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

DG9-Conjugated Morpholino-Based Exon 51-Skipping Therapy for Duchenne Muscular Dystrophy

Authors:
Shah MNA, Sutanto LE, Yokota T
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

Authors:
Haque US, Kohut M, Yokota T
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

Authors:
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
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

Authors:
Mendez AR, Pybus C, Greenberg DE
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

Authors:
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
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

Authors:
Kliuchnikov E, Maksudov F, Pierson D, Marx KA, Chanda A, Barsegov V
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

Authors:
Melo D, Maruyama R, Yokota T
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.

Using muscle homing peptide CyPep10 to deliver phosphorodiamidate morpholino oligomers in the mdx mouse

Authors:
Schneider AE, Tanganyika-de Winter CL, Jirka SMG, Tan X, Thompson EG, Ha K, Mitra A, Garcia S, Luimes M, Oliver RA, Guerlavais V, Aartsma-Rus A
Citation:
Mol Ther Nucleic Acids. 2025 Jul 17;36(3):102625. doi: 10.1016/j.omtn.2025.102625. PMID: 40777739; PMCID: PMC12329520
Epub:
Not Epub
Abstract:
The severe muscle wasting disorder Duchenne muscular dystrophy (DMD) is characterized by the absence of dystrophin, a protein...
Delivery Method:
CP10 and acetyl-R6Gly peptide-linked; intravenous (i.v.)
Organism or Cell Type:
mdx mice
Citation Extract:
Schneider AE, Tanganyika-de Winter CL, Jirka SMG, Tan X, Thompson EG, Ha K, Mitra A, Garcia S, Luimes M, Oliver RA, Guerlavais V, Aartsma-Rus A. Using muscle homing peptide CyPep10 to deliver phosphorodiamidate morpholino oligomers in the mdx mouse. Mol Ther Nucleic Acids. 2025 Jul 17;36(3):102625. doi: 10.1016/j.omtn.2025.102625. PMID: 40777739; PMCID: PMC12329520.

Exon skipping peptide-conjugated morpholinos downregulate dynamin 2 to rescue centronuclear myopathy

Authors:
Moschovaki-Filippidou F, de Carvalho Neves J, Diedhiou N, Jad Y, Böhm J, Wood MJA, Varela MA, Laporte J
Citation:
Brain. 2025 Jul 3:awaf249. doi: 10.1093/brain/awaf249. Epub ahead of print. PMID: 40608957
Epub:
Not Epub
Abstract:
Centronuclear myopathies (CNM) are rare congenital disorders characterized by muscle weakness and disorganization of myofibers...
Delivery Method:
Pip8b2 peptide-linked; intravenous (i.v.) and intramuscular (i.m.) injection into the tibialis anterior (TA) for mice
Organism or Cell Type:
C2C12 mouse myoblasts; Dnm2R369W/+ mice
Citation Extract:
Moschovaki-Filippidou F, de Carvalho Neves J, Diedhiou N, Jad Y, Böhm J, Wood MJA, Varela MA, Laporte J. Exon skipping peptide-conjugated morpholinos downregulate dynamin 2 to rescue centronuclear myopathy. Brain. 2025 Jul 3:awaf249. doi: 10.1093/brain/awaf249. Epub ahead of print. PMID: 40608957.

Regulatory T Cell Function Is Not Affected by Antisense Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer (PPMO)-Mediated TMPRSS2 Truncation

Authors:
Gunne S, Sailer FK, Keutmann L, Schwerdtner M, Moulton HM, Böttcher-Friebertshäuser E, Schiffmann S
Citation:
Drugs and Drug Candidates. 2025; 4(2):25. https://doi.org/10.3390/ddc4020025
Epub:
Not Epub
Abstract:
Background: TMPRSS2 plays an important role in the viral entry mechanisms of influenza viruses and coronaviruses. Therefore,...
Delivery Method:
peptide-linked
Organism or Cell Type:
Human regulatory T cells (Treg) and responder T cells
Citation Extract:
Gunne S, Sailer FK, Keutmann L, Schwerdtner M, Moulton HM, Böttcher-Friebertshäuser E, Schiffmann S. Regulatory T Cell Function Is Not Affected by Antisense Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer (PPMO)-Mediated TMPRSS2 Truncation. Drugs and Drug Candidates. 2025; 4(2):25. https://doi.org/10.3390/ddc4020025.

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