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

A peptide conjugate enables systemic injection of the morpholino inducer and more durable induction of T3H38 ribozyme-controlled AAV transgene in mice

Authors:
Tang X, Wang H, Yin Y, Zhong G
Citation:
Gene Ther. 2025 Feb 12. doi: 10.1038/s41434-025-00519-8. Epub ahead of print. PMID: 39939797
Epub:
Not Epub
Abstract:
Genetic switches that allow for precise control over transgene expression timing or levels may improve the safety and expand...
Link:
https://www.nature.com/articles/s41434-025-00519-8
Delivery Method:
peptide-linked, Vivo-Morpholino
Organism or Cell Type:
mouse
Citation Extract:
Tang X, Wang H, Yin Y, Zhong G. A peptide conjugate enables systemic injection of the morpholino inducer and more durable induction of T3H38 ribozyme-controlled AAV transgene in mice. Gene Ther. 2025 Feb 12. doi: 10.1038/s41434-025-00519-8. Epub ahead of print. PMID: 39939797.

Improved induction of ribozyme-controlled AAV transgene via peptide-conjugated morpholino oligos

Authors:
Cheng T, Chen B, Zou W
Citation:
Gene Ther. 2025 Feb 26. doi: 10.1038/s41434-025-00520-1. Epub ahead of print. PMID: 40011707
Epub:
Not Epub
Abstract:
Genetic switches that allow for precise control over transgene expression timing or levels may improve the safety and expand...
Link:
https://www.nature.com/articles/s41434-025-00520-1
Delivery Method:
peptide-linked
Organism or Cell Type:
mouse
Citation Extract:
Cheng T, Chen B, Zou W. Improved induction of ribozyme-controlled AAV transgene via peptide-conjugated morpholino oligos. Gene Ther. 2025 Feb 26. doi: 10.1038/s41434-025-00520-1. Epub ahead of print. PMID: 40011707.

Morpholino oligomer delivery via bath immersion for use in reverse genetic studies on the early development of eastern oysters (Crassostrea virginica)

Authors:
Xu L, Small JM, Hood SM, Zhao M, Plough LV, Wong T-T
Citation:
Aquaculture. Available online 5 February 2025, 742261
Epub:
Not Epub
Abstract:
The eastern oyster genome and expanding omics data have provided valuable insights into this species. However, the limited...
Link:
https://www.sciencedirect.com/science/article/pii/S0044848625001474
Delivery Method:
Vivo-Morpholino, peptide-linked, bathing
Organism or Cell Type:
Crassostrea virginica (eastern oyster)
Citation Extract:
Xu L, Small JM, Hood SM, Zhao M, Plough LV, Wong T-T. Morpholino oligomer delivery via bath immersion for use in reverse genetic studies on the early development of eastern oysters (Crassostrea virginica). Aquaculture. Available online 5 February 2025, 742261.

Unveiling the Role of TMPRSS2 in the Proteolytic Activation of Pandemic and Zoonotic Influenza Viruses and Coronaviruses in Human Airway Cells

Authors:
Schwerdtner M, Schmacke LC, Nave J, Limburg H, Steinmetzer T, Stein DA, Moulton HM, Böttcher-Friebertshäuser E
Citation:
Viruses. 2024 Nov 20;16(11):1798. doi: 10.3390/v16111798. PMID: 39599912; PMCID: PMC11599139
Epub:
Not Epub
Abstract:
The zoonotic transmission of influenza A viruses (IAVs) and coronaviruses (CoVs) may result in severe disease. Cleavage of...
Link:
https://pmc.ncbi.nlm.nih.gov/articles/PMC11599139/
Delivery Method:
(RXR)4 peptide-linked
Organism or Cell Type:
cell culture: Calu-3
Citation Extract:
Schwerdtner M, Schmacke LC, Nave J, Limburg H, Steinmetzer T, Stein DA, Moulton HM, Böttcher-Friebertshäuser E. Unveiling the Role of TMPRSS2 in the Proteolytic Activation of Pandemic and Zoonotic Influenza Viruses and Coronaviruses in Human Airway Cells. Viruses. 2024 Nov 20;16(11):1798. doi: 10.3390/v16111798. PMID: 39599912; PMCID: PMC11599139.

Alternative splicing dysregulation across tissue and therapeutic approaches in a mouse model of myotonic dystrophy type 1

Authors:
Hicks SM, Frias JA, Mishra SK, Scotti M, Muscato DR, Valero MC, Adams LM, Cleary JD, Nakamori M, Wang E, Berglund JA
Citation:
Mol Ther Nucleic Acids. 2024 Sep 13;35(4):102338. doi: 10.1016/j.omtn.2024.102338. PMID: 39391766; PMCID: PMC11465180
Epub:
Not Epub
Abstract:
Myotonic dystrophy type 1 (DM1), the leading cause of adult-onset muscular dystrophy, is caused by a CTG repeat expansion....
Link:
https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(24)00225-7
Delivery Method:
peptide-linked; intramuscular
Organism or Cell Type:
HSA(LR) mice
Citation Extract:
Hicks SM, Frias JA, Mishra SK, Scotti M, Muscato DR, Valero MC, Adams LM, Cleary JD, Nakamori M, Wang E, Berglund JA. Alternative splicing dysregulation across tissue and therapeutic approaches in a mouse model of myotonic dystrophy type 1. Mol Ther Nucleic Acids. 2024 Sep 13;35(4):102338. doi: 10.1016/j.omtn.2024.102338. PMID: 39391766; PMCID: PMC11465180.

Inhibition of SARS-CoV-2 growth in the lungs of mice by a peptide-conjugated morpholino oligomer targeting viral RNA

Authors:
Sakai A, Singh G, Khoshbakht M, Bittner S, Löhr CV, Diaz-Tapia R, Warang P, White K, Le Luo L, Tolbert B, Blanco M, Chow A, Guttman M, Li C, Bao Y, Ho J, Maurer-Stroh S, Chatterjee A, Chanda S, García-Sastre A, Schotsaert M, Teijaro JR, Moulton HM, Stein DA
Citation:
Molecular Therapy: Nucleic Acid (2024), doi: https://doi.org/10.1016/j.omtn.2024.102331
Epub:
Not Epub
Abstract:
Further development of direct-acting antiviral agents against human SARS-CoV-2 infections remains a public health priority....
Link:
https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(24)00218-X
Delivery Method:
peptide-linked; intranasal and intratracheal
Organism or Cell Type:
cell culture: Hela-ACE2; mice: K18-hACE2
Citation Extract:
Sakai A, Singh G, Khoshbakht M, Bittner S, Löhr CV, Diaz-Tapia R, Warang P, White K, Le Luo L, Tolbert B, Blanco M, Chow A, Guttman M, Li C, Bao Y, Ho J, Maurer-Stroh S, Chatterjee A, Chanda S, García-Sastre A, Schotsaert M, Teijaro JR, Moulton HM, Stein DA. Inhibition of SARS-CoV-2 growth in the lungs of mice by a peptide-conjugated morpholino oligomer targeting viral RNA. Molecular Therapy: Nucleic Acid (2024), doi: https://doi.org/10.1016/j.omtn.2024.102331.

Enhancing Peptide and PMO Delivery to Mouse Airway Epithelia by Chemical Conjugation with the Amphiphilic Peptide S10

Authors:
Auger M, Sorroza-Martinez L, Brahiti N, Huppé C-A, Faucher-Giguère L, Arbi I, Hervault M, Cheng X, Gaillet B, Couture F, Guay D, Soultan A-H
Citation:
Molecular Therapy: Nucleic Acid (2024), doi: https://doi.org/10.1016/j.omtn.2024.102290
Epub:
Not Epub
Abstract:
Delivery of antisense oligonucleotides (ASOs) to airway epithelial cells is arduous due to the physiological barriers that...
Link:
https://www.sciencedirect.com/science/article/pii/S216225312400177X
Delivery Method:
intranasal peptide-linked
Organism or Cell Type:
cell culture: HeLa EGFP-654, mice: CAG-EGFP-654
Citation Extract:
Auger M, Sorroza-Martinez L, Brahiti N, Huppé C-A, Faucher-Giguère L, Arbi I, Hervault M, Cheng X, Gaillet B, Couture F, Guay D, Soultan A-H. Enhancing Peptide and PMO Delivery to Mouse Airway Epithelia by Chemical Conjugation with the Amphiphilic Peptide S10. Molecular Therapy: Nucleic Acid (2024), doi: https://doi.org/10.1016/j.omtn.2024.102290.

Comparing cell penetration of biotherapeutics across human cell lines

Authors:
Batistatou N and Kritzer J
Citation:
bioRxiv [preprint] 2024.03.28.587257; doi: https://doi.org/10.1101/2024.03.28.587257
Epub:
Not Epub
Abstract:
A major obstacle in biotherapeutics development is maximizing cell penetration. Ideally, assays would allow for optimization of...
Link:
https://www.biorxiv.org/content/10.1101/2024.03.28.587257v1
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: Hela, Saos-2, U2OS, MIA PaCa-2, HEK293-T, BxPC-3, HepG2
Citation Extract:
Batistatou N and Kritzer J. Comparing cell penetration of biotherapeutics across human cell lines. bioRxiv [preprint] 2024.03.28.587257; doi: https://doi.org/10.1101/2024.03.28.587257.

Efficient systemic CNS delivery of a therapeutic antisense oligonucleotide with a blood-brain barrier-penetrating ApoE-derived peptide

Authors:
Yeoh YQ, Amin A, Cuic B, Tomas D, Turner BJ, Shabanpoor F
Citation:
Biomed Pharmacother. 2024 Jun;175:116737. doi: 10.1016/j.biopha.2024.116737. Epub 2024 May 14. PMID: 38749176
Epub:
Not Epub
Abstract:
Antisense oligonucleotide (ASO) has emerged as a promising therapeutic approach for treating central nervous system (CNS)...
Link:
https://doi.org/10.1016/j.biopha.2024.116737
Delivery Method:
BBB-penetrating peptide-linked
Organism or Cell Type:
SMA Type-I patient-derived fibroblasts, SMN2 transgenic mice
Citation Extract:
Yeoh YQ, Amin A, Cuic B, Tomas D, Turner BJ, Shabanpoor F. Efficient systemic CNS delivery of a therapeutic antisense oligonucleotide with a blood-brain barrier-penetrating ApoE-derived peptide. Biomed Pharmacother. 2024 Jun;175:116737. doi: 10.1016/j.biopha.2024.116737. Epub 2024 May 14. PMID: 38749176.

ACE2 acts as a novel regulator of TMPRSS2-catalyzed proteolytic activation of influenza A virus in airway cells

Authors:
Heindl MR, Rupp A-L, Schwerdtner M, Bestle D, Harbig A, De Rocher A, Schmacke LC, Staker B, Steinmetzer T, Stein DA, Moulton HM, Böttcher-Friebertshäuser E
Citation:
J Virol. 2024 Apr 16;98(4):e0010224. doi: 10.1128/jvi.00102-24
Epub:
Not Epub
Abstract:
The transmembrane serine protease 2 (TMPRSS2) activates the outer structural proteins of a number of respiratory viruses...
Link:
https://journals.asm.org/doi/10.1128/jvi.00102-24
Delivery Method:
(RXR)4 peptide-linked
Organism or Cell Type:
cell culture: Calu-3
Citation Extract:
Heindl MR, Rupp A-L, Schwerdtner M, Bestle D, Harbig A, De Rocher A, Schmacke LC, Staker B, Steinmetzer T, Stein DA, Moulton HM, Böttcher-Friebertshäuser E. ACE2 acts as a novel regulator of TMPRSS2-catalyzed proteolytic activation of influenza A virus in airway cells . J Virol. 2024 Apr 16;98(4):e0010224. doi: 10.1128/jvi.00102-24.

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