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

Cell-penetrating peptide-conjugated, splice-switching oligonucleotides mitigate the phenotype in BTK/Tec double deficient X-linked agammaglobulinemia model

Authors:
Bestas B, Estupiñán HY, Wang Q, Kharazi S, He C, K Mohammad D, Gupta D, Wiklander OPB, Lehto T, Lundin KE, Berglöf A, Karlsson MCI, Abendroth F, El Andaloussi S, Gait MJ, Wood MJA, Leumann CJ, Stetsenko DA, Månsson R, Wengel J, Zain R, Smith CIE
Citation:
RSC Chem Biol. 2025 Mar 31;6(5):761-771. doi: 10.1039/d4cb00312h. PMID: 40171248; PMCID: PMC11955834
Epub:
Not Epub
Abstract:
Splice-switching oligonucleotides (SSOs) have been developed as a treatment for various disorders, including Duchenne muscular...
Delivery Method:
Pip6a peptide-linked; intravenous (i.v.) injection
Organism or Cell Type:
primary B cells from BTK transgenic mice, BTK transgenic mice, BTK/Tec double-deficient mice
Citation Extract:
Bestas B, Estupiñán HY, Wang Q, Kharazi S, He C, K Mohammad D, Gupta D, Wiklander OPB, Lehto T, Lundin KE, Berglöf A, Karlsson MCI, Abendroth F, El Andaloussi S, Gait MJ, Wood MJA, Leumann CJ, Stetsenko DA, Månsson R, Wengel J, Zain R, Smith CIE. Cell-penetrating peptide-conjugated, splice-switching oligonucleotides mitigate the phenotype in BTK/Tec double deficient X-linked agammaglobulinemia model. RSC Chem Biol. 2025 Mar 31;6(5):761-771. doi: 10.1039/d4cb00312h. PMID: 40171248; PMCID: PMC11955834.

An antisense oligomer conjugate with unpredicted bactericidal activity against Fusobacterium nucleatum

Authors:
Cosi V, Jung J, Popella L, Ponath F, Ghosh C, Barquist L, Vogel J
Citation:
mBio. 2025 Apr 29:e0052425. doi: 10.1128/mbio.00524-25. Epub ahead of print. PMID: 40298409
Epub:
Not Epub
Abstract:
Fusobacteria are commensal members of the oral microbiome that can spread from their primary niche and colonize distal sites...
Delivery Method:
(RXR)4XB peptide-linked
Organism or Cell Type:
Fusobacterium nucleatum
Citation Extract:
Cosi V, Jung J, Popella L, Ponath F, Ghosh C, Barquist L, Vogel J. An antisense oligomer conjugate with unpredicted bactericidal activity against Fusobacterium nucleatum. mBio. 2025 Apr 29:e0052425. doi: 10.1128/mbio.00524-25. Epub ahead of print. PMID: 40298409.

Cell-penetrating peptide-conjugated, splice-switching oligonucleotides mitigate the phenotype in BTK/Tec double deficient X-linked agammaglobulinemia model

Authors:
Bestas B, Estupiñán HY, Wang Q, Kharazi S, He C, K Mohammad D, Gupta D, Wiklander OPB, Lehto T, Lundin KE, Berglöf A, Karlsson MCI, Abendroth F, El Andaloussi S, Gait MJ, Wood MJA, Leumann CJ, Stetsenko DA, Månsson R, Wengel J, Zain R, Smith CIE
Citation:
RSC Chem Biol. 2025 Mar 31. doi: 10.1039/d4cb00312h. Epub ahead of print. PMID: 40171248; PMCID: PMC11955834
Epub:
Not Epub
Abstract:
Splice-switching oligonucleotides (SSOs) have been developed as a treatment for various disorders, including Duchenne muscular...
Delivery Method:
Pip6a peptide-linked; intravenous (i.v.) injection
Organism or Cell Type:
cell culture: 2OS luciferase reporter cell line containing a mutated BTK intron 4, primary B cells with mutant BTK transcript; mice: BAC transgenic mice with full-length mutated human BTK pre-mRNA
Citation Extract:
Bestas B, Estupiñán HY, Wang Q, Kharazi S, He C, K Mohammad D, Gupta D, Wiklander OPB, Lehto T, Lundin KE, Berglöf A, Karlsson MCI, Abendroth F, El Andaloussi S, Gait MJ, Wood MJA, Leumann CJ, Stetsenko DA, Månsson R, Wengel J, Zain R, Smith CIE. Cell-penetrating peptide-conjugated, splice-switching oligonucleotides mitigate the phenotype in BTK/Tec double deficient X-linked agammaglobulinemia model. RSC Chem Biol. 2025 Mar 31. doi: 10.1039/d4cb00312h. Epub ahead of print. PMID: 40171248; PMCID: PMC11955834.

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

Authors:
Ghosh C, Popella L, Dhamodharan V, Jung J, Dietzsch J, Barquist L, Höbartner C, Vogel J
Citation:
RNA. 2024 Feb 27:rna.079969.124. doi: 10.1261/rna.079969.124. Online ahead of print
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:
Salmonella
Citation Extract:
Ghosh C, Popella L, Dhamodharan V, Jung J, Dietzsch J, Barquist L, Höbartner C, Vogel J. A comparative analysis of peptide-delivered antisense antibiotics employing diverse nucleotide mimics. RNA. 2024 Feb 27:rna.079969.124. doi: 10.1261/rna.079969.124. Online ahead of print.

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.

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.

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.

Peptide-Conjugated Phosphorodiamidate Morpholino Oligomers Retain Activity against Multidrug-Resistant Pseudomonas aeruginosa In Vitro and In Vivo

Authors:
Moustafa DA, Wu AW, Zamora D, Daly SM, Sturge CR, Pybus C, Geller BL, Goldberg JB, Greenberg DE
Citation:
mBio. 2021;12(1):e02411-20. doi:10.1128/mBio.02411-20
Epub:
Not Epub
Abstract:
Most antimicrobials currently in the clinical pipeline are modifications of existing classes of antibiotics and are considered...
Delivery Method:
peptide-linked
Organism or Cell Type:
Pseudomonas aeruginosa as biofilm or murine pneumonial infection
Citation Extract:
Moustafa DA, Wu AW, Zamora D, Daly SM, Sturge CR, Pybus C, Geller BL, Goldberg JB, Greenberg DE. Peptide-Conjugated Phosphorodiamidate Morpholino Oligomers Retain Activity against Multidrug-Resistant Pseudomonas aeruginosa In Vitro and In Vivo. mBio. 2021;12(1):e02411-20. doi:10.1128/mBio.02411-20.

Peptide Conjugated Morpholinos for Management of The Huanglongbing Pathosystem

Authors:
Sandoval-Mojica AF, Altman S, Hunter WB, Pelz-Stelinski KS
Citation:
Pest Manag Sci. 2020 May 1. doi: 10.1002/ps.5877. [Epub ahead of print]
Epub:
Yes
Abstract:
BACKGROUND: 'Candidatus Liberibacter asiaticus' (CLas) is the causal agent of the devastating citrus disease...
Delivery Method:
peptide-linked
Organism or Cell Type:
Candidatus Liberibacter asiaticus, Wolbachia
Citation Extract:
Sandoval-Mojica AF, Altman S, Hunter WB, Pelz-Stelinski KS. Peptide Conjugated Morpholinos for Management of The Huanglongbing Pathosystem. Pest Manag Sci. 2020 May 1. doi: 10.1002/ps.5877. [Epub ahead of print].

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].

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