-
1
-
-
0035371211
-
Molecular genetics and prospects for therapy of the inherited retinal dystrophies
-
Bessant DA, Ali RR, Bhattacharya SS. Molecular genetics and prospects for therapy of the inherited retinal dystrophies. Curr Opin Genet Dev 2001; 11: 307-316.
-
(2001)
Curr Opin Genet Dev
, vol.11
, pp. 307-316
-
-
Bessant, D.A.1
Ali, R.R.2
Bhattacharya, S.S.3
-
2
-
-
33846957381
-
Perspective on genes and mutations causing retinitis pigmentosa
-
Daiger SP, Bowne SJ, Sullivan LS. Perspective on genes and mutations causing retinitis pigmentosa. Arch Ophthalmol 2007; 125: 151-158.
-
(2007)
Arch Ophthalmol
, vol.125
, pp. 151-158
-
-
Daiger, S.P.1
Bowne, S.J.2
Sullivan, L.S.3
-
3
-
-
0036500230
-
On the genetics of retinitis pigmentosa and on mutation-independent approaches to therapeutic intervention
-
Farrar GJ, Kenna PF, Humphries P. On the genetics of retinitis pigmentosa and on mutation-independent approaches to therapeutic intervention. EMBO J 2002; 21: 857-864.
-
(2002)
EMBO J
, vol.21
, pp. 857-864
-
-
Farrar, G.J.1
Kenna, P.F.2
Humphries, P.3
-
5
-
-
63449125884
-
Prospects for retinal gene replacement therapy
-
Smith AJ, Bainbridge JW, Ali RR. Prospects for retinal gene replacement therapy. Trends Genet 2009; 25: 156-165.
-
(2009)
Trends Genet
, vol.25
, pp. 156-165
-
-
Smith, A.J.1
Bainbridge, J.W.2
Ali, R.R.3
-
6
-
-
0026053969
-
The retinal degeneration slow (rds) gene product is a photoreceptor disc membrane-associated glycoprotein
-
Travis GH, Sutcliffe JG, Bok D. The retinal degeneration slow (rds) gene product is a photoreceptor disc membrane-associated glycoprotein. Neuron 1991; 6: 61-70.
-
(1991)
Neuron
, vol.6
, pp. 61-70
-
-
Travis, G.H.1
Sutcliffe, J.G.2
Bok, D.3
-
7
-
-
46549086021
-
The spectrum of retinal dystrophies caused by mutations in the peripherin/RDS gene
-
Boon CJ, den Hollander AI, Hoyng CB, Cremers FP, Klevering BJ, Keunen JE. The spectrum of retinal dystrophies caused by mutations in the peripherin/RDS gene. Prog Retin Eye Res 2008; 27: 213-235.
-
(2008)
Prog Retin Eye Res
, vol.27
, pp. 213-235
-
-
Boon, C.J.1
Den Hollander, A.I.2
Hoyng, C.B.3
Cremers, F.P.4
Klevering, B.J.5
Keunen, J.E.6
-
9
-
-
0034870596
-
Molecular genetics of macular degeneration
-
Musarella MA. Molecular genetics of macular degeneration. Doc Ophthalmol 2001; 102: 165-177.
-
(2001)
Doc Ophthalmol
, vol.102
, pp. 165-177
-
-
Musarella, M.A.1
-
10
-
-
0035162582
-
Prevalence of mutations causing retinitis pigmentosa and other inherited retinopathies
-
Sohocki MM, Daiger SP, Bowne SJ, Rodriquez JA, Northrup H, Heckenlively JR et al. Prevalence of mutations causing retinitis pigmentosa and other inherited retinopathies. Hum Mutat 2001; 17: 42-51.
-
(2001)
Hum Mutat
, vol.17
, pp. 42-51
-
-
Sohocki, M.M.1
Daiger, S.P.2
Bowne, S.J.3
Rodriquez, J.A.4
Northrup, H.5
Heckenlively, J.R.6
-
11
-
-
33746681394
-
Prevalence of disease-causing mutations in families with autosomal dominant retinitis pigmentosa: A screen of known genes in 200 families
-
Sullivan LS, Bowne SJ, Birch DG, Hughbanks-Wheaton D, Heckenlively JR, Lewis RA et al. Prevalence of disease-causing mutations in families with autosomal dominant retinitis pigmentosa: a screen of known genes in 200 families. Invest Ophthalmol Vis Sci 2006; 47: 3052-3064.
-
(2006)
Invest Ophthalmol Vis Sci
, vol.47
, pp. 3052-3064
-
-
Sullivan, L.S.1
Bowne, S.J.2
Birch, D.G.3
Hughbanks-Wheaton, D.4
Heckenlively, J.R.5
Lewis, R.A.6
-
12
-
-
44249120315
-
Effect of gene therapy on visual function in Leber's congenital amaurosis
-
Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med 2008; 358: 2231-2239.
-
(2008)
N Engl J Med
, vol.358
, pp. 2231-2239
-
-
Bainbridge, J.W.1
Smith, A.J.2
Barker, S.S.3
Robbie, S.4
Henderson, R.5
Balaggan, K.6
-
13
-
-
54949104686
-
Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adenoassociated virus gene vector: Short-term results of a phase i trial
-
Hauswirth WW, Aleman TS, Kaushal S, Cideciyan AV, Schwartz SB, Wang L et al. Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adenoassociated virus gene vector: short-term results of a phase I trial. Hum Gene Ther 2008; 19: 979-990.
-
(2008)
Hum Gene Ther
, vol.19
, pp. 979-990
-
-
Hauswirth, W.W.1
Aleman, T.S.2
Kaushal, S.3
Cideciyan, A.V.4
Schwartz, S.B.5
Wang, L.6
-
14
-
-
44249085878
-
Safety and efficacy of gene transfer for Leber's congenital amaurosis
-
Maguire AM, Simonelli F, Pierce EA, Pugh Jr EN, Mingozzi F, Bennicelli J et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 2008; 358: 2240-2248.
-
(2008)
N Engl J Med
, vol.358
, pp. 2240-2248
-
-
Maguire, A.M.1
Simonelli, F.2
Pierce, E.A.3
Pugh Jr., E.N.4
Mingozzi, F.5
Bennicelli, J.6
-
15
-
-
16444376307
-
Assessing adenoviral hammerhead ribozyme and small hairpin RNA cassettes in neurons: Inhibition of endogenous caspase-3 activity and protection from apoptotic cell death
-
Bantounas I, Glover CP, Kelly S, Iseki S, Phylactou LA, Uney JB. Assessing adenoviral hammerhead ribozyme and small hairpin RNA cassettes in neurons: inhibition of endogenous caspase-3 activity and protection from apoptotic cell death. J Neurosci Res 2005; 79: 661-669.
-
(2005)
J Neurosci Res
, vol.79
, pp. 661-669
-
-
Bantounas, I.1
Glover, C.P.2
Kelly, S.3
Iseki, S.4
Phylactou, L.A.5
Uney, J.B.6
-
16
-
-
0031965901
-
Ribozyme-targeted destruction of RNA associated with autosomal- dominant retinitis pigmentosa
-
Drenser KA, Timmers AM, Hauswirth WW, Lewin AS. Ribozyme-targeted destruction of RNA associated with autosomal- dominant retinitis pigmentosa. Invest Ophthalmol Vis Sci 1998; 39: 681-689.
-
(1998)
Invest Ophthalmol Vis Sci
, vol.39
, pp. 681-689
-
-
Drenser, K.A.1
Timmers, A.M.2
Hauswirth, W.W.3
Lewin, A.S.4
-
17
-
-
33751232603
-
Preservation of photoreceptor morphology and function in P23H rats using an allele independent ribozyme
-
Gorbatyuk M, Justilien V, Liu J, Hauswirth WW, Lewin AS. Preservation of photoreceptor morphology and function in P23H rats using an allele independent ribozyme. Exp Eye Res 2007; 84: 44-52.
-
(2007)
Exp Eye Res
, vol.84
, pp. 44-52
-
-
Gorbatyuk, M.1
Justilien, V.2
Liu, J.3
Hauswirth, W.W.4
Lewin, A.S.5
-
18
-
-
0034633619
-
Ribozyme rescue of photoreceptor cells in P23H transgenic rats: Long-term survival and late-stage therapy
-
Lavail MM, Yasumura D, Matthes MT, Drenser KA, Flannery JG, Lewin AS et al. Ribozyme rescue of photoreceptor cells in P23H transgenic rats: long-term survival and late-stage therapy. Proc Natl Acad Sci USA 2000; 97: 11488-11493.
-
(2000)
Proc Natl Acad Sci USA
, vol.97
, pp. 11488-11493
-
-
Lavail, M.M.1
Yasumura, D.2
Matthes, M.T.3
Drenser, K.A.4
Flannery, J.G.5
Lewin, A.S.6
-
19
-
-
0031880563
-
Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa
-
Lewin AS, Drenser KA, HauswirthWW, Nishikawa S, Yasumura D, Flannery JG et al. Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa. Nat Med 1998; 4: 967-971.
-
(1998)
Nat Med
, vol.4
, pp. 967-971
-
-
Lewin, A.S.1
Drenser, K.A.2
Hauswirth, W.W.3
Nishikawa, S.4
Yasumura, D.5
Flannery, J.G.6
-
20
-
-
33749445653
-
Preferential silencing of a common dominant rhodopsin mutation does not inhibit retinal degeneration in a transgenic model
-
Tessitore A, Parisi F, Denti MA, Allocca M, Di Vicino U, Domenici L et al. Preferential silencing of a common dominant rhodopsin mutation does not inhibit retinal degeneration in a transgenic model. Mol Ther 2006; 14: 692-699.
-
(2006)
Mol Ther
, vol.14
, pp. 692-699
-
-
Tessitore, A.1
Parisi, F.2
Denti, M.A.3
Allocca, M.4
Di Vicino, U.5
Domenici, L.6
-
21
-
-
33947595161
-
Suppression of mouse rhodopsin expression in vivo by AAV mediated siRNA delivery
-
Gorbatyuk M, Justilien V, Liu J, Hauswirth WW, Lewin AS. Suppression of mouse rhodopsin expression in vivo by AAV mediated siRNA delivery. Vision Res 2007; 47: 1202-1208.
-
(2007)
Vision Res
, vol.47
, pp. 1202-1208
-
-
Gorbatyuk, M.1
Justilien, V.2
Liu, J.3
Hauswirth, W.W.4
Lewin, A.S.5
-
22
-
-
34347246364
-
RNA interference-mediated suppression and replacement of human rhodopsin in vivo
-
O'Reilly M, Palfi A, Chadderton N, Millington-Ward S, Ader M, Cronin T et al. RNA interference-mediated suppression and replacement of human rhodopsin in vivo. Am J Hum Genet 2007; 81: 127-135.
-
(2007)
Am J Hum Genet
, vol.81
, pp. 127-135
-
-
O'Reilly, M.1
Palfi, A.2
Chadderton, N.3
Millington-Ward, S.4
Ader, M.5
Cronin, T.6
-
23
-
-
38749085254
-
A transgenic mouse model for gene therapy of rhodopsin-linked retinitis pigmentosa
-
O'Reilly M, Millington-Ward S, Palfi A, Chadderton N, Cronin T, McNally N et al. A transgenic mouse model for gene therapy of rhodopsin-linked retinitis pigmentosa. Vision Res 2008; 48: 386-391.
-
(2008)
Vision Res
, vol.48
, pp. 386-391
-
-
O'Reilly, M.1
Millington-Ward, S.2
Palfi, A.3
Chadderton, N.4
Cronin, T.5
McNally, N.6
-
24
-
-
33645224383
-
RNAi-based suppression and replacement of rdsperipherin in retinal organotypic culture
-
Palfi A, Ader M, Kiang AS, Millington-Ward S, Clark G, O'Reilly M et al. RNAi-based suppression and replacement of rdsperipherin in retinal organotypic culture. Hum Mutat 2006; 27: 260-268.
-
(2006)
Hum Mutat
, vol.27
, pp. 260-268
-
-
Palfi, A.1
Ader, M.2
Kiang, A.S.3
Millington-Ward, S.4
Clark, G.5
O'Reilly, M.6
-
25
-
-
46249091156
-
Therapeutic benefit derived from RNAi-mediated ablation of IMPDH1 transcripts in a murine model of autosomal dominant retinitis pigmentosa (RP10)
-
Tam LC, Kiang AS, Kennan A, Kenna PF, Chadderton N, Ader M et al. Therapeutic benefit derived from RNAi-mediated ablation of IMPDH1 transcripts in a murine model of autosomal dominant retinitis pigmentosa (RP10). Hum Mol Genet 2008; 17: 2084-2100.
-
(2008)
Hum Mol Genet
, vol.17
, pp. 2084-2100
-
-
Tam, L.C.1
Kiang, A.S.2
Kennan, A.3
Kenna, P.F.4
Chadderton, N.5
Ader, M.6
-
26
-
-
63949087144
-
Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy
-
Chadderton N, Millington-Ward S, Palfi A, O'Reilly M, Tuohy G, Humphries MM et al. Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy. Mol Ther 2009; 17: 593-599.
-
(2009)
Mol Ther
, vol.17
, pp. 593-599
-
-
Chadderton, N.1
Millington-Ward, S.2
Palfi, A.3
O'Reilly, M.4
Tuohy, G.5
Humphries, M.M.6
-
27
-
-
33745299723
-
Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways
-
Grimm D, Streetz KL, Jopling CL, Storm TA, Pandey K, Davis CR et al. Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature 2006; 441: 537-541.
-
(2006)
Nature
, vol.441
, pp. 537-541
-
-
Grimm, D.1
Streetz, K.L.2
Jopling, C.L.3
Storm, T.A.4
Pandey, K.5
Davis, C.R.6
-
28
-
-
0037685280
-
Expression profiling reveals off-target gene regulation by RNAi
-
Jackson AL, Bartz SR, Schelter J, Kobayashi SV, Burchard J, Mao M et al. Expression profiling reveals off-target gene regulation by RNAi. Nat Biotechnol 2003; 21: 635-637.
-
(2003)
Nat Biotechnol
, vol.21
, pp. 635-637
-
-
Jackson, A.L.1
Bartz, S.R.2
Schelter, J.3
Kobayashi, S.V.4
Burchard, J.5
Mao, M.6
-
29
-
-
1842608548
-
MicroRNA precursors in motion: Exportin-5 mediates their nuclear export
-
Kim VN. MicroRNA precursors in motion: exportin-5 mediates their nuclear export. Trends Cell Biol 2004; 14: 156-159.
-
(2004)
Trends Cell Biol
, vol.14
, pp. 156-159
-
-
Kim, V.N.1
-
30
-
-
44449121785
-
Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi
-
McBride JL, Boudreau RL, Harper SQ, Staber PD, Monteys AM, Martins I et al. Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi. Proc Natl Acad Sci USA 2008; 105: 5868-5873.
-
(2008)
Proc Natl Acad Sci USA
, vol.105
, pp. 5868-5873
-
-
McBride, J.L.1
Boudreau, R.L.2
Harper, S.Q.3
Staber, P.D.4
Monteys, A.M.5
Martins, I.6
-
31
-
-
67651102784
-
An efficient method to enhance gene silencing by using precursor microRNA designed small hairpin RNAs
-
Shan Z, Lin Q, Deng C, Li X, Huang W, Tan H et al. An efficient method to enhance gene silencing by using precursor microRNA designed small hairpin RNAs. Mol Biol Rep 2008; 36: 1483-1489.
-
(2008)
Mol Biol Rep
, vol.36
, pp. 1483-1489
-
-
Shan, Z.1
Lin, Q.2
Deng, C.3
Li, X.4
Huang, W.5
Tan, H.6
-
32
-
-
58149232358
-
Artificial microRNAs as siRNA shuttles: Improved safety as compared to shRNAs in vitro and in vivo
-
Boudreau RL, Martins I, Davidson BL. Artificial microRNAs as siRNA shuttles: improved safety as compared to shRNAs in vitro and in vivo. Mol Ther 2009; 17: 169-175.
-
(2009)
Mol Ther
, vol.17
, pp. 169-175
-
-
Boudreau, R.L.1
Martins, I.2
Davidson, B.L.3
-
33
-
-
23744485151
-
Towards mutationindependent silencing of genes involved in retinal degeneration by RNA interference
-
Cashman SM, Binkley EA, Kumar-Singh R. Towards mutationindependent silencing of genes involved in retinal degeneration by RNA interference. Gene Therapy 2005; 12: 1223-1228.
-
(2005)
Gene Therapy
, vol.12
, pp. 1223-1228
-
-
Cashman, S.M.1
Binkley, E.A.2
Kumar-Singh, R.3
-
34
-
-
2342471249
-
Enhanced gene silencing of HIV-1 specific siRNA using microRNA designed hairpins
-
Boden D, Pusch O, Silbermann R, Lee F, Tucker L, Ramratnam B. Enhanced gene silencing of HIV-1 specific siRNA using microRNA designed hairpins. Nucleic Acids Res 2004; 32: 1154-1158.
-
(2004)
Nucleic Acids Res
, vol.32
, pp. 1154-1158
-
-
Boden, D.1
Pusch, O.2
Silbermann, R.3
Lee, F.4
Tucker, L.5
Ramratnam, B.6
-
35
-
-
0033947697
-
Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy
-
Ali RR, Sarra GM, Stephens C, Alwis MD, Bainbridge JW, Munro PM et al. Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy. Nat Genet 2000; 25: 306-310.
-
(2000)
Nat Genet
, vol.25
, pp. 306-310
-
-
Ali, R.R.1
Sarra, G.M.2
Stephens, C.3
Alwis, M.D.4
Bainbridge, J.W.5
Munro, P.M.6
-
36
-
-
35348844777
-
Rapid and stable knockdown of an endogenous gene in retinal pigment epithelium
-
Paskowitz DM, Greenberg KP, Yasumura D, Grimm D, Yang H, Duncan JL et al. Rapid and stable knockdown of an endogenous gene in retinal pigment epithelium. Hum Gene Ther 2007; 18: 871-880.
-
(2007)
Hum Gene Ther
, vol.18
, pp. 871-880
-
-
Paskowitz, D.M.1
Greenberg, K.P.2
Yasumura, D.3
Grimm, D.4
Yang, H.5
Duncan, J.L.6
-
37
-
-
4043057946
-
RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia
-
Xia H, Mao Q, Eliason SL, Harper SQ, Martins IH, Orr HT et al. RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia. Nat Med 2004; 10: 816-820.
-
(2004)
Nat Med
, vol.10
, pp. 816-820
-
-
Xia, H.1
Mao, Q.2
Eliason, S.L.3
Harper, S.Q.4
Martins, I.H.5
Orr, H.T.6
-
38
-
-
40249098715
-
Assessment of ocular transduction using singlestranded and self-complementary recombinant adeno-associated virus serotype 2/8
-
Natkunarajah M, Trittibach P, McIntosh J, Duran Y, Barker SE, Smith AJ et al. Assessment of ocular transduction using singlestranded and self-complementary recombinant adeno-associated virus serotype 2/8. Gene Therapy 2008; 15: 463-467.
-
(2008)
Gene Therapy
, vol.15
, pp. 463-467
-
-
Natkunarajah, M.1
Trittibach, P.2
McIntosh, J.3
Duran, Y.4
Barker, S.E.5
Smith, A.J.6
-
39
-
-
0029914677
-
Gene transfer into the mouse retina mediated by an adeno-associated viral vector
-
Ali RR, Reichel MB, Thrasher AJ, Levinsky RJ, Kinnon C, Kanuga N et al. Gene transfer into the mouse retina mediated by an adeno-associated viral vector. Hum Mol Genet 1996; 5: 591-594.
-
(1996)
Hum Mol Genet
, vol.5
, pp. 591-594
-
-
Ali, R.R.1
Reichel, M.B.2
Thrasher, A.J.3
Levinsky, R.J.4
Kinnon, C.5
Kanuga, N.6
-
40
-
-
17644415316
-
Long-term preservation of retinal function in the RCS rat model of retinitis pigmentosa following lentivirus-mediated gene therapy
-
Tschernutter M, Schlichtenbrede FC, Howe S, Balaggan KS, Munro PM, Bainbridge JW et al. Long-term preservation of retinal function in the RCS rat model of retinitis pigmentosa following lentivirus-mediated gene therapy. Gene Therapy 2005; 12: 694-701.
-
(2005)
Gene Therapy
, vol.12
, pp. 694-701
-
-
Tschernutter, M.1
Schlichtenbrede, F.C.2
Howe, S.3
Balaggan, K.S.4
Munro, P.M.5
Bainbridge, J.W.6
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