-
1
-
-
84872863485
-
Nanoparticle-based delivery of RNAi therapeutics: Progress and challenges
-
Zhou, J., Shum, K.-T., Burnett, J.C. and Rossi, J.J. (2013) Nanoparticle-based delivery of RNAi therapeutics: progress and challenges. Pharmaceuticals, 6, 85-107.
-
(2013)
Pharmaceuticals
, vol.6
, pp. 85-107
-
-
Zhou, J.1
Shum, K.-T.2
Burnett, J.C.3
Rossi, J.J.4
-
2
-
-
84918578407
-
Multivalent N-Acetylgalactosamine-Conjugated siRNA localizes in hepatocytes and elicits robust RNAi-mediated gene silencing
-
Nair, J.K., Willoughby, J.L.S., Chan, A., Charisse, K., Alam, M.R., Wang, Q., Hoekstra, M., Kandasamy, P., Kel'in, A.V., Milstein, S. et al. (2014) Multivalent N-Acetylgalactosamine-Conjugated siRNA localizes in hepatocytes and elicits robust RNAi-mediated gene silencing. JACS, 136, 16958-16961.
-
(2014)
JACS
, vol.136
, pp. 16958-16961
-
-
Nair, J.K.1
Willoughby, J.L.S.2
Chan, A.3
Charisse, K.4
Alam, M.R.5
Wang, Q.6
Hoekstra, M.7
Kandasamy, P.8
Kel'in, A.V.9
Milstein, S.10
-
3
-
-
85016271522
-
Clinical proof of concept for a novel hepatocyte-targeting GalNAc-siRNA conjugate
-
Zimmermann, T.S., Karsten, V., Chan, A., Chiesa, J., Boyce, M., Bettencourt, B.B., Hutabarat, R., Nochur, S., Vaishnaw, A. and Gollob, J. (2017) Clinical proof of concept for a novel hepatocyte-targeting GalNAc-siRNA conjugate. Mol. Ther., 25, 71-78.
-
(2017)
Mol. Ther.
, vol.25
, pp. 71-78
-
-
Zimmermann, T.S.1
Karsten, V.2
Chan, A.3
Chiesa, J.4
Boyce, M.5
Bettencourt, B.B.6
Hutabarat, R.7
Nochur, S.8
Vaishnaw, A.9
Gollob, J.10
-
4
-
-
85047830699
-
Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo
-
Hassler, M.R., Turanov, A.A., Alterman, J.F., Haraszti, R.A., Coles, A.H., Osborn, M.F., Echeverria, D., Nikan, M., Salomon, W.E., Roux, L. et al. (2018) Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo. Nucleic Acids Res., 46, 2185-2196.
-
(2018)
Nucleic Acids Res.
, vol.46
, pp. 2185-2196
-
-
Hassler, M.R.1
Turanov, A.A.2
Alterman, J.F.3
Haraszti, R.A.4
Coles, A.H.5
Osborn, M.F.6
Echeverria, D.7
Nikan, M.8
Salomon, W.E.9
Roux, L.10
-
5
-
-
85037980916
-
Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc-siRNA conjugates
-
Nair, J.K., Attarwala, H., Sehgal, A., Wang, Q., Aluri, K., Zhang, X., Gao, M., Liu, J., Indrakanti, R., Schofield, S. et al. (2017) Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc-siRNA conjugates. Nucleic Acids Res., 45, 10969-10977.
-
(2017)
Nucleic Acids Res.
, vol.45
, pp. 10969-10977
-
-
Nair, J.K.1
Attarwala, H.2
Sehgal, A.3
Wang, Q.4
Aluri, K.5
Zhang, X.6
Gao, M.7
Liu, J.8
Indrakanti, R.9
Schofield, S.10
-
6
-
-
85042001178
-
Advanced siRNA designs further improve in vivo performance of GalNAc-siRNA conjugates
-
Foster, D.J., Brown, C.R., Shaikh, S., Trapp, C., Schlegel, M.K., Qian, K., Sehgal, A., Rajeev, K.G., Jadhav, V., Manoharan, M. et al. (2018) Advanced siRNA designs further improve in vivo performance of GalNAc-siRNA conjugates. Mol. Ther., 26, 708-717.
-
(2018)
Mol. Ther.
, vol.26
, pp. 708-717
-
-
Foster, D.J.1
Brown, C.R.2
Shaikh, S.3
Trapp, C.4
Schlegel, M.K.5
Qian, K.6
Sehgal, A.7
Rajeev, K.G.8
Jadhav, V.9
Manoharan, M.10
-
7
-
-
9144247189
-
Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs
-
Soutschek, J., Akinc, A., Bramlage, B., Charisse, K., Constien, R., Donoghue, M., Elbashir, S., Geick, A., Hadwiger, P., Harborth, J. et al. (2004) Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature, 432, 173-178.
-
(2004)
Nature
, vol.432
, pp. 173-178
-
-
Soutschek, J.1
Akinc, A.2
Bramlage, B.3
Charisse, K.4
Constien, R.5
Donoghue, M.6
Elbashir, S.7
Geick, A.8
Hadwiger, P.9
Harborth, J.10
-
8
-
-
13944261143
-
Fully 2'-modified oligonucleotide duplexes with improved in vitro potency and stability compared to unmodified small interfering RNA
-
Allerson, C.R., Sioufi, N., Jarres, R., Prakash, T.P., Naik, N., Berdeja, A., Wanders, L., Griffey, R.H., Swayze, E.E. and Bhat, B. (2005) Fully 2'-modified oligonucleotide duplexes with improved in vitro potency and stability compared to unmodified small interfering RNA. J. Med. Chem., 48, 901-904.
-
(2005)
J. Med. Chem.
, vol.48
, pp. 901-904
-
-
Allerson, C.R.1
Sioufi, N.2
Jarres, R.3
Prakash, T.P.4
Naik, N.5
Berdeja, A.6
Wanders, L.7
Griffey, R.H.8
Swayze, E.E.9
Bhat, B.10
-
9
-
-
44149112583
-
Nucleoside modifications modulate activation of the protein kinase PKR in an RNA structure-specific manner
-
Nallagatla, S.R. and Bevilacqua, P.C. (2008) Nucleoside modifications modulate activation of the protein kinase PKR in an RNA structure-specific manner. RNA, 14, 1201-1213.
-
(2008)
RNA
, vol.14
, pp. 1201-1213
-
-
Nallagatla, S.R.1
Bevilacqua, P.C.2
-
10
-
-
33745614074
-
Position-specific chemical modification of siRNAs reduces "off-target" transcript silencing
-
Jackson, A.L., Burchard, J., Leake, D., Reynolds, A., Schelter, J., Guo, J., Johnson, J.M., Lim, L., Karpilow, J., Nichols, K. et al. (2006) Position-specific chemical modification of siRNAs reduces "off-target" transcript silencing. RNA, 12, 1197-1205.
-
(2006)
RNA
, vol.12
, pp. 1197-1205
-
-
Jackson, A.L.1
Burchard, J.2
Leake, D.3
Reynolds, A.4
Schelter, J.5
Guo, J.6
Johnson, J.M.7
Lim, L.8
Karpilow, J.9
Nichols, K.10
-
11
-
-
84937252963
-
Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides
-
Geary, R.S., Norris, D., Yu, R. and Bennett, C.F. (2015) Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides. Adv. Drug. Deliv. Rev., 87, 46-51.
-
(2015)
Adv. Drug. Deliv. Rev.
, vol.87
, pp. 46-51
-
-
Geary, R.S.1
Norris, D.2
Yu, R.3
Bennett, C.F.4
-
12
-
-
0036760718
-
Developments in RNA chemistry, a personal view
-
Eckstein, F. (2002) Developments in RNA chemistry, a personal view. Biochimie, 84, 841-848.
-
(2002)
Biochimie
, vol.84
, pp. 841-848
-
-
Eckstein, F.1
-
13
-
-
15844371219
-
Structural basis for 5'-end-specific recognition of guide RNA by the A
-
Ma, J.B., Yuan, Y.R., Meister, G., Pei, Y., Tuschl, T. and Patel, D.J. (2005) Structural basis for 5'-end-specific recognition of guide RNA by the A. fulgidus Piwi protein. Nature, 434, 666-670.
-
(2005)
Fulgidus Piwi Protein. Nature
, vol.434
, pp. 666-670
-
-
Ma, J.B.1
Yuan, Y.R.2
Meister, G.3
Pei, Y.4
Tuschl, T.5
Patel, D.J.6
-
14
-
-
77953479619
-
Structural basis for 5'-nucleotide base-specific recognition of guide RNA by human AGO2
-
Frank, F., Sonenberg, N. and Nagar, B. (2010) Structural basis for 5'-nucleotide base-specific recognition of guide RNA by human AGO2. Nature, 465, 818-822.
-
(2010)
Nature
, vol.465
, pp. 818-822
-
-
Frank, F.1
Sonenberg, N.2
Nagar, B.3
-
15
-
-
85026287048
-
5′-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo
-
Haraszti, R.A., Roux, L., Coles, A.H., Turanov, A.A., Alterman, J.F., Echeverria, D., Godinho, B.M., Aronin, N. and Khvorova, A. (2017) 5′-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo. Nucleic Acids Res., 45, 7581-7592.
-
(2017)
Nucleic Acids Res.
, vol.45
, pp. 7581-7592
-
-
Haraszti, R.A.1
Roux, L.2
Coles, A.H.3
Turanov, A.A.4
Alterman, J.F.5
Echeverria, D.6
Godinho, B.M.7
Aronin, N.8
Khvorova, A.9
-
16
-
-
84964582919
-
5′-(E)-Vinylphosphonate: A stable phosphate mimic can improve the RNAi activity of siRNA-GalNAc conjugates
-
Parmar, R., Willoughby, J.L.S., Liu, J., Foster, D.J., Brigham, B., Theile, C.S., Charisse, K., Akinc, A., Guidry, E., Pei, Y. et al. (2016) 5′-(E)-Vinylphosphonate: a stable phosphate mimic can improve the RNAi activity of siRNA-GalNAc conjugates. Chembiochem., 17, 987-989.
-
(2016)
Chembiochem.
, vol.17
, pp. 987-989
-
-
Parmar, R.1
Willoughby, J.L.S.2
Liu, J.3
Foster, D.J.4
Brigham, B.5
Theile, C.S.6
Charisse, K.7
Akinc, A.8
Guidry, E.9
Pei, Y.10
-
17
-
-
84926320054
-
Hepatocyte-specific delivery of siRNAs conjugated to novel non-nucleosidic trivalent N-Acetylgalactosamine elicits robust gene silencing in vivo
-
Rajeev, K.G., Nair, J.K., Jayaraman, M., Charisse, K., Taneja, N., O'Shea, J., Willoughby, J.L.S., Yucius, K., Nguyen, T., Shulga-Morskaya, s. et al. (2015) Hepatocyte-specific delivery of siRNAs conjugated to novel non-nucleosidic trivalent N-Acetylgalactosamine elicits robust gene silencing in vivo. Chembiochem., 16, 903-908.
-
(2015)
Chembiochem.
, vol.16
, pp. 903-908
-
-
Rajeev, K.G.1
Nair, J.K.2
Jayaraman, M.3
Charisse, K.4
Taneja, N.5
O'Shea, J.6
Willoughby, J.L.S.7
Yucius, K.8
Nguyen, T.9
Shulga-Morskaya, S.10
-
18
-
-
84929574453
-
SiRNA conjugates carrying sequentially assembled trivalent N-Acetylgalactosamine linked through nucleosides elicit robust gene silencing in vivo in hepatocytes
-
Matsuda, S., Keiser, K., Nair, J.K., Charisse, K., Manoharan, R.M., Kretschmer, P., Peng, C.G., Kel'in, A.V., Kandasamy, P., Willoughby, J.L.S. et al. (2015) siRNA conjugates carrying sequentially assembled trivalent N-Acetylgalactosamine linked through nucleosides elicit robust gene silencing in vivo in hepatocytes. ACS Chem. Biol., 10, 1181-1187.
-
(2015)
ACS Chem. Biol.
, vol.10
, pp. 1181-1187
-
-
Matsuda, S.1
Keiser, K.2
Nair, J.K.3
Charisse, K.4
Manoharan, R.M.5
Kretschmer, P.6
Peng, C.G.7
Kel'in, A.V.8
Kandasamy, P.9
Willoughby, J.L.S.10
-
19
-
-
84905584822
-
Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice
-
Prakash, T.P., Graham, M.J., Yu, J., Carty, R., Low, A., Chappell, A., Schmidt, K., Zhao, C., Aghajan, M., Murray, H.F. et al. (2014) Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice. Nucleic Acids Res., 42, 8796-8807.
-
(2014)
Nucleic Acids Res.
, vol.42
, pp. 8796-8807
-
-
Prakash, T.P.1
Graham, M.J.2
Yu, J.3
Carty, R.4
Low, A.5
Chappell, A.6
Schmidt, K.7
Zhao, C.8
Aghajan, M.9
Murray, H.F.10
-
20
-
-
85039034524
-
Asialoglycoprotein receptor 1 mediates productive uptake of N-acetylgalactosamine-conjugated and unconjugated phosphorothioate antisense oligonucleotides into liver hepatocytes
-
Tanowitz, M., Hettrick, L., Revenko, A., Kinberger, G.A., Prakash, T.P. and Seth, P.P. (2017) Asialoglycoprotein receptor 1 mediates productive uptake of N-acetylgalactosamine-conjugated and unconjugated phosphorothioate antisense oligonucleotides into liver hepatocytes. Nucleic Acids Res., 45, 12388-12400.
-
(2017)
Nucleic Acids Res.
, vol.45
, pp. 12388-12400
-
-
Tanowitz, M.1
Hettrick, L.2
Revenko, A.3
Kinberger, G.A.4
Prakash, T.P.5
Seth, P.P.6
-
21
-
-
85015608983
-
Preclinical and clinical advances of GalNAc-decorated nucleic acid therapeutics
-
Huang, H. (2017) Preclinical and clinical advances of GalNAc-decorated nucleic acid therapeutics. Mol. Ther. Nucleic Acids, 6, 116-132.
-
(2017)
Mol. Ther. Nucleic Acids
, vol.6
, pp. 116-132
-
-
Huang, H.1
-
22
-
-
85048360649
-
Improving siRNA delivery in vivo through lipid conjugation
-
Osborn, M.F. and Khvorova, A. (2018) Improving siRNA delivery In vivo through lipid conjugation. Nucleic Acid Ther., 28, 128-136.
-
(2018)
Nucleic Acid Ther.
, vol.28
, pp. 128-136
-
-
Osborn, M.F.1
Khvorova, A.2
-
23
-
-
35148815670
-
Mechanisms and optimization of in vivo delivery of lipophilic siRNAs
-
Wolfrum, C., Shi, S., Jayaprakash, K.N., Jayaraman, M., Wang, G., Pandey, R.K., Rajeev, K.G., Nakayama, T., Charrise, K., Ndungo, E.M. et al. (2007) Mechanisms and optimization of in vivo delivery of lipophilic siRNAs. Nat. Biotechnol., 25, 1149-1157.
-
(2007)
Nat. Biotechnol.
, vol.25
, pp. 1149-1157
-
-
Wolfrum, C.1
Shi, S.2
Jayaprakash, K.N.3
Jayaraman, M.4
Wang, G.5
Pandey, R.K.6
Rajeev, K.G.7
Nakayama, T.8
Charrise, K.9
Ndungo, E.M.10
-
24
-
-
41149179660
-
Efficient in vivo delivery of siRNA to the liver by conjugation of α-tocopherol
-
Nishina, K., Unno, T., Uno, Y., Kubodera, T., Kanouchi, T., Mizusawa, H. and Yokota, T. (2008) Efficient in vivo delivery of siRNA to the liver by conjugation of α-tocopherol. Mol. Ther., 16, 734-740.
-
(2008)
Mol. Ther.
, vol.16
, pp. 734-740
-
-
Nishina, K.1
Unno, T.2
Uno, Y.3
Kubodera, T.4
Kanouchi, T.5
Mizusawa, H.6
Yokota, T.7
-
25
-
-
52449107112
-
Effects of cholesterol-tagged small interfering RNAs targeting 12/15-lipoxygenase on parameters of diabetic nephropathy in a mouse model of type 1 diabetes
-
Yuan, H., Lanting, L., Xu, Z.-G., Li, S.-L., Swiderski, P., Putta, S., Jonnalagadda, M., Kato, M. and Natarajan, R. (2008) Effects of cholesterol-tagged small interfering RNAs targeting 12/15-lipoxygenase on parameters of diabetic nephropathy in a mouse model of type 1 diabetes. Am. J. Physiol. Renal Physiol., 295, F605-F617.
-
(2008)
Am. J. Physiol. Renal Physiol.
, vol.295
, pp. F605-F617
-
-
Yuan, H.1
Lanting, L.2
Xu, Z.-G.3
Li, S.-L.4
Swiderski, P.5
Putta, S.6
Jonnalagadda, M.7
Kato, M.8
Natarajan, R.9
-
26
-
-
84982735874
-
Silencing myostatin using cholesterol-conjugated siRNAs induces muscle growth
-
Khan, T., Weber, H., DiMuzio, J., Matter, A., Dogdas, B., Shah, T., Thankappan, A., Disa, J., Jadhav, V., Lubbers, L. et al. (2016) Silencing myostatin using cholesterol-conjugated siRNAs induces muscle growth. Mol. Ther. Nucleic Acids, 5, e342.
-
(2016)
Mol. Ther. Nucleic Acids
, vol.5
, pp. e342
-
-
Khan, T.1
Weber, H.2
DiMuzio, J.3
Matter, A.4
Dogdas, B.5
Shah, T.6
Thankappan, A.7
Disa, J.8
Jadhav, V.9
Lubbers, L.10
-
27
-
-
85058728860
-
RNAi modulation of placental sFLT1 for the treatment of preeclampsia
-
Turanov, A.A., Lo, A., Hassler, M.R., Makris, A., Ashar-Patel, A., Alterman, J.F., Coles, A.H., Haraszti, R.A., Roux, L., Godinho, B.M.D.C. et al. (2018) RNAi modulation of placental sFLT1 for the treatment of preeclampsia. Nat. Biotechnol., doi:10.1038/nbt.4297.
-
(2018)
Nat. Biotechnol.
-
-
Turanov, A.A.1
Lo, A.2
Hassler, M.R.3
Makris, A.4
Ashar-Patel, A.5
Alterman, J.F.6
Coles, A.H.7
Haraszti, R.A.8
Roux, L.9
Godinho, B.M.D.C.10
-
28
-
-
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. PNAS, 104, 17204-17209.
-
(2007)
PNAS
, 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
-
29
-
-
84958582499
-
Transdermal delivery of siRNA through microneedle array
-
Deng, Y., Chen, J., Zhao, Y., Yan, X., Zhang, L., Choy, K., Hu, J., Sant, H.J., Gale, B.K. and Tang, T. (2016) Transdermal delivery of siRNA through microneedle array. Sci. Rep., 6, 1-8.
-
(2016)
Sci. Rep.
, vol.6
, pp. 1-8
-
-
Deng, Y.1
Chen, J.2
Zhao, Y.3
Yan, X.4
Zhang, L.5
Choy, K.6
Hu, J.7
Sant, H.J.8
Gale, B.K.9
Tang, T.10
-
30
-
-
58249094519
-
Cell host & microbe article durable protection from herpes simplex virus-2 transmission following intravaginal application of siRNAs targeting both a viral and host gene
-
Wu, Y., Navarro, F., Lal, A., Basar, E., Pandey, R.K., Manoharan, M., Feng, Y., Lee, S.J., Lieberman, J. and Palliser, D. (2009) Cell host & microbe article durable protection from herpes simplex virus-2 transmission following intravaginal application of siRNAs targeting both a viral and host gene. Cell Host Microbe, 5, 84-94.
-
(2009)
Cell Host Microbe
, vol.5
, pp. 84-94
-
-
Wu, Y.1
Navarro, F.2
Lal, A.3
Basar, E.4
Pandey, R.K.5
Manoharan, M.6
Feng, Y.7
Lee, S.J.8
Lieberman, J.9
Palliser, D.10
-
31
-
-
84948734979
-
Hydrophobically modified siRNAs silence Huntingtin mRNA in primary neurons and mouse brain
-
Alterman, J.F., Hall, L.M., Coles, A.H., Hassler, M.R., Didiot, M.-C., Chase, K., Abraham, J., Sottosanti, E., Johnson, E., Sapp, E. et al. (2015) Hydrophobically modified siRNAs silence Huntingtin mRNA in primary neurons and mouse brain. Mol. Ther. Nucleic Acids, 4, e266.
-
(2015)
Mol. Ther. Nucleic Acids
, vol.4
, pp. e266
-
-
Alterman, J.F.1
Hall, L.M.2
Coles, A.H.3
Hassler, M.R.4
Didiot, M.-C.5
Chase, K.6
Abraham, J.7
Sottosanti, E.8
Johnson, E.9
Sapp, E.10
-
32
-
-
85015168960
-
Docosahexaenoic acid conjugation enhances distribution and safety of siRNA upon local administration in mouse brain
-
Nikan, M., Osborn, M.F., Coles, A.H., Godinho, B.M., Hall, L.M., Haraszti, R.A., Hassler, M.R., Echeverria, D., Aronin, N. and Khvorova, A. (2016) Docosahexaenoic acid conjugation enhances distribution and safety of siRNA upon local administration in mouse brain. Mol. Ther. Nucleic Acids, 5, e344.
-
(2016)
Mol. Ther. Nucleic Acids
, vol.5
, pp. e344
-
-
Nikan, M.1
Osborn, M.F.2
Coles, A.H.3
Godinho, B.M.4
Hall, L.M.5
Haraszti, R.A.6
Hassler, M.R.7
Echeverria, D.8
Aronin, N.9
Khvorova, A.10
-
33
-
-
85021057285
-
Synthesis and evaluation of parenchymal retention and efficacy of a metabolically stable O-Phosphocholine-N-docosahexaenoyl-l-serine siRNA conjugate in mouse brain
-
Nikan, M., Osborn, M.F., Coles, A.H., Biscans, A., Godinho, B.M., Haraszti, R.A., Sapp, E., Echeverria, D., DiFiglia, M., Aronin, N. et al. (2017) Synthesis and evaluation of parenchymal retention and efficacy of a metabolically stable O-Phosphocholine-N-docosahexaenoyl-l-serine siRNA conjugate in mouse brain. Bioconjug. Chem., 28, 758-1766.
-
(2017)
Bioconjug. Chem.
, vol.28
, pp. 758-1766
-
-
Nikan, M.1
Osborn, M.F.2
Coles, A.H.3
Biscans, A.4
Godinho, B.M.5
Haraszti, R.A.6
Sapp, E.7
Echeverria, D.8
DiFiglia, M.9
Aronin, N.10
-
34
-
-
85035318115
-
Pharmacokinetic profiling of conjugated therapeutic oligonucleotides: A high-throughput method based upon serial blood microsampling coupled to Peptide Nucleic Acid hybridization assay
-
Godinho, B.M.D.C., Gilbert, J.W., Haraszti, R.A., Coles, A.H., Biscans, A., Roux, L., Nikan, M., Echeverria, D., Hassler, M. and Khvorova, A. (2017) Pharmacokinetic profiling of conjugated therapeutic oligonucleotides: a high-throughput method based upon serial blood microsampling coupled to Peptide Nucleic Acid hybridization assay. Nculeic Acid Ther., 27, 323-334.
-
(2017)
Nculeic Acid Ther.
, vol.27
, pp. 323-334
-
-
Godinho, B.M.D.C.1
Gilbert, J.W.2
Haraszti, R.A.3
Coles, A.H.4
Biscans, A.5
Roux, L.6
Nikan, M.7
Echeverria, D.8
Hassler, M.9
Khvorova, A.10
-
36
-
-
84964556021
-
A high-throughput method for direct detection of therapeutic oligonucleotide-induced gene silencing in vivo
-
Coles, A.H., Osborn, M.F., Alterman, J.F., Turanov, A.A., Godinho, B.M., Kennington, L., Chase, K., Aronin, N. and Khvorova, A. (2016) A high-throughput method for direct detection of therapeutic oligonucleotide-induced gene silencing in vivo. Nucleic Acid Ther., 26, 86-92.
-
(2016)
Nucleic Acid Ther.
, vol.26
, pp. 86-92
-
-
Coles, A.H.1
Osborn, M.F.2
Alterman, J.F.3
Turanov, A.A.4
Godinho, B.M.5
Kennington, L.6
Chase, K.7
Aronin, N.8
Khvorova, A.9
-
37
-
-
38549173564
-
Membrane lipids: Where they are and how they behave
-
van Meer, G., Voelker, D.R. and Feigenson, G.W. (2008) Membrane lipids: where they are and how they behave. Nat. Rev. Mol. Cell Biol., 9, 112-124.
-
(2008)
Nat. Rev. Mol. Cell Biol.
, vol.9
, pp. 112-124
-
-
Van Meer, G.1
Voelker, D.R.2
Feigenson, G.W.3
-
38
-
-
0014147006
-
Composition of phospholipids and of phospholipid fatty acids of human plasma
-
Phillips, G.B. and Dodges, J.T. (1967) Composition of phospholipids and of phospholipid fatty acids of human plasma. J. Lipid Res., 8, 676-681.
-
(1967)
J. Lipid Res.
, vol.8
, pp. 676-681
-
-
Phillips, G.B.1
Dodges, J.T.2
-
39
-
-
20044396377
-
Activity of stabilized short interfering RNA in a mouse model of hepatitis B virus replication
-
Morrissey, D.V., Blanchard, K., Shaw, L., Jensen, K., Lockridge, J.A., Dickinson, B., McSwiggen, J.A., Vargeese, C., Bowman, K., Shaffer, C.S. et al. (2005) Activity of stabilized short interfering RNA in a mouse model of hepatitis B virus replication. Hepatology, 41, 1349-1356.
-
(2005)
Hepatology
, vol.41
, pp. 1349-1356
-
-
Morrissey, D.V.1
Blanchard, K.2
Shaw, L.3
Jensen, K.4
Lockridge, J.A.5
Dickinson, B.6
McSwiggen, J.A.7
Vargeese, C.8
Bowman, K.9
Shaffer, C.S.10
-
40
-
-
24344500241
-
Sequence, chemical, and structural variation of small interfering RNAs and short hairpin RNAs and the effect on mammalian gene silencing
-
Harbort, J., Elbashir, S.M., Vandenburgh, K., H., M., Scaringe, S.A., Weber, K. and Tuschl, T. (2003) Sequence, chemical, and structural variation of small interfering RNAs and short hairpin RNAs and the effect on mammalian gene silencing. Antisense Nucleic Acid Drug Dev., 13, 83-105.
-
(2003)
Antisense Nucleic Acid Drug Dev.
, vol.13
, pp. 83-105
-
-
Harbort, J.1
Elbashir, S.M.2
Vandenburgh, K.H.M.3
Scaringe, S.A.4
Weber, K.5
Tuschl, T.6
-
41
-
-
0019418445
-
Reversed-phase high performance liquid chromatography of phosphatidylcholine: A simple method for determining relative hydrophobic interaction of various molecular species
-
Smith, M. and Jungalwala, F.B. (1981) Reversed-phase high performance liquid chromatography of phosphatidylcholine: a simple method for determining relative hydrophobic interaction of various molecular species. J. Lipid Res., 22, 697-704.
-
(1981)
J. Lipid Res.
, vol.22
, pp. 697-704
-
-
Smith, M.1
Jungalwala, F.B.2
-
42
-
-
1542336952
-
Rational siRNA design for RNA interference
-
Reynolds, A., Leake, D., Boese, Q., Scaringe, S., Marshall, W.S. and Khvorova, A. (2004) Rational siRNA design for RNA interference. Nat. Biotechnol., 22, 326-330.
-
(2004)
Nat. Biotechnol.
, vol.22
, pp. 326-330
-
-
Reynolds, A.1
Leake, D.2
Boese, Q.3
Scaringe, S.4
Marshall, W.S.5
Khvorova, A.6
-
43
-
-
0029166689
-
Renal uptake of an 18-mer phosphorothioate oligonucleotide
-
Oberbauer, R., Schreiner, G.F. and Meyer, T.W. (1995) Renal uptake of an 18-mer phosphorothioate oligonucleotide. Kidney Int., 48, 1226-1232.
-
(1995)
Kidney Int.
, vol.48
, pp. 1226-1232
-
-
Oberbauer, R.1
Schreiner, G.F.2
Meyer, T.W.3
-
44
-
-
67649271530
-
Antisense oligonucleotide pharmacokinetics and metabolism
-
Geary, R.S. (2009) Antisense oligonucleotide pharmacokinetics and metabolism. Expert Opin. Drug Metab. Toxicol., 5, 381-391.
-
(2009)
Expert Opin. Drug Metab. Toxicol.
, vol.5
, pp. 381-391
-
-
Geary, R.S.1
-
45
-
-
34547900273
-
Forty mouse strain survey of body composition
-
Reed, D.R., Bachmanov, A.A. and Tordoff, M.G. (2007) Forty mouse strain survey of body composition. Physiol. Behav., 91, 593-600.
-
(2007)
Physiol. Behav.
, vol.91
, pp. 593-600
-
-
Reed, D.R.1
Bachmanov, A.A.2
Tordoff, M.G.3
-
46
-
-
0033384970
-
Overgrowth of skin in growth hormone transgenic mice depends on the presence of male gonads
-
Wanke, R., Milz, S., Rieger, N., Ogiolda, L., Renner-Müller, I., Brem, G., Hermanns, W. and Wolf, E. (1999) Overgrowth of skin in growth hormone transgenic mice depends on the presence of male gonads. J. Investig. Dermatol., 113, 967-971.
-
(1999)
J. Investig. Dermatol.
, vol.113
, pp. 967-971
-
-
Wanke, R.1
Milz, S.2
Rieger, N.3
Ogiolda, L.4
Renner-Müller, I.5
Brem, G.6
Hermanns, W.7
Wolf, E.8
-
47
-
-
84945922777
-
Plasmodium berghei ANKA causes intestinal malaria associated with dysbiosis
-
Taniguchi, T., Miyauchi, E., Nakamura, S., Hirai, M., Suzue, K., Imai, T., Nomura, T., Handa, T., Okada, H., Shimokawa, C. et al. (2015) Plasmodium berghei ANKA causes intestinal malaria associated with dysbiosis. Sci. Rep., 5, 1-12.
-
(2015)
Sci. Rep.
, vol.5
, pp. 1-12
-
-
Taniguchi, T.1
Miyauchi, E.2
Nakamura, S.3
Hirai, M.4
Suzue, K.5
Imai, T.6
Nomura, T.7
Handa, T.8
Okada, H.9
Shimokawa, C.10
-
48
-
-
84940759062
-
Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: A phase II multi-dose study
-
Suhr, O.B., Coelho, T., Buades, Pouget, Conceicao, J., Berk, I., Schmidt, J., Waddington-Cruz, H., Campistol, J.M., Bettencourt, B.R. et al. (2015) Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study. Orphanet J. Rare Dis., 10, 1-9.
-
(2015)
Orphanet J. Rare Dis.
, vol.10
, pp. 1-9
-
-
Suhr, O.B.1
Coelho, T.2
Buades, P.3
Conceicao, J.4
Berk, I.5
Schmidt, J.6
Waddington-Cruz, H.7
Campistol, J.M.8
Bettencourt, B.R.9
-
49
-
-
43049176170
-
A combinatorial library of lipid-like materials for delivery of RNAi therapeutics
-
Akinc, A., Zumbuehl, A., Goldberg, M., Leshchiner, E.S., Busini, V., Hossain, N., Bacallado, S.A., N., N.D., Fuller, J., Alvarez, R. et al. (2008) A combinatorial library of lipid-like materials for delivery of RNAi therapeutics. Nat. Biotechnol., 26, 561-569.
-
(2008)
Nat. Biotechnol.
, vol.26
, pp. 561-569
-
-
Akinc, A.1
Zumbuehl, A.2
Goldberg, M.3
Leshchiner, E.S.4
Busini, V.5
Hossain, N.6
Bacallado, S.A.7
NN, D.8
Fuller, J.9
Alvarez, R.10
-
50
-
-
84973889249
-
Influence of particle size on the in vivo potency of lipid nanoparticle formulations of siRNA
-
Chen, S., Tam, Y.Y.C., Lin, P.J.C., Sung, M.M.H., Tam, Y.K. and Cullis, P.R. (2016) Influence of particle size on the in vivo potency of lipid nanoparticle formulations of siRNA. J. Control Release, 235, 236-244.
-
(2016)
J. Control Release
, vol.235
, pp. 236-244
-
-
Chen, S.1
Tam, Y.Y.C.2
Lin, P.J.C.3
Sung, M.M.H.4
Tam, Y.K.5
Cullis, P.R.6
-
51
-
-
85014615816
-
Antisense oligonucleotides used to target the DUX4 mRNA as therapeutic approaches in FaciosScapuloHumeral Muscular Dystrophy (FSHD)
-
Ansseau, E., Vanderplanck, C., Wauters, A., Harper, S.Q., Coppée, F. and Belayew, A. (2017) Antisense oligonucleotides used to target the DUX4 mRNA as therapeutic approaches in FaciosScapuloHumeral Muscular Dystrophy (FSHD). Genes, 8, 1-21.
-
(2017)
Genes
, vol.8
, pp. 1-21
-
-
Ansseau, E.1
Vanderplanck, C.2
Wauters, A.3
Harper, S.Q.4
Coppée, F.5
Belayew, A.6
|