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

Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy

Authors:
Hammond SM, Hazell G, Shabanpoor F, Saleh AF, Bowerman M, Sleigh JN, Meijboom KE, Zhou H, Muntoni F, Talbot K, Gait MJ, Wood MJ
Citation:
Proc Natl Acad Sci U S A. 2016 Sep 27;113(39):10962-7. doi: 10.1073/pnas.1605731113. Epub 2016 Sep 12
Epub:
Yes
Abstract:
The development of antisense oligonucleotide therapy is an important advance in the identification of corrective therapy for...
Link:
http://www.pnas.org/content/early/2016/09/07/1605731113.long
Delivery Method:
Pip6a peptide-linked; intravenous (i.v.)
Organism or Cell Type:
SH-SY5Y cells; SMA mice
Citation Extract:
Hammond SM, Hazell G, Shabanpoor F, Saleh AF, Bowerman M, Sleigh JN, Meijboom KE, Zhou H, Muntoni F, Talbot K, Gait MJ, Wood MJ. Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy. Proc Natl Acad Sci U S A. 2016 Sep 27;113(39):10962-7. doi: 10.1073/pnas.1605731113. Epub 2016 Sep 12.

Fructose Promotes Uptake and Activity of Oligonucleotides With Different Chemistries in a Context-dependent Manner in mdx Mice

Authors:
Cao L, Han G, Lin C, Gu B, Gao X, Moulton HM, Seow Y, Yin H
Citation:
Mol Ther Nucleic Acids. 2016 Jun 28;5(6):e329. doi: 10.1038/mtna.2016.46
Epub:
Not Epub
Abstract:
Antisense oligonucleotide (AO)-mediated exon-skipping therapeutics shows great promise in correcting frame-disrupting mutations...
Link:
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5022132/
Delivery Method:
bare PMO and (RxR)4 peptide-linked; intravenous (i.v.)
Organism or Cell Type:
mdx mice
Citation Extract:
Cao L, Han G, Lin C, Gu B, Gao X, Moulton HM, Seow Y, Yin H. Fructose Promotes Uptake and Activity of Oligonucleotides With Different Chemistries in a Context-dependent Manner in mdx Mice. Mol Ther Nucleic Acids. 2016 Jun 28;5(6):e329. doi: 10.1038/mtna.2016.46.

Translational Inhibition of CTX-M Extended Spectrum β-Lactamase in Clinical Strains of Escherichia coli by Synthetic Antisense Oligonucleotides Partially Restores Sensitivity to Cefotaxime

Authors:
Readman JB, Dickson G, Coldham NG
Citation:
Front Microbiol. 2016 Mar 24;7:373. eCollection 2016
Epub:
Not Epub
Abstract:
Synthetic antisense oligomers are DNA mimics that can specifically inhibit gene expression at the translational level by...
Link:
http://journal.frontiersin.org/article/10.3389/fmicb.2016.00373/full
Delivery Method:
peptide-linked
Organism or Cell Type:
Escherichia coli
Citation Extract:
Readman JB, Dickson G, Coldham NG. Translational Inhibition of CTX-M Extended Spectrum β-Lactamase in Clinical Strains of Escherichia coli by Synthetic Antisense Oligonucleotides Partially Restores Sensitivity to Cefotaxime. Front Microbiol. 2016 Mar 24;7:373. eCollection 2016.

mRNA and microRNA transcriptomics analyses in a murine model of dystrophin loss and therapeutic restoration

Authors:
Roberts TC, Blomberg KE, Smith CI, El Andaloussi S, Wood MJ
Citation:
Genom Data. 2015 Dec 2;7:88-9. doi: 10.1016/j.gdata.2015.11.025. eCollection 2016 Mar
Epub:
Not Epub
Abstract:
Duchenne muscular dystrophy (DMD) is a pediatric, X-linked, progressive muscle-wasting disorder caused by loss of function...
Link:
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4778658/
Delivery Method:
Pip6e peptide-linked; intravenous (i.v.)
Organism or Cell Type:
mdx mice
Citation Extract:
Roberts TC, Blomberg KE, Smith CI, El Andaloussi S, Wood MJ. mRNA and microRNA transcriptomics analyses in a murine model of dystrophin loss and therapeutic restoration. Genom Data. 2015 Dec 2;7:88-9. doi: 10.1016/j.gdata.2015.11.025. eCollection 2016 Mar.

Deletion of Dystrophin In-Frame Exon 5 Leads to a Severe Phenotype: Guidance for Exon Skipping Strategies

Authors:
Toh ZY, Thandar Aung-Htut M, Pinniger G, Adams AM, Krishnaswarmy S, Wong BL, Fletcher S, Wilton SD
Citation:
PLoS One. 2016 Jan 8;11(1):e0145620. doi: 10.1371/journal.pone.0145620. eCollection 2016
Epub:
Not Epub
Abstract:
Duchenne and Becker muscular dystrophy severity depends upon the nature and location of the DMD gene lesion and generally...
Link:
http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0145620
Delivery Method:
peptide-linked; intraperitoneal (i.p.)
Organism or Cell Type:
C57BL/10 & mdx mice
Citation Extract:
Toh ZY, Thandar Aung-Htut M, Pinniger G, Adams AM, Krishnaswarmy S, Wong BL, Fletcher S, Wilton SD. Deletion of Dystrophin In-Frame Exon 5 Leads to a Severe Phenotype: Guidance for Exon Skipping Strategies. PLoS One. 2016 Jan 8;11(1):e0145620. doi: 10.1371/journal.pone.0145620. eCollection 2016.

Meta- and Orthogonal Integration of Influenza “OMICs” Data Defines a Role for UBR4 in Virus Budding

Authors:
Tripathi S, Pohl MO, Zhou Y, Rodriguez-Frandsen A, Wang G, Stein DA, Moulton HM, DeJesus P, Che J, Mulder LCF, Yángüez E, Andenmatten D, Pache L, Manicassamy B, Albrecht RA, Gonzalez MG, Nguyen Q, Brass A, Elledge S, White M, Shapira S, Hacohen N, Karlas A, Meyer TF, Shales M, Gatorano A, Johnson JR, Jang G, Johnson T, Verschueren E, Sanders D, Krogan N, Shaw M, König R, Stertz S, García-Sastre A, Chanda SK
Citation:
Cell Host & Microbe. 2015;18(6):723-35. doi:10.1016/j.chom.2015.11.002
Epub:
Not Epub
Abstract:
Several systems-level datasets designed to dissect host-pathogen interactions during influenza A infection have been reported....
Link:
http://www.cell.com/cell-host-microbe/abstract/S1931-3128%2815%2900456-4
Delivery Method:
intranasal peptide-linked
Organism or Cell Type:
BALB/c mice
Citation Extract:
Tripathi S, Pohl MO, Zhou Y, Rodriguez-Frandsen A, Wang G, Stein DA, Moulton HM, DeJesus P, Che J, Mulder LCF, Yángüez E, Andenmatten D, Pache L, Manicassamy B, Albrecht RA, Gonzalez MG, Nguyen Q, Brass A, Elledge S, White M, Shapira S, Hacohen N, Karlas A, Meyer TF, Shales M, Gatorano A, Johnson JR, Jang G, Johnson T, Verschueren E, Sanders D, Krogan N, Shaw M, König R, Stertz S, García-Sastre A, Chanda SK. Meta- and Orthogonal Integration of Influenza “OMICs” Data Defines a Role for UBR4 in Virus Budding. Cell Host & Microbe. 2015;18(6):723-35. doi:10.1016/j.chom.2015.11.002.

Identification of novel, therapy-responsive protein biomarkers in a mouse model of Duchenne muscular dystrophy by aptamer-based serum proteomics

Authors:
Coenen-Stass AML, McClorey G, Manzano R, Betts CA, Blain A, Saleh AF, Gait MJ, Lochmüller H, Wood MJA, Roberts TC
Citation:
Sci Rep. 2015;5:17104 doi:10.1038/srep17014
Epub:
Not Epub
Abstract:
There is currently an urgent need for biomarkers that can be used to monitor the efficacy of experimental therapies for...
Link:
http://www.nature.com/articles/srep17014
Delivery Method:
Pip6a peptide-linked; intravenous (i.v.)
Organism or Cell Type:
C57/Bl10 & mdx mice
Citation Extract:
Coenen-Stass AML, McClorey G, Manzano R, Betts CA, Blain A, Saleh AF, Gait MJ, Lochmüller H, Wood MJA, Roberts TC. Identification of novel, therapy-responsive protein biomarkers in a mouse model of Duchenne muscular dystrophy by aptamer-based serum proteomics. Sci Rep. 2015;5:17104 doi:10.1038/srep17014.

Spiroindolone That Inhibits PfATPase4 Is a Potent, Cidal Inhibitor of Toxoplasma gondii Tachyzoites In Vitro and In Vivo

Authors:
Zhou Y, Fomovska A, Muench S, Lai BS, Mui E, McLeod R
Citation:
Antimicrob Agents Chemother. 2014;58(3):1789-92. doi: 10.1128/AAC.02225-13. Epub 2013 Dec 23. PMID: 24366743
Epub:
Not Epub
Abstract:
Here, we show that spiroindolone, an effective treatment for plasmodia, is also active against Toxoplasma gondii tachyzoites....
Link:
http://aac.asm.org/content/early/2013/12/17/AAC.02225-13.abstract
Delivery Method:
peptide-linked
Organism or Cell Type:
Toxoplasma gondii
Citation Extract:
Zhou Y, Fomovska A, Muench S, Lai BS, Mui E, McLeod R. Spiroindolone That Inhibits PfATPase4 Is a Potent, Cidal Inhibitor of Toxoplasma gondii Tachyzoites In Vitro and In Vivo. Antimicrob Agents Chemother. 2014;58(3):1789-92. doi: 10.1128/AAC.02225-13. Epub 2013 Dec 23. PMID: 24366743 .

Targeting protein translation, RNA splicing, and degradation by morpholino-based conjugates in Plasmodium falciparum

Authors:
Garg A, Wesolowski D, Alonso D, Deitsch KW, Ben Mamoun C, Altman S
Citation:
Proc Natl Acad Sci U S A. 2015 Sep 22;112(38):11935-40. doi: 10.1073/pnas.1515864112. Epub 2015 Sep 8.
Epub:
Yes
Abstract:
Identification and genetic validation of new targets from available genome sequences are critical steps toward the development...
Link:
http://www.pnas.org/content/early/2015/09/02/1515864112.abstract
Delivery Method:
Vivo-Morpholino & peptide-linked
Organism or Cell Type:
Plasmodium falciparum
Citation Extract:
Garg A, Wesolowski D, Alonso D, Deitsch KW, Ben Mamoun C, Altman S. Targeting protein translation, RNA splicing, and degradation by morpholino-based conjugates in Plasmodium falciparum. Proc Natl Acad Sci U S A. 2015 Sep 22;112(38):11935-40. doi: 10.1073/pnas.1515864112. Epub 2015 Sep 8..

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...
Link:
http://link.springer.com/protocol/10.1007%2F978-1-4939-2806-4_20
Delivery Method:
Pip6a peptide-linked
Organism or Cell Type:
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.

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