-
1
-
-
79955158683
-
Systemic administration of PRO051 in Duchenne's muscular dystrophy
-
Goemans, NM, Tulinius, M, van den Akker, JT, Burm, BE, Ekhart, PF, Heuvelmans, N et al. (2011). Systemic administration of PRO051 in Duchenne's muscular dystrophy. N Engl J Med 364: 1513-1522.
-
(2011)
N Engl J Med
, vol.364
, pp. 1513-1522
-
-
Goemans, N.M.1
Tulinius, M.2
Van Den Akker, J.T.3
Burm, B.E.4
Ekhart, P.F.5
Heuvelmans, N.6
-
2
-
-
80051690306
-
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: An open-label, phase 2, dose-escalation study
-
Cirak, S, Arechavala-Gomeza, V, Guglieri, M, Feng, L, Torelli, S, Anthony, K et al. (2011). Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. Lancet 378: 595-605.
-
(2011)
Lancet
, vol.378
, pp. 595-605
-
-
Cirak, S.1
Arechavala-Gomeza, V.2
Guglieri, M.3
Feng, L.4
Torelli, S.5
Anthony, K.6
-
4
-
-
79960981599
-
Targeting RNA to treat neuromuscular disease
-
Muntoni, F and Wood, MJ (2011). Targeting RNA to treat neuromuscular disease. Nat Rev Drug Discov 10: 621-637.
-
(2011)
Nat Rev Drug Discov
, vol.10
, pp. 621-637
-
-
Muntoni, F.1
Wood, M.J.2
-
5
-
-
61549103435
-
Disability and survival in Duchenne muscular dystrophy
-
Kohler, M, Clarenbach, CF, Bahler, C, Brack, T, Russi, EW and Bloch, KE (2009). Disability and survival in Duchenne muscular dystrophy. J Neurol Neurosurg Psychiatry 80: 320-325.
-
(2009)
J Neurol Neurosurg Psychiatry
, vol.80
, pp. 320-325
-
-
Kohler, M.1
Clarenbach, C.F.2
Bahler, C.3
Brack, T.4
Russi, E.W.5
Bloch, K.E.6
-
6
-
-
0023718118
-
An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus
-
Monaco, AP, Bertelson, CJ, Liechti-Gallati, S, Moser, H and Kunkel, LM (1988). An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus. Genomics 2: 90-95.
-
(1988)
Genomics
, vol.2
, pp. 90-95
-
-
Monaco, A.P.1
Bertelson, C.J.2
Liechti-Gallati, S.3
Moser, H.4
Kunkel, L.M.5
-
7
-
-
0037160782
-
The muscular dystrophies
-
Emery, AE (2002). The muscular dystrophies. Lancet 359: 687-695.
-
(2002)
Lancet
, vol.359
, pp. 687-695
-
-
Emery, A.E.1
-
8
-
-
0036279927
-
Bioavailability and efficacy of antisense morpholino oligomers targeted to c-myc and cytochrome P-450 3A2 following oral administration in rats
-
Arora, V, Knapp, DC, Reddy, MT, Weller, DD and Iversen, PL (2002). Bioavailability and efficacy of antisense morpholino oligomers targeted to c-myc and cytochrome P-450 3A2 following oral administration in rats. J Pharm Sci 91: 1009-1018.
-
(2002)
J Pharm Sci
, vol.91
, pp. 1009-1018
-
-
Arora, V.1
Knapp, D.C.2
Reddy, M.T.3
Weller, D.D.4
Iversen, P.L.5
-
9
-
-
0035119622
-
Absolute bioavailability of 2′-O-(2-methoxyethyl)-modified antisense oligonucleotides following intraduodenal instillation in rats
-
Geary, RS, Khatsenko, O, Bunker, K, Crooke, R, Moore, M, Burckin, T et al. (2001). Absolute bioavailability of 2′-O-(2-methoxyethyl)-modified antisense oligonucleotides following intraduodenal instillation in rats. J Pharmacol Exp Ther 296: 898-904.
-
(2001)
J Pharmacol Exp Ther
, vol.296
, pp. 898-904
-
-
Geary, R.S.1
Khatsenko, O.2
Bunker, K.3
Crooke, R.4
Moore, M.5
Burckin, T.6
-
10
-
-
2642556483
-
Oral bioavailability and multiple dose tolerability of an antisense oligonucleotide tablet formulated with sodium caprate
-
Raoof, AA, Chiu, P, Ramtoola, Z, Cumming, IK, Teng, C, Weinbach, SP et al. (2004). Oral bioavailability and multiple dose tolerability of an antisense oligonucleotide tablet formulated with sodium caprate. J Pharm Sci 93: 1431-1439.
-
(2004)
J Pharm Sci
, vol.93
, pp. 1431-1439
-
-
Raoof, A.A.1
Chiu, P.2
Ramtoola, Z.3
Cumming, I.K.4
Teng, C.5
Weinbach, S.P.6
-
11
-
-
38349171887
-
Oral delivery of antisense oligonucleotides in man
-
Tillman, LG, Geary, RS and Hardee, GE (2008). Oral delivery of antisense oligonucleotides in man. J Pharm Sci 97: 225-236.
-
(2008)
J Pharm Sci
, vol.97
, pp. 225-236
-
-
Tillman, L.G.1
Geary, R.S.2
Hardee, G.E.3
-
12
-
-
0024353559
-
The molecular basis of muscular dystrophy in the mdx mouse: A point mutation
-
Sicinski, P, Geng, Y, Ryder-Cook, AS, Barnard, EA, Darlison, MG and Barnard, PJ (1989). The molecular basis of muscular dystrophy in the mdx mouse: a point mutation. Science 244: 1578-1580.
-
(1989)
Science
, vol.244
, pp. 1578-1580
-
-
Sicinski, P.1
Geng, Y.2
Ryder-Cook, A.S.3
Barnard, E.A.4
Darlison, M.G.5
Barnard, P.J.6
-
13
-
-
84878264662
-
Dose-dependent pharmacokinetic profiles of 2'-O-methyl phosphorothioate antisense oligonucleotidesin mdx mice
-
Verhaart, IE, Tanganyika-de Winter, CL, Karnaoukh, TG, Kolfschoten, IG, de Kimpe, SJ, van Deutekom, JC et al. (2013). Dose-dependent pharmacokinetic profiles of 2'-O-methyl phosphorothioate antisense oligonucleotidesin mdx mice. Nucleic Acid Ther 23: 228-237.
-
(2013)
Nucleic Acid Ther
, vol.23
, pp. 228-237
-
-
Verhaart, I.E.1
Tanganyika-De Winter, C.L.2
Karnaoukh, T.G.3
Kolfschoten, I.G.4
De Kimpe, S.J.5
Van Deutekom, J.C.6
-
14
-
-
84868371403
-
Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment
-
Betts, C, Saleh, AF, Arzumanov, AA, Hammond, SM, Godfrey, C, Coursindel, T et al. (2012). Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment. Mol Ther Nucleic Acids 1: e38.
-
(2012)
Mol Ther Nucleic Acids
, vol.1
, pp. e38
-
-
Betts, C.1
Saleh, A.F.2
Arzumanov, A.A.3
Hammond, S.M.4
Godfrey, C.5
Coursindel, T.6
-
15
-
-
0013181060
-
Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy
-
Mann, CJ, Honeyman, K, McClorey, G, Fletcher, S and Wilton, SD (2002). Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy. J Gene Med 4: 644-654.
-
(2002)
J Gene Med
, vol.4
, pp. 644-654
-
-
Mann, C.J.1
Honeyman, K.2
McClorey, G.3
Fletcher, S.4
Wilton, S.D.5
-
16
-
-
84874737072
-
Size discrimination in rat and mouse gastric emptying
-
Jang, SF, Goins, BA, Phillips, WT, Santoyo, C, Rice-Ficht, A and McConville, JT (2013). Size discrimination in rat and mouse gastric emptying. Biopharm Drug Dispos 34: 107-124.
-
(2013)
Biopharm Drug Dispos
, vol.34
, pp. 107-124
-
-
Jang, S.F.1
Goins, B.A.2
Phillips, W.T.3
Santoyo, C.4
Rice-Ficht, A.5
McConville, J.T.6
-
17
-
-
0036830625
-
Effect of sodium caprate on the intestinal absorption of two modified antisense oligonucleotides in pigs
-
Raoof, AA, Ramtoola, Z, McKenna, B, Yu, RZ, Hardee, G and Geary, RS (2002). Effect of sodium caprate on the intestinal absorption of two modified antisense oligonucleotides in pigs. Eur J Pharm Sci 17: 131-138.
-
(2002)
Eur J Pharm Sci
, vol.17
, pp. 131-138
-
-
Raoof, A.A.1
Ramtoola, Z.2
McKenna, B.3
Yu, R.Z.4
Hardee, G.5
Geary, R.S.6
-
18
-
-
69949107887
-
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: A single-blind, placebo-controlled, dose-escalation, proof-of-concept study
-
Kinali, M, Arechavala-Gomeza, V, Feng, L, Cirak, S, Hunt, D, Adkin, C et al. (2009). Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study. Lancet Neurol 8: 918-928.
-
(2009)
Lancet Neurol
, vol.8
, pp. 918-928
-
-
Kinali, M.1
Arechavala-Gomeza, V.2
Feng, L.3
Cirak, S.4
Hunt, D.5
Adkin, C.6
-
19
-
-
84859439270
-
Phase I clinical trial of Smad7 knockdown using antisense oligonucleotide in patients with active Crohn's disease
-
Monteleone, G, Fantini, MC, Onali, S, Zorzi, F, Sancesario, G, Bernardini, S et al. (2012). Phase I clinical trial of Smad7 knockdown using antisense oligonucleotide in patients with active Crohn's disease. Mol Ther 20: 870-876.
-
(2012)
Mol Ther
, vol.20
, pp. 870-876
-
-
Monteleone, G.1
Fantini, M.C.2
Onali, S.3
Zorzi, F.4
Sancesario, G.5
Bernardini, S.6
-
20
-
-
84893724116
-
Biodistribution and molecular studies on orally administered nanoparticle-AON complexes encapsulated with alginate aiming at inducing dystrophin rescue in mdx mice
-
Falzarano, MS, Passarelli, C, Bassi, E, Fabris, M, Perrone, D, Sabatelli, P et al. (2013). Biodistribution and molecular studies on orally administered nanoparticle-AON complexes encapsulated with alginate aiming at inducing dystrophin rescue in mdx mice. Biomed Res Int 2013: 527418.
-
(2013)
Biomed Res Int
, vol.2013
, pp. 527418
-
-
Falzarano, M.S.1
Passarelli, C.2
Bassi, E.3
Fabris, M.4
Perrone, D.5
Sabatelli, P.6
-
21
-
-
65349121206
-
In vivo comparison of 2′-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping
-
Heemskerk, HA, de Winter, CL, de Kimpe, SJ, van Kuik-Romeijn, P, Heuvelmans, N, Platenburg, GJ et al. (2009). In vivo comparison of 2′-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping. J Gene Med 11: 257-266.
-
(2009)
J Gene Med
, vol.11
, pp. 257-266
-
-
Heemskerk, H.A.1
De Winter, C.L.2
De Kimpe, S.J.3
Van Kuik-Romeijn, P.4
Heuvelmans, N.5
Platenburg, G.J.6
-
22
-
-
71549130146
-
Safety and efficacy of sodium caprate in promoting oral drug absorption: from in vitro to the clinic
-
Maher, S, Leonard, TW, Jacobsen, J and Brayden, DJ (2009). Safety and efficacy of sodium caprate in promoting oral drug absorption: from in vitro to the clinic. Adv Drug Deliv Rev 61: 1427-1449.
-
(2009)
Adv Drug Deliv Rev
, vol.61
, pp. 1427-1449
-
-
Maher, S.1
Leonard, T.W.2
Jacobsen, J.3
Brayden, D.J.4
-
23
-
-
0036570927
-
Development of an ultrasensitive noncompetitive hybridization-ligation enzyme-linked immunosorbent assay for the determination of phosphorothioate oligodeoxynucleotide in plasma
-
Yu, RZ, Baker, B, Chappell, A, Geary, RS, Cheung, E and Levin, AA (2002). Development of an ultrasensitive noncompetitive hybridization-ligation enzyme-linked immunosorbent assay for the determination of phosphorothioate oligodeoxynucleotide in plasma. Anal Biochem 304: 19-25.
-
(2002)
Anal Biochem
, vol.304
, pp. 19-25
-
-
Yu, R.Z.1
Baker, B.2
Chappell, A.3
Geary, R.S.4
Cheung, E.5
Levin, A.A.6
|