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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 268 scientific papers returned from the database with the search filters currently being used below.
There are 268 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
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Citation:
2025. RSC Chemical Biology. 10.1039/D4CB00312H 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. 2025. RSC Chemical Biology. 10.1039/D4CB00312H. |
Long-Term Dystrophin Replacement Therapy in Duchenne Muscular Dystrophy Causes Cardiac Inflammation
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Citation:
J Am Coll Cardiol Basic Trans Science. null2025, 0 (0). https://doi.org/10.1016/j.jacbts.2024.12.015 Epub:
Not Epub Abstract:
Micro-dystrophin replacement gene therapy is currently under clinical trials in Duchenne muscular dystrophy (DMD) patients.... Delivery Method:
peptide-linked combined therapy with AAV-U7; retro-orbital & intravenous (i.v.) Organism or Cell Type:
mice: C57BL/6J, dystrophin−/−; utrophin−/− dKO, mdx (utrophin−/−) Citation Extract: Forand A, Moog S, Mougenot N, Lemaitre M, Sevoz-Couche C, Guesmia Z, Virtanen L, Giordani L, Muchir A, Pietri-Rouxel F. Long-Term Dystrophin Replacement Therapy in Duchenne Muscular Dystrophy Causes Cardiac Inflammation. J Am Coll Cardiol Basic Trans Science. null2025, 0 (0). https://doi.org/10.1016/j.jacbts.2024.12.015. |
Phosphorodiamidate Morpholino Oligomers-Loaded Nanobubbles for Ultrasound-Mediated Delivery to the Myocardium in Muscular Dystrophy
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Citation:
ACS Omega Article ASAP. doi: 10.1021/acsomega.4c10896 Epub:
Not Epub Abstract:
Microbubbles (MBs) and nanobubbles (NBs) can oscillate and collapse in response to ultrasound exposure, resulting in contrast... Delivery Method:
R8 peptide-linked; intravenous (i.v.) injection Organism or Cell Type:
mice: C57BL/10ScSnmdx Citation Extract: Endo-Takahashi Y, Sakurai A, Oguri Y, Katagiri F, Akiyama S, Sashida S, Yamaguchi T, Marunouchi T, Suzuki R, Maruyama K, Tanonaka K, Nomizu M, Negishi Y. Phosphorodiamidate Morpholino Oligomers-Loaded Nanobubbles for Ultrasound-Mediated Delivery to the Myocardium in Muscular Dystrophy. ACS Omega Article ASAP. doi: 10.1021/acsomega.4c10896. |
Targeting a Novel Site in Exon 51 with Antisense Oligonucleotides Induces Enhanced Exon Skipping in a Mouse Model of Duchenne Muscular Dystrophy
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Citation:
Nucleic Acid Ther. 2025 Feb 7. doi: 10.1089/nat.2024.0049. Epub ahead of print. PMID: 39916530 Epub:
Not Epub Abstract:
Exon skipping with antisense oligonucleotides (ASOs) can correct disease-causing mutations of Duchenne muscular dystrophy (DMD... Delivery Method:
peptide-linked or bare oligo intravenous (i.v.) injection Organism or Cell Type:
hDMDdel52/mdx mice Citation Extract: Oppeneer T, Qi Y, Henshaw J, Larimore K, Melton A, Puoliväli J, Carter C, Fant P, Brennan S, Wetzel LA, Sigg MA, Crawford BE, Magat J, Froelich S, Woloszynek JC, O'Neill CA. Targeting a Novel Site in Exon 51 with Antisense Oligonucleotides Induces Enhanced Exon Skipping in a Mouse Model of Duchenne Muscular Dystrophy. Nucleic Acid Ther. 2025 Feb 7. doi: 10.1089/nat.2024.0049. Epub ahead of print. PMID: 39916530. |
A peptide conjugate enables systemic injection of the morpholino inducer and more durable induction of T3H38 ribozyme-controlled AAV transgene in mice
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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... 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
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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... 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)
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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... 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
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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... 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
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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.... 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
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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.... 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. |