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Volumn 507, Issue , 2012, Pages 255-274

Gene delivery to the Retina: From mouse to man

Author keywords

Animal model; Gene therapy; Gene transfer; Retina; Subretinal injection; Vector

Indexed keywords

NUCLEIC ACID;

EID: 84857539197     PISSN: 00766879     EISSN: 15577988     Source Type: Book Series    
DOI: 10.1016/B978-0-12-386509-0.00013-2     Document Type: Chapter
Times cited : (15)

References (64)
  • 6
    • 80053295514 scopus 로고    scopus 로고
    • AAV-mediated gene replacement either alone or in combination with physical and pharmacological agents results in partial and transient protection from photoreceptor degeneration associated with {beta}PDE deficiency
    • M. Allocca, A. Manfredi, and C. Iodice AAV-mediated gene replacement either alone or in combination with physical and pharmacological agents results in partial and transient protection from photoreceptor degeneration associated with {beta}PDE deficiency Invest. Ophthalmol. Vis. Sci. 52 8 2011 5713 5719
    • (2011) Invest. Ophthalmol. Vis. Sci. , vol.52 , Issue.8 , pp. 5713-5719
    • Allocca, M.1    Manfredi, A.2    Iodice, C.3
  • 7
    • 77952983302 scopus 로고    scopus 로고
    • Safety and efficacy of subretinal re-administration of an AAV2 vector in large animal models: Implications for studies in humans
    • D. Amado, F. Mingozzi, and D. Hui Safety and efficacy of subretinal re-administration of an AAV2 vector in large animal models: Implications for studies in humans Sci. Transl. Med. 2 2010 21ra16
    • (2010) Sci. Transl. Med. , vol.2
    • Amado, D.1    Mingozzi, F.2    Hui, D.3
  • 8
    • 34248200353 scopus 로고    scopus 로고
    • Single-stranded oligonucleotide-mediated in vivo gene repair in the rd1 retina
    • C. Andrieu-Soler, M. Halhal, and J. Boatright Single-stranded oligonucleotide-mediated in vivo gene repair in the rd1 retina Mol. Vis. 13 2007 682 706
    • (2007) Mol. Vis. , vol.13 , pp. 682-706
    • Andrieu-Soler, C.1    Halhal, M.2    Boatright, J.3
  • 9
    • 28444452653 scopus 로고    scopus 로고
    • Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosis
    • M.L. Batten, Y. Imanishi, and D.C. Tu Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosis PLoS Med. 2 2005 e333
    • (2005) PLoS Med. , vol.2 , pp. 333
    • Batten, M.L.1    Imanishi, Y.2    Tu, D.C.3
  • 14
    • 77956208538 scopus 로고    scopus 로고
    • Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse
    • S.E. Boye, S.L. Boye, and J. Pang Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse PLoS One 5 2010 e11306
    • (2010) PLoS One , vol.5 , pp. 11306
    • Boye, S.E.1    Boye, S.L.2    Pang, J.3
  • 15
    • 65449178420 scopus 로고    scopus 로고
    • A partial structural and functional rescue of a retinitis pigmentosa model with compacted DNA nanoparticles
    • X. Cai, Z. Nash, and S.M. Conley A partial structural and functional rescue of a retinitis pigmentosa model with compacted DNA nanoparticles PLoS One 4 2009 e5290
    • (2009) PLoS One , vol.4 , pp. 5290
    • Cai, X.1    Nash, Z.2    Conley, S.M.3
  • 16
    • 79959619541 scopus 로고    scopus 로고
    • Gene therapy in the retinal degeneration slow model of retinitis pigmentosa
    • X. Cai, S.M. Conley, and M.I. Naash Gene therapy in the retinal degeneration slow model of retinitis pigmentosa Adv. Exp. Med. Biol. 664 2010 611 619
    • (2010) Adv. Exp. Med. Biol. , vol.664 , pp. 611-619
    • Cai, X.1    Conley, S.M.2    Naash, M.I.3
  • 17
    • 79960832400 scopus 로고    scopus 로고
    • Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy
    • L.S. Carvalho, J. Xu, and R.A. Pearson Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy Hum. Mol. Genet. 20 16 2011 3161 3175
    • (2011) Hum. Mol. Genet. , vol.20 , Issue.16 , pp. 3161-3175
    • Carvalho, L.S.1    Xu, J.2    Pearson, R.A.3
  • 18
    • 63949087144 scopus 로고    scopus 로고
    • Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy
    • N. Chadderton, S. Millington-Ward, and A. Palfi Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy Mol. Ther. 17 2009 593 599
    • (2009) Mol. Ther. , vol.17 , pp. 593-599
    • Chadderton, N.1    Millington-Ward, S.2    Palfi, A.3
  • 19
    • 77954620685 scopus 로고    scopus 로고
    • Nanoparticles for retinal gene therapy
    • S.M. Conley, and M.I. Naash Nanoparticles for retinal gene therapy Prog. Retin. Eye Res. 29 2010 376 397
    • (2010) Prog. Retin. Eye Res. , vol.29 , pp. 376-397
    • Conley, S.M.1    Naash, M.I.2
  • 21
    • 68249112028 scopus 로고    scopus 로고
    • AAV-mediated tyrosinase gene transfer restores melanogenesis and retinal function in a model of oculo-cutaneous albinism type i (OCA1)
    • A. Gargiulo, C. Bonetti, and S. Montefusco AAV-mediated tyrosinase gene transfer restores melanogenesis and retinal function in a model of oculo-cutaneous albinism type I (OCA1) Mol. Ther. 17 2009 1347 1354
    • (2009) Mol. Ther. , vol.17 , pp. 1347-1354
    • Gargiulo, A.1    Bonetti, C.2    Montefusco, S.3
  • 22
    • 77950863181 scopus 로고    scopus 로고
    • AAV-mediated knockdown of peripherin-2 in vivo using miRNA-based hairpins
    • A. Georgiadis, M. Tschernutter, and J.W. Bainbridge AAV-mediated knockdown of peripherin-2 in vivo using miRNA-based hairpins Gene Ther. 17 2010 486 493
    • (2010) Gene Ther. , vol.17 , pp. 486-493
    • Georgiadis, A.1    Tschernutter, M.2    Bainbridge, J.W.3
  • 24
  • 25
    • 34648823212 scopus 로고    scopus 로고
    • Retinoschisin gene therapy and natural history in the Rs1h-KO mouse: Long-term rescue from retinal degeneration
    • DOI 10.1167/iovs.07-0203
    • S. Kjellstrom, R.A. Bush, and Y. Zeng Retinoschisin gene therapy and natural history in the Rs1h-KO mouse: Long-term rescue from retinal degeneration Invest. Ophthalmol. Vis. Sci. 48 2007 3837 3845 (Pubitemid 351261125)
    • (2007) Investigative Ophthalmology and Visual Science , vol.48 , Issue.8 , pp. 3837-3845
    • Kjellstrom, S.1    Bush, R.A.2    Zeng, Y.3    Takada, Y.4    Sieving, P.A.5
  • 26
    • 52049107645 scopus 로고    scopus 로고
    • Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy
    • J. Kong, S.R. Kim, and K. Binley Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy Gene Ther. 15 2008 1311 1320
    • (2008) Gene Ther. , vol.15 , pp. 1311-1320
    • Kong, J.1    Kim, S.R.2    Binley, K.3
  • 27
    • 0030016358 scopus 로고    scopus 로고
    • Encapsidated adenovirus minichromosomes allow delivery and expression of a 14 kb dystrophin cDNA to muscle cells
    • DOI 10.1093/hmg/5.7.913
    • R. Kumar-Singh, and J.S. Chamberlain Encapsidated adenovirus minichromosomes allow delivery and expression of a 14 kb dystrophin cDNA to muscle cells Hum. Mol. Genet. 5 1996 913 921 (Pubitemid 26232270)
    • (1996) Human Molecular Genetics , vol.5 , Issue.7 , pp. 913-921
    • Kumar-Singh, R.1    Chamberlain, J.S.2
  • 28
    • 0034633619 scopus 로고    scopus 로고
    • Ribozyme rescue of photoreceptor cells in P23H transgenic rats: Long-term survival and late stage therapy
    • M.M. LaVail, D. Yasumura, and M.T. Matthes Ribozyme rescue of photoreceptor cells in P23H transgenic rats: Long-term survival and late stage therapy Proc. Natl. Acad. Sci. USA 97 2000 11488 11493
    • (2000) Proc. Natl. Acad. Sci. USA , vol.97 , pp. 11488-11493
    • Lavail, M.M.1    Yasumura, D.2    Matthes, M.T.3
  • 34
    • 70350620424 scopus 로고    scopus 로고
    • Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: A phase 1 dose-escalation trial
    • A.M. Maguire, K.A. High, and A. Auricchio Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: A phase 1 dose-escalation trial Lancet 374 2009 1597 1605
    • (2009) Lancet , vol.374 , pp. 1597-1605
    • Maguire, A.M.1    High, K.A.2    Auricchio, A.3
  • 35
    • 70349971731 scopus 로고    scopus 로고
    • Gene therapy for red-green colour blindness in adult primates
    • K. Mancuso, W.W. Hauswirth, and Q. Li Gene therapy for red-green colour blindness in adult primates Nature 461 7265 2009 784 787
    • (2009) Nature , vol.461 , Issue.7265 , pp. 784-787
    • Mancuso, K.1    Hauswirth, W.W.2    Li, Q.3
  • 36
    • 79955372235 scopus 로고    scopus 로고
    • AAV delivery of wild-type rhodopsin preserves retinal function in a mouse model of autosomal dominant retinitis pigmentosa
    • H. Mao, T. James Jr., and A. Schwein AAV delivery of wild-type rhodopsin preserves retinal function in a mouse model of autosomal dominant retinitis pigmentosa Hum. Gene Ther. 22 2011 567 575
    • (2011) Hum. Gene Ther. , vol.22 , pp. 567-575
    • Mao, H.1    James, Jr.T.2    Schwein, A.3
  • 37
    • 78650918670 scopus 로고    scopus 로고
    • Restoration of cone vision in the CNGA3-/- mouse model of congenital complete lack of cone photoreceptor function
    • S. Michalakis, R. Muhlfriedel, and N. Tanimoto Restoration of cone vision in the CNGA3-/- mouse model of congenital complete lack of cone photoreceptor function Mol. Ther. 18 2010 2057 2063
    • (2010) Mol. Ther. , vol.18 , pp. 2057-2063
    • Michalakis, S.1    Muhlfriedel, R.2    Tanimoto, N.3
  • 38
    • 84857588654 scopus 로고    scopus 로고
    • Long-term preservation of cones and improvement in visual function following gene therapy in a mouse model of Leber congenital amaurosis (LCA) caused by GC1 deficiency
    • M. Mihelec, R.A. Pearson, and S.J. Robbie Long-term preservation of cones and improvement in visual function following gene therapy in a mouse model of Leber congenital amaurosis (LCA) caused by GC1 deficiency Hum. Gene Ther. 20 16 2011 3161 3175
    • (2011) Hum. Gene Ther. , vol.20 , Issue.16 , pp. 3161-3175
    • Mihelec, M.1    Pearson, R.A.2    Robbie, S.J.3
  • 39
    • 79953314650 scopus 로고    scopus 로고
    • Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosa
    • S. Millington-Ward, N. Chadderton, and M. O'Reilly Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosa Mol. Ther. 19 2011 642 649
    • (2011) Mol. Ther. , vol.19 , pp. 642-649
    • Millington-Ward, S.1    Chadderton, N.2    O'Reilly, M.3
  • 41
    • 79952097561 scopus 로고    scopus 로고
    • Zinc-finger-based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa
    • C. Mussolino, D. Sanges, and E. Marrocco Zinc-finger-based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa EMBO Mol. Med. 3 2011 118 128
    • (2011) EMBO Mol. Med. , vol.3 , pp. 118-128
    • Mussolino, C.1    Sanges, D.2    Marrocco, E.3
  • 46
    • 33645224383 scopus 로고    scopus 로고
    • RNAi-based suppression and replacement of rds-peripherin in retinal organotypic culture
    • A. Palfi, M. Ader, and A.S. Kiang RNAi-based suppression and replacement of rds-peripherin in retinal organotypic culture Hum. Mutat. 27 2006 260 268
    • (2006) Hum. Mutat. , vol.27 , pp. 260-268
    • Palfi, A.1    Ader, M.2    Kiang, A.S.3
  • 48
    • 53449093170 scopus 로고    scopus 로고
    • AAV-mediated gene therapy for retinal degeneration in the rd10 mouse containing a recessive PDEbeta mutation
    • J.J. Pang, S.L. Boye, and A. Kumar AAV-mediated gene therapy for retinal degeneration in the rd10 mouse containing a recessive PDEbeta mutation Invest. Ophthalmol. Vis. Sci. 49 2008 4278 4283
    • (2008) Invest. Ophthalmol. Vis. Sci. , vol.49 , pp. 4278-4283
    • Pang, J.J.1    Boye, S.L.2    Kumar, A.3
  • 49
    • 77954493492 scopus 로고    scopus 로고
    • Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency
    • J. Pang, S.E. Boye, and B. Lei Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency Gene Ther. 17 2010 815 826
    • (2010) Gene Ther. , vol.17 , pp. 815-826
    • Pang, J.1    Boye, S.E.2    Lei, B.3
  • 50
    • 79955059185 scopus 로고    scopus 로고
    • Achromatopsia as a potential candidate for gene therapy
    • J.J. Pang, J. Alexander, and B. Lei Achromatopsia as a potential candidate for gene therapy Adv. Exp. Med. Biol. 664 2010 639 646
    • (2010) Adv. Exp. Med. Biol. , vol.664 , pp. 639-646
    • Pang, J.J.1    Alexander, J.2    Lei, B.3
  • 51
    • 79551620165 scopus 로고    scopus 로고
    • Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa
    • J.J. Pang, X. Dai, and S.E. Boye Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa Mol. Ther. 19 2011 234 242
    • (2011) Mol. Ther. , vol.19 , pp. 234-242
    • Pang, J.J.1    Dai, X.2    Boye, S.E.3
  • 53
    • 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
    • G.-M. Sarra, C. Stephens, and M. de Alwis 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 2001 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
  • 54
    • 79955030162 scopus 로고    scopus 로고
    • Gene therapy prevents photoreceptor death and preserves retinal function in a Bardet-Biedl syndrome mouse model
    • D.L. Simons, S.L. Boye, and W.W. Hauswirth Gene therapy prevents photoreceptor death and preserves retinal function in a Bardet-Biedl syndrome mouse model Proc. Natl. Acad. Sci. USA 108 2011 6276 6281
    • (2011) Proc. Natl. Acad. Sci. USA , vol.108 , pp. 6276-6281
    • Simons, D.L.1    Boye, S.L.2    Hauswirth, W.W.3
  • 55
    • 74349104948 scopus 로고    scopus 로고
    • Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations
    • X. Sun, B. Pawlyk, and X. Xu Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations Gene Ther. 17 2011 117 131
    • (2011) Gene Ther. , vol.17 , pp. 117-131
    • Sun, X.1    Pawlyk, B.2    Xu, X.3
  • 57
    • 0032816002 scopus 로고    scopus 로고
    • Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer
    • M. Takahashi, H. Miyoshi, and I.M. Verma Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer J. Virol. 73 1999 7812 7816 (Pubitemid 29383266)
    • (1999) Journal of Virology , vol.73 , Issue.9 , pp. 7812-7816
    • Takahashi, M.1    Miyoshi, H.2    Verma, I.M.3    Gage, F.H.4
  • 59
    • 79959610722 scopus 로고    scopus 로고
    • Protection of Photoreceptors in a Mouse Model of RP10
    • L.C. Tam, A.S. Kiang, and N. Chadderton Protection of Photoreceptors in a Mouse Model of RP10 Adv. Exp. Med. Biol. 664 2010 559 565
    • (2010) Adv. Exp. Med. Biol. , vol.664 , pp. 559-565
    • Tam, L.C.1    Kiang, A.S.2    Chadderton, N.3
  • 60
    • 66149101630 scopus 로고    scopus 로고
    • Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: Effective rescue of mouse models of partial and complete Aipl1 deficiency
    • M. Tan, A. Smith, and B. Pawlyk Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: Effective rescue of mouse models of partial and complete Aipl1 deficiency Hum. Mol. Genet. 18 2009 2099 2114
    • (2009) Hum. Mol. Genet. , vol.18 , pp. 2099-2114
    • Tan, M.1    Smith, A.2    Pawlyk, B.3
  • 61
    • 83155177072 scopus 로고    scopus 로고
    • Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey
    • L. Vandenberghe, P. Bell, and A. Maguire Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey Sci. Transl. Med. 3 2011 88ra54
    • (2011) Sci. Transl. Med. , vol.3
    • Vandenberghe, L.1    Bell, P.2    Maguire, A.3
  • 62
    • 33745608443 scopus 로고    scopus 로고
    • Lentiviral expression of retinal guanylate cyclase-1 (RetGC1) restores vision in an avian model of childhood blindness
    • M.L. Williams, J.E. Coleman, and S.E. Haire Lentiviral expression of retinal guanylate cyclase-1 (RetGC1) restores vision in an avian model of childhood blindness PLoS Med. 3 2006 e201
    • (2006) PLoS Med. , vol.3 , pp. 201
    • Williams, M.L.1    Coleman, J.E.2    Haire, S.E.3
  • 64
    • 79956020619 scopus 로고    scopus 로고
    • Whirlin replacement restores the formation of the USH2 protein complex in whirlin knockout photoreceptors
    • J. Zou, L. Luo, and Z. Shen Whirlin replacement restores the formation of the USH2 protein complex in whirlin knockout photoreceptors Invest. Ophthalmol. Vis. Sci. 52 2011 2343 2351
    • (2011) Invest. Ophthalmol. Vis. Sci. , vol.52 , pp. 2343-2351
    • Zou, J.1    Luo, L.2    Shen, Z.3


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