-
1
-
-
33750834619
-
Infusion of autologous retrodifferentiated stem cells into patients with beta-thalassemia
-
Abuljadayel IS, Quereshi H, Ahsan T, Rizvi S, Ahmed T, Khan SM, Akhtar J, Dhoot G (2006) Infusion of autologous retrodifferentiated stem cells into patients with beta-thalassemia. Scienti ficWorldJournal 6:1278-1297
-
(2006)
Scienti FicWorldJournal
, vol.6
, pp. 1278-1297
-
-
Abuljadayel, I.S.1
Quereshi, H.2
Ahsan, T.3
Rizvi, S.4
Ahmed, T.5
Khan, S.M.6
Akhtar, J.7
Dhoot, G.8
-
2
-
-
69849098671
-
Hematopoietic stem cell gene therapy for adenosine deaminase de ficient-SCID
-
Aiuti A, Brigida I, Ferrua F, Cappelli B, Chiesa R, Marktel S, Roncarolo MG (2009) Hematopoietic stem cell gene therapy for adenosine deaminase de ficient-SCID. Immunol Res 44:150-159
-
(2009)
Immunol Res
, vol.44
, pp. 150-159
-
-
Aiuti, A.1
Brigida, I.2
Ferrua, F.3
Cappelli, B.4
Chiesa, R.5
Marktel, S.6
Roncarolo, M.G.7
-
3
-
-
79953117041
-
Evidence for the cure of HIV infection by CCR5Delta32/Delta32 stem cell transplantation
-
Allers K, Hutter G, Hofmann J, Loddenkemper C, Rieger K, Thiel E, Schneider T (2011) Evidence for the cure of HIV infection by CCR5Delta32/Delta32 stem cell transplantation. Blood 117:2791-2799
-
(2011)
Blood
, vol.117
, pp. 2791-2799
-
-
Allers, K.1
Hutter, G.2
Hofmann, J.3
Loddenkemper, C.4
Rieger, K.5
Thiel, E.6
Schneider, T.7
-
4
-
-
25144501447
-
Custom zinc-finger nucleases for use in human cells
-
Alwin S, Gere MB, Guhl E, Effertz K, Barbas CF 3rd, Segal DJ, Weitzman MD, Cathomen T (2005) Custom zinc-finger nucleases for use in human cells. Mol Ther 12:610-617
-
(2005)
Mol Ther
, vol.12
, pp. 610-617
-
-
Alwin, S.1
Gere, M.B.2
Guhl, E.3
Effertz, K.4
Barbas Iii., C.F.5
Segal, D.J.6
Weitzman, M.D.7
Cathomen, T.8
-
5
-
-
28944449341
-
Engineering of large numbers of highly specific homing endonucleases that induce recombination on novel DNA targets
-
Arnould S, Chames P, Perez C, Lacroix E, Duclert A, Epinat JC, Stricher F, Petit AS, Patin A, Guillier S, Rolland S, Prieto J, Blanco FJ, Bravo J, Montoya G, Serrano L, Duchateau P, Paques F (2006) Engineering of large numbers of highly specific homing endonucleases that induce recombination on novel DNA targets. J Mol Biol 355:443-458
-
(2006)
J Mol Biol
, vol.355
, pp. 443-458
-
-
Arnould, S.1
Chames, P.2
Perez, C.3
Lacroix, E.4
Duclert, A.5
Epinat, J.C.6
Stricher, F.7
Petit, A.S.8
Patin, A.9
Guillier, S.10
Rolland, S.11
Prieto, J.12
Blanco, F.J.13
Bravo, J.14
Montoya, G.15
Serrano, L.16
Duchateau, P.17
Paques, F.18
-
6
-
-
34447109864
-
Engineered I-CreI derivatives cleaving sequences from the human XPC gene can induce highly Efficient gene correction in mammalian cells
-
Arnould S, Perez C, Cabaniols JP, Smith J, Gouble A, Grizot S, Epinat JC, Duclert A, Duchateau P, Paques F (2007) Engineered I-CreI derivatives cleaving sequences from the human XPC gene can induce highly Efficient gene correction in mammalian cells. J Mol Biol 371:49-65
-
(2007)
J Mol Biol
, vol.371
, pp. 49-65
-
-
Arnould, S.1
Perez, C.2
Cabaniols, J.P.3
Smith, J.4
Gouble, A.5
Grizot, S.6
Epinat, J.C.7
Duclert, A.8
Duchateau, P.9
Paques, F.10
-
7
-
-
78650432023
-
The I-CreI meganuclease and its engineered derivatives: Applications from cell modification to gene therapy
-
Arnould S, Delenda C, Grizot S, Desseaux C, Paques F, Silva GH, Smith J (2011) The I-CreI meganuclease and its engineered derivatives: applications from cell modification to gene therapy. Protein Eng Des Sel 24:27-31
-
(2011)
Protein Eng des Sel
, vol.24
, pp. 27-31
-
-
Arnould, S.1
Delenda, C.2
Grizot, S.3
Desseaux, C.4
Paques, F.5
Silva, G.H.6
Smith, J.7
-
8
-
-
29744458522
-
A phase I/II clinical trial of beta-globin gene therapy for beta-thalassemia
-
Bank A, Dorazio R, Leboulch P (2005) A phase I/II clinical trial of beta-globin gene therapy for beta-thalassemia. Ann N Y Acad Sci 1054:308-316
-
(2005)
Ann N y Acad Sci
, vol.1054
, pp. 308-316
-
-
Bank, A.1
Dorazio, R.2
Leboulch, P.3
-
9
-
-
0034652141
-
Positive and negative regulation of endogenous genes by designed transcription factors
-
Beerli RR, Dreier B, Barbas CF 3rd (2000) Positive and negative regulation of endogenous genes by designed transcription factors. Proc Natl Acad Sci USA 97:1495-1500
-
(2000)
Proc Natl Acad Sci USA
, vol.97
, pp. 1495-1500
-
-
Beerli, R.R.1
Dreier, B.2
Barbas Iii., C.F.3
-
10
-
-
77950572073
-
Targeted gene addition to human mesenchymal stromal cells as a cell-based plasma-soluble protein delivery platform
-
Benabdallah BF, Allard E, Yao S, Friedman G, Gregory PD, Eliopoulos N, Fradette J, Spees JL, Haddad E, Holmes MC, Beausejour CM (2010) Targeted gene addition to human mesenchymal stromal cells as a cell-based plasma-soluble protein delivery platform. Cytotherapy 12:394-399
-
(2010)
Cytotherapy
, vol.12
, pp. 394-399
-
-
Benabdallah, B.F.1
Allard, E.2
Yao, S.3
Friedman, G.4
Gregory, P.D.5
Eliopoulos, N.6
Fradette, J.7
Spees, J.L.8
Haddad, E.9
Holmes, M.C.10
Beausejour, C.M.11
-
11
-
-
33646182222
-
Efficient gene targeting in Drosophila with zinc-finger nucleases
-
Beumer K, Bhattacharyya G, Bibikova M, Trautman JK, Carroll D (2006) Efficient gene targeting in Drosophila with zinc-finger nucleases. Genetics 172:2391-2403
-
(2006)
Genetics
, vol.172
, pp. 2391-2403
-
-
Beumer, K.1
Bhattacharyya, G.2
Bibikova, M.3
Trautman, J.K.4
Carroll, D.5
-
12
-
-
58149374562
-
Efficient gene targeting in Drosophila by direct embryo injection with zinc-finger nucleases
-
Beumer KJ, Trautman JK, Bozas A, Liu JL, Rutter J, Gall JG, Carroll D (2008) Efficient gene targeting in Drosophila by direct embryo injection with zinc-finger nucleases. Proc Natl Acad Sci USA 105:19821-19826
-
(2008)
Proc Natl Acad Sci USA
, vol.105
, pp. 19821-19826
-
-
Beumer, K.J.1
Trautman, J.K.2
Bozas, A.3
Liu, J.L.4
Rutter, J.5
Gall, J.G.6
Carroll, D.7
-
13
-
-
0034749283
-
Stimulation of homologous recombination through targeted cleavage by chimeric nucleases
-
Bibikova M, Carroll D, Segal DJ, Trautman JK, Smith J, Kim YG, Chandrasegaran S (2001) Stimulation of homologous recombination through targeted cleavage by chimeric nucleases. Mol Cell Biol 21:289-297
-
(2001)
Mol Cell Biol
, vol.21
, pp. 289-297
-
-
Bibikova, M.1
Carroll, D.2
Segal, D.J.3
Trautman, J.K.4
Smith, J.5
Kim, Y.G.6
Chandrasegaran, S.7
-
14
-
-
0036021389
-
Targeted chromosomal cleavage and mutagenesis in Drosophila using zinc-finger nucleases
-
Bibikova M, Golic M, Golic KG, Carroll D (2002) Targeted chromosomal cleavage and mutagenesis in Drosophila using zinc-finger nucleases. Genetics 161:1169-1175
-
(2002)
Genetics
, vol.161
, pp. 1169-1175
-
-
Bibikova, M.1
Golic, M.2
Golic, K.G.3
Carroll, D.4
-
16
-
-
72149110399
-
Breaking the code of DNA binding specificity of TAL-type III effectors
-
Boch J, Scholze H, Schornack S, Landgraf A, Hahn S, Kay S, Lahaye T, Nickstadt A, Bonas U (2009) Breaking the code of DNA binding specificity of TAL-type III effectors. Science 326:1509-1512
-
(2009)
Science
, vol.326
, pp. 1509-1512
-
-
Boch, J.1
Scholze, H.2
Schornack, S.3
Landgraf, A.4
Hahn, S.5
Kay, S.6
Lahaye, T.7
Nickstadt, A.8
Bonas, U.9
-
17
-
-
70349313064
-
Genetic analysis of zinc-finger nucleaseinduced gene targeting in Drosophila
-
Bozas A, Beumer KJ, Trautman JK, Carroll D (2009) Genetic analysis of zinc-finger nucleaseinduced gene targeting in Drosophila. Genetics 182:641-651
-
(2009)
Genetics
, vol.182
, pp. 641-651
-
-
Bozas, A.1
Beumer, K.J.2
Trautman, J.K.3
Carroll, D.4
-
18
-
-
78149482538
-
Stem-cell gene therapy for the Wiskott-Aldrich syndrome
-
Boztug K, Schmidt M, Schwarzer A, Banerjee PP, Diez IA, Dewey RA, Bohm M, Nowrouzi A, Ball CR, Glimm H, Naundorf S, Kuhlcke K, Blasczyk R, Kondratenko I, Marodi L, Orange JS, von Kalle C, Klein C (2010) Stem-cell gene therapy for the Wiskott-Aldrich syndrome. N Engl J Med 363:1918-1927
-
(2010)
N Engl J Med
, vol.363
, pp. 1918-1927
-
-
Boztug, K.1
Schmidt, M.2
Schwarzer, A.3
Banerjee, P.P.4
Diez, I.A.5
Dewey, R.A.6
Bohm, M.7
Nowrouzi, A.8
Ball, C.R.9
Glimm, H.10
Naundorf, S.11
Kuhlcke, K.12
Blasczyk, R.13
Kondratenko, I.14
Marodi, L.15
Orange, J.S.16
Von Kalle, C.17
Klein, C.18
-
19
-
-
67649757162
-
Chromosomal translocations induced at specified loci in human stem cells
-
Brunet E, Simsek D, Tomishima M, Dekelver R, Choi VM, Gregory P, Urnov F, Weinstock DM, Jasin M (2009) Chromosomal translocations induced at specified loci in human stem cells. Proc Natl Acad Sci USA 106:10620-10625
-
(2009)
Proc Natl Acad Sci USA
, vol.106
, pp. 10620-10625
-
-
Brunet, E.1
Simsek, D.2
Tomishima, M.3
Dekelver, R.4
Choi, V.M.5
Gregory, P.6
Urnov, F.7
Weinstock, D.M.8
Jasin, M.9
-
20
-
-
78549267889
-
Targeted genome modification in mice using zinc-finger nucleases
-
Carbery ID, Ji D, Harrington A, Brown V, Weinstein EJ, Liaw L, Cui X (2010) Targeted genome modification in mice using zinc-finger nucleases. Genetics 186:451-459
-
(2010)
Genetics
, vol.186
, pp. 451-459
-
-
Carbery, I.D.1
Ji, D.2
Harrington, A.3
Brown, V.4
Weinstein, E.J.5
Liaw, L.6
Cui, X.7
-
21
-
-
0034724857
-
Gene therapy of human severe combined immunode ficiency (SCID)-X1 disease
-
Fischer A
-
Cavazzana-Calvo M, Hacein-Bey S, De Saint Basile G, Gross F, Yvon E, Nusbaum P, Selz F, Hue C, Certain S, Casanova JL, Bousso P, Deist FL, Fischer A (2000) Gene therapy of human severe combined immunode ficiency (SCID)-X1 disease. Science 288:669-672
-
(2000)
Science
, vol.288
, pp. 669-672
-
-
Cavazzana-Calvo, M.1
Hacein-Bey, S.2
De Saint Basile, G.3
Gross, F.4
Yvon, E.5
Nusbaum, P.6
Selz, F.7
Hue, C.8
Certain, S.9
Casanova, J.L.10
Bousso, P.11
Deist, F.L.12
-
23
-
-
79960064013
-
Efficient design and assembly of custom TALEN and other TAL effectorbased constructs for DNA targeting
-
Cermak T, Doyle EL, Christian M, Wang L, Zhang Y, Schmidt C, Baller JA, Somia NV, Bogdanove AJ, Voytas DF (2011) Efficient design and assembly of custom TALEN and other TAL effectorbased constructs for DNA targeting. Nucleic Acids Res 39:e82
-
(2011)
Nucleic Acids Res
, vol.39
-
-
Cermak, T.1
Doyle, E.L.2
Christian, M.3
Wang, L.4
Zhang, Y.5
Schmidt, C.6
Baller, J.A.7
Somia, N.V.8
Bogdanove, A.J.9
Voytas, D.F.10
-
24
-
-
78951479577
-
Targeting DNA double-strand breaks with TAL effector nucleases
-
Christian M, Cermak T, Doyle EL, Schmidt C, Zhang F, Hummel A, Bogdanove AJ, Voytas DF (2010) Targeting DNA double-strand breaks with TAL effector nucleases. Genetics 186:757-761
-
(2010)
Genetics
, vol.186
, pp. 757-761
-
-
Christian, M.1
Cermak, T.2
Doyle, E.L.3
Schmidt, C.4
Zhang, F.5
Hummel, A.6
Bogdanove, A.J.7
Voytas, D.F.8
-
25
-
-
77953139680
-
Gene correction by homologous recombination with zinc finger nucleases in primary cells from a mouse model of a generic recessive genetic disease
-
Connelly JP, Barker JC, Pruett-Miller S, Porteus MH (2010) Gene correction by homologous recombination with zinc finger nucleases in primary cells from a mouse model of a generic recessive genetic disease. Mol Ther 18:1103-1110
-
(2010)
Mol Ther
, vol.18
, pp. 1103-1110
-
-
Connelly, J.P.1
Barker, J.C.2
Pruett-Miller, S.3
Porteus, M.H.4
-
26
-
-
78651322138
-
Targeted integration in rat and mouse embryos with zinc-finger nucleases
-
Cui X, Ji D, Fisher DA, Wu Y, Briner DM, Weinstein EJ (2011) Targeted integration in rat and mouse embryos with zinc-finger nucleases. Nat Biotechnol 29:64-67
-
(2011)
Nat Biotechnol
, vol.29
, pp. 64-67
-
-
Cui, X.1
Ji, D.2
Fisher, D.A.3
Wu, Y.4
Briner, D.M.5
Weinstein, E.J.6
-
27
-
-
77955157281
-
Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome
-
Dekelver RC, Choi VM, Moehle EA, Paschon DE, Hockemeyer D, Meijsing SH, Sancak Y, Cui X, Steine EJ, Miller JC, Tam P, Bartsevich VV, Meng X, Rupniewski I, Gopalan SM, Sun HC, Pitz KJ, Rock JM, Zhang L, Davis GD, Rebar EJ, Cheeseman IM, Yamamoto KR, Sabatini DM, Jaenisch R, Gregory PD, Urnov FD (2010) Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome. Genome Res 20:1133-1142
-
(2010)
Genome Res
, vol.20
, pp. 1133-1142
-
-
Dekelver, R.C.1
Choi, V.M.2
Moehle, E.A.3
Paschon, D.E.4
Hockemeyer, D.5
Meijsing, S.H.6
Sancak, Y.7
Cui, X.8
Steine, E.J.9
Miller, J.C.10
Tam, P.11
Bartsevich, V.V.12
Meng, X.13
Rupniewski, I.14
Gopalan, S.M.15
Sun, H.C.16
Pitz, K.J.17
Rock, J.M.18
Zhang, L.19
Davis, G.D.20
Rebar, E.J.21
Cheeseman, I.M.22
Yamamoto, K.R.23
Sabatini, D.M.24
Jaenisch, R.25
Gregory, P.D.26
Urnov, F.D.27
more..
-
28
-
-
44949155482
-
Heritable targeted gene disruption in zebra fish using designed zinc-finger nucleases
-
Doyon Y, McCammon JM, Miller JC, Faraji F, Ngo C, Katibah GE, Amora R, Hocking TD, Zhang L, Rebar EJ, Gregory PD, Urnov FD, Amacher SL (2008) Heritable targeted gene disruption in zebra fish using designed zinc-finger nucleases. Nat Biotechnol 26:702-708
-
(2008)
Nat Biotechnol
, vol.26
, pp. 702-708
-
-
Doyon, Y.1
McCammon, J.M.2
Miller, J.C.3
Faraji, F.4
Ngo, C.5
Katibah, G.E.6
Amora, R.7
Hocking, T.D.8
Zhang, L.9
Rebar, E.J.10
Gregory, P.D.11
Urnov, F.D.12
Amacher, S.L.13
-
29
-
-
79952280348
-
Rapid and Efficient clathrin-mediated endocytosis revealed in genome-edited mammalian cells
-
Doyon JB, Zeitler B, Cheng J, Cheng AT, Cherone JM, Santiago Y, Lee AH, Vo TD, Doyon Y, Miller JC, Paschon DE, Zhang L, Rebar EJ, Gregory PD, Urnov FD, Drubin DG (2011a) Rapid and Efficient clathrin-mediated endocytosis revealed in genome-edited mammalian cells. Nat Cell Biol 13:331-337
-
(2011)
Nat Cell Biol
, vol.13
, pp. 331-337
-
-
Doyon, J.B.1
Zeitler, B.2
Cheng, J.3
Cheng, A.T.4
Cherone, J.M.5
Santiago, Y.6
Lee, A.H.7
Vo, T.D.8
Doyon, Y.9
Miller, J.C.10
Paschon, D.E.11
Zhang, L.12
Rebar, E.J.13
Gregory, P.D.14
Urnov, F.D.15
Drubin, D.G.16
-
30
-
-
78650912707
-
Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures
-
Doyon Y, Vo TD, Mendel MC, Greenberg SG, Wang J, Xia DF, Miller JC, Urnov FD, Gregory PD, Holmes MC (2011b) Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures. Nat Methods 8:74-79
-
(2011)
Nat Methods
, vol.8
, pp. 74-79
-
-
Doyon, Y.1
Vo, T.D.2
Mendel, M.C.3
Greenberg, S.G.4
Wang, J.5
Xia, D.F.6
Miller, J.C.7
Urnov, F.D.8
Gregory, P.D.9
Holmes, M.C.10
-
31
-
-
0035800769
-
Development of zinc finger domains for recognition of the 5 ¢-ANN-3 ¢ family of DNA sequences and their use in the construction of artificial transcription factors
-
Dreier B, Beerli RR, Segal DJ, Flippin JD, Barbas CF 3rd (2001) Development of zinc finger domains for recognition of the 5 ¢-ANN-3 ¢ family of DNA sequences and their use in the construction of artificial transcription factors. J Biol Chem 276:29466-29478
-
(2001)
J Biol Chem
, vol.276
, pp. 29466-29478
-
-
Dreier, B.1
Beerli, R.R.2
Segal, D.J.3
Flippin, J.D.4
Barbas Iii., C.F.5
-
32
-
-
27444444925
-
Development of zinc finger domains for recognition of the 5 ¢-CNN-3 ¢ family DNA sequences and their use in the construction of artificial transcription factors
-
Dreier B, Fuller RP, Segal DJ, Lund CV, Blancafort P, Huber A, Koksch B, Barbas CF 3rd (2005) Development of zinc finger domains for recognition of the 5 ¢-CNN-3 ¢ family DNA sequences and their use in the construction of artificial transcription factors. J Biol Chem 280:35588-35597
-
(2005)
J Biol Chem
, vol.280
, pp. 35588-35597
-
-
Dreier, B.1
Fuller, R.P.2
Segal, D.J.3
Lund, C.V.4
Blancafort, P.5
Huber, A.6
Koksch, B.7
Barbas Iii., C.F.8
-
33
-
-
33744480822
-
A bacterial one-hybrid selection system for interrogating zinc finger-DNA interactions
-
Durai S, Bosley A, Abulencia AB, Chandrasegaran S, Ostermeier M (2006) A bacterial one-hybrid selection system for interrogating zinc finger-DNA interactions. Comb Chem High Throughput Screen 9:301-311
-
(2006)
Comb Chem High Throughput Screen
, vol.9
, pp. 301-311
-
-
Durai, S.1
Bosley, A.2
Abulencia, A.B.3
Chandrasegaran, S.4
Ostermeier, M.5
-
34
-
-
79958695683
-
Efficient immunoglobulin gene disruption and targeted replacement in rabbit using zinc finger nucleases
-
Flisikowska T, Thorey IS, Offner S, Ros F, Lifke V, Zeitler B, Rottmann O, Vincent A, Zhang L, Jenkins S, Niersbach H, Kind AJ, Gregory PD, Schnieke AE, Platzer J (2011) Efficient immunoglobulin gene disruption and targeted replacement in rabbit using zinc finger nucleases. PLoS One 6:e21045
-
(2011)
PLoS One
, vol.6
-
-
Flisikowska, T.1
Thorey, I.S.2
Offner, S.3
Ros, F.4
Lifke, V.5
Zeitler, B.6
Rottmann, O.7
Vincent, A.8
Zhang, L.9
Jenkins, S.10
Niersbach, H.11
Kind, A.J.12
Gregory, P.D.13
Schnieke, A.E.14
Platzer, J.15
-
35
-
-
84861409753
-
Rapid mutation of endogenous zebra fish genes using zinc finger nucleases made by Oligomerized Pool ENgineering (OPEN)
-
Foley JE, Yeh JR, Maeder ML, Reyon D, Sander JD, Peterson RT, Joung JK (2009) Rapid mutation of endogenous zebra fish genes using zinc finger nucleases made by Oligomerized Pool ENgineering (OPEN). PLoS One 4:e4348
-
(2009)
PLoS One
, vol.4
-
-
Foley, J.E.1
Yeh, J.R.2
Maeder, M.L.3
Reyon, D.4
Sander, J.D.5
Peterson, R.T.6
Joung, J.K.7
-
36
-
-
80052766645
-
An unbiased genome-wide analysis of zinc-finger nuclease specificity
-
Gabriel R, Lombardo A, Arens A, Miller JC, Genovese P, Kaeppel C, Nowrouzi A, Bartholomae CC, Wang J, Friedman G, Holmes MC, Gregory PD, Glimm H, Schmidt M, Naldini L, von Kalle C (2011) An unbiased genome-wide analysis of zinc-finger nuclease specificity. Nat Biotechnol 29:816-823
-
(2011)
Nat Biotechnol
, vol.29
, pp. 816-823
-
-
Gabriel, R.1
Lombardo, A.2
Arens, A.3
Miller, J.C.4
Genovese, P.5
Kaeppel, C.6
Nowrouzi, A.7
Bartholomae, C.C.8
Wang, J.9
Friedman, G.10
Holmes, M.C.11
Gregory, P.D.12
Glimm, H.13
Schmidt, M.14
Naldini, L.15
Von Kalle, C.16
-
37
-
-
33748413936
-
Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning
-
Gaspar HB, Bjorkegren E, Parsley K, Gilmour KC, King D, Sinclair J, Zhang F, Giannakopoulos A, Adams S, Fairbanks LD, Gaspar J, Henderson L, Xu-Bayford JH, Davies EG, Veys PA, Kinnon C, Thrasher AJ (2006) Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning. Mol Ther 14:505-513
-
(2006)
Mol Ther
, vol.14
, pp. 505-513
-
-
Gaspar, H.B.1
Bjorkegren, E.2
Parsley, K.3
Gilmour, K.C.4
King, D.5
Sinclair, J.6
Zhang, F.7
Giannakopoulos, A.8
Adams, S.9
Fairbanks, L.D.10
Gaspar, J.11
Henderson, L.12
Xu-Bayford, J.H.13
Davies, E.G.14
Veys, P.A.15
Kinnon, C.16
Thrasher, A.J.17
-
38
-
-
67749106611
-
Knockout rats via embryo microinjection of zinc-finger nucleases
-
Geurts AM, Cost GJ, Freyvert Y, Zeitler B, Miller JC, Choi VM, Jenkins SS, Wood A, Cui X, Meng X, Vincent A, Lam S, Michalkiewicz M, Schilling R, Foeckler J, Kalloway S, Weiler H, Menoret S, Anegon I, Davis GD, Zhang L, Rebar EJ, Gregory PD, Urnov FD, Jacob HJ, Buelow R (2009) Knockout rats via embryo microinjection of zinc-finger nucleases. Science 325:433
-
(2009)
Science
, vol.325
, pp. 433
-
-
Geurts, A.M.1
Cost, G.J.2
Freyvert, Y.3
Zeitler, B.4
Miller, J.C.5
Choi, V.M.6
Jenkins, S.S.7
Wood, A.8
Cui, X.9
Meng, X.10
Vincent, A.11
Lam, S.12
Michalkiewicz, M.13
Schilling, R.14
Foeckler, J.15
Kalloway, S.16
Weiler, H.17
Menoret, S.18
Anegon, I.19
Davis, G.D.20
Zhang, L.21
Rebar, E.J.22
Gregory, P.D.23
Urnov, F.D.24
Jacob, H.J.25
Buelow, R.26
more..
-
39
-
-
70349407359
-
Efficient targeting of a SCID gene by an engineered single-chain homing endonuclease
-
Grizot S, Smith J, Daboussi F, Prieto J, Redondo P, Merino N, Villate M, Thomas S, Lemaire L, Montoya G, Blanco FJ, Paques F, Duchateau P (2009) Efficient targeting of a SCID gene by an engineered single-chain homing endonuclease. Nucleic Acids Res 37:5405-5419
-
(2009)
Nucleic Acids Res
, vol.37
, pp. 5405-5419
-
-
Grizot, S.1
Smith, J.2
Daboussi, F.3
Prieto, J.4
Redondo, P.5
Merino, N.6
Villate, M.7
Thomas, S.8
Lemaire, L.9
Montoya, G.10
Blanco, F.J.11
Paques, F.12
Duchateau, P.13
-
40
-
-
77951234910
-
Generation of redesigned homing endonucleases comprising DNA-binding domains derived from two different scaffolds
-
Grizot S, Epinat JC, Thomas S, Duclert A, Rolland S, Paques F, Duchateau P (2010) Generation of redesigned homing endonucleases comprising DNA-binding domains derived from two different scaffolds. Nucleic Acids Res 38:2006-2018
-
(2010)
Nucleic Acids Res
, vol.38
, pp. 2006-2018
-
-
Grizot, S.1
Epinat, J.C.2
Thomas, S.3
Duclert, A.4
Rolland, S.5
Paques, F.6
Duchateau, P.7
-
41
-
-
0142084745
-
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
-
Hacein-Bey-Abina S, Von Kalle C, Schmidt M, Mccormack MP, Wulffraat N, Leboulch P, Lim A, Osborne CS, Pawliuk R, Morillon E, Sorensen R, Forster A, Fraser P, Cohen JI, De Saint Basile G, Alexander I, Wintergerst U, Frebourg T, Aurias A, Stoppa-Lyonnet D, Romana S, Radford-Weiss I, Gross F, Valensi F, Delabesse E, Macintyre E, Sigaux F, Soulier J, Leiva LE, Wissler M, Prinz C, Rabbitts TH, Le Deist F, Fischer A, Cavazzana-Calvo M (2003) LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302:415-419
-
(2003)
Science
, vol.302
, pp. 415-419
-
-
Hacein-Bey-Abina, S.1
Von Kalle, C.2
Schmidt, M.3
McCormack, M.P.4
Wulffraat, N.5
Leboulch, P.6
Lim, A.7
Osborne, C.S.8
Pawliuk, R.9
Morillon, E.10
Sorensen, R.11
Forster, A.12
Fraser, P.13
Cohen, J.I.14
De Saint Basile, G.15
Alexander, I.16
Wintergerst, U.17
Frebourg, T.18
Aurias, A.19
Stoppa-Lyonnet, D.20
Romana, S.21
Radford-Weiss, I.22
Gross, F.23
Valensi, F.24
Delabesse, E.25
MacIntyre, E.26
Sigaux, F.27
Soulier, J.28
Leiva, L.E.29
Wissler, M.30
Prinz, C.31
Rabbitts, T.H.32
Le Deist, F.33
Fischer, A.34
Cavazzana-Calvo, M.35
more..
-
42
-
-
51349090473
-
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
-
Hacein-Bey-Abina S, Garrigue A, Wang GP, Soulier J, Lim A, Morillon E, Clappier E, Caccavelli L, Delabesse E, Beldjord K, Asna fiV, Macintyre E, Dal Cortivo L, Radford I, Brousse N, Sigaux F, Moshous D, Hauer J, Borkhardt A, Belohradsky BH, Wintergerst U, Velez MC, Leiva L, Sorensen R, Wulffraat N, Blanche S, Bushman FD, Fischer A, Cavazzana-Calvo M (2008) Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest 118:3132-3142
-
(2008)
J Clin Invest
, vol.118
, pp. 3132-3142
-
-
Hacein-Bey-Abina, S.1
Garrigue, A.2
Wang, G.P.3
Soulier, J.4
Lim, A.5
Morillon, E.6
Clappier, E.7
Caccavelli, L.8
Delabesse, E.9
Beldjord, K.10
Asnafi, V.11
MacIntyre, E.12
Dal Cortivo, L.13
Radford, I.14
Brousse, N.15
Sigaux, F.16
Moshous, D.17
Hauer, J.18
Borkhardt, A.19
Belohradsky, B.H.20
Wintergerst, U.21
Velez, M.C.22
Leiva, L.23
Sorensen, R.24
Wulffraat, N.25
Blanche, S.26
Bushman, F.D.27
Fischer, A.28
Cavazzana-Calvo, M.29
more..
-
43
-
-
70249114821
-
Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases
-
Hockemeyer D, Soldner F, Beard C, Gao Q, Mitalipova M, Dekelver RC, Katibah GE, Amora R, Boydston EA, Zeitler B, Meng X, Miller JC, Zhang L, Rebar EJ, Gregory PD, Urnov FD, Jaenisch R (2009) Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases. Nat Biotechnol 27:851-857
-
(2009)
Nat Biotechnol
, vol.27
, pp. 851-857
-
-
Hockemeyer, D.1
Soldner, F.2
Beard, C.3
Gao, Q.4
Mitalipova, M.5
Dekelver, R.C.6
Katibah, G.E.7
Amora, R.8
Boydston, E.A.9
Zeitler, B.10
Meng, X.11
Miller, J.C.12
Zhang, L.13
Rebar, E.J.14
Gregory, P.D.15
Urnov, F.D.16
Jaenisch, R.17
-
44
-
-
78650735673
-
Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo
-
Holt N, Wang J, Kim K, Friedman G, Wang X, Taupin V, Crooks GM, Kohn DB, Gregory PD, Holmes MC, Cannon PM (2010) Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nat Biotechnol 28:839-847
-
(2010)
Nat Biotechnol
, vol.28
, pp. 839-847
-
-
Holt, N.1
Wang, J.2
Kim, K.3
Friedman, G.4
Wang, X.5
Taupin, V.6
Crooks, G.M.7
Kohn, D.B.8
Gregory, P.D.9
Holmes, M.C.10
Cannon, P.M.11
-
45
-
-
60549090253
-
Long-term control of HIV by CCR5 Delta32/Delta32 stem-cell transplantation
-
Hutter G, Nowak D, Mossner M, Ganepola S, Mussig A, Allers K, Schneider T, Hofmann J, Kucherer C, Blau O, Blau IW, Hofmann WK, Thiel E (2009) Long-term control of HIV by CCR5 Delta32/Delta32 stem-cell transplantation. N Engl J Med 360:692-698
-
(2009)
N Engl J Med
, vol.360
, pp. 692-698
-
-
Hutter, G.1
Nowak, D.2
Mossner, M.3
Ganepola, S.4
Mussig, A.5
Allers, K.6
Schneider, T.7
Hofmann, J.8
Kucherer, C.9
Blau, O.10
Blau, I.W.11
Hofmann, W.K.12
Thiel, E.13
-
46
-
-
77949327872
-
Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils
-
Kang EM, Choi U, Theobald N, Linton G, Long Priel DA, Kuhns D, Malech HL (2010) Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils. Blood 115:783-791
-
(2010)
Blood
, vol.115
, pp. 783-791
-
-
Kang, E.M.1
Choi, U.2
Theobald, N.3
Linton, G.4
Long Priel, D.A.5
Kuhns, D.6
Malech, H.L.7
-
47
-
-
77953135742
-
Engineering of human pluripotent stem cells by AAV-mediated gene targeting
-
Khan IF, Hirata RK, Wang PR, Li Y, Kho J, Nelson A, Huo Y, Zavaljevski M, Ware C, Russell DW (2010) Engineering of human pluripotent stem cells by AAV-mediated gene targeting. Mol Ther 18:1192-1199
-
(2010)
Mol Ther
, vol.18
, pp. 1192-1199
-
-
Khan, I.F.1
Hirata, R.K.2
Wang, P.R.3
Li, Y.4
Kho, J.5
Nelson, A.6
Huo, Y.7
Zavaljevski, M.8
Ware, C.9
Russell, D.W.10
-
48
-
-
79960046970
-
AAV-mediated gene targeting methods for human cells
-
Khan IF, Hirata RK, Russell DW (2011) AAV-mediated gene targeting methods for human cells. Nat Protoc 6:482-501
-
(2011)
Nat Protoc
, vol.6
, pp. 482-501
-
-
Khan, I.F.1
Hirata, R.K.2
Russell, D.W.3
-
50
-
-
67650045497
-
Targeted genome editing in human cells with zinc finger nucleases constructed via modular assembly
-
Kim HJ, Lee HJ, Kim H, Cho SW, Kim JS (2009) Targeted genome editing in human cells with zinc finger nucleases constructed via modular assembly. Genome Res 19:1279-1288
-
(2009)
Genome Res
, vol.19
, pp. 1279-1288
-
-
Kim, H.J.1
Lee, H.J.2
Kim, H.3
Cho, S.W.4
Kim, J.S.5
-
51
-
-
74949133880
-
Targeted chromosomal deletions in human cells using zinc finger nucleases
-
Lee HJ, Kim E, Kim JS (2010) Targeted chromosomal deletions in human cells using zinc finger nucleases. Genome Res 20:81-89
-
(2010)
Genome Res
, vol.20
, pp. 81-89
-
-
Lee, H.J.1
Kim, E.2
Kim, J.S.3
-
53
-
-
35948946526
-
Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery
-
Lombardo A, Genovese P, Beausejour CM, Colleoni S, Lee YL, Kim KA, Ando D, Urnov FD, Galli C, Gregory PD, Holmes MC, Naldini L (2007) Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat Biotechnol 25:1298-1306
-
(2007)
Nat Biotechnol
, vol.25
, pp. 1298-1306
-
-
Lombardo, A.1
Genovese, P.2
Beausejour, C.M.3
Colleoni, S.4
Lee, Y.L.5
Kim, K.A.6
Ando, D.7
Urnov, F.D.8
Galli, C.9
Gregory, P.D.10
Holmes, M.C.11
Naldini, L.12
-
54
-
-
47349097567
-
Rapid "open-source" engineering of customized zinc-finger nucleases for highly Efficient gene modification
-
Maeder ML, Thibodeau-Beganny S, Osiak A, Wright DA, Anthony RM, Eichtinger M, Jiang T, Foley JE, Winfrey RJ, Townsend JA, Unger-Wallace E, Sander JD, Muller-Lerch F, Fu F, Pearlberg J, Gobel C, Dassie JP, Pruett-Miller SM, Porteus MH, Sgroi DC, Iafrate AJ, Dobbs D, Mccray PB Jr, Cathomen T, Voytas DF, Joung JK (2008) Rapid "open-source" engineering of customized zinc-finger nucleases for highly Efficient gene modification. Mol Cell 31:294-301
-
(2008)
Mol Cell
, vol.31
, pp. 294-301
-
-
Maeder, M.L.1
Thibodeau-Beganny, S.2
Osiak, A.3
Wright, D.A.4
Anthony, R.M.5
Eichtinger, M.6
Jiang, T.7
Foley, J.E.8
Winfrey, R.J.9
Townsend, J.A.10
Unger-Wallace, E.11
Sander, J.D.12
Muller-Lerch, F.13
Fu, F.14
Pearlberg, J.15
Gobel, C.16
Dassie, J.P.17
Pruett-Miller, S.M.18
Porteus, M.H.19
Sgroi, D.C.20
Iafrate, A.J.21
Dobbs, D.22
Mccray Jr., P.B.23
Cathomen, T.24
Voytas, D.F.25
Joung, J.K.26
more..
-
55
-
-
79952302385
-
De novo-engineered transcription activator-like effector (TALE) hybrid nuclease with novel DNA binding specificity creates double-strand breaks
-
Mahfouz MM, Li L, Shamimuzzaman M, Wibowo A, Fang X, Zhu JK (2011) De novo-engineered transcription activator-like effector (TALE) hybrid nuclease with novel DNA binding specificity creates double-strand breaks. Proc Natl Acad Sci USA 108:2623-2628
-
(2011)
Proc Natl Acad Sci USA
, vol.108
, pp. 2623-2628
-
-
Mahfouz, M.M.1
Li, L.2
Shamimuzzaman, M.3
Wibowo, A.4
Fang, X.5
Zhu, J.K.6
-
56
-
-
12644293807
-
Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease
-
Malech HL, Maples PB, Whiting-Theobald N, Linton GF, Sekhsaria S, Vowells SJ, Li F, Miller JA, Decarlo E, Holland SM, Leitman SF, Carter CS, Butz RE, Read EJ, Fleisher TA, Schneiderman RD, van Epps DE, Spratt SK, Maack CA, Rokovich JA, Cohen LK, Gallin JI (1997) Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease. Proc Natl Acad Sci USA 94:12133-12138
-
(1997)
Proc Natl Acad Sci USA
, vol.94
, pp. 12133-12138
-
-
Malech, H.L.1
Maples, P.B.2
Whiting-Theobald, N.3
Linton, G.F.4
Sekhsaria, S.5
Vowells, S.J.6
Li, F.7
Miller, J.A.8
Decarlo, E.9
Holland, S.M.10
Leitman, S.F.11
Carter, C.S.12
Butz, R.E.13
Read, E.J.14
Fleisher, T.A.15
Schneiderman, R.D.16
Van Epps, D.E.17
Spratt, S.K.18
Maack, C.A.19
Rokovich, J.A.20
Cohen, L.K.21
Gallin, J.I.22
more..
-
57
-
-
77749273530
-
Generation of knockout rats with X-linked severe combined immunodeficiency (X-SCID) using zinc-finger nucleases
-
Mashimo T, Takizawa A, Voigt B, Yoshimi K, Hiai H, Kuramoto T, Serikawa T (2010) Generation of knockout rats with X-linked severe combined immunodeficiency (X-SCID) using zinc-finger nucleases. PLoS One 5:e8870
-
(2010)
PLoS One
, vol.5
-
-
Mashimo, T.1
Takizawa, A.2
Voigt, B.3
Yoshimi, K.4
Hiai, H.5
Kuramoto, T.6
Serikawa, T.7
-
58
-
-
44949162060
-
Targeted gene inactivation in zebra fish using engineered zinc-finger nucleases
-
Meng X, Noyes MB, Zhu LJ, Lawson ND, Wolfe SA (2008) Targeted gene inactivation in zebra fish using engineered zinc-finger nucleases. Nat Biotechnol 26:695-701
-
(2008)
Nat Biotechnol
, vol.26
, pp. 695-701
-
-
Meng, X.1
Noyes, M.B.2
Zhu, L.J.3
Lawson, N.D.4
Wolfe, S.A.5
-
59
-
-
77957005768
-
Gene targeting by homologous recombination in mouse zygotes mediated by zinc-finger nucleases
-
Meyer M, de Angelis MH, Wurst W, Kuhn R (2010) Gene targeting by homologous recombination in mouse zygotes mediated by zinc-finger nucleases. Proc Natl Acad Sci USA 107:15022-15026
-
(2010)
Proc Natl Acad Sci USA
, vol.107
, pp. 15022-15026
-
-
Meyer, M.1
De Angelis, M.H.2
Wurst, W.3
Kuhn, R.4
-
60
-
-
34447319080
-
An improved zinc-finger nuclease architecture for highly specific genome editing
-
Miller JC, Holmes MC, Wang J, Guschin DY, Lee YL, Rupniewski I, Beausejour CM, Waite AJ, Wang NS, Kim KA, Gregory PD, Pabo CO, Rebar EJ (2010) An improved zinc-finger nuclease architecture for highly specific genome editing. Nat Biotechnol 25:778-785
-
(2010)
Nat Biotechnol
, vol.25
, pp. 778-785
-
-
Miller, J.C.1
Holmes, M.C.2
Wang, J.3
Guschin, D.Y.4
Lee, Y.L.5
Rupniewski, I.6
Beausejour, C.M.7
Waite, A.J.8
Wang, N.S.9
Kim, K.A.10
Gregory, P.D.11
Pabo, C.O.12
Rebar, E.J.13
-
61
-
-
67649858822
-
Zinc-finger directed double-strand breaks within CAG repeat tracts promote repeat instability in human cells
-
Mittelman D, Moye C, Morton J, Sykoudis K, Lin Y, Carroll D, Wilson JH (2009) Zinc-finger directed double-strand breaks within CAG repeat tracts promote repeat instability in human cells. Proc Natl Acad Sci USA 106:9607-9612
-
(2009)
Proc Natl Acad Sci USA
, vol.106
, pp. 9607-9612
-
-
Mittelman, D.1
Moye, C.2
Morton, J.3
Sykoudis, K.4
Lin, Y.5
Carroll, D.6
Wilson, J.H.7
-
62
-
-
33847682924
-
Targeted gene addition into a specified location in the human genome using designed zinc finger nucleases
-
Moehle EA, Rock JM, Lee YL, Jouvenot Y, Dekelver RC, Gregory PD, Urnov FD, Holmes MC (2007) Targeted gene addition into a specified location in the human genome using designed zinc finger nucleases. Proc Natl Acad Sci USA 104:3055-3060
-
(2007)
Proc Natl Acad Sci USA
, vol.104
, pp. 3055-3060
-
-
Moehle, E.A.1
Rock, J.M.2
Lee, Y.L.3
Jouvenot, Y.4
Dekelver, R.C.5
Gregory, P.D.6
Urnov, F.D.7
Holmes, M.C.8
-
63
-
-
33750804666
-
Induction and repair of zinc-finger nuclease-targeted double-strand breaks in Caenorhabditis elegans somatic cells
-
Morton J, Davis MW, Jorgensen EM, Carroll D (2006) Induction and repair of zinc-finger nuclease-targeted double-strand breaks in Caenorhabditis elegans somatic cells. Proc Natl Acad Sci USA 103:16370-16375
-
(2006)
Proc Natl Acad Sci USA
, vol.103
, pp. 16370-16375
-
-
Morton, J.1
Davis, M.W.2
Jorgensen, E.M.3
Carroll, D.4
-
64
-
-
72149090954
-
A simple cipher governs DNA recognition by TAL effectors
-
Moscou MJ, Bogdanove AJ (2009) A simple cipher governs DNA recognition by TAL effectors. Science 326:1501
-
(2009)
Science
, vol.326
, pp. 1501
-
-
Moscou, M.J.1
Bogdanove, A.J.2
-
65
-
-
79551491956
-
Molecular basis of engineered meganuclease targeting of the endogenous human RAG1 locus
-
Munoz IG, Prieto J, Subramanian S, Coloma J, Redondo P, Villate M, Merino N, Marenchino M, D'Abramo M, Gervasio FL, Grizot S, Daboussi F, Smith J, Chion-Sotinel I, Paques F, Duchateau P, Alibes A, Stricher F, Serrano L, Blanco FJ, Montoya G (2011) Molecular basis of engineered meganuclease targeting of the endogenous human RAG1 locus. Nucleic Acids Res 39:729-743
-
(2011)
Nucleic Acids Res
, vol.39
, pp. 729-743
-
-
Munoz, I.G.1
Prieto, J.2
Subramanian, S.3
Coloma, J.4
Redondo, P.5
Villate, M.6
Merino, N.7
Marenchino, M.8
D'Abramo, M.9
Gervasio, F.L.10
Grizot, S.11
Daboussi, F.12
Smith, J.13
Chion-Sotinel, I.14
Paques, F.15
Duchateau, P.16
Alibes, A.17
Stricher, F.18
Serrano, L.19
Blanco, F.J.20
Montoya, G.21
more..
-
66
-
-
80053039555
-
A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity
-
Mussolino C, Morbitzer R, Lutge F, Dannemann N, Lahaye T, Cathomen T (2011) A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity. Nucleic Acids Res 39(21):9283-9293
-
(2011)
Nucleic Acids Res
, vol.39
, Issue.21
, pp. 9283-9293
-
-
Mussolino, C.1
Morbitzer, R.2
Lutge, F.3
Dannemann, N.4
Lahaye, T.5
Cathomen, T.6
-
67
-
-
77954738991
-
Targeted mutagenesis in the sea urchin embryo using zinc-finger nucleases
-
Ochiai H, Fujita K, Suzuki K, Nishikawa M, Shibata T, Sakamoto N, Yamamoto T (2010) Targeted mutagenesis in the sea urchin embryo using zinc-finger nucleases. Genes Cells 15:875-885
-
(2010)
Genes Cells
, vol.15
, pp. 875-885
-
-
Ochiai, H.1
Fujita, K.2
Suzuki, K.3
Nishikawa, M.4
Shibata, T.5
Sakamoto, N.6
Yamamoto, T.7
-
68
-
-
33645734405
-
Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1
-
Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U, Glimm H, Kuhlcke K, Schilz A, Kunkel H, Naundorf S, Brinkmann A, Deichmann A, Fischer M, Ball C, Pilz I, Dunbar C, Du Y, Jenkins NA, Copeland NG, Luthi U, Hassan M, Thrasher AJ, Hoelzer D, von Kalle C, Seger R, Grez M (2006) Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med 12:401-409
-
(2006)
Nat Med
, vol.12
, pp. 401-409
-
-
Ott, M.G.1
Schmidt, M.2
Schwarzwaelder, K.3
Stein, S.4
Siler, U.5
Koehl, U.6
Glimm, H.7
Kuhlcke, K.8
Schilz, A.9
Kunkel, H.10
Naundorf, S.11
Brinkmann, A.12
Deichmann, A.13
Fischer, M.14
Ball, C.15
Pilz, I.16
Dunbar, C.17
Du, Y.18
Jenkins, N.A.19
Copeland, N.G.20
Luthi, U.21
Hassan, M.22
Thrasher, A.J.23
Hoelzer, D.24
Von Kalle, C.25
Seger, R.26
Grez, M.27
more..
-
69
-
-
78651337233
-
Genomic safe harbors permit high beta-globin transgene expression in thalassemia induced pluripotent stem cells
-
Papapetrou EP, Lee G, Malani N, Setty M, Riviere I, Tirunagari LM, Kadota K, Roth SL, Giardina P, Viale A, Leslie C, Bushman FD, Studer L, Sadelain M (2011) Genomic safe harbors permit high beta-globin transgene expression in thalassemia induced pluripotent stem cells. Nat Biotechnol 29:73-78
-
(2011)
Nat Biotechnol
, vol.29
, pp. 73-78
-
-
Papapetrou, E.P.1
Lee, G.2
Malani, N.3
Setty, M.4
Riviere, I.5
Tirunagari, L.M.6
Kadota, K.7
Roth, S.L.8
Giardina, P.9
Viale, A.10
Leslie, C.11
Bushman, F.D.12
Studer, L.13
Sadelain, M.14
-
70
-
-
33847246298
-
Meganucleases and DNA double-strand break-induced recombination: Perspectives for gene therapy
-
Paques F, Duchateau P (2007) Meganucleases and DNA double-strand break-induced recombination: perspectives for gene therapy. Curr Gene Ther 7:49-66
-
(2007)
Curr Gene Ther
, vol.7
, pp. 49-66
-
-
Paques, F.1
Duchateau, P.2
-
71
-
-
80052293623
-
Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection
-
Pattanayak V, Ramirez CL, Joung JK, Liu DR (2011) Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection. Nat Methods 8:765-770
-
(2011)
Nat Methods
, vol.8
, pp. 765-770
-
-
Pattanayak, V.1
Ramirez, C.L.2
Joung, J.K.3
Liu, D.R.4
-
72
-
-
46949095221
-
Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases
-
Perez EE, Wang J, Miller JC, Jouvenot Y, Kim KA, Liu O, Wang N, Lee G, Bartsevich VV, Lee YL, Guschin DY, Rupniewski I, Waite AJ, Carpenito C, Carroll RG, Orange JS, Urnov FD, Rebar EJ, Ando D, Gregory PD, Riley JL, Holmes MC, June CH (2008) Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases. Nat Biotechnol 26:808-816
-
(2008)
Nat Biotechnol
, vol.26
, pp. 808-816
-
-
Perez, E.E.1
Wang, J.2
Miller, J.C.3
Jouvenot, Y.4
Kim, K.A.5
Liu, O.6
Wang, N.7
Lee, G.8
Bartsevich, V.V.9
Lee, Y.L.10
Guschin, D.Y.11
Rupniewski, I.12
Waite, A.J.13
Carpenito, C.14
Carroll, R.G.15
Orange, J.S.16
Urnov, F.D.17
Rebar, E.J.18
Ando, D.19
Gregory, P.D.20
Riley, J.L.21
Holmes, M.C.22
June, C.H.23
more..
-
73
-
-
30344473645
-
Mammalian gene targeting with designed zinc finger nucleases
-
Porteus MH (2006) Mammalian gene targeting with designed zinc finger nucleases. Mol Ther 13:438-446
-
(2006)
Mol Ther
, vol.13
, pp. 438-446
-
-
Porteus, M.H.1
-
74
-
-
0038523969
-
Chimeric nucleases stimulate gene targeting in human cells
-
Porteus MH, Baltimore D (2003) Chimeric nucleases stimulate gene targeting in human cells. Science 300:763
-
(2003)
Science
, vol.300
, pp. 763
-
-
Porteus, M.H.1
Baltimore, D.2
-
75
-
-
33746515537
-
Human SMC5/6 complex promotes sister chromatid homologous recombination by recruiting the SMC1/3 cohesin complex to double-strand breaks
-
Potts PR, Porteus MH, Yu H (2006) Human SMC5/6 complex promotes sister chromatid homologous recombination by recruiting the SMC1/3 cohesin complex to double-strand breaks. EMBO J 25:3377-3388
-
(2006)
EMBO J
, vol.25
, pp. 3377-3388
-
-
Potts, P.R.1
Porteus, M.H.2
Yu, H.3
-
76
-
-
41149127259
-
Comparison of zinc finger nucleases for use in gene targeting in mammalian cells
-
Pruett-Miller SM, Connelly JP, Maeder ML, Joung JK, Porteus MH (2008) Comparison of zinc finger nucleases for use in gene targeting in mammalian cells. Mol Ther 16:707-717
-
(2008)
Mol Ther
, vol.16
, pp. 707-717
-
-
Pruett-Miller, S.M.1
Connelly, J.P.2
Maeder, M.L.3
Joung, J.K.4
Porteus, M.H.5
-
77
-
-
61449266636
-
Attenuation of zinc finger nuclease toxicity by small-molecule regulation of protein levels
-
Pruett-Miller SM, Reading DW, Porter SN, Porteus MH (2009) Attenuation of zinc finger nuclease toxicity by small-molecule regulation of protein levels. PLoS Genet 5:e1000376
-
(2009)
PLoS Genet
, vol.5
-
-
Pruett-Miller, S.M.1
Reading, D.W.2
Porter, S.N.3
Porteus, M.H.4
-
78
-
-
42949083192
-
Unexpected failure rates for modular assembly of engineered zinc fingers
-
Ramirez CL, Foley JE, Wright DA, Muller-Lerch F, Rahman SH, Cornu TI, Winfrey RJ, Sander JD, Fu F, Townsend JA, Cathomen T, Voytas DF, Joung JK (2008) Unexpected failure rates for modular assembly of engineered zinc fingers. Nat Methods 5:374-375
-
(2008)
Nat Methods
, vol.5
, pp. 374-375
-
-
Ramirez, C.L.1
Foley, J.E.2
Wright, D.A.3
Muller-Lerch, F.4
Rahman, S.H.5
Cornu, T.I.6
Winfrey, R.J.7
Sander, J.D.8
Fu, F.9
Townsend, J.A.10
Cathomen, T.11
Voytas, D.F.12
Joung, J.K.13
-
79
-
-
0029942895
-
Phage display methods for selecting zinc finger proteins with novel DNA-binding specificities
-
Rebar EJ, Greisman HA, Pabo CO (1996) Phage display methods for selecting zinc finger proteins with novel DNA-binding specificities. Methods Enzymol 267:129-149
-
(1996)
Methods Enzymol
, vol.267
, pp. 129-149
-
-
Rebar, E.J.1
Greisman, H.A.2
Pabo, C.O.3
-
80
-
-
84860747716
-
FLASH assembly of TALENs for high-throughput genome editing
-
Apr 8. doi: 10.1038/nbt.2170
-
Reyon D, Tsai SQ, Khayter C, Foden JA, Sander JD, Joung JK (2012) FLASH assembly of TALENs for high-throughput genome editing. Nat Biotechnol. Apr 8. doi: 10.1038/nbt.2170
-
(2012)
Nat Biotechnol
-
-
Reyon, D.1
Tsai, S.Q.2
Khayter, C.3
Foden, J.A.4
Sander, J.D.5
Joung, J.K.6
-
81
-
-
0028061666
-
Introduction of double-strand breaks into the genome of mouse cells by expression of a rare-cutting endonuclease
-
Rouet P, Smih F, Jasin M (1994) Introduction of double-strand breaks into the genome of mouse cells by expression of a rare-cutting endonuclease. Mol Cell Biol 14:8096-8106
-
(1994)
Mol Cell Biol
, vol.14
, pp. 8096-8106
-
-
Rouet, P.1
Smih, F.2
Jasin, M.3
-
82
-
-
78650863981
-
Selection-free zinc-finger-nuclease engineering by context-dependent assembly (CoDA)
-
Sander JD, Dahlborg EJ, Goodwin MJ, Cade L, Zhang F, Cifuentes D, Curtin SJ, Blackburn JS, Thibodeau-Beganny S, Qi Y, Pierick CJ, Hoffman E, Maeder ML, Khayter C, Reyon D, Dobbs D, Langenau DM, Stupar RM, Giraldez AJ, Voytas DF, Peterson RT, Yeh JR, Joung JK (2011) Selection-free zinc-finger-nuclease engineering by context-dependent assembly (CoDA). Nat Methods 8:67-69
-
(2011)
Nat Methods
, vol.8
, pp. 67-69
-
-
Sander, J.D.1
Dahlborg, E.J.2
Goodwin, M.J.3
Cade, L.4
Zhang, F.5
Cifuentes, D.6
Curtin, S.J.7
Blackburn, J.S.8
Thibodeau-Beganny, S.9
Qi, Y.10
Pierick, C.J.11
Hoffman, E.12
Maeder, M.L.13
Khayter, C.14
Reyon, D.15
Dobbs, D.16
Langenau, D.M.17
Stupar, R.M.18
Giraldez, A.J.19
Voytas, D.F.20
Peterson, R.T.21
Yeh, J.R.22
Joung, J.K.23
more..
-
83
-
-
84861170955
-
A transcription activator-like effector toolbox for genome engineering
-
Jan 5 doi: 10.1038/nprot.2011.431
-
Sanjana NE, Cong L, Zhou Y, Cunniff MM, Feng G, Zhang F (2012) A transcription activator-like effector toolbox for genome engineering. Nat Protoc. Jan 5;7(1):171-92. doi: 10.1038/nprot.2011.431
-
(2012)
Nat Protoc
, vol.7
, Issue.1
, pp. 171-192
-
-
Sanjana, N.E.1
Cong, L.2
Zhou, Y.3
Cunniff, M.M.4
Feng, G.5
Zhang, F.6
-
84
-
-
33845914020
-
A combinatorial approach to create artificial homing endonucleases cleaving chosen sequences
-
Smith J, Grizot S, Arnould S, Duclert A, Epinat JC, Chames P, Prieto J, Redondo P, Blanco FJ, Bravo J, Montoya G, Paques F, Duchateau P (2006) A combinatorial approach to create artificial homing endonucleases cleaving chosen sequences. Nucleic Acids Res 34:e149
-
(2006)
Nucleic Acids Res
, vol.34
-
-
Smith, J.1
Grizot, S.2
Arnould, S.3
Duclert, A.4
Epinat, J.C.5
Chames, P.6
Prieto, J.7
Redondo, P.8
Blanco, F.J.9
Bravo, J.10
Montoya, G.11
Paques, F.12
Duchateau, P.13
-
85
-
-
77957755672
-
Autonomous zinc-finger nuclease pairs for targeted chromosomal deletion
-
Sollu C, Pars K, Cornu TI, Thibodeau-Beganny S, Maeder ML, Joung JK, Heilbronn R, Cathomen T (2010) Autonomous zinc-finger nuclease pairs for targeted chromosomal deletion. Nucleic Acids Res 38:8269-8276
-
(2010)
Nucleic Acids Res
, vol.38
, pp. 8269-8276
-
-
Sollu, C.1
Pars, K.2
Cornu, T.I.3
Thibodeau-Beganny, S.4
Maeder, M.L.5
Joung, J.K.6
Heilbronn, R.7
Cathomen, T.8
-
86
-
-
34447323568
-
Structure-based redesign of the dimerization interface reduces the toxicity of zinc-finger nucleases
-
Szczepek M, Brondani V, Buchel J, Serrano L, Segal DJ, Cathomen T (2007) Structure-based redesign of the dimerization interface reduces the toxicity of zinc-finger nucleases. Nat Biotechnol 25:786-793
-
(2007)
Nat Biotechnol
, vol.25
, pp. 786-793
-
-
Szczepek, M.1
Brondani, V.2
Buchel, J.3
Serrano, L.4
Segal, D.J.5
Cathomen, T.6
-
87
-
-
77956988605
-
Targeted mutagenesis in the silkworm Bombyx mori using zinc finger nuclease mRNA injection
-
Takasu Y, Kobayashi I, Beumer K, Uchino K, Sezutsu H, Sajwan S, Carroll D, Tamura T, Zurovec M (2010) Targeted mutagenesis in the silkworm Bombyx mori using zinc finger nuclease mRNA injection. Insect Biochem Mol Biol 40:759-765
-
(2010)
Insect Biochem Mol Biol
, vol.40
, pp. 759-765
-
-
Takasu, Y.1
Kobayashi, I.2
Beumer, K.3
Uchino, K.4
Sezutsu, H.5
Sajwan, S.6
Carroll, D.7
Tamura, T.8
Zurovec, M.9
-
88
-
-
18944373328
-
Highly Efficient endogenous human gene correction using designed zinc-finger nucleases
-
Urnov FD, Miller JC, Lee YL, Beausejour CM, Rock JM, Augustus S, Jamieson AC, Porteus MH, Gregory PD, Holmes MC (2005) Highly Efficient endogenous human gene correction using designed zinc-finger nucleases. Nature 435:646-651
-
(2005)
Nature
, vol.435
, pp. 646-651
-
-
Urnov, F.D.1
Miller, J.C.2
Lee, Y.L.3
Beausejour, C.M.4
Rock, J.M.5
Augustus, S.6
Jamieson, A.C.7
Porteus, M.H.8
Gregory, P.D.9
Holmes, M.C.10
-
89
-
-
33644693285
-
High-frequency homologous recombination in plants mediated by zincfinger nucleases
-
Wright DA, Townsend JA, Winfrey RJ Jr, Irwin PA, Rajagopal J, Lonosky PM, Hall BD, Jondle MD, Voytas DF (2005) High-frequency homologous recombination in plants mediated by zincfinger nucleases. Plant J 44:693-705
-
(2005)
Plant J
, vol.44
, pp. 693-705
-
-
Wright, D.A.1
Townsend, J.A.2
Winfrey Jr., R.J.3
Irwin, P.A.4
Rajagopal, J.5
Lonosky, P.M.6
Hall, B.D.7
Jondle, M.D.8
Voytas, D.F.9
-
90
-
-
79955569746
-
Efficient targeted gene disruption in the soma and germ line of the frog Xenopus tropicalis using engineered zinc-finger nucleases
-
Young JJ, Cherone JM, Doyon Y, Ankoudinova I, Faraji FM, Lee AH, Ngo C, Guschin DY, Paschon DE, Miller JC, Zhang L, Rebar EJ, Gregory PD, Urnov FD, Harland RM, Zeitler B (2011) Efficient targeted gene disruption in the soma and germ line of the frog Xenopus tropicalis using engineered zinc-finger nucleases. Proc Natl Acad Sci USA 108:7052-7057
-
(2011)
Proc Natl Acad Sci USA
, vol.108
, pp. 7052-7057
-
-
Young, J.J.1
Cherone, J.M.2
Doyon, Y.3
Ankoudinova, I.4
Faraji, F.M.5
Lee, A.H.6
Ngo, C.7
Guschin, D.Y.8
Paschon, D.E.9
Miller, J.C.10
Zhang, L.11
Rebar, E.J.12
Gregory, P.D.13
Urnov, F.D.14
Harland, R.M.15
Zeitler, B.16
-
91
-
-
67649170378
-
Gene targeting of a disease-related gene in human induced pluripotent stem and embryonic stem cells
-
Zou J, Maeder ML, Mali P, Pruett-Miller SM, Thibodeau-Beganny S, Chou BK, Chen G, Ye Z, Park IH, Daley GQ, Porteus MH, Joung JK, Cheng L (2009) Gene targeting of a disease-related gene in human induced pluripotent stem and embryonic stem cells. Cell Stem Cell 5:97-110
-
(2009)
Cell Stem Cell
, vol.5
, pp. 97-110
-
-
Zou, J.1
Maeder, M.L.2
Mali, P.3
Pruett-Miller, S.M.4
Thibodeau-Beganny, S.5
Chou, B.K.6
Chen, G.7
Ye, Z.8
Park, I.H.9
Daley, G.Q.10
Porteus, M.H.11
Joung, J.K.12
Cheng, L.13
-
92
-
-
79957587075
-
Oxidase-de ficient neutrophils from X-linked chronic granulomatous disease iPS cells: Functional correction by zinc finger nuclease-mediated safe harbor targeting
-
Zou J, Sweeney CL, Chou BK, Choi U, Pan J, Wang H, Dowey SN, Cheng L, Malech HL (2011) Oxidase-de ficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease-mediated safe harbor targeting. Blood 117:5561-5572
-
(2011)
Blood
, vol.117
, pp. 5561-5572
-
-
Zou, J.1
Sweeney, C.L.2
Chou, B.K.3
Choi, U.4
Pan, J.5
Wang, H.6
Dowey, S.N.7
Cheng, L.8
Malech, H.L.9
|