-
1
-
-
84902096048
-
Development and applications of CRISPR-Cas9 for genome engineering
-
Hsu PD, Lander ES, Zhang F. Development and applications of CRISPR-Cas9 for genome engineering. Cell 2014; 157: 1262-1278
-
(2014)
Cell
, vol.157
, pp. 1262-1278
-
-
Hsu, P.D.1
Lander, E.S.2
Zhang, F.3
-
2
-
-
84892765883
-
Genomescale CRISPR-Cas9 knockout screening in human cells
-
Shalem O, Sanjana NE, Hartenian E, Shi X, Scott DA, Mikkelsen TS et al. Genomescale CRISPR-Cas9 knockout screening in human cells. Science 2014; 343: 84-87
-
(2014)
Science
, vol.343
, pp. 84-87
-
-
Shalem, O.1
Sanjana, N.E.2
Hartenian, E.3
Shi, X.4
Scott, D.A.5
Mikkelsen, T.S.6
-
3
-
-
84892749369
-
Genetic screens in human cells using the CRISPR-Cas9 system
-
Wang T, Wei JJ, Sabatini DM, Lander ES. Genetic screens in human cells using the CRISPR-Cas9 system. Science 2014; 343: 80-84
-
(2014)
Science
, vol.343
, pp. 80-84
-
-
Wang, T.1
Wei, J.J.2
Sabatini, D.M.3
Lander, E.S.4
-
4
-
-
84884687531
-
Type I-E CRISPR-cas systems discriminate target from non-target DNA through base pairing-independent PAM recognition
-
Westra ER, Semenova E, Datsenko KA, Jackson RN, Wiedenheft B, Severinov K et al. Type I-E CRISPR-cas systems discriminate target from non-target DNA through base pairing-independent PAM recognition. PLoS Genet 2013; 9: e1003742
-
(2013)
PLoS Genet
, vol.9
, pp. e1003742
-
-
Westra, E.R.1
Semenova, E.2
Datsenko, K.A.3
Jackson, R.N.4
Wiedenheft, B.5
Severinov, K.6
-
5
-
-
84924033062
-
Efficient and allele-specific genome editing of disease loci in human iPSCs
-
Smith C, Abalde-Atristain L, He C, Brodsky BR, Braunstein EM, Chaudhari P et al. Efficient and allele-specific genome editing of disease loci in human iPSCs. Mol Ther 2014; 23: 570-577
-
(2014)
Mol Ther
, vol.23
, pp. 570-577
-
-
Smith, C.1
Abalde-Atristain, L.2
He, C.3
Brodsky, B.R.4
Braunstein, E.M.5
Chaudhari, P.6
-
6
-
-
84903555108
-
Allele-specific genome editing and correction of disease-associated phenotypes in rats using the CRISPR-Cas platform
-
Yoshimi K, Kaneko T, Voigt B, Mashimo T. Allele-specific genome editing and correction of disease-associated phenotypes in rats using the CRISPR-Cas platform. Nat Commun 2014; 5: 4240
-
(2014)
Nat Commun
, vol.5
, pp. 4240
-
-
Yoshimi, K.1
Kaneko, T.2
Voigt, B.3
Mashimo, T.4
-
7
-
-
83155167571
-
Development of allele-specific therapeutic siRNA in Meesmann epithelial corneal dystrophy
-
Liao H, Irvine AD, Macewen CJ, Weed KH, Porter L, Corden LD et al. Development of allele-specific therapeutic siRNA in Meesmann epithelial corneal dystrophy. PLoS One 2011; 6: e28582
-
(2011)
PLoS One
, vol.6
, pp. e28582
-
-
Liao, H.1
Irvine, A.D.2
Macewen, C.J.3
Weed, K.H.4
Porter, L.5
Corden, L.D.6
-
8
-
-
0030475766
-
Keratin 12-deficient mice have fragile corneal epithelia
-
Kao WW, Liu CY, Converse RL, Shiraishi A, Kao CW, Ishizaki M et al. Keratin 12-deficient mice have fragile corneal epithelia. Invest Ophthalmol Vis Sci 1996; 37: 2572-2584
-
(1996)
Invest Ophthalmol Vis Sci
, vol.37
, pp. 2572-2584
-
-
Kao, W.W.1
Liu, C.Y.2
Converse, R.L.3
Shiraishi, A.4
Kao, C.W.5
Ishizaki, M.6
-
9
-
-
84894086032
-
Development of allele-specific gene-silencing siRNAs for TGFBI Arg124Cys in lattice corneal dystrophy type I
-
Courtney DG, Atkinson SD, Moore JE, Maurizi E, Serafini C, Pellegrini G et al. Development of allele-specific gene-silencing siRNAs for TGFBI Arg124Cys in lattice corneal dystrophy type I. Invest Ophthalmol Vis Sci 2014; 55: 977-985
-
(2014)
Invest Ophthalmol Vis Sci
, vol.55
, pp. 977-985
-
-
Courtney, D.G.1
Atkinson, S.D.2
Moore, J.E.3
Maurizi, E.4
Serafini, C.5
Pellegrini, G.6
-
10
-
-
84901610878
-
SiRNA silencing of the mutant keratin 12 allele in corneal limbal epithelial cells grown from patients with Meesmann?s epithelial corneal dystrophy
-
Courtney DG, Atkinson SD, Allen EHA, Moore JE, Walsh CP, Pedrioli DML et al. siRNA silencing of the mutant keratin 12 allele in corneal limbal epithelial cells grown from patients with Meesmann?s epithelial corneal dystrophy. Invest Ophthalmol Vis Sci 2014; 55: 3352-3360
-
(2014)
Invest Ophthalmol Vis Sci
, vol.55
, pp. 3352-3360
-
-
Courtney, D.G.1
Atkinson, S.D.2
Eha, A.3
Moore, J.E.4
Walsh, C.P.5
Dml, P.6
-
11
-
-
84874741259
-
Allele-specific siRNA silencing for the common keratin 12 founder mutation in Meesmann epithelial corneal dystrophy
-
Allen EHA, Atkinson SD, Liao H, Moore JE, Leslie Pedrioli DM, Smith FJD et al. Allele-specific siRNA silencing for the common keratin 12 founder mutation in Meesmann epithelial corneal dystrophy. Invest Ophthalmol Vis Sci 2013; 54: 494-502
-
(2013)
Invest Ophthalmol Vis Sci
, vol.54
, pp. 494-502
-
-
Eha, A.1
Atkinson, S.D.2
Liao, H.3
Moore, J.E.4
Leslie Pedrioli, D.M.5
Fjd, S.6
-
12
-
-
84890033064
-
Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients
-
Schwank G, Koo B-K, Sasselli V, Dekkers JF, Heo I, Demircan T et al. Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients. Cell Stem Cell 2013; 13: 653-658
-
(2013)
Cell Stem Cell
, vol.13
, pp. 653-658
-
-
Schwank, G.1
Koo, B.-K.2
Sasselli, V.3
Dekkers, J.F.4
Heo, I.5
Demircan, T.6
-
13
-
-
84902095353
-
Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
-
Yin H, Xue W, Chen S, Bogorad RL, Benedetti E, Grompe M et al. Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype. Nat Biotechnol 2014; 32: 551-553
-
(2014)
Nat Biotechnol
, vol.32
, pp. 551-553
-
-
Yin, H.1
Xue, W.2
Chen, S.3
Bogorad, R.L.4
Benedetti, E.5
Grompe, M.6
-
14
-
-
84890050551
-
Correction of a genetic disease in mouse via use of CRISPR-Cas9
-
Wu Y, Liang D, Wang Y, Bai M, Tang W, Bao S et al. Correction of a genetic disease in mouse via use of CRISPR-Cas9. Cell Stem Cell 2013; 13: 659-662
-
(2013)
Cell Stem Cell
, vol.13
, pp. 659-662
-
-
Wu, Y.1
Liang, D.2
Wang, Y.3
Bai, M.4
Tang, W.5
Bao, S.6
-
15
-
-
0035809198
-
An inducible mouse model for epidermolysis bullosa simplex: Implications for gene therapy
-
Cao T, Longley MA, Wang X, Roop DR. An inducible mouse model for epidermolysis bullosa simplex: implications for gene therapy. J Cell Biol 2001; 152: 651-656
-
(2001)
J Cell Biol
, vol.152
, pp. 651-656
-
-
Cao, T.1
Ma, L.2
Wang, X.3
Roop, D.R.4
-
16
-
-
0036364632
-
High-level gene transfer to the cornea using electroporation
-
Blair-Parks K, Weston BC, Dean DA. High-level gene transfer to the cornea using electroporation. J Gene Med 4: 92-100
-
J Gene Med
, vol.4
, pp. 92-100
-
-
Blair-Parks, K.1
Weston, B.C.2
Dean, D.A.3
-
17
-
-
84887010498
-
Genome engineering using the CRISPR-Cas9 system
-
Ran FA, Hsu PD, Wright J, Agarwala V, Scott DA, Zhang F. Genome engineering using the CRISPR-Cas9 system. Nat Protoc 2013; 8: 2281-2308
-
(2013)
Nat Protoc
, vol.8
, pp. 2281-2308
-
-
Ran, F.A.1
Hsu, P.D.2
Wright, J.3
Agarwala, V.4
Scott, D.A.5
Zhang, F.6
-
18
-
-
80052865748
-
Development of allele-specific therapeutic siRNA for keratin 5 mutations in epidermolysis bullosa simplex
-
Atkinson SD, McGilligan VE, Liao H, Szeverenyi I, Smith FJD, Moore CBT et al. Development of allele-specific therapeutic siRNA for keratin 5 mutations in epidermolysis bullosa simplex. J Invest Dermatol 2011; 131: 2079-2086
-
(2011)
J Invest Dermatol
, vol.131
, pp. 2079-2086
-
-
Atkinson, S.D.1
McGilligan, V.E.2
Liao, H.3
Szeverenyi, I.4
Fjd, S.5
Cbt, M.6
-
20
-
-
0035710746
-
Analysis of relative gene expression data using real-time quantitative PCR and the 2 (-Delta Delta C (T)) Method
-
Livak KJ, Schmittgen TD. Analysis of relative gene expression data using real-time quantitative PCR and the 2 (-Delta Delta C (T)) Method. Methods 2001; 25: 402-408
-
(2001)
Methods
, vol.25
, pp. 402-408
-
-
Livak, K.J.1
Schmittgen, T.D.2
-
21
-
-
1842483281
-
Determination of stable housekeeping genes, differentially regulated target genes and sample integrity: BestKeeper - Excel-based tool using pair-wise correlations
-
Pfaffl MW, Tichopad A, Prgomet C, Neuvians TP. Determination of stable housekeeping genes, differentially regulated target genes and sample integrity: BestKeeper - Excel-based tool using pair-wise correlations. Biotechnol Lett 2004; 26: 509-515
-
(2004)
Biotechnol Lett
, vol.26
, pp. 509-515
-
-
Pfaffl, M.W.1
Tichopad, A.2
Prgomet, C.3
Neuvians, T.P.4
|