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Morpholino Publication Database

This database contains citations and abstracts for research using Morpholino oligos, as well as some review articles incorporating Morpholino data. You can search the content using the filter boxes below.

There are 261 scientific papers returned from the database with the search filters currently being used below.

In Vitro Assays to Assess Exon Skipping in Duchenne Muscular Dystrophy

Authors:
Boisguerin P, O'Donovan L, Gait MJ, Lebleu B
Citation:
Methods Mol Biol. 2015;1324:317-29. doi: 10.1007/978-1-4939-2806-4_20
Epub:
Not Epub
Abstract:
Cell-penetrating peptide (CPP)-mediated delivery of phosphorodiamidate morpholino oligomers (PMO) results in efficient exon...
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: skeletal H2k cells and primary cardiomyocytes from mdx mice
Citation Extract:
Boisguerin P, O'Donovan L, Gait MJ, Lebleu B. In Vitro Assays to Assess Exon Skipping in Duchenne Muscular Dystrophy. Methods Mol Biol. 2015;1324:317-29. doi: 10.1007/978-1-4939-2806-4_20.

Development and Application of an Ultrasensitive Hybridization-Based ELISA Method for the Determination of Peptide-Conjugated Phosphorodiamidate Morpholino Oligonucleotides

Authors:
Burki U, Keane J, Blain A, O'Donovan L, Gait MJ, Laval SH, Straub V
Citation:
Nucleic Acid Ther. 2015 Jul 15. [Epub ahead of print]
Epub:
Yes
Abstract:
Antisense oligonucleotide (AON)-induced exon skipping is one of the most promising strategies for treating Duchenne muscular...
Delivery Method:
peptide-linked
Citation Extract:
Burki U, Keane J, Blain A, O'Donovan L, Gait MJ, Laval SH, Straub V. Development and Application of an Ultrasensitive Hybridization-Based ELISA Method for the Determination of Peptide-Conjugated Phosphorodiamidate Morpholino Oligonucleotides. Nucleic Acid Ther. 2015 Jul 15. [Epub ahead of print].

Implications for Cardiac Function Following Rescue of the Dystrophic Diaphragm in a Mouse Model of Duchenne Muscular Dystrophy

Authors:
Betts CA, Saleh AF, Carr CA, Muses S, Wells KE, Hammond SM, Godfrey C, McClorey G, Woffindale C, Clarke K, Wells DJ, Gait MJ, Wood MJA
Citation:
Sci Rep. 2015;5:11632. doi:10.1038/srep11632
Epub:
Not Epub
Abstract:
Duchenne muscular dystrophy (DMD) is caused by absence of the integral structural protein, dystrophin, which renders muscle...
Delivery Method:
peptide-linked
Organism or Cell Type:
mice, mdx
Citation Extract:
Betts CA, Saleh AF, Carr CA, Muses S, Wells KE, Hammond SM, Godfrey C, McClorey G, Woffindale C, Clarke K, Wells DJ, Gait MJ, Wood MJA. Implications for Cardiac Function Following Rescue of the Dystrophic Diaphragm in a Mouse Model of Duchenne Muscular Dystrophy. Sci Rep. 2015;5:11632. doi:10.1038/srep11632.

Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice

Authors:
Lu-Nguyen NB, Jarmin SA, Saleh AF, Popplewell L, Gait MJ, Dickson G
Citation:
Mol Ther. 2015;[Epub ahead of print] doi:10.1038/mt.2015.88
Epub:
Yes
Abstract:
The fatal X-linked Duchenne muscular dystrophy (DMD), characterized by progressive muscle wasting and muscle weakness, is...
Delivery Method:
peptide-linked
Organism or Cell Type:
mice, mdx
Citation Extract:
Lu-Nguyen NB, Jarmin SA, Saleh AF, Popplewell L, Gait MJ, Dickson G. Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice. Mol Ther. 2015;[Epub ahead of print] doi:10.1038/mt.2015.88.

Inhibition of hepatitis E virus replication by peptide-conjugated morpholino oligomers

Authors:
Nan Y, Ma Z, Kannan H, Stein DA, Iversen PI, Meng X-J, Zhang Y-J
Citation:
Antiviral Res. 2015;[Epub ahead of print] doi:10.1016/j.antiviral.2015.06.006
Epub:
Yes
Abstract:
Hepatitis E virus (HEV) infection is a cause of hepatitis in humans worldwide and has been associated with a mortality rate of...
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: liver cells
Citation Extract:
Nan Y, Ma Z, Kannan H, Stein DA, Iversen PI, Meng X-J, Zhang Y-J. Inhibition of hepatitis E virus replication by peptide-conjugated morpholino oligomers. Antiviral Res. 2015;[Epub ahead of print] doi:10.1016/j.antiviral.2015.06.006.

Self-assembly into nanoparticles is essential for receptor mediated uptake of therapeutic antisense oligonucleotides

Authors:
Ahmed K, Aoki Y, Koo T, McClorey G, Benner L, Coenen-Stass A, O'Donovan L, Lehto T, Garciaguerra A, Nordin JZ, Saleh AF, Behlke MA, Morris , Goyenvalle A, Dugovic B, Leumann CJ, Gordon S, Gait MJ, El-Andaloussi S, Wood MJA
Citation:
Nano Lett. 2015;[Epub ahead of print] doi:10.1021/acs.nanolett.5b00490
Epub:
Yes
Abstract:
Antisense oligonucleotides (ASOs) have the potential of revolutionizing medicine due to their ability to manipulate gene...
Delivery Method:
peptide-coupled
Organism or Cell Type:
cell culture, mice
Citation Extract:
Ahmed K, Aoki Y, Koo T, McClorey G, Benner L, Coenen-Stass A, O'Donovan L, Lehto T, Garciaguerra A, Nordin JZ, Saleh AF, Behlke MA, Morris , Goyenvalle A, Dugovic B, Leumann CJ, Gordon S, Gait MJ, El-Andaloussi S, Wood MJA. Self-assembly into nanoparticles is essential for receptor mediated uptake of therapeutic antisense oligonucleotides. Nano Lett. 2015;[Epub ahead of print] doi:10.1021/acs.nanolett.5b00490.

Combination antisense treatment for destructive exon skipping of myostatin and open reading frame rescue of dystrophin in neonatal mdx mice

Authors:
Lu-Nguyen NB, Jarmin SA, Saleh AF, Popplewell L, Gait MJ, Dickson G
Citation:
Mol Ther. 2015 May 11. doi: 10.1038/mt.2015.88. [Epub ahead of print]
Epub:
Yes
Abstract:
The fatal X-linked Duchenne muscular dystrophy (DMD), characterized by progressive muscle wasting and muscle weakness, is...
Delivery Method:
peptide-coupled
Organism or Cell Type:
mice, mdx
Citation Extract:
Lu-Nguyen NB, Jarmin SA, Saleh AF, Popplewell L, Gait MJ, Dickson G. Combination antisense treatment for destructive exon skipping of myostatin and open reading frame rescue of dystrophin in neonatal mdx mice. Mol Ther. 2015 May 11. doi: 10.1038/mt.2015.88. [Epub ahead of print].

Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice

Authors:
Betts CA, Saleh AF, Carr CA, Hammond SM, Coenen-Stass AM, Godfrey C, McClorey G, Varela MA, Roberts TC, Clarke K, Gait MJ, Wood MJ
Citation:
Sci Rep. 2015 Mar 11;5:8986. doi: 10.1038/srep08986
Epub:
Not Epub
Abstract:
Duchenne muscular dystrophy (DMD) is a fatal neuromuscular disorder caused by mutations in the Dmd gene. In addition to...
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Betts CA, Saleh AF, Carr CA, Hammond SM, Coenen-Stass AM, Godfrey C, McClorey G, Varela MA, Roberts TC, Clarke K, Gait MJ, Wood MJ. Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice. Sci Rep. 2015 Mar 11;5:8986. doi: 10.1038/srep08986.

High-throughput screening identifies small molecules that enhance the pharmacological effects of oligonucleotides

Authors:
Yang B, Ming X, Cao C, Laing B, Yuan A, Porter MA, Hull-Ryde EA, Maddry J, Suto M, Janzen WP, Juliano RL
Citation:
Nucl Acids Res. 2015;[Epub ahead of print] doi:10.1093/nar/gkv060
Epub:
Yes
Abstract:
The therapeutic use of antisense and siRNA oligonucleotides has been constrained by the limited ability of these membrane-...
Delivery Method:
peptide-coupled
Organism or Cell Type:
cell culture: A375 melanoma cells
Citation Extract:
Yang B, Ming X, Cao C, Laing B, Yuan A, Porter MA, Hull-Ryde EA, Maddry J, Suto M, Janzen WP, Juliano RL. High-throughput screening identifies small molecules that enhance the pharmacological effects of oligonucleotides. Nucl Acids Res. 2015;[Epub ahead of print] doi:10.1093/nar/gkv060 .

Peptide Nanoparticle Delivery of Charge-Neutral Splice-Switching Morpholino Oligonucleotides

Authors:
Järver P, Zaghloul EM, Arzumanov AA, Saleh AF, McClorey G, Hammond SM, Hällbrink M, Langel Ü, Smith CI, Wood MJ, Gait MJ, Andaloussi SE
Citation:
Nucleic Acid Ther. 2015 Jan 16. [Epub ahead of print]
Epub:
Yes
Abstract:
Oligonucleotide analogs have provided novel therapeutics targeting various disorders. However, their poor cellular uptake...
Delivery Method:
peptide nanoparticle
Organism or Cell Type:
cell culture: U2OS cells, murine H2K mdx myotubes, human SMA type 1 fibroblasts
Citation Extract:
Järver P, Zaghloul EM, Arzumanov AA, Saleh AF, McClorey G, Hammond SM, Hällbrink M, Langel Ü, Smith CI, Wood MJ, Gait MJ, Andaloussi SE. Peptide Nanoparticle Delivery of Charge-Neutral Splice-Switching Morpholino Oligonucleotides. Nucleic Acid Ther. 2015 Jan 16. [Epub ahead of print].

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