-
1
-
-
25844487226
-
Diseases of unstable repeat expansion: Mechanisms and common principles
-
Gatchel, J.R. and Zoghbi, H.Y. (2005) Diseases of unstable repeat expansion: mechanisms and common principles. Nat. Rev. Genet., 6, 743-755.
-
(2005)
Nat. Rev. Genet.
, vol.6
, pp. 743-755
-
-
Gatchel, J.R.1
Zoghbi, H.Y.2
-
2
-
-
77949512140
-
RNA targeting therapeutics: Molecular mechanisms of antisense oligonucleotides as a therapeutic platform
-
Bennett, C.F. and Swayze, E.E. (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
-
3
-
-
33645127443
-
Genetic medicines: Treatment strategies for hereditary disorders
-
O'Connor, T.P. and Crystal, R.G. (2006) Genetic medicines: treatment strategies for hereditary disorders. Nat. Rev. Genet., 7, 261-276.
-
(2006)
Nat. Rev. Genet.
, vol.7
, pp. 261-276
-
-
O'Connor, T.P.1
Crystal, R.G.2
-
4
-
-
0027480960
-
A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes
-
MacDonald, M.E., Ambrose, C.M., Duyao, M.P., Myers, R.H., Lin, C., Srinidhi, L., Barnes, G., Taylor, S.A., James, M., Groot, N. et al. (1993) A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes. Cell, 72, 971-983.
-
(1993)
Cell
, vol.72
, pp. 971-983
-
-
Macdonald, M.E.1
Ambrose, C.M.2
Duyao, M.P.3
Myers, R.H.4
Lin, C.5
Srinidhi, L.6
Barnes, G.7
Taylor, S.A.8
James, M.9
Groot, N.10
-
5
-
-
33846225133
-
Huntington's disease
-
Walker, F.O. (2007) Huntington's disease. Lancet, 369, 218-228.
-
(2007)
Lancet
, vol.369
, pp. 218-228
-
-
Walker, F.O.1
-
6
-
-
0029055717
-
Targeted disruption of the Huntington's disease gene results in embryonic lethality and behavioral and morphological changes in heterozygotes
-
Nasir, J., Floresco, S.B., O'Kusky, J.R., Diewert, V.M., Richman, J.M., Zeisler, J., Borowski, A., Marth, J.D., Phillips, A.G. and Hayden, M.R. (1995) Targeted disruption of the Huntington's disease gene results in embryonic lethality and behavioral and morphological changes in heterozygotes. Cell, 81, 811-823.
-
(1995)
Cell
, vol.81
, pp. 811-823
-
-
Nasir, J.1
Floresco, S.B.2
O'Kusky, J.R.3
Diewert, V.M.4
Richman, J.M.5
Zeisler, J.6
Borowski, A.7
Marth, J.D.8
Phillips, A.G.9
Hayden, M.R.10
-
7
-
-
33845720388
-
Huntingtin inhibits caspase-3 activation
-
Zhang, Y., Leavitt, B.R., van Raamsdonk, J.M., Dragatsis, I., Goldowitz, D., MacDonald, M.E., Hayden, M.R. and Friedlander, R.M. (2006) Huntingtin inhibits caspase-3 activation. EMBO J., 25, 5896-5906.
-
(2006)
EMBO J.
, vol.25
, pp. 5896-5906
-
-
Zhang, Y.1
Leavitt, B.R.2
Van Raamsdonk, J.M.3
Dragatsis, I.4
Goldowitz, D.5
Macdonald, M.E.6
Hayden, M.R.7
Friedlander, R.M.8
-
8
-
-
0035919701
-
Loss of huntingtin-mediated BDNF gene transcription in Huntington's Disease
-
Zuccato, C., Ciammola, A., Rigamonti, D., Leavitt, B.R., Goffredo, D., Conti, L., MacDonald, M.E., Friedlander, R.M., Silani, V., Hayden, M.R. et al. (2001) Loss of huntingtin-mediated BDNF gene transcription in Huntington's Disease. Science, 293, 493-498.
-
(2001)
Science
, vol.293
, pp. 493-498
-
-
Zuccato, C.1
Ciammola, A.2
Rigamonti, D.3
Leavitt, B.R.4
Goffredo, D.5
Conti, L.6
Macdonald, M.E.7
Friedlander, R.M.8
Silani, V.9
Hayden, M.R.10
-
9
-
-
84993912315
-
Increased apoptosis and early embryonic lethality in mice nullizygous for the Huntington's disease gene homologue
-
Zeitlin, S., Liu, J.P., Chapman, D.L., Papaioannou, V.E. and Efstratiadis, A. (1995) Increased apoptosis and early embryonic lethality in mice nullizygous for the Huntington's disease gene homologue. Nat. Genet., 11, 155-163.
-
(1995)
Nat. Genet.
, vol.11
, pp. 155-163
-
-
Zeitlin, S.1
Liu, J.P.2
Chapman, D.L.3
Papaioannou, V.E.4
Efstratiadis, A.5
-
10
-
-
84862663712
-
Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis
-
Kordasiewicz, H.B., Stanek, L.M., Wancewicz, E.V., Mazur, C., McAlonis, M.M., Pytel, K.A., Artates, J.W., Weiss, A., Cheng, S.H., Shihabuddin, L.S. et al. (2012) Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis. Neuron, 74, 1031-1044.
-
(2012)
Neuron
, vol.74
, pp. 1031-1044
-
-
Kordasiewicz, H.B.1
Stanek, L.M.2
Wancewicz, E.V.3
Mazur, C.4
McAlonis, M.M.5
Pytel, K.A.6
Artates, J.W.7
Weiss, A.8
Cheng, S.H.9
Shihabuddin, L.S.10
-
11
-
-
36749033738
-
Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits
-
DiFiglia, M., Sena-Esteves, M., Chase, K., Sapp, E., Pfister, E., Sass, M., Yoder, J., Reeves, P., Pandey, R.K., Rajeev, K.G. et al. (2007) Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits. Proc. Natl Acad. Sci. USA, 104, 17204-17209.
-
(2007)
Proc. Natl Acad. Sci. USA
, vol.104
, pp. 17204-17209
-
-
Difiglia, M.1
Sena-Esteves, M.2
Chase, K.3
Sapp, E.4
Pfister, E.5
Sass, M.6
Yoder, J.7
Reeves, P.8
Pandey, R.K.9
Rajeev, K.G.10
-
12
-
-
82955199935
-
Preclinical safety of RNAimediated HTT suppression in the rhesus macaque as a potential therapy for huntington's disease
-
McBride, J.L., Pitzer, M.R., Boudreau, R.L., Dufour, B., Hobbs, T., Ojeda, S.R. and Davidson, B.L. (2011) Preclinical safety of RNAimediated HTT suppression in the rhesus macaque as a potential therapy for huntington's disease. Mol. Ther., 19, 2152-2162.
-
(2011)
Mol. Ther.
, vol.19
, pp. 2152-2162
-
-
McBride, J.L.1
Pitzer, M.R.2
Boudreau, R.L.3
Dufour, B.4
Hobbs, T.5
Ojeda, S.R.6
Davidson, B.L.7
-
13
-
-
82955233704
-
Rational design of therapeutic siRNAs: Minimizing off-targeting potential to improve the safety of RNAi therapy for Huntington's disease
-
Boudreau, R.L., Spengler, R.M. and Davidson, B.L. (2011) Rational design of therapeutic siRNAs: minimizing off-targeting potential to improve the safety of RNAi therapy for Huntington's disease. Mol. Ther., 19, 2169-2177.
-
(2011)
Mol. Ther.
, vol.19
, pp. 2169-2177
-
-
Boudreau, R.L.1
Spengler, R.M.2
Davidson, B.L.3
-
14
-
-
78649379362
-
Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat
-
Gagnon, K.T., Pendergraff, H.M., Deleavey, G.F., Swayze, E.E., Potier, P., Randolph, J., Roesch, E.B., Chattopadhyaya, J., Damha, M.J., Bennett, C.F. et al. (2010) Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat. Biochemistry, 49, 10166-10178.
-
(2010)
Biochemistry
, vol.49
, pp. 10166-10178
-
-
Gagnon, K.T.1
Pendergraff, H.M.2
Deleavey, G.F.3
Swayze, E.E.4
Potier, P.5
Randolph, J.6
Roesch, E.B.7
Chattopadhyaya, J.8
Damha, M.J.9
Bennett, C.F.10
-
15
-
-
78649359013
-
Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism
-
Hu, J., Liu, J. and Corey, D.R. (2010) Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism. Chem. Biol., 17, 1183-1188.
-
(2010)
Chem. Biol.
, vol.17
, pp. 1183-1188
-
-
Hu, J.1
Liu, J.2
Corey, D.R.3
-
16
-
-
66149181896
-
Allelespecific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs
-
Hu, J., Matsui, M., Gagnon, K.T., Schwartz, J.C., Gabillet, S., Arar, K., Wu, J., Bezprozvanny, I. and Corey, D.R. (2009) Allelespecific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs. Nat. Biotechnol., 27, 478-484.
-
(2009)
Nat. Biotechnol.
, vol.27
, pp. 478-484
-
-
Hu, J.1
Matsui, M.2
Gagnon, K.T.3
Schwartz, J.C.4
Gabillet, S.5
Arar, K.6
Wu, J.7
Bezprozvanny, I.8
Corey, D.R.9
-
17
-
-
84865688581
-
Single-stranded RNAs Use RNAi to potently and allele-selectively inhibit mutant huntingtin expression
-
Yu, D., Pendergraff, H., Liu, J., Kordasiewicz, H.B., Cleveland, D.W., Swayze, E.E., Lima, W.F., Crooke, S.T., Prakash, T.P. and Corey, D.R. (2012) Single-stranded RNAs Use RNAi to potently and allele-selectively inhibit mutant huntingtin expression. Cell, 150, 895-908.
-
(2012)
Cell
, vol.150
, pp. 895-908
-
-
Yu, D.1
Pendergraff, H.2
Liu, J.3
Kordasiewicz, H.B.4
Cleveland, D.W.5
Swayze, E.E.6
Lima, W.F.7
Crooke, S.T.8
Prakash, T.P.9
Corey, D.R.10
-
18
-
-
47549105506
-
Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts
-
van Bilsen, P.H., Jaspers, L., Lombardi, M.S., Odekerken, J.C., Burright, E.N. and Kaemmerer, W.F. (2008) Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts. Hum. Gene. Ther., 19, 710-719.
-
(2008)
Hum. Gene. Ther.
, vol.19
, pp. 710-719
-
-
Van Bilsen, P.H.1
Jaspers, L.2
Lombardi, M.S.3
Odekerken, J.C.4
Burright, E.N.5
Kaemmerer, W.F.6
-
19
-
-
33749438388
-
Designing siRNA that distinguish between genes that differ by a single nucleotide
-
Schwarz, D.S., Ding, H., Kennington, L., Moore, J.T., Schelter, J., Burchard, J., Linsley, P.S., Aronin, N., Xu, Z. and Zamore, P.D. (2006) Designing siRNA that distinguish between genes that differ by a single nucleotide. PLoS Genet., 2, e140.
-
(2006)
PLoS Genet.
, vol.2
-
-
Schwarz, D.S.1
Ding, H.2
Kennington, L.3
Moore, J.T.4
Schelter, J.5
Burchard, J.6
Linsley, P.S.7
Aronin, N.8
Xu, Z.9
Zamore, P.D.10
-
20
-
-
67349263503
-
A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference
-
Lombardi, M.S., Jaspers, L., Spronkmans, C., Gellera, C., Taroni, F., Di Maria, E., Donato, S.D. and Kaemmerer, W.F. (2009) A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference. Exp. Neurol., 217, 312-319.
-
(2009)
Exp. Neurol.
, vol.217
, pp. 312-319
-
-
Lombardi, M.S.1
Jaspers, L.2
Spronkmans, C.3
Gellera, C.4
Taroni, F.5
Di Maria, E.6
Donato, S.D.7
Kaemmerer, W.F.8
-
21
-
-
0026774939
-
Selective inhibition of mutant Ha-ras mRNA expression by antisense oligonucleotides
-
Monia, B.P., Johnston, J.F., Ecker, D.J., Zounes, M.A., Lima, W.F. and Freier, S.M. (1992) Selective inhibition of mutant Ha-ras mRNA expression by antisense oligonucleotides. J. Biol. Chem., 267, 19954-19962.
-
(1992)
J. Biol. Chem.
, vol.267
, pp. 19954-19962
-
-
Monia, B.P.1
Johnston, J.F.2
Ecker, D.J.3
Zounes, M.A.4
Lima, W.F.5
Freier, S.M.6
-
22
-
-
82955237522
-
Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene/ allele-specific silencing of mutant huntingtin
-
Carroll, J.B., Warby, S.C., Southwell, A.L., Doty, C.N., Greenlee, S., Skotte, N., Hung, G., Bennett, C.F., Freier, S.M. and Hayden, M.R. (2011) Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene/ allele-specific silencing of mutant huntingtin. Mol. Ther., 19, 2178-2185.
-
(2011)
Mol. Ther.
, vol.19
, pp. 2178-2185
-
-
Carroll, J.B.1
Warby, S.C.2
Southwell, A.L.3
Doty, C.N.4
Greenlee, S.5
Skotte, N.6
Hung, G.7
Bennett, C.F.8
Freier, S.M.9
Hayden, M.R.10
-
23
-
-
0023750525
-
Morphometric demonstration of atrophic changes in the cerebral cortex
-
de la Monte, S.M., Vonsattel, J.P. and Richardson, E.P. Jr (1988) Morphometric demonstration of atrophic changes in the cerebral cortex, white matter, and neostriatum in Huntington's disease. J. Neuropathol. Exp. Neurol., 47, 516-525.
-
(1988)
White Matter, and Neostriatum in Huntington's Disease. J. Neuropathol. Exp. Neurol.
, vol.47
, pp. 516-525
-
-
De La Monte, S.M.1
Vonsattel, J.P.2
Richardson Jr., E.P.3
-
24
-
-
67349159137
-
Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients
-
Pfister, E.L., Kennington, L., Straubhaar, J., Wagh, S., Liu, W., DiFiglia, M., Landwehrmeyer, B., Vonsattel, J.-P., Zamore, P.D. and Aronin, N. (2009) Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients. Curr. Biol., 19, 774-778.
-
(2009)
Curr. Biol.
, vol.19
, pp. 774-778
-
-
Pfister, E.L.1
Kennington, L.2
Straubhaar, J.3
Wagh, S.4
Liu, W.5
Difiglia, M.6
Landwehrmeyer, B.7
Vonsattel, J.-P.8
Zamore, P.D.9
Aronin, N.10
-
25
-
-
0037470145
-
Efficient reduction of target RNAs by small interfering RNA and RNase H-dependent antisense agents
-
Vickers, T.A., Koo, S., Bennett, C.F., Crooke, S.T., Dean, N.M. and Baker, B.F. (2003) Efficient reduction of target RNAs by small interfering RNA and RNase H-dependent antisense agents. J. Biol. Chem., 278, 7108-7118.
-
(2003)
J. Biol. Chem.
, vol.278
, pp. 7108-7118
-
-
Vickers, T.A.1
Koo, S.2
Bennett, C.F.3
Crooke, S.T.4
Dean, N.M.5
Baker, B.F.6
-
26
-
-
84894521728
-
-
Crooke, S.T. (ed.), CRC Press, Boca Raton
-
Swayze, E.E. and Bhat, B. (2007) In: Crooke, S.T. (ed.), Antisense Drug Technology: Principles, Strategies, and Applications, 2nd edn. CRC Press, Boca Raton, pp. 143-182.
-
(2007)
Antisense Drug Technology: Principles, Strategies, and Applications, 2nd Edn.
, pp. 143-182
-
-
Swayze, E.E.1
Bhat, B.2
-
27
-
-
84876466100
-
An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: A phase 1, randomised, first-in-man study
-
Miller, T.M., Pestronk, A., David, W., Rothstein, J., Simpson, E., Appel, S.H., Andres, P.L., Mahoney, K., Allred, P., Alexander, K. et al. (2013) An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study. Lancet Neurol., 12, 435-442.
-
(2013)
Lancet Neurol.
, vol.12
, pp. 435-442
-
-
Miller, T.M.1
Pestronk, A.2
David, W.3
Rothstein, J.4
Simpson, E.5
Appel, S.H.6
Andres, P.L.7
Mahoney, K.8
Allred, P.9
Alexander, K.10
-
28
-
-
84890098975
-
Getting a fix on SMA
-
Dolgin, E. (2012) Getting a fix on SMA. Nat. Med., 18, 1605-1605.
-
(2012)
Nat. Med.
, vol.18
, pp. 1605-1605
-
-
Dolgin, E.1
-
29
-
-
0033053332
-
Crystal structure and improved antisense properties of 20-O-(2-methoxyethyl)-RNA
-
Teplova, M., Minasov, G., Tereshko, V., Inamati, G.B., Cook, P.D., Manoharan, M. and Egli, M. (1999) Crystal structure and improved antisense properties of 20-O-(2-methoxyethyl)-RNA. Nat. Struct. Biol., 6, 535-539.
-
(1999)
Nat. Struct. Biol.
, vol.6
, pp. 535-539
-
-
Teplova, M.1
Minasov, G.2
Tereshko, V.3
Inamati, G.B.4
Cook, P.D.5
Manoharan, M.6
Egli, M.7
-
30
-
-
59449093219
-
Short antisense oligonucleotides with novel 20-40 conformationaly restricted nucleoside analogues show improved potency without increased toxicity in animals
-
Seth, P.P., Siwkowski, A., Allerson, C.R., Vasquez, G., Lee, S., Prakash, T.P., Wancewicz, E.V., Witchell, D. and Swayze, E.E. (2009) Short antisense oligonucleotides with novel 20-40 conformationaly restricted nucleoside analogues show improved potency without increased toxicity in animals. J. Med. Chem., 52, 10-13.
-
(2009)
J. Med. Chem.
, vol.52
, pp. 10-13
-
-
Seth, P.P.1
Siwkowski, A.2
Allerson, C.R.3
Vasquez, G.4
Lee, S.5
Prakash, T.P.6
Wancewicz, E.V.7
Witchell, D.8
Swayze, E.E.9
-
31
-
-
79959479529
-
Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes
-
Koller, E., Vincent, T.M., Chappell, A., De, S., Manoharan, M. and Bennett, C.F. (2011) Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes. Nucleic Acids Res., 39, 4795-4807.
-
(2011)
Nucleic Acids Res.
, vol.39
, pp. 4795-4807
-
-
Koller, E.1
Vincent, T.M.2
Chappell, A.3
De, S.4
Manoharan, M.5
Bennett, C.F.6
-
32
-
-
0034232357
-
The G x U wobble base pair. A fundamental building block of RNA structure crucial to RNA function in diverse biological systems
-
Varani, G. and McClain, W.H. (2000) The G x U wobble base pair. A fundamental building block of RNA structure crucial to RNA function in diverse biological systems. EMBO Rep., 1, 18-23.
-
(2000)
EMBO Rep.
, vol.1
, pp. 18-23
-
-
Varani, G.1
McClain, W.H.2
-
33
-
-
0035913728
-
Long-range cooperativity due to C5-propynylation of oligopyrimidines enhances specific recognition by uridine of ribo-adenosine over ribo-guanosine
-
Barnes, T.W. III and Turner, D.H. (2001) Long-range cooperativity due to C5-propynylation of oligopyrimidines enhances specific recognition by uridine of ribo-adenosine over ribo-guanosine. J. Am. Chem. Soc., 123, 9186-9187.
-
(2001)
J. Am. Chem. Soc.
, vol.123
, pp. 9186-9187
-
-
Barnes III, T.W.1
Turner, D.H.2
-
34
-
-
0033554848
-
Thermodynamics of RNA-RNA duplexes with 2-or 4-thiouridines: Implications for antisense design and targeting a group i intron
-
Testa, S.M., Disney, M.D., Turner, D.H. and Kierzek, R. (1999) Thermodynamics of RNA-RNA duplexes with 2-or 4-thiouridines: implications for antisense design and targeting a group I intron. Biochemistry, 38, 16655-16662.
-
(1999)
Biochemistry
, vol.38
, pp. 16655-16662
-
-
Testa, S.M.1
Disney, M.D.2
Turner, D.H.3
Kierzek, R.4
-
35
-
-
80054756151
-
Synthesis, improved antisense activity and structural rationale for the divergent RNA affinities of 30-fluoro hexitol nucleic acid (FHNA and Ara-FHNA) modified oligonucleotides
-
Egli, M., Pallan, P.S., Allerson, C.R., Prakash, T.P., Berdeja, A., Yu, J., Lee, S., Watt, A., Gaus, H., Bhat, B. et al. (2011) Synthesis, improved antisense activity and structural rationale for the divergent RNA affinities of 30-fluoro hexitol nucleic acid (FHNA and Ara-FHNA) modified oligonucleotides. J. Am. Chem. Soc., 133, 16642-16649.
-
(2011)
J. Am. Chem. Soc.
, vol.133
, pp. 16642-16649
-
-
Egli, M.1
Pallan, P.S.2
Allerson, C.R.3
Prakash, T.P.4
Berdeja, A.5
Yu, J.6
Lee, S.7
Watt, A.8
Gaus, H.9
Bhat, B.10
-
36
-
-
2342427578
-
Determination of the role of the human RNase H1 in the pharmacology of DNA-like antisense drugs
-
Wu, H., Lima, W.F., Zhang, H., Fan, A., Sun, H. and Crooke, S.T. (2004) Determination of the role of the human RNase H1 in the pharmacology of DNA-like antisense drugs. J. Biol. Chem., 279, 17181-17189.
-
(2004)
J. Biol. Chem.
, vol.279
, pp. 17181-17189
-
-
Wu, H.1
Lima, W.F.2
Zhang, H.3
Fan, A.4
Sun, H.5
Crooke, S.T.6
-
37
-
-
4344589884
-
Structural requirements at the catalytic site of the heteroduplex substrate for human RNase H1 catalysis
-
Lima, W.F., Nichols, J.G., Wu, H., Prakash, T.P., Migawa, M.T., Wyrzykiewicz, T.K., Bhat, B. and Crooke, S.T. (2004) Structural requirements at the catalytic site of the heteroduplex substrate for human RNase H1 catalysis. J. Biol. Chem., 279, 36317-36326.
-
(2004)
J. Biol. Chem.
, vol.279
, pp. 36317-36326
-
-
Lima, W.F.1
Nichols, J.G.2
Wu, H.3
Prakash, T.P.4
Migawa, M.T.5
Wyrzykiewicz, T.K.6
Bhat, B.7
Crooke, S.T.8
-
38
-
-
35348978302
-
Structure of human RNase H1 complexed with an RNA/DNA hybrid: Insight into HIV reverse transcription
-
Nowotny, M., Gaidamakov, S.A., Ghirlando, R., Cerritelli, S.M., Crouch, R.J. and Yang, W. (2007) Structure of human RNase H1 complexed with an RNA/DNA hybrid: insight into HIV reverse transcription. Mol. Cell, 28, 264-276.
-
(2007)
Mol. Cell
, vol.28
, pp. 264-276
-
-
Nowotny, M.1
Gaidamakov, S.A.2
Ghirlando, R.3
Cerritelli, S.M.4
Crouch, R.J.5
Yang, W.6
-
39
-
-
0033213981
-
Properties of cloned and expressed human RNase H1
-
Wu, H., Lima, W.F. and Crooke, S.T. (1999) Properties of cloned and expressed human RNase H1. J. Biol. Chem., 274, 28270-28278.
-
(1999)
J. Biol. Chem.
, vol.274
, pp. 28270-28278
-
-
Wu, H.1
Lima, W.F.2
Crooke, S.T.3
-
40
-
-
33845883545
-
Human RNase H1 discriminates between subtle variations in the structure of the heteroduplex substrate
-
Lima, W.F., Rose, J.B., Nichols, J.G., Wu, H., Migawa, M.T., Wyrzykiewicz, T.K., Siwkowski, A.M. and Crooke, S.T. (2007) Human RNase H1 discriminates between subtle variations in the structure of the heteroduplex substrate. Mol. Pharmacol., 71, 83-91.
-
(2007)
Mol. Pharmacol.
, vol.71
, pp. 83-91
-
-
Lima, W.F.1
Rose, J.B.2
Nichols, J.G.3
Wu, H.4
Migawa, M.T.5
Wyrzykiewicz, T.K.6
Siwkowski, A.M.7
Crooke, S.T.8
-
41
-
-
0028846258
-
5'-Me-DNA-A new oligonucleotide analog: Synthesis and biochemical properties
-
Saha, A.K., Caulfield, T.J., Hobbs, C., Upson, D.A., Waychunas, C. and Yawman, A.M. (1995) 5'-Me-DNA-a new oligonucleotide analog: synthesis and biochemical properties. J. Org. Chem., 60, 788-789.
-
(1995)
J. Org. Chem.
, vol.60
, pp. 788-789
-
-
Saha, A.K.1
Caulfield, T.J.2
Hobbs, C.3
Upson, D.A.4
Waychunas, C.5
Yawman, A.M.6
-
42
-
-
33645545684
-
20-fluoroarabino-and arabinonucleic acid show different conformations, resulting in deviating RNA affinities and processing of their heteroduplexes with RNA by RNase H
-
Li, F., Sarkhel, S., Wilds, C.J., Wawrzak, Z., Prakash, T.P., Manoharan, M. and Egli, M. (2006) 20-fluoroarabino-and arabinonucleic acid show different conformations, resulting in deviating RNA affinities and processing of their heteroduplexes with RNA by RNase H. Biochemistry, 45, 4141-4152.
-
(2006)
Biochemistry
, vol.45
, pp. 4141-4152
-
-
Li, F.1
Sarkhel, S.2
Wilds, C.J.3
Wawrzak, Z.4
Prakash, T.P.5
Manoharan, M.6
Egli, M.7
-
43
-
-
0025762698
-
Properties of 2'-fluorothymidine-containing oligonucleotides: Interaction with restriction endonuclease EcoRV
-
Williams, D.M., Benseler, F. and Eckstein, F. (1991) Properties of 2'-fluorothymidine-containing oligonucleotides: interaction with restriction endonuclease EcoRV. Biochemistry, 30, 4001-4009.
-
(1991)
Biochemistry
, vol.30
, pp. 4001-4009
-
-
Williams, D.M.1
Benseler, F.2
Eckstein, F.3
-
44
-
-
84864199342
-
Structure and nuclease resistance of 20, 40-constrained 20-O-methoxyethyl (cMOE) and 20-O-ethyl (cEt) modified DNAs
-
Pallan, P.S., Allerson, C.R., Berdeja, A., Seth, P.P., Swayze, E.E., Prakash, T.P. and Egli, M. (2012) Structure and nuclease resistance of 20, 40-constrained 20-O-methoxyethyl (cMOE) and 20-O-ethyl (cEt) modified DNAs. Chem. Commun., 48, 8195-8197.
-
(2012)
Chem. Commun.
, vol.48
, pp. 8195-8197
-
-
Pallan, P.S.1
Allerson, C.R.2
Berdeja, A.3
Seth, P.P.4
Swayze, E.E.5
Prakash, T.P.6
Egli, M.7
-
45
-
-
84871214247
-
A fully humanized transgenic mouse model of Huntington disease
-
Southwell, A.L., Warby, S.C., Carroll, J.B., Doty, C.N., Skotte, N.H., Zhang, W., Villanueva, E.B., Kovalik, V., Xie, Y., Pouladi, M.A. et al. (2013) A fully humanized transgenic mouse model of Huntington disease. Hum. Mol. Genet., 22, 18-34.
-
(2013)
Hum. Mol. Genet.
, vol.22
, pp. 18-34
-
-
Southwell, A.L.1
Warby, S.C.2
Carroll, J.B.3
Doty, C.N.4
Skotte, N.H.5
Zhang, W.6
Villanueva, E.B.7
Kovalik, V.8
Xie, Y.9
Pouladi, M.A.10
-
46
-
-
77955643169
-
Molecular mechanisms and potential therapeutical targets in Huntington's disease
-
Zuccato, C., Valenza, M. and Cattaneo, E. (2010) Molecular mechanisms and potential therapeutical targets in Huntington's disease. Physiol. Rev., 90, 905-981.
-
(2010)
Physiol. Rev.
, vol.90
, pp. 905-981
-
-
Zuccato, C.1
Valenza, M.2
Cattaneo, E.3
-
47
-
-
80052383976
-
Targeting several CAG expansion diseases by a single antisense oligonucleotide
-
Evers, M.M., Pepers, B.A., van Deutekom, J.C.T., Mulders, S.A.M., den Dunnen, J.T., Aartsma-Rus, A., van Ommen, G.-J.B. and van Roon-Mom, W.M.C. (2011) Targeting several CAG expansion diseases by a single antisense oligonucleotide. PLoS One, 6, e24308.
-
(2011)
PLoS One
, vol.6
-
-
Evers, M.M.1
Pepers, B.A.2
Van Deutekom, J.C.T.3
Mulders, S.A.M.4
Den Dunnen, J.T.5
Aartsma-Rus, A.6
Van Ommen, G.-J.B.7
Van Roon-Mom, W.M.C.8
-
48
-
-
84873463075
-
Aberrant splicing of HTT generates the pathogenic Exon 1 protein in Huntington disease
-
Sathasivam, K., Neueder, A., Gipson, T.A., Landles, C., Benjamin, A.C., Bondulich, M.K., Smith, D.L., Faull, R.L.M., Roos, R.A.C., Howland, D. et al. (2013) Aberrant splicing of HTT generates the pathogenic Exon 1 protein in Huntington disease. Proc. Natl Acad. Sci. USA, 110, 2366-2370.
-
(2013)
Proc. Natl Acad. Sci. USA
, vol.110
, pp. 2366-2370
-
-
Sathasivam, K.1
Neueder, A.2
Gipson, T.A.3
Landles, C.4
Benjamin, A.C.5
Bondulich, M.K.6
Smith, D.L.7
Faull, R.L.M.8
Roos, R.A.C.9
Howland, D.10
-
49
-
-
67349263503
-
A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference
-
Lombardi, M.S., Jaspers, L., Spronkmans, C., Gellera, C., Taroni, F., Di Maria, E., Donato, S.D. and Kaemmerer, W.F. (2009) A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference. Exp. Neurol., 217, 312-319.
-
(2009)
Exp. Neurol.
, vol.217
, pp. 312-319
-
-
Lombardi, M.S.1
Jaspers, L.2
Spronkmans, C.3
Gellera, C.4
Taroni, F.5
Di Maria, E.6
Donato, S.D.7
Kaemmerer, W.F.8
|