eubanks, J.P.2    Powers, M.R.3
  • 43
    • 33744771291 scopus 로고    scopus 로고
    • Characterization of peripherin/rds and rom-1 transport in rod photoreceptors of transgenic and knockout animals
    • Lee, E. S., Burnside, B., and Flannery, J. G. (2006) Characterization of peripherin/rds and rom-1 transport in rod photoreceptors of transgenic and knockout animals. Invest. Ophthalmol. Vis. Sci. 47, 2150-2160
    • (2006) Invest. Ophthalmol. Vis. Sci. , vol.47 , pp. 2150-2160
    • Lee, E.S.1    Burnside, B.2    Flannery, J.G.3
  • 44
    • 33645831156 scopus 로고    scopus 로고
    • Mouse opsin promoter-directed Cre recombinase expression in transgenic mice
    • Le, Y. Z., Zheng, L., Zheng, W., Ash, J. D., Agbaga, M. P., Zhu, M., and Anderson, R. E. (2006) Mouse opsin promoter-directed Cre recombinase expression in transgenic mice. Mol. Vis. 12, 389-398
    • (2006) Mol. Vis. , vol.12 , pp. 389-398
    • Le, Y.Z.1    Zheng, L.2    Zheng, W.3    Ash, J.D.4    Agbaga, M.P.5    Zhu, M.6    Anderson, R.E.7
  • 45
    • 37049007445 scopus 로고    scopus 로고
    • Effect of Rds abundance on cone outer segment morphogenesis, photoreceptor gene expression, and outer limiting membrane integrity
    • Farjo, R., Fliesler, S. J., and Naash, M. I. (2007) Effect of Rds abundance on cone outer segment morphogenesis, photoreceptor gene expression, and outer limiting membrane integrity. J. Comp. Neurol. 504, 619-630
    • (2007) J. Comp. Neurol. , vol.504 , pp. 619-630
    • Farjo, R.1    Fliesler, S.J.2    Naash, M.I.3
  • 46
    • 0035182556 scopus 로고    scopus 로고
    • In vivo gene transfer to the mouse eye using an HIV-based lentiviral vector; efficient long-term transduction of corneal endothelium and retinal pigment epithelium
    • Bainbridge, J. W., Stephens, C., Parsley, K., Demaison, C., Halfyard, A., Thrasher, A. J., and Ali, R. R. (2001) In vivo gene transfer to the mouse eye using an HIV-based lentiviral vector; efficient long-term transduction of corneal endothelium and retinal pigment epithelium. Gene Ther. 8, 1665-1668
    • (2001) Gene Ther. , vol.8 , pp. 1665-1668
    • Bainbridge, J.W.1    Stephens, C.2    Parsley, K.3    Demaison, C.4    Halfyard, A.5    Thrasher, A.J.6    Ali, R.R.7
  • 47
    • 33745905638 scopus 로고    scopus 로고
    • Efficiency of lentiviral transduction during development in normal and rd mice
    • Pang, J., Cheng, M., Haire, S. E., Barker, E., Planelles, V., and Blanks, J. C. (2006) Efficiency of lentiviral transduction during development in normal and rd mice. Mol. Vis. 12, 756-767
    • (2006) Mol. Vis. , vol.12 , pp. 756-767
    • Pang, J.1    Cheng, M.2    Haire, S.E.3    Barker, E.4    Planelles, V.5    Blanks, J.C.6
  • 48
    • 33845510734 scopus 로고    scopus 로고
    • The role of Rds in outer segment morphogenesis and human retinal disease
    • Farjo, R., and Naash, M. I. (2006) The role of Rds in outer segment morphogenesis and human retinal disease. Ophthalmic Genet. 27, 117-122
    • (2006) Ophthalmic Genet. , vol.27 , pp. 117-122
    • Farjo, R.1    Naash, M.I.2


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