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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 12371 scientific papers returned from the database with the search filters currently being used below.
There are 12371 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:
RSC Chem Biol. 2025 Mar 31;6(5):761-771. doi: 10.1039/d4cb00312h. PMID: 40171248; PMCID: PMC11955834 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:
primary B cells from BTK transgenic mice, BTK transgenic mice, BTK/Tec double-deficient mice 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. RSC Chem Biol. 2025 Mar 31;6(5):761-771. doi: 10.1039/d4cb00312h. PMID: 40171248; PMCID: PMC11955834. |
Protein-O-fucosylation of coreceptors may be required for Nodal signaling in Xenopus
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Citation:
Mol Cells. 2025 May;48(5):100207. doi: 10.1016/j.mocell.2025.100207. Epub 2025 Mar 3. PMID: 40043779; PMCID: PMC11964751 Epub:
Not Epub Abstract:
Nodal-related ligands of TGF-β family play pivotal roles for mesoderm induction and body axis formation during vertebrate early... Delivery Method:
microinjection Organism or Cell Type:
Xenopus laevis Citation Extract: Kim YJ, Nho SJ, Lee SY, Yeo CY. Protein-O-fucosylation of coreceptors may be required for Nodal signaling in Xenopus. Mol Cells. 2025 May;48(5):100207. doi: 10.1016/j.mocell.2025.100207. Epub 2025 Mar 3. PMID: 40043779; PMCID: PMC11964751. |
An antisense oligomer conjugate with unpredicted bactericidal activity against Fusobacterium nucleatum
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Citation:
mBio. 2025 Apr 29:e0052425. doi: 10.1128/mbio.00524-25. Epub ahead of print. PMID: 40298409 Epub:
Not Epub Abstract:
Fusobacteria are commensal members of the oral microbiome that can spread from their primary niche and colonize distal sites... Delivery Method:
(RXR)4XB peptide-linked Organism or Cell Type:
Fusobacterium nucleatum Citation Extract: Cosi V, Jung J, Popella L, Ponath F, Ghosh C, Barquist L, Vogel J. An antisense oligomer conjugate with unpredicted bactericidal activity against Fusobacterium nucleatum. mBio. 2025 Apr 29:e0052425. doi: 10.1128/mbio.00524-25. Epub ahead of print. PMID: 40298409. |
Role of ZFHX4 in orofacial clefting based on human genetic data and zebrafish models
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Citation:
Eur J Hum Genet. 2025 May;33(5):595-606. doi: 10.1038/s41431-024-01775-9. Epub 2024 Dec 19. PMID: 39702590; PMCID: PMC7617551 Epub:
Not Epub Abstract:
Orofacial clefting (OFC) is a frequent congenital anomaly and can occur either in the context of underlying syndromes or in... Delivery Method:
microinjection Organism or Cell Type:
zebrafish Citation Extract: Ishorst N, Hölzel S, Greve C, Yilmaz Ö, Lindenberg T, Lambertz J, Drichel D, Zametica B, Mingardo E, Kalanithy JC, Channab K, Kibris D, Henne S, Degenhardt F, Siewert A, Dixon M, Kruse T, Ongkosuwito E, Girisha KM, Pande S, Nowak S, Hagelueken G, Geyer M, Carels C, van Rooij IALM, Ludwig KU, Odermatt B, Mangold E. Role of ZFHX4 in orofacial clefting based on human genetic data and zebrafish models. Eur J Hum Genet. 2025 May;33(5):595-606. doi: 10.1038/s41431-024-01775-9. Epub 2024 Dec 19. PMID: 39702590; PMCID: PMC7617551. |
Disruption of the moonlighting function of CTF18 in a patient with T-lymphopenia
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Citation:
Front Immunol. 2025 Feb 14;16:1539848. doi: 10.3389/fimmu.2025.1539848. PMID: 40028343; PMCID: PMC11868726 Epub:
Not Epub Abstract:
Introduction: Newborn screening for immunodeficiency has led to the identification of numerous cases for which the causal... Delivery Method:
microinjection Organism or Cell Type:
zebrafish Citation Extract: Sertori R, Truong B, Singh MK, Shinton S, Price R, Sharo A, Shultes P, Sunderam U, Rana S, Srinivasan R, Datta S, Font-Burgada J, Brenner SE, Puck JM, Wiest DL. Disruption of the moonlighting function of CTF18 in a patient with T-lymphopenia. Front Immunol. 2025 Feb 14;16:1539848. doi: 10.3389/fimmu.2025.1539848. PMID: 40028343; PMCID: PMC11868726. |
Dynactin knockdown leads to synuclein aggregation by blocking autophagy in a zebrafish model of Parkinson's disease
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Citation:
Braz J Med Biol Res. 2025 Mar 24;58:e14282. doi: 10.1590/1414-431X2025e14282. PMID: 40136228 Epub:
Not Epub Abstract:
Axons of dopaminergic neurons projecting from substantia nigra to striatum are severely affected in the early stage of... Delivery Method:
microinjection Organism or Cell Type:
zebrafish Citation Extract: Wu Y, Guo Q, Gan J, Duan L, Zhao H, Tai H, Yang C, Li Y, Xu Z, Yao Y, Nie Z, Yang M, Li S, Li J, Su B. Dynactin knockdown leads to synuclein aggregation by blocking autophagy in a zebrafish model of Parkinson's disease. Braz J Med Biol Res. 2025 Mar 24;58:e14282. doi: 10.1590/1414-431X2025e14282. PMID: 40136228. |
AOC 1044 induces exon 44 skipping and restores dystrophin protein in preclinical models of Duchenne muscular dystrophy
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Citation:
Nucleic Acids Res. 2025 Mar 20;53(6):gkaf241. doi: 10.1093/nar/gkaf241. PMID: 40183632; PMCID: PMC11969676 Epub:
Not Epub Abstract:
Duchenne muscular dystrophy (DMD) is a severe disorder caused by mutations in the dystrophin gene, resulting in loss of... Delivery Method:
antibody-linked; intravenous (i.v.) injection Organism or Cell Type:
Primary human skeletal muscle cells, hDMDdel45/mdx transgenic mice, Cynomolgus monkeys Citation Extract: Etxaniz U, Marks I, Albin T, Diaz M, Bhardwaj R, Anderson A, Tyaglo O, Hoang T, Missinato MA, Svensson K, Badillo B, Kovach PR, Leung L, Cochran M, Kwon HW, Ahad Shah MN, Maruyama R, Yokota T, Doppalapudi VR, Darimont B, Younis HS, Flanagan WM, Levin AA, Huang H, Karamanlidis G. AOC 1044 induces exon 44 skipping and restores dystrophin protein in preclinical models of Duchenne muscular dystrophy. Nucleic Acids Res. 2025 Mar 20;53(6):gkaf241. doi: 10.1093/nar/gkaf241. PMID: 40183632; PMCID: PMC11969676. |
Splicing correction by peptide-conjugated morpholinos as a novel treatment for late-onset Pompe disease
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Citation:
Mol Ther Nucleic Acids. 2025 Mar 27;36(2):102524. doi: 10.1016/j.omtn.2025.102524. PMID: 40255904; PMCID: PMC12008586 Epub:
Not Epub Abstract:
Late-onset Pompe disease (LOPD) is overwhelmingly caused by a single mutation that disrupts splicing of acid-alpha glucosidase... Delivery Method:
Ac-N-Arg6-Gly-OH peptide-linked Organism or Cell Type:
IPSC-derived muscle cells and skeletal muscle tissue; Late-onset Pompe disease mouse model Citation Extract: Oliver RA, Ahern ME, Castaneda PG, Jinadasa T, Bardhan A, Morgan KY, Ha K, Adhikari K, Jungels N, Liberman N, Mitra A, Greer CD, Wright AM, Thompson EG, Garcia S, Copson E, Allu S, Tan X, Callahan AJ, Cai BZ, Guerlavais V, Kim KJ, Malmberg AB. Splicing correction by peptide-conjugated morpholinos as a novel treatment for late-onset Pompe disease. Mol Ther Nucleic Acids. 2025 Mar 27;36(2):102524. doi: 10.1016/j.omtn.2025.102524. PMID: 40255904; PMCID: PMC12008586. |
Role of UBC9 in the inflammatory response and pathogen susceptibility in zebrafish
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Citation:
Fish Shellfish Immunol. 2025 Jul;162:110337. doi: 10.1016/j.fsi.2025.110337. Epub 2025 Apr 14. PMID: 40239932 Epub:
Not Epub Abstract:
UBC9 is a key enzyme involved in SUMOylation, a post-translational modification that targets protein function, stability,... Delivery Method:
microinjection Organism or Cell Type:
zebrafish Citation Extract: Shah PT, Guo F, Feng J, Wu C, Xing L. Role of UBC9 in the inflammatory response and pathogen susceptibility in zebrafish. Fish Shellfish Immunol. 2025 Jul;162:110337. doi: 10.1016/j.fsi.2025.110337. Epub 2025 Apr 14. PMID: 40239932. |
Mitochondrial dysfunction is driven by imbalanced fission and fusion of mitochondria in myofibrillar myopathy type 5
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Citation:
Hum Mol Genet. 2025 Apr 17:ddaf051. doi: 10.1093/hmg/ddaf051. Epub ahead of print. PMID: 40244302 Epub:
Not Epub Abstract:
Myofibrillar myopathy type 5 (MFM5) is a dominantly inherited myopathy caused by mutations in the FLNC gene. The underlying... Delivery Method:
microinjection Organism or Cell Type:
zebrafish Citation Extract: Wu W, Lv X, Feng Y, Yang M, Yang G, Zhao D, Yan C, Lin P. Mitochondrial dysfunction is driven by imbalanced fission and fusion of mitochondria in myofibrillar myopathy type 5. Hum Mol Genet. 2025 Apr 17:ddaf051. doi: 10.1093/hmg/ddaf051. Epub ahead of print. PMID: 40244302. |