-
1
-
-
84857097177
-
RNA-guided genetic silencing systems in bacteria and archaea
-
Wiedenheft B, Sternberg SH, Doudna JA (2012) RNA-guided genetic silencing systems in bacteria and archaea. Nature 482(7385):331-338.
-
(2012)
Nature
, vol.482
, Issue.7385
, pp. 331-338
-
-
Wiedenheft, B.1
Sternberg, S.H.2
Doudna, J.A.3
-
2
-
-
80755187812
-
CRISPR-Cas systems in bacteria and archaea: Versatile small RNAs for adaptive defense and regulation
-
Bhaya D, Davison M, Barrangou R (2011) CRISPR-Cas systems in bacteria and archaea: Versatile small RNAs for adaptive defense and regulation. Annu Rev Genet 45:273-297.
-
(2011)
Annu Rev Genet
, vol.45
, pp. 273-297
-
-
Bhaya, D.1
Davison, M.2
Barrangou, R.3
-
3
-
-
79959319829
-
CRISPR-based adaptive immune systems
-
Terns MP, Terns RM (2011) CRISPR-based adaptive immune systems. Curr Opin Microbiol 14(3):321-327.
-
(2011)
Curr Opin Microbiol
, vol.14
, Issue.3
, pp. 321-327
-
-
Terns, M.P.1
Terns, R.M.2
-
4
-
-
79956157571
-
Evolution and classification of the CRISPR-Cas systems
-
Makarova KS, et al. (2011) Evolution and classification of the CRISPR-Cas systems. Nat Rev Microbiol 9(6):467-477.
-
(2011)
Nat Rev Microbiol
, vol.9
, Issue.6
, pp. 467-477
-
-
Makarova, K.S.1
-
5
-
-
34047118522
-
CRISPR provides acquired resistance against viruses in prokaryotes
-
Barrangou R, et al. (2007) CRISPR provides acquired resistance against viruses in prokaryotes. Science 315(5819):1709-1712.
-
(2007)
Science
, vol.315
, Issue.5819
, pp. 1709-1712
-
-
Barrangou, R.1
-
6
-
-
84865070369
-
A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity
-
Jinek M, et al. (2012) A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science 337(6096):816-821.
-
(2012)
Science
, vol.337
, Issue.6096
, pp. 816-821
-
-
Jinek, M.1
-
7
-
-
84873729095
-
Multiplex genome engineering using CRISPR/Cas systems
-
Cong L, et al. (2013) Multiplex genome engineering using CRISPR/Cas systems. Science 339(6121):819-823.
-
(2013)
Science
, vol.339
, Issue.6121
, pp. 819-823
-
-
Cong, L.1
-
8
-
-
84873734105
-
RNA-guided human genome engineering via Cas9
-
Mali P, et al. (2013) RNA-guided human genome engineering via Cas9. Science 339(6121):823-826.
-
(2013)
Science
, vol.339
, Issue.6121
, pp. 823-826
-
-
Mali, P.1
-
9
-
-
84902185906
-
Genome engineering with targetable nucleases
-
Carroll D (2014) Genome engineering with targetable nucleases. Annu Rev Biochem 83:409-439.
-
(2014)
Annu Rev Biochem
, vol.83
, pp. 409-439
-
-
Carroll, D.1
-
10
-
-
84856430123
-
Gene editing: Not just for translation anymore
-
McMahon MA, Rahdar M, Porteus M (2012) Gene editing: Not just for translation anymore. Nat Methods 9(1):28-31.
-
(2012)
Nat Methods
, vol.9
, Issue.1
, pp. 28-31
-
-
McMahon, M.A.1
Rahdar, M.2
Porteus, M.3
-
11
-
-
84902096048
-
Development and applications of CRISPR-Cas9 for genome engineering
-
Hsu PD, Lander ES, Zhang F (2014) Development and applications of CRISPR-Cas9 for genome engineering. Cell 157(6):1262-1278.
-
(2014)
Cell
, vol.157
, Issue.6
, pp. 1262-1278
-
-
Hsu, P.D.1
Lander, E.S.2
Zhang, F.3
-
12
-
-
84929666410
-
Expanding the Biologist's Toolkit with CRISPR-Cas9
-
Sternberg SH, Doudna JA (2015) Expanding the Biologist's Toolkit with CRISPR-Cas9. Mol Cell 58(4):568-574.
-
(2015)
Mol Cell
, vol.58
, Issue.4
, pp. 568-574
-
-
Sternberg, S.H.1
Doudna, J.A.2
-
13
-
-
84914689918
-
Xylose phosphorylation functions as a molecular switch to regulate proteoglycan biosynthesis
-
Wen J, et al. (2014) Xylose phosphorylation functions as a molecular switch to regulate proteoglycan biosynthesis. Proc Natl Acad Sci USA 111(44):15723-15728.
-
(2014)
Proc Natl Acad Sci USA
, vol.111
, Issue.44
, pp. 15723-15728
-
-
Wen, J.1
-
14
-
-
84928797239
-
DNA sequence-specific binding of CENP-B enhances the fidelity of human centromere function
-
Fachinetti D, et al. (2015) DNA sequence-specific binding of CENP-B enhances the fidelity of human centromere function. Dev Cell 33(3):314-327.
-
(2015)
Dev Cell
, vol.33
, Issue.3
, pp. 314-327
-
-
Fachinetti, D.1
-
15
-
-
84877707375
-
One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering
-
Wang H, et al. (2013) One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering. Cell 153(4):910-918.
-
(2013)
Cell
, vol.153
, Issue.4
, pp. 910-918
-
-
Wang, H.1
-
16
-
-
84883819602
-
Heritable gene targeting in the mouse and rat using a CRISPR-Cas system
-
Li D, et al. (2013) Heritable gene targeting in the mouse and rat using a CRISPR-Cas system. Nat Biotechnol 31(8):681-683.
-
(2013)
Nat Biotechnol
, vol.31
, Issue.8
, pp. 681-683
-
-
Li, D.1
-
17
-
-
84927514894
-
In vivo genome editing using Staphylococcus aureus Cas9
-
Ran FA, et al. (2015) In vivo genome editing using Staphylococcus aureus Cas9. Nature 520(7546):186-191.
-
(2015)
Nature
, vol.520
, Issue.7546
, pp. 186-191
-
-
Ran, F.A.1
-
18
-
-
84912101598
-
CRISPR-Cas9 knockin mice for genome editing and cancer modeling
-
Platt RJ, et al. (2014) CRISPR-Cas9 knockin mice for genome editing and cancer modeling. Cell 159(2):440-455.
-
(2014)
Cell
, vol.159
, Issue.2
, pp. 440-455
-
-
Platt, R.J.1
-
19
-
-
84925008880
-
Genome-wide CRISPR screen in a mouse model of tumor growth and metastasis
-
Chen S, et al. (2015) Genome-wide CRISPR screen in a mouse model of tumor growth and metastasis. Cell 160(6):1246-1260.
-
(2015)
Cell
, vol.160
, Issue.6
, pp. 1246-1260
-
-
Chen, S.1
-
20
-
-
84923106217
-
Therapeutic genome editing: Prospects and challenges
-
Cox DBT, Platt RJ, Zhang F (2015) Therapeutic genome editing: Prospects and challenges. Nat Med 21(2):121-131.
-
(2015)
Nat Med
, vol.21
, Issue.2
, pp. 121-131
-
-
Cox, D.B.T.1
Platt, R.J.2
Zhang, F.3
-
21
-
-
84928775846
-
Biotechnology. A prudent path forward for genomic engineering and germline gene modification
-
Baltimore D, et al. (2015) Biotechnology. A prudent path forward for genomic engineering and germline gene modification. Science 348(6230):36-38.
-
(2015)
Science
, vol.348
, Issue.6230
, pp. 36-38
-
-
Baltimore, D.1
-
22
-
-
84937905397
-
Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells
-
Hendel A, et al. (2015) Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells. Nat Biotechnol 33(9):985-989.
-
(2015)
Nat Biotechnol
, vol.33
, Issue.9
, pp. 985-989
-
-
Hendel, A.1
-
23
-
-
84880570576
-
High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells
-
Fu Y, et al. (2013) High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells. Nat Biotechnol 31(9):822-826.
-
(2013)
Nat Biotechnol
, vol.31
, Issue.9
, pp. 822-826
-
-
Fu, Y.1
-
24
-
-
84884165315
-
DNA targeting specificity of RNA-guided Cas9 nucleases
-
Hsu PD, et al. (2013) DNA targeting specificity of RNA-guided Cas9 nucleases. Nat Biotechnol 31(9):827-832.
-
(2013)
Nat Biotechnol
, vol.31
, Issue.9
, pp. 827-832
-
-
Hsu, P.D.1
-
25
-
-
84884155038
-
High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity
-
Pattanayak V, et al. (2013) High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity. Nat Biotechnol 31(9):839-843.
-
(2013)
Nat Biotechnol
, vol.31
, Issue.9
, pp. 839-843
-
-
Pattanayak, V.1
-
26
-
-
84903545084
-
Genome-wide analysis reveals characteristics of off-target sites bound by the Cas9 endonuclease
-
Kuscu C, Arslan S, Singh R, Thorpe J, Adli M (2014) Genome-wide analysis reveals characteristics of off-target sites bound by the Cas9 endonuclease. Nat Biotechnol 32(7):677-683.
-
(2014)
Nat Biotechnol
, vol.32
, Issue.7
, pp. 677-683
-
-
Kuscu, C.1
Arslan, S.2
Singh, R.3
Thorpe, J.4
Adli, M.5
-
27
-
-
84923266604
-
GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases
-
Tsai SQ, et al. (2015) GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases. Nat Biotechnol 33(2):187-197.
-
(2015)
Nat Biotechnol
, vol.33
, Issue.2
, pp. 187-197
-
-
Tsai, S.Q.1
-
28
-
-
84983792922
-
Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery
-
Lin S, Staahl BT, Alla RK, Doudna JA (2014) Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery. eLife 3:e04766.
-
(2014)
ELife
, vol.3
, pp. e04766
-
-
Lin, S.1
Staahl, B.T.2
Alla, R.K.3
Doudna, J.A.4
-
29
-
-
0038385504
-
RNA interference in mammalian cells by chemically-modified RNA
-
Braasch DA, et al. (2003) RNA interference in mammalian cells by chemically-modified RNA. Biochemistry 42(26):7967-7975.
-
(2003)
Biochemistry
, vol.42
, Issue.26
, pp. 7967-7975
-
-
Braasch, D.A.1
-
30
-
-
0041331496
-
SiRNA function in RNAi: A chemical modification analysis
-
Chiu Y-L, Rana TM (2003) siRNA function in RNAi: A chemical modification analysis. RNA 9(9):1034-1048.
-
(2003)
RNA
, vol.9
, Issue.9
, pp. 1034-1048
-
-
Chiu, Y.-L.1
Rana, T.M.2
-
31
-
-
84915763945
-
Phosphorothioates, essential components of therapeutic oligonucleotides
-
Eckstein F (2014) Phosphorothioates, essential components of therapeutic oligonucleotides. Nucleic Acid Ther 24(6):374-387.
-
(2014)
Nucleic Acid Ther
, vol.24
, Issue.6
, pp. 374-387
-
-
Eckstein, F.1
-
32
-
-
1842579613
-
Mutation detection using Surveyor nuclease
-
Qiu P, et al. (2004) Mutation detection using Surveyor nuclease. Biotechniques 36(4):702-707.
-
(2004)
Biotechniques
, vol.36
, Issue.4
, pp. 702-707
-
-
Qiu, P.1
-
33
-
-
77949512140
-
RNA targeting therapeutics: Molecular mechanisms of antisense oligonucleotides as a therapeutic platform
-
Bennett CF, Swayze EE (2010) RNA targeting therapeutics: Molecular mechanisms of antisense oligonucleotides as a therapeutic platform. Annu Rev Pharmacol Toxicol 50:259-293.
-
(2010)
Annu Rev Pharmacol Toxicol
, vol.50
, pp. 259-293
-
-
Bennett, C.F.1
Swayze, E.E.2
-
34
-
-
77949297395
-
Synthesis and biophysical evaluation of 2', 4'-constrained 2'O-methoxyethyl and 2', 4'-constrained 2'O-ethyl nucleic acid analogues
-
Seth PP, et al. (2010) Synthesis and biophysical evaluation of 2', 4'-constrained 2'O-methoxyethyl and 2', 4'-constrained 2'O-ethyl nucleic acid analogues. J Org Chem 75(5):1569-1581.
-
(2010)
J Org Chem
, vol.75
, Issue.5
, pp. 1569-1581
-
-
Seth, P.P.1
-
35
-
-
84933574487
-
STRUCTURAL BIOLOGY. A Cas9-guide RNA complex preorganized for target DNA recognition
-
Jiang F, Zhou K, Ma L, Gressel S, Doudna JA (2015) STRUCTURAL BIOLOGY. A Cas9-guide RNA complex preorganized for target DNA recognition. Science 348(6242):1477-1481.
-
(2015)
Science
, vol.348
, Issue.6242
, pp. 1477-1481
-
-
Jiang, F.1
Zhou, K.2
Ma, L.3
Gressel, S.4
Doudna, J.A.5
-
36
-
-
84896929630
-
Improving CRISPR-Cas nuclease specificity using truncated guide RNAs
-
Fu Y, Sander JD, Reyon D, Cascio VM, Joung JK (2014) Improving CRISPR-Cas nuclease specificity using truncated guide RNAs. Nat Biotechnol 32(3):279-284.
-
(2014)
Nat Biotechnol
, vol.32
, Issue.3
, pp. 279-284
-
-
Fu, Y.1
Sander, J.D.2
Reyon, D.3
Cascio, V.M.4
Joung, J.K.5
-
37
-
-
0346003765
-
Solidphase chemical synthesis of phosphonoacetate and thiophosphonoacetate oligodeoxynucleotides
-
Dellinger DJ, Sheehan DM, Christensen NK, Lindberg JG, Caruthers MH (2003) Solidphase chemical synthesis of phosphonoacetate and thiophosphonoacetate oligodeoxynucleotides. J Am Chem Soc 125(4):940-950.
-
(2003)
J Am Chem Soc
, vol.125
, Issue.4
, pp. 940-950
-
-
Dellinger, D.J.1
Sheehan, D.M.2
Christensen, N.K.3
Lindberg, J.G.4
Caruthers, M.H.5
-
38
-
-
9144247189
-
Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs
-
Soutschek J, et al. (2004) Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature 432(7014):173-178.
-
(2004)
Nature
, vol.432
, Issue.7014
, pp. 173-178
-
-
Soutschek, J.1
-
39
-
-
33746667851
-
Antisense oligonucleotide therapy for neurodegenerative disease
-
Smith RA, et al. (2006) Antisense oligonucleotide therapy for neurodegenerative disease. J Clin Invest 116(8):2290-2296.
-
(2006)
J Clin Invest
, vol.116
, Issue.8
, pp. 2290-2296
-
-
Smith, R.A.1
-
40
-
-
79952348568
-
Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy
-
Passini MA, et al. (2011) Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy. Sci Transl Med 3(72):72ra18.
-
(2011)
Sci Transl Med
, vol.3
, Issue.72
, pp. 72ra18
-
-
Passini, M.A.1
-
41
-
-
84865688581
-
Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression
-
Yu D, et al. (2012) Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression. Cell 150(5):895-908.
-
(2012)
Cell
, vol.150
, Issue.5
, pp. 895-908
-
-
Yu, D.1
-
42
-
-
79959479529
-
Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes
-
Koller E, et al. (2011) Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes. Nucleic Acids Res 39(11):4795-4807.
-
(2011)
Nucleic Acids Res
, vol.39
, Issue.11
, pp. 4795-4807
-
-
Koller, E.1
-
43
-
-
84856389509
-
RNA-based therapeutics: Current progress and future prospects
-
Burnett JC, Rossi JJ (2012) RNA-based therapeutics: Current progress and future prospects. Chem Biol 19(1):60-71.
-
(2012)
Chem Biol
, vol.19
, Issue.1
, pp. 60-71
-
-
Burnett, J.C.1
Rossi, J.J.2
-
44
-
-
11144282529
-
Antisense and siRNA as agonists of Toll-like receptors
-
Agrawal S, Kandimalla ER (2004) Antisense and siRNA as agonists of Toll-like receptors. Nat Biotechnol 22(12):1533-1537.
-
(2004)
Nat Biotechnol
, vol.22
, Issue.12
, pp. 1533-1537
-
-
Agrawal, S.1
Kandimalla, E.R.2
-
45
-
-
44349170706
-
Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection
-
Zincarelli C, Soltys S, Rengo G, Rabinowitz JE (2008) Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. Mol Ther 16(6):1073-1080.
-
(2008)
Mol Ther
, vol.16
, Issue.6
, pp. 1073-1080
-
-
Zincarelli, C.1
Soltys, S.2
Rengo, G.3
Rabinowitz, J.E.4
-
46
-
-
79954622209
-
Therapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges
-
Mingozzi F, High KA (2011) Therapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges. Nat Rev Genet 12(5):341-355.
-
(2011)
Nat Rev Genet
, vol.12
, Issue.5
, pp. 341-355
-
-
Mingozzi, F.1
High, K.A.2
-
47
-
-
84890119907
-
Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS
-
Foust KD, et al. (2013) Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS. Mol Ther 21(12):2148-2159.
-
(2013)
Mol Ther
, vol.21
, Issue.12
, pp. 2148-2159
-
-
Foust, K.D.1
-
48
-
-
84961290469
-
Motor neuron cell-nonautonomous rescue of spinal muscular atrophy phenotypes in mild and severe transgenic mouse models
-
Hua Y, et al. (2015) Motor neuron cell-nonautonomous rescue of spinal muscular atrophy phenotypes in mild and severe transgenic mouse models. Genes Dev 29(3):288-297.
-
(2015)
Genes Dev
, vol.29
, Issue.3
, pp. 288-297
-
-
Hua, Y.1
-
49
-
-
84908352138
-
Genome-scale CRISPR-mediated control of gene repression and activation
-
Gilbert LA, et al. (2014) Genome-scale CRISPR-mediated control of gene repression and activation. Cell 159(3):647-661.
-
(2014)
Cell
, vol.159
, Issue.3
, pp. 647-661
-
-
Gilbert, L.A.1
-
50
-
-
84894063115
-
Dynamic imaging of genomic loci in living human cells by an optimized CRISPR/Cas system
-
Chen B, et al. (2013) Dynamic imaging of genomic loci in living human cells by an optimized CRISPR/Cas system. Cell 155(7):1479-1491.
-
(2013)
Cell
, vol.155
, Issue.7
, pp. 1479-1491
-
-
Chen, B.1
-
51
-
-
84908328232
-
A protein-tagging system for signal amplification in gene expression and fluorescence imaging
-
Tanenbaum ME, Gilbert LA, Qi LS, Weissman JS, Vale RD (2014) A protein-tagging system for signal amplification in gene expression and fluorescence imaging. Cell 159(3):635-646.
-
(2014)
Cell
, vol.159
, Issue.3
, pp. 635-646
-
-
Tanenbaum, M.E.1
Gilbert, L.A.2
Qi, L.S.3
Weissman, J.S.4
Vale, R.D.5
-
52
-
-
21244497878
-
Positional effect of chemical modifications on short interference RNA activity in mammalian cells
-
Prakash TP, et al. (2005) Positional effect of chemical modifications on short interference RNA activity in mammalian cells. J Med Chem 48(13):4247-4253.
-
(2005)
J Med Chem
, vol.48
, Issue.13
, pp. 4247-4253
-
-
Prakash, T.P.1
|