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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 280 scientific papers returned from the database with the search filters currently being used below.
There are 280 scientific papers returned from the database with the search filters currently being used below.
Cardiac and skeletal muscle delivery of biotherapeutics with a blood vessel epicardial substance-targeting peptide
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Citation:
Biomaterials. 2026 Jan 4;329:123986. doi: 10.1016/j.biomaterials.2026.123986. Epub ahead of print. PMID: 41506143 Epub:
Not Epub Abstract:
Although peptide-based delivery strategies show promise for muscle and heart diseases, delivery of biotherapeutics to both... Delivery Method:
BV2 peptide-linked, exosome; injection Organism or Cell Type:
mdx and dystrophin/utrophin double-knockout (DKO) mice Citation Extract: Wang B, Cao J, Wu J, Zhao Y, Zhang Y, Abendroth F, Lin C, Zhong L, Yu H, Seow Y, Ou M, Vázquez O, Mei L, Yin H, Han G. Cardiac and skeletal muscle delivery of biotherapeutics with a blood vessel epicardial substance-targeting peptide. Biomaterials. 2026 Jan 4;329:123986. doi: 10.1016/j.biomaterials.2026.123986. Epub ahead of print. PMID: 41506143. |
Targeted antisense oligonucleotide treatment rescues developmental alterations in spinal muscular atrophy organoids
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Citation:
Nat Commun. 2025 Dec 21. doi: 10.1038/s41467-025-67725-1. Epub ahead of print. PMID: 41423447 Epub:
Not Epub Abstract:
Spinal muscular atrophy (SMA) is a severe neurological disease caused by mutations in the SMN1 gene, characterized by early... Delivery Method:
R6 peptide-linked Organism or Cell Type:
SMA type 1 patient-derived spinal cord and cerebral organoids Citation Extract: Faravelli I, Rinchetti P, Tambalo M, Simutin I, Mapelli L, Mancinelli S, Miotto M, Rizzuti M, D'Angelo A, Cordiglieri C, Forotti G, Peano C, Kunderfranco P, Calandriello L, Comi GP, Paraboschi E, Pali E, Beatrice F, D'Angelo E, Przedborski S, Nizzardo M, Lodato S, Corti S. Targeted antisense oligonucleotide treatment rescues developmental alterations in spinal muscular atrophy organoids. Nat Commun. 2025 Dec 21. doi: 10.1038/s41467-025-67725-1. Epub ahead of print. PMID: 41423447. |
Timing matters: exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy
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Citation:
bioRxiv [preprint] 2025.12.23.696144; doi: https://doi.org/10.64898/2025.12.23.696144 Epub:
Not Epub Abstract:
Exon skipping is a leading therapeutic approach for Duchenne muscular dystrophy (DMD), a progressive muscle wasting disorder... Delivery Method:
Pip6a peptide-linked; intravenous (i.v.) Organism or Cell Type:
mdx mice Citation Extract: Stenler S, Huang J, van Westering TLE, Coenen-Stass AML, Krjutškov K, Andaloussi SEL, Wood MJA, Roberts TC. Timing matters: exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy. bioRxiv [preprint] 2025.12.23.696144; doi: https://doi.org/10.64898/2025.12.23.696144. |
DG9-Conjugated Morpholino-Based Exon 51-Skipping Therapy for Duchenne Muscular Dystrophy
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Citation:
Methods Mol Biol. 2026;2963:1-14. doi: 10.1007/978-1-0716-4738-7_1. PMID: 41028673 Epub:
Not Epub Abstract:
Duchenne muscular dystrophy (DMD) is a severe genetic disorder caused by mutations in the DMD gene that leads to the loss of... Delivery Method:
DG9 peptide-linked; retro-orbital Organism or Cell Type:
mdx mice Citation Extract: Shah MNA, Sutanto LE, Yokota T. DG9-Conjugated Morpholino-Based Exon 51-Skipping Therapy for Duchenne Muscular Dystrophy. Methods Mol Biol. 2026;2963:1-14. doi: 10.1007/978-1-0716-4738-7_1. PMID: 41028673. |
DG9-Conjugated Morpholino Rescues Phenotype in Spinal Muscular Atrophy Mice
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Citation:
Methods Mol Biol. 2026;2963:15-40. doi: 10.1007/978-1-0716-4738-7_2. PMID: 41028674 Epub:
Not Epub Abstract:
Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease characterized by decreased expression of the survival... Delivery Method:
DG9 peptide-linked, subcutaneous (s.c.) Organism or Cell Type:
SMA mice Citation Extract: Haque US, Kohut M, Yokota T. DG9-Conjugated Morpholino Rescues Phenotype in Spinal Muscular Atrophy Mice. Methods Mol Biol. 2026;2963:15-40. doi: 10.1007/978-1-0716-4738-7_2. PMID: 41028674. |
Integrative Multi-Omics Analysis In Vivo Identifies Influenza A Virus Host Factors
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Citation:
iScience. 22 September 2025 Epub:
Not Epub Abstract:
Influenza A virus (IAV) infection remodels cellular processes to support viral replication. Modulation of host factors by the... Delivery Method:
intranasal peptide-linked Organism or Cell Type:
Balb/CJ mice; mouse embryonic fibroblasts (MEFs) Citation Extract: Shriver LP, Polacco B, Eckhardt M, Chang MW, Martínez-Romero C, Adkins-Travis K, Cahalane C, Haas K, Stein DA, Fourati S, Martin-Sancho L, Metreveli G, Johnson JR, Muñoz-Moreno R, Newton BW, Kaake RM, Zhou Y, Jimenez-Morales D, VonDollen J, Verschueren E, Swaney DL, Nguyen TT, Stevenson EJ, Stancliffe E, Patti GJ, Albrecht R, Moulton HM, Pache L, Sekaly RP, Benner C, Hultquist JF, Chanda SK, Garcia Sastre A, Krogan NJ. Integrative Multi-Omics Analysis In Vivo Identifies Influenza A Virus Host Factors. iScience. 22 September 2025. |
Antisense phosphorodiamidate morpholino oligomers retain activity in Burkholderia cepacia complex biofilm
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Citation:
Frontiers in Microbiology. Volume 16 - 2025, 2025. doi: 10.3389/fmicb.2025.1660799 Epub:
Not Epub Abstract:
Background: Members of the Burkholderia cepacia complex (Bcc) are known to cause severe pulmonary infections in... Delivery Method:
peptide-linked Organism or Cell Type:
Burkholderia cepacia Citation Extract: Mendez AR, Pybus C, Greenberg DE. Antisense phosphorodiamidate morpholino oligomers retain activity in Burkholderia cepacia complex biofilm. Frontiers in Microbiology. Volume 16 - 2025, 2025. doi: 10.3389/fmicb.2025.1660799. |
In Vivo Phage Display for the Identification of Muscle Homing Peptides to Improve the Delivery of Phosphorodiamidate Morpholino Oligomers for Duchenne Muscular Dystrophy Therapy
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Citation:
Nucleic Acid Ther. 2025 Aug 29. doi: 10.1177/21593337251371708. Epub ahead of print. PMID: 40879532 Epub:
Not Epub Abstract:
The severe X-linked degenerative neuromuscular disease Duchenne muscular dystrophy (DMD) is caused by the loss of dystrophin... Delivery Method:
peptide-linked Organism or Cell Type:
mdx mice Citation Extract: Schneider AE, Winter CLT, Mei H, Jirka SMG, Tan X, Thompson EG, Ha K, Mitra A, Garcia S, Luimes M, Oliver R, Morgan KY, Guerlavais V, Aartsma-Rus A. In Vivo Phage Display for the Identification of Muscle Homing Peptides to Improve the Delivery of Phosphorodiamidate Morpholino Oligomers for Duchenne Muscular Dystrophy Therapy. Nucleic Acid Ther. 2025 Aug 29. doi: 10.1177/21593337251371708. Epub ahead of print. PMID: 40879532. |
Molecular Properties and Intramolecular interactions of Peptide-Conjugated Phosphorodiamidate Morpholino Oligonucleotides
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Citation:
Molecular Therapy - Nucleic Acids (2025) Epub:
Not Epub Abstract:
We combined Circular Dichroism (CD) and viscosity measurements with MD simulations and classification and regression approaches... Delivery Method:
peptide-linked Citation Extract: Kliuchnikov E, Maksudov F, Pierson D, Marx KA, Chanda A, Barsegov V. Molecular Properties and Intramolecular interactions of Peptide-Conjugated Phosphorodiamidate Morpholino Oligonucleotides. Molecular Therapy - Nucleic Acids (2025). |
Systemic Injection of Peptide-PMOs into Humanized DMD Mice and Detection by RT-PCR and ELISA
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Citation:
Methods Mol Biol. 2025;2964:231-242. doi: 10.1007/978-1-0716-4730-1_15. PMID: 40720022 Epub:
Not Epub Abstract:
Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder due to the lack of dystrophin production. The disease is... Delivery Method:
peptide-linked; retro-orbital injection Organism or Cell Type:
humanized DMD mice Citation Extract: Melo D, Maruyama R, Yokota T. Systemic Injection of Peptide-PMOs into Humanized DMD Mice and Detection by RT-PCR and ELISA. Methods Mol Biol. 2025;2964:231-242. doi: 10.1007/978-1-0716-4730-1_15. PMID: 40720022. |