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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 257 scientific papers returned from the database with the search filters currently being used below.

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].

Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy

Authors:
Shabanpoor F, McClorey G, Saleh AF, Järver P, Wood MJA, Gait MJ
Citation:
Nucl Acids Res. 2015 Jan;43(1):29-39. doi: 10.1093/nar/gku1256. Epub 2014 Dec 2
Epub:
Not Epub
Abstract:
The potential for therapeutic application of splice-switching oligonucleotides (SSOs) to modulate pre-mRNA splicing is...
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: mouse H2K/mdx myotubes, mice (mdx)
Citation Extract:
Shabanpoor F, McClorey G, Saleh AF, Järver P, Wood MJA, Gait MJ. Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy. Nucl Acids Res. 2015 Jan;43(1):29-39. doi: 10.1093/nar/gku1256. Epub 2014 Dec 2.

Parallel Synthesis of Cell-Penetrating Peptide Conjugates of PMO Toward Exon Skipping Enhancement in Duchenne Muscular Dystrophy

Authors:
O'Donovan L, Okamoto I, Arzumanov AA, Williams DL, Deuss P, Gait MJ
Citation:
Nucleic Acid Ther. 2014 Nov 20. [Epub ahead of print]
Epub:
Yes
Abstract:
We describe two new methods of parallel chemical synthesis of libraries of peptide conjugates of phosphorodiamidate morpholino...
Delivery Method:
peptide-coupled
Organism or Cell Type:
cell culture: murine H2k mdx myoblast
Citation Extract:
O'Donovan L, Okamoto I, Arzumanov AA, Williams DL, Deuss P, Gait MJ. Parallel Synthesis of Cell-Penetrating Peptide Conjugates of PMO Toward Exon Skipping Enhancement in Duchenne Muscular Dystrophy. Nucleic Acid Ther. 2014 Nov 20. [Epub ahead of print].

Wild-type mouse models to screen antisense oligonucleotides for exon-skipping efficacy in duchenne muscular dystrophy

Authors:
Cao L, Han G, Gu B, Yin H
Citation:
PLoS One. 2014 Nov 3;9(11):e111079. doi: 10.1371/journal.pone.0111079. eCollection 2014
Epub:
Not Epub
Abstract:
A readily available animal model is essential for rapidly identifying effective treatments for Duchenne muscular dystrophy (DMD...
Delivery Method:
injection and peptide-coupled
Organism or Cell Type:
mice
Citation Extract:
Cao L, Han G, Gu B, Yin H. Wild-type mouse models to screen antisense oligonucleotides for exon-skipping efficacy in duchenne muscular dystrophy. PLoS One. 2014 Nov 3;9(11):e111079. doi: 10.1371/journal.pone.0111079. eCollection 2014.

Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease

Authors:
Clayton NP, Nelson CA, Weeden T, Taylor KM, Moreland RJ, Scheule RK, Phillips L, Leger AJ, Cheng SH, Wentworth BM
Citation:
Mol Ther Nucleic Acids. 2014 Oct 28;3:e206. doi: 10.1038/mtna.2014.57
Epub:
Not Epub
Abstract:
Pompe disease is an autosomal recessive disorder caused by a deficiency of acid α-glucosidase (GAA; EC 3.2.1.20) and the...
Delivery Method:
injection & electroporation or peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Clayton NP, Nelson CA, Weeden T, Taylor KM, Moreland RJ, Scheule RK, Phillips L, Leger AJ, Cheng SH, Wentworth BM. Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease. Mol Ther Nucleic Acids. 2014 Oct 28;3:e206. doi: 10.1038/mtna.2014.57.

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