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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.

The ETS transcription factor ELF1 regulates a broadly antiviral program distinct from the type I interferon response

Authors:
Seifert LL, Si C, Saha D, Sadic M, de Vries M, Ballentine S, Briley A, Wang G, Valero-Jimenez AM, Mohamed A, Schaefer U, Moulton HM, García-Sastre A, Tripathi S, Rosenberg BR, Dittmann M
Citation:
PLoS Pathog. 2019 Nov 4;15(11):e1007634. doi: 10.1371/journal.ppat.1007634. PMID: 31682641; PMCID: PMC6932815
Epub:
Not Epub
Abstract:
Induction of vast transcriptional programs is a central event of innate host responses to viral infections. Here we report a...
Delivery Method:
intranasal peptide-linked
Organism or Cell Type:
BALB/cJ mice
Citation Extract:
Seifert LL, Si C, Saha D, Sadic M, de Vries M, Ballentine S, Briley A, Wang G, Valero-Jimenez AM, Mohamed A, Schaefer U, Moulton HM, García-Sastre A, Tripathi S, Rosenberg BR, Dittmann M. The ETS transcription factor ELF1 regulates a broadly antiviral program distinct from the type I interferon response. PLoS Pathog. 2019 Nov 4;15(11):e1007634. doi: 10.1371/journal.ppat.1007634. PMID: 31682641; PMCID: PMC6932815.

Multiplex in situ hybridization within a single transcript: RNAscope reveals dystrophin mRNA dynamics

Authors:
Hildyard JCW, Rawson F, Wells DJ, Piercy RJ
Citation:
bioRxiv. 2019;[preprint] doi:10.1101/791780
Epub:
Not Epub
Abstract:
Dystrophin plays a vital role in maintaining muscle health, yet low mRNA expression, lengthy transcription time and the...
Delivery Method:
PIP6a peptide-linked
Organism or Cell Type:
mdx mice
Citation Extract:
Hildyard JCW, Rawson F, Wells DJ, Piercy RJ. Multiplex in situ hybridization within a single transcript: RNAscope reveals dystrophin mRNA dynamics. bioRxiv. 2019;[preprint] doi:10.1101/791780 .

Hexose potentiates peptide-conjugated morpholino oligomer efficacy in cardiac muscles of dystrophic mice in an age-dependent manner

Authors:
Han G, Gu B, Lin C, Ning H, Song J, Gao X, Moulton HM, Yin HF
Citation:
Mol Ther Nucl Acids. 2019 Sep 23;18:341-350. doi: 10.1016/j.omtn.2019.09.012. [Epub ahead of print]
Epub:
Not Epub
Abstract:
Insufficient delivery of oligonucleotides to muscle and heart remains a barrier for clinical implementation of antisense...
Delivery Method:
peptide-linked; intravenous (i.v.)
Organism or Cell Type:
mice mdx
Citation Extract:
Han G, Gu B, Lin C, Ning H, Song J, Gao X, Moulton HM, Yin HF. Hexose potentiates peptide-conjugated morpholino oligomer efficacy in cardiac muscles of dystrophic mice in an age-dependent manner. Mol Ther Nucl Acids. 2019 Sep 23;18:341-350. doi: 10.1016/j.omtn.2019.09.012. [Epub ahead of print].

Reduction of integrin alpha 4 activity through splice modulating antisense oligonucleotides

Authors:
Aung-Htut MT, Comerford I, Johnsen R, Foyle K, Fletcher S, Wilton SD
Citation:
Sci Rep. 2019;9(1):12994. doi:10.1038/s41598-019-49385-6
Epub:
Not Epub
Abstract:
With recent approvals of antisense oligonucleotides as therapeutics, there is an increasing interest in expanding the...
Delivery Method:
in vitro lipofectamine (fibroblasts) electroporation (Jurkat) , in vivo peptide-linked
Organism or Cell Type:
cell culture: primary dermal fibroblasts, Jurkat; autoimmune encephalomyelitis mouse model of multiple sclerosis
Citation Extract:
Aung-Htut MT, Comerford I, Johnsen R, Foyle K, Fletcher S, Wilton SD. Reduction of integrin alpha 4 activity through splice modulating antisense oligonucleotides. Sci Rep. 2019;9(1):12994. doi:10.1038/s41598-019-49385-6.

Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice

Authors:
Klein AF, Varela MA, Arandel L, Holland A, Naouar N, Arzumanov A, Seoane D, Revillod L, Bassez G, Ferry A, Jauvin D, Gourdon G, Puymirat J, Gait MJ, Furling D, Wood MJA
Citation:
J Clin Invest. 2019 Nov 1;129(11):4739-4744. doi: 10.1172/JCI128205
Epub:
Not Epub
Abstract:
Antisense oligonucleotides (ASOs) targeting pathologic RNAs have shown promising therapeutic corrections for many genetic...
Delivery Method:
Pip6a peptide-linked; intravenous (i.v.) injection for mice
Organism or Cell Type:
patient-derived muscle cells; DM1 mice
Citation Extract:
Klein AF, Varela MA, Arandel L, Holland A, Naouar N, Arzumanov A, Seoane D, Revillod L, Bassez G, Ferry A, Jauvin D, Gourdon G, Puymirat J, Gait MJ, Furling D, Wood MJA. Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice. J Clin Invest. 2019 Nov 1;129(11):4739-4744. doi: 10.1172/JCI128205.

Chimeras of Cell-Penetrating Peptides Demonstrate Synergistic Improvement in Antisense Efficacy

Authors:
Fadzen CM, Holden RL, Wolfe JM, Choo ZN, Schissel CK, Yao M, Hanson GJ, Pentelute BL
Citation:
Biochemistry. 2019 Sep 24;58(38):3980-3989. doi: 10.1021/acs.biochem.9b00413. Epub 2019 Sep 6. PMID: 31450889
Epub:
Yes
Abstract:
Phosphorodiamidate morpholino oligonucleotides (PMOs) make up a promising class of therapeutics for genetic disease. PMOs...
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: HeLa-654
Citation Extract:
Fadzen CM, Holden RL, Wolfe JM, Choo ZN, Schissel CK, Yao M, Hanson GJ, Pentelute BL. Chimeras of Cell-Penetrating Peptides Demonstrate Synergistic Improvement in Antisense Efficacy. Biochemistry. 2019 Sep 24;58(38):3980-3989. doi: 10.1021/acs.biochem.9b00413. Epub 2019 Sep 6. PMID: 31450889.

TMPRSS2 is the major activating protease of influenza A virus in primary human airway cells and influenza B virus in human type II pneumocytes

Authors:
Limburg H, Harbig A, Bestle D, Stein DA, Moulton HM, Jaeger J, Janga H, Hardes K, Koepke J, Schulte L, Koczulla AR, Schmeck B, Klenk HD, Böttcher-Friebertshäuser E
Citation:
J Virol. 2019 Oct 15;93(21):e00649-19. doi: 10.1128/JVI.00649-19. PMID: 31391268; PMCID: PMC6803253
Epub:
Yes
Abstract:
Cleavage of influenza virus hemagglutinin (HA) by host cell proteases is essential for virus infectivity and spread. We...
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: primary human bronchial epithelial cells (HBEC), primary type II alveolar epithelial cells (AECII) and Calu-3 cells
Citation Extract:
Limburg H, Harbig A, Bestle D, Stein DA, Moulton HM, Jaeger J, Janga H, Hardes K, Koepke J, Schulte L, Koczulla AR, Schmeck B, Klenk HD, Böttcher-Friebertshäuser E. TMPRSS2 is the major activating protease of influenza A virus in primary human airway cells and influenza B virus in human type II pneumocytes. J Virol. 2019 Oct 15;93(21):e00649-19. doi: 10.1128/JVI.00649-19. PMID: 31391268; PMCID: PMC6803253.

Global analysis of polysome-associated mRNA in vesicular stomatitis virus infected cells

Authors:
Neidermyer WJ Jr, Whelan SPJ
Citation:
PLoS Pathog. 2019;15(6):e1007875. doi:10.1371/journal.ppat.1007875
Epub:
Not Epub
Abstract:
Infection of mammalian cells with vesicular stomatitis virus (VSV) results in the inhibition of cellular translation while...
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: HeLa
Citation Extract:
Neidermyer WJ Jr, Whelan SPJ. Global analysis of polysome-associated mRNA in vesicular stomatitis virus infected cells. PLoS Pathog. 2019;15(6):e1007875. doi:10.1371/journal.ppat.1007875.

AcrAB-TolC Inhibition by Peptide-conjugated Phosphorodiamidate Morpholino Oligomers Restores Antibiotic Activity in vitro and in vivo

Authors:
Sturge CR, Felder-Scott CF, Pifer R, Pybus C, Jain R, Geller B, Greenberg D
Citation:
ACS Infect Dis. 2019 May 23. doi: 10.1021/acsinfecdis.9b00123. [Epub ahead of print]
Epub:
Yes
Abstract:
Overexpression of bacterial efflux pumps are a driver of increasing antibiotic resistance in gram-negative pathogens. The AcrAB...
Delivery Method:
peptide-linked
Organism or Cell Type:
Enterobacteriaceae strains, Klebsiella pneumoniae, Pseudomonas aeruginosa
Citation Extract:
Sturge CR, Felder-Scott CF, Pifer R, Pybus C, Jain R, Geller B, Greenberg D. AcrAB-TolC Inhibition by Peptide-conjugated Phosphorodiamidate Morpholino Oligomers Restores Antibiotic Activity in vitro and in vivo. ACS Infect Dis. 2019 May 23. doi: 10.1021/acsinfecdis.9b00123. [Epub ahead of print].

Scavenger receptor class A1 mediates uptake of morpholino antisense oligonucleotide into dystrophic skeletal muscle

Authors:
Miyatake S, Mizobe Y, Tsoumpra M, Lim KRQ, Hara Y, Shabanpoor F, Yokota T, Takeda S, Aoki Y
Citation:
Mol Ther Nucl Acids. 2019 Jan 25;14:520-535. doi: 10.1016/j.omtn.2019.01.008.
Epub:
Not Epub
Abstract:
Exon-skipping using phosphorodiamidate morpholino oligomers (PMOs) is a promising treatment strategy for Duchenne muscular...
Delivery Method:
Pip6a peptide-linked, cell bathing, intramuscular (i.m.) injection for mice
Organism or Cell Type:
H2K-mdx 52 myoblast cells; mdx52 & SR-A1/2 and dystrophin double knockout mice
Citation Extract:
Miyatake S, Mizobe Y, Tsoumpra M, Lim KRQ, Hara Y, Shabanpoor F, Yokota T, Takeda S, Aoki Y. Scavenger receptor class A1 mediates uptake of morpholino antisense oligonucleotide into dystrophic skeletal muscle. Mol Ther Nucl Acids. 2019 Jan 25;14:520-535. doi: 10.1016/j.omtn.2019.01.008. .

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