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

P23 Evaluation of safety parameters and dystrophin expression by sequential administration of exon-skipping and gene therapy in a DMDmdx mouse model

Authors:
Potter R, Cooper Olson GC, Smith L, Greve J, Haile A, Wier C, Snedeker J, Burch P, Hunter B, Malmberg A, Rodino-Klapac L
Citation:
Neuromuscul Disord. 2023;33(Supp 1):S102-3. doi:10.1016/j.nmd.2023.07.150
Epub:
Not Epub
Abstract:
There are promising treatment approaches emerging for Duchenne muscular dystrophy (DMD): exon skipping with peptide-conjugated...
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Potter R, Cooper Olson GC, Smith L, Greve J, Haile A, Wier C, Snedeker J, Burch P, Hunter B, Malmberg A, Rodino-Klapac L. P23 Evaluation of safety parameters and dystrophin expression by sequential administration of exon-skipping and gene therapy in a DMDmdx mouse model. Neuromuscul Disord. 2023;33(Supp 1):S102-3. doi:10.1016/j.nmd.2023.07.150.

Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression

Authors:
González-Martínez I, Cerro-Herreros E, Moreno N, García-Rey A, Espinosa-Espinosa J, Carrascosa-Sàez M, Piqueras-Losilla D, Arzumanov A, Seoane-Miraz D, Jad Y, Raz R, Wood MJ, Varela MA, Llamusí B, Artero R
Citation:
Mol Ther Nucleic Acids. 2023 Sep 5;34:102024. doi: 10.1016/j.omtn.2023.09.001. eCollection 2023 Dec 12
Epub:
Not Epub
Abstract:
Myotonic dystrophy type 1 (DM1) is a rare neuromuscular disease caused by a CTG repeat expansion in the DMPK gene that...
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: DM1, mice
Citation Extract:
González-Martínez I, Cerro-Herreros E, Moreno N, García-Rey A, Espinosa-Espinosa J, Carrascosa-Sàez M, Piqueras-Losilla D, Arzumanov A, Seoane-Miraz D, Jad Y, Raz R, Wood MJ, Varela MA, Llamusí B, Artero R. Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression. Mol Ther Nucleic Acids. 2023 Sep 5;34:102024. doi: 10.1016/j.omtn.2023.09.001. eCollection 2023 Dec 12.

Modulation of Pro-Inflammatory IL-6 Trans-Signaling Axis by Splice Switching Oligonucleotides as a Therapeutic Modality in Inflammation

Authors:
Gupta D, Orehek S, Turunen J, O’Donovan L, Gait MJ, El-Andaloussi S, Wood MJA
Citation:
Cells. 2023;12:2285. doi:10.3390/ cells12182285
Epub:
Not Epub
Abstract:
Interleukin-6 (IL-6) is a pleiotropic cytokine that plays a crucial role in maintaining normal homeostatic processes under the...
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Gupta D, Orehek S, Turunen J, O’Donovan L, Gait MJ, El-Andaloussi S, Wood MJA. Modulation of Pro-Inflammatory IL-6 Trans-Signaling Axis by Splice Switching Oligonucleotides as a Therapeutic Modality in Inflammation. Cells. 2023;12:2285. doi:10.3390/ cells12182285.

Sequence specificity defines the effectiveness of PPMOs targeting Pseudomonas aeruginosa

Authors:
Nanayakkara AK, Moustafa DA, Pifer R, Goldberg JB, Greenberg DE
Citation:
Antimicrob Agents Chemother. 2023 Aug 23:e0024523. doi: 10.1128/aac.00245-23. Online ahead of print
Epub:
Yes
Abstract:
Development of new therapeutics against antibiotic resistant pathogenic bacteria is recognized as a priority across the globe....
Delivery Method:
peptide-linked
Organism or Cell Type:
Pseudomonas aeruginosa (bacterium)
Citation Extract:
Nanayakkara AK, Moustafa DA, Pifer R, Goldberg JB, Greenberg DE. Sequence specificity defines the effectiveness of PPMOs targeting Pseudomonas aeruginosa. Antimicrob Agents Chemother. 2023 Aug 23:e0024523. doi: 10.1128/aac.00245-23. Online ahead of print.

The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of neuromuscular disorders

Authors:
Li X, Kheirabadi M, Dougherty PG, Kamer KJ, Shen X, Estrella NL, Peddigari S, Pathak A, Blake SL, Sizensky E, Genio CD, Gaur AB, Dhanabal M, Girgenrath M, Sethuraman N, Qian Z
Citation:
Mol Ther Nucleic Acids. 2023 Jun 29;33:273-285. doi: 10.1016/j.omtn.2023.06.022. eCollection 2023 Sep 12
Epub:
Not Epub
Abstract:
Biological therapeutic agents are highly targeted and potent but limited in their ability to reach intracellular targets. These...
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Li X, Kheirabadi M, Dougherty PG, Kamer KJ, Shen X, Estrella NL, Peddigari S, Pathak A, Blake SL, Sizensky E, Genio CD, Gaur AB, Dhanabal M, Girgenrath M, Sethuraman N, Qian Z. The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of neuromuscular disorders. Mol Ther Nucleic Acids. 2023 Jun 29;33:273-285. doi: 10.1016/j.omtn.2023.06.022. eCollection 2023 Sep 12.

A comparative analysis of peptide-delivered antisense antibiotics employing diverse nucleotide mimics

Authors:
Ghosh C, Popella L, Dhamodharan V, Jung J, Barquist L, Höbartner C, Vogel J
Citation:
bioRxiv. 2023;[preprint] doi:10.1101/2023.07.11.548539
Epub:
Not Epub
Abstract:
Antisense oligomer (ASO)-based antibiotics that target mRNAs of essential bacterial genes have great potential for...
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: Salmonella enterica serovar Typhimurium strain SL1344
Citation Extract:
Ghosh C, Popella L, Dhamodharan V, Jung J, Barquist L, Höbartner C, Vogel J. A comparative analysis of peptide-delivered antisense antibiotics employing diverse nucleotide mimics. bioRxiv. 2023;[preprint] doi:10.1101/2023.07.11.548539.

Inhibition of Interferon Alpha by a Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer Increases Influenza Virus Replication in Chicken Embryo Fibroblast (DF-1) Cells; An Alternative Strategy to Increasing Vaccine Production in Cell Culture

Authors:
Prescott M, Moulton H, Pastey M
Citation:
J Med Microbiol. 2024 Feb;73(2). doi: 10.1099/jmm.0.001807. PMID: 38353513
Epub:
Not Epub
Abstract:
Influenza is a global health issue causing substantial health and economic burdens in affected populations. Routine, annual...
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: DF-1 chick embryo fibroblasts
Citation Extract:
Prescott M, Moulton H, Pastey M. Inhibition of Interferon Alpha by a Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer Increases Influenza Virus Replication in Chicken Embryo Fibroblast (DF-1) Cells; An Alternative Strategy to Increasing Vaccine Production in Cell Culture. J Med Microbiol. 2024 Feb;73(2). doi: 10.1099/jmm.0.001807. PMID: 38353513.

In-Cell Penetration Selection-Mass Spectrometry Produces Noncanonical Peptides for Antisense Delivery

Authors:
Schissel CK, Farquhar CE, Loas A, Malmberg AB, Pentelute BL
Citation:
ACS Chem Biol. 2023 Mar 1. doi: 10.1021/acschembio.2c00920. Online ahead of print
Epub:
Yes
Abstract:
Peptide-mediated delivery of macromolecules in cells has significant potential therapeutic benefits, but no therapy employing...
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: HeLa
Citation Extract:
Schissel CK, Farquhar CE, Loas A, Malmberg AB, Pentelute BL. In-Cell Penetration Selection-Mass Spectrometry Produces Noncanonical Peptides for Antisense Delivery. ACS Chem Biol. 2023 Mar 1. doi: 10.1021/acschembio.2c00920. Online ahead of print.

DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration

Authors:
Aslesh T, Erkut E, Ren J, Lim KRQ, Woo S, Hatlevig S, Moulton HM, Gosgnach S, Greer J, Maruyama R, Yokota T
Citation:
JCI Insight. 2023;8(5):e160516. doi: 10.1172/jci.insight.160516. Online ahead of print
Epub:
Not Epub
Abstract:
Antisense oligonucleotide (AO)-mediated therapy is a promising strategy to treat several neurological diseases including spinal...
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Aslesh T, Erkut E, Ren J, Lim KRQ, Woo S, Hatlevig S, Moulton HM, Gosgnach S, Greer J, Maruyama R, Yokota T. DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration. JCI Insight. 2023;8(5):e160516. doi: 10.1172/jci.insight.160516. Online ahead of print.

PPMOs: A Case Study for Cell-Penetrating Peptide Application

Authors:
Bittner S, Moulton ZX, Moulton HM
Citation:
In Cell-Penetrating Peptides (eds M. Oba and Y. Demizu). 2023:ch 15. doi:10.1002/9783527835997.ch15
Epub:
Not Epub
Abstract:
The current state of the art for in vivo antisense morpholino technology is peptide-conjugated phosphorodiamidate morpholino...
Delivery Method:
peptide-linked
Citation Extract:
Bittner S, Moulton ZX, Moulton HM. PPMOs: A Case Study for Cell-Penetrating Peptide Application. In Cell-Penetrating Peptides (eds M. Oba and Y. Demizu). 2023:ch 15. doi:10.1002/9783527835997.ch15.

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