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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 12378 scientific papers returned from the database with the search filters currently being used below.

Role of ZFHX4 in orofacial clefting based on human genetic data and zebrafish models

Authors:
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
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...
Link:
https://pmc.ncbi.nlm.nih.gov/articles/PMC7617551/
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

Authors:
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
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...
Link:
https://pmc.ncbi.nlm.nih.gov/articles/PMC11868726/
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

Authors:
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
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...
Link:
https://www.scielo.br/j/bjmbr/a/84zWhTkPhBbPgw4vykvwJkF/
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

Authors:
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
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...
Link:
https://pmc.ncbi.nlm.nih.gov/articles/PMC11969676/
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

Authors:
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
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...
Link:
https://pmc.ncbi.nlm.nih.gov/articles/PMC12008586/
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

Authors:
Shah PT, Guo F, Feng J, Wu C, Xing L
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,...
Link:
https://www.sciencedirect.com/science/article/abs/pii/S1050464825002268
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

Authors:
Wu W, Lv X, Feng Y, Yang M, Yang G, Zhao D, Yan C, Lin P
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...
Link:
https://academic.oup.com/hmg/advance-article-abstract/doi/10.1093/hmg/ddaf051/8115486
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.

Solute carrier family 2 member 2 (glucose transporter 2): a common factor of hepatocyte and hepatocellular carcinoma differentiation

Authors:
Kim Y, Yeuni Y, Heo HJ, Kim ES, Myung K, Baryawno N, Kim YH, Oh CK
Citation:
PLoS One. 2025 Apr 25;20(4):e0321020. doi: 10.1371/journal.pone.0321020. PMID: 40279337; PMCID: PMC12026939
Epub:
Not Epub
Abstract:
GLUT2 (SLC2A2), a vital glucose transporter in liver, pancreas, and kidney tissues, regulates blood glucose levels and energy...
Link:
https://pmc.ncbi.nlm.nih.gov/articles/PMC12026939/
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Kim Y, Yeuni Y, Heo HJ, Kim ES, Myung K, Baryawno N, Kim YH, Oh CK. Solute carrier family 2 member 2 (glucose transporter 2): a common factor of hepatocyte and hepatocellular carcinoma differentiation. PLoS One. 2025 Apr 25;20(4):e0321020. doi: 10.1371/journal.pone.0321020. PMID: 40279337; PMCID: PMC12026939.

The Rho GEF Trio functions in contact inhibition of locomotion of neural crest cells by interacting with Ptk7

Authors:
Till K, Borchers A
Citation:
Development. 2025 May 1;152(9):dev204446. doi: 10.1242/dev.204446. Epub 2025 May 6. PMID: 40326503
Epub:
Not Epub
Abstract:
Neural crest (NC) cells are highly migratory cells that contribute to a wide range of vertebrate tissues and must respond to a...
Link:
https://journals.biologists.com/dev/article/152/9/dev204446/367882/The-Rho-GEF-Trio-functions-in-contact-inhibition
Delivery Method:
microinjection
Organism or Cell Type:
Xenopus laevis
Citation Extract:
Till K, Borchers A. The Rho GEF Trio functions in contact inhibition of locomotion of neural crest cells by interacting with Ptk7. Development. 2025 May 1;152(9):dev204446. doi: 10.1242/dev.204446. Epub 2025 May 6. PMID: 40326503.

Timely neurogenesis drives the transition from nematic to crystalline nuclear packing during retinal morphogenesis

Authors:
Ferme LC, Ryan AQ, Haase R, Modes CD, Norden C
Citation:
Sci Adv. 2025 May 9;11(19):eadu6843. doi: 10.1126/sciadv.adu6843. Epub 2025 May 9. PMID: 40344072; PMCID: PMC12063663
Epub:
Not Epub
Abstract:
Correct organogenesis depends on the timely coordination of developmental processes, such as cell proliferation,...
Link:
https://pmc.ncbi.nlm.nih.gov/articles/PMC12063663/
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Ferme LC, Ryan AQ, Haase R, Modes CD, Norden C. Timely neurogenesis drives the transition from nematic to crystalline nuclear packing during retinal morphogenesis. Sci Adv. 2025 May 9;11(19):eadu6843. doi: 10.1126/sciadv.adu6843. Epub 2025 May 9. PMID: 40344072; PMCID: PMC12063663.

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