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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.

Enhanced delivery of peptide-morpholino oligonucleotides with a small molecule to correct splicing defects in the lung

Authors:
Dang Y, van Heusden C, Nickerson V, Chung F, Wang Y, Quinney NL, Gentzsch M, Randell SH, Moulton HM, Kole R, Ni A, Juliano RL, Kreda SM
Citation:
Nucleic Acids Res. 2021;49(11):6100–13. doi: 10.1093/nar/gkab488. Online ahead of print
Epub:
Not Epub
Abstract:
Pulmonary diseases offer many targets for oligonucleotide therapeutics. However, effective delivery of oligonucleotides to the...
Link:
https://academic.oup.com/nar/advance-article/doi/10.1093/nar/gkab488/6295539
Delivery Method:
peptide-linked plus endosome escape reagent (OEC)
Organism or Cell Type:
cell culture: HeLa EGFP654, Human Bronchial Epithelial, UNCCF8T, EGFP654 Mouse Tracheal Epithelial, mouse
Citation Extract:
Dang Y, van Heusden C, Nickerson V, Chung F, Wang Y, Quinney NL, Gentzsch M, Randell SH, Moulton HM, Kole R, Ni A, Juliano RL, Kreda SM. Enhanced delivery of peptide-morpholino oligonucleotides with a small molecule to correct splicing defects in the lung. Nucleic Acids Res. 2021;49(11):6100–13. doi: 10.1093/nar/gkab488. Online ahead of print.

CX3CR1 is a Receptor for Human Respiratory Syncytial Virus in Cotton Rats

Authors:
Green G, Johnson SM, Costello H, Brakel K, Harder O, Oomens AG, Peeples ME, Moulton HM, Niewiesk S
Citation:
J Virol. 2021 Jul 26;95(16):e0001021. doi: 10.1128/JVI.00010-21. Epub 2021 Jul 26. PMID: 34037420; PMCID: PMC8312862
Epub:
Not Epub
Abstract:
Respiratory syncytial virus (RSV) has been reported to use CX3CR1 in vitro as a receptor on cultured primary human airway...
Link:
https://pmc.ncbi.nlm.nih.gov/articles/PMC8312862/
Delivery Method:
peptide-linked
Organism or Cell Type:
Sigmodon sp. (Cotton rat)
Citation Extract:
Green G, Johnson SM, Costello H, Brakel K, Harder O, Oomens AG, Peeples ME, Moulton HM, Niewiesk S. CX3CR1 is a Receptor for Human Respiratory Syncytial Virus in Cotton Rats. J Virol. 2021 Jul 26;95(16):e0001021. doi: 10.1128/JVI.00010-21. Epub 2021 Jul 26. PMID: 34037420; PMCID: PMC8312862.

A targeted antisense therapeutic approach for Hutchinson–Gilford progeria syndrome

Authors:
Erdos MR, Cabral WA, Tavarez UL, Cao K, Gvozdenovic-Jeremic J, Narisu N, Zerfas PM, Crumley S, Boku Y, Hanson G, Mourich DV, Kole R, Eckhaus MA, Gordon LB, Collins FS
Citation:
Nat Med. 2021 Mar;27(3):536-545. doi: 10.1038/s41591-021-01274-0. Epub 2021 Mar 11.
Epub:
Not Epub
Abstract:
Hutchinson–Gilford progeria syndrome (HGPS) is a rare accelerated aging disorder characterized by premature death from...
Link:
https://www.nature.com/articles/s41591-021-01274-0
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: human progeric fibroblasts, mice
Citation Extract:
Erdos MR, Cabral WA, Tavarez UL, Cao K, Gvozdenovic-Jeremic J, Narisu N, Zerfas PM, Crumley S, Boku Y, Hanson G, Mourich DV, Kole R, Eckhaus MA, Gordon LB, Collins FS. A targeted antisense therapeutic approach for Hutchinson–Gilford progeria syndrome. Nat Med. 2021 Mar;27(3):536-545. doi: 10.1038/s41591-021-01274-0. Epub 2021 Mar 11..

Modular Synthesis of Trifunctional Peptide-oligonucleotide Conjugates via Native Chemical Ligation

Authors:
Dastpeyman M, Karas JA, Amin A, Turner BJ, Shabanpoor F
Citation:
Front Chem. 2021;9:9. doi:10.3389/fchem.2021.627329
Epub:
Not Epub
Abstract:
Cell penetrating peptides (CPPs) are being increasingly used as efficient vectors for intracellular delivery of biologically...
Link:
https://www.frontiersin.org/articles/10.3389/fchem.2021.627329/full
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: spinal muscular atrophy (SMA) patient fibroblasts
Citation Extract:
Dastpeyman M, Karas JA, Amin A, Turner BJ, Shabanpoor F. Modular Synthesis of Trifunctional Peptide-oligonucleotide Conjugates via Native Chemical Ligation. Front Chem. 2021;9:9. doi:10.3389/fchem.2021.627329.

Endosomal escape cell-penetrating peptides significantly enhance pharmacological effectiveness and CNS activity of systemically administered antisense oligonucleotides

Authors:
Dastpeyman M, Sharifi R, Amin A, Karas JA, Cuic B, Pan Y, Nicolazzo JA, Turner BJ, Shabanpoor F
Citation:
Int J Pharm. 2021 Feb 25:120398. doi: 10.1016/j.ijpharm.2021.120398. Online ahead of print
Epub:
Yes
Abstract:
Antisense oligonucleotides (ASOs) are an emerging class of gene-specific therapeutics for diseases associated with the central...
Link:
https://www.sciencedirect.com/science/article/abs/pii/S0378517321002027
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Dastpeyman M, Sharifi R, Amin A, Karas JA, Cuic B, Pan Y, Nicolazzo JA, Turner BJ, Shabanpoor F. Endosomal escape cell-penetrating peptides significantly enhance pharmacological effectiveness and CNS activity of systemically administered antisense oligonucleotides. Int J Pharm. 2021 Feb 25:120398. doi: 10.1016/j.ijpharm.2021.120398. Online ahead of print.

Targeting the 5' untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy

Authors:
Winkelsas AM, Grunseich C, Harmison GG, Chwalenia K, Rinaldi C, Hammond SM, Johnson K, Bowerman M, Arya S, Talbot K, Wood MJ, Fischbeck KH
Citation:
Mol Ther Nucleic Acids. 2021 Jan 5;23:731-742. doi: 10.1016/j.omtn.2020.12.027. eCollection 2021 Mar 5
Epub:
Not Epub
Abstract:
Spinal muscular atrophy (SMA) is a neuromuscular disorder caused by mutations in the survival motor neuron 1 (SMN1) gene. All...
Link:
https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(20)30408-X
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: iPSC
Citation Extract:
Winkelsas AM, Grunseich C, Harmison GG, Chwalenia K, Rinaldi C, Hammond SM, Johnson K, Bowerman M, Arya S, Talbot K, Wood MJ, Fischbeck KH. Targeting the 5' untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy. Mol Ther Nucleic Acids. 2021 Jan 5;23:731-742. doi: 10.1016/j.omtn.2020.12.027. eCollection 2021 Mar 5.

Immortalized Canine Dystrophic Myoblast Cell Lines for Development of Peptide-Conjugated Splice-Switching Oligonucleotides

Authors:
Tone Y, Mamchaoui K, Tsoumpra MK, Hashimoto Y, Terada R, Maruyama R, Gait MJ, Arzumanov AA, McClorey G, Imamura M, Takeda S, Yokota T, Wood MJA, Mouly V, Aoki Y
Citation:
Nucleic Acid Ther. 2021 Feb 9. doi: 10.1089/nat.2020.0907. Online ahead of print
Epub:
Yes
Abstract:
Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disease caused by frameshift or nonsense mutations in the DMD gene...
Link:
https://www.liebertpub.com/doi/10.1089/nat.2020.0907
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture:CXMDJ immortalized myoblasts
Citation Extract:
Tone Y, Mamchaoui K, Tsoumpra MK, Hashimoto Y, Terada R, Maruyama R, Gait MJ, Arzumanov AA, McClorey G, Imamura M, Takeda S, Yokota T, Wood MJA, Mouly V, Aoki Y. Immortalized Canine Dystrophic Myoblast Cell Lines for Development of Peptide-Conjugated Splice-Switching Oligonucleotides. Nucleic Acid Ther. 2021 Feb 9. doi: 10.1089/nat.2020.0907. Online ahead of print.

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...
Link:
https://mbio.asm.org/content/12/1/e02411-20
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.

Discovery and mechanistic characterization of a novel AhR receptor ligand that induces apoptosis in cancer cells

Authors:
O'Donnell E, Jang HS, Liefwalker D, Kerkvliet N, Kolluri SK
Citation:
Research Square. 2020;[preprint] doi:10.21203/rs.3.rs-131951/v1
Epub:
Not Epub
Abstract:
Background: The aryl hydrocarbon receptor (AhR) is a ligand-activated transcription factor and a member of the bHLH/PAS (basic...
Link:
https://www.researchsquare.com/article/rs-131951/v1
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: Hepa 1
Citation Extract:
O'Donnell E, Jang HS, Liefwalker D, Kerkvliet N, Kolluri SK. Discovery and mechanistic characterization of a novel AhR receptor ligand that induces apoptosis in cancer cells. Research Square. 2020;[preprint] doi:10.21203/rs.3.rs-131951/v1.

MOTS‐c promotes phosphorodiamidate morpholino oligomer uptake and efficacy in dystrophic mice

Authors:
Ran N, Lin C, Leng L, Han G, Geng M, Wu Y, Bittner S, Moulton H, Yin H
Citation:
EMBO Mol Med. 2020;13:e12993 doi:10.15252/emmm.202012993
Epub:
Not Epub
Abstract:
Antisense oligonucleotide (AO)‐mediated exon‐skipping therapies show promise in Duchenne muscular dystrophy (DMD), a...
Link:
https://www.embopress.org/doi/full/10.15252/emmm.202012993
Delivery Method:
unlinked MOTS‐c peptide
Organism or Cell Type:
mouse mdx
Citation Extract:
Ran N, Lin C, Leng L, Han G, Geng M, Wu Y, Bittner S, Moulton H, Yin H. MOTS‐c promotes phosphorodiamidate morpholino oligomer uptake and efficacy in dystrophic mice. EMBO Mol Med. 2020;13:e12993 doi:10.15252/emmm.202012993.

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