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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.

Cell-penetrating peptide-conjugated, splice-switching oligonucleotides mitigate the phenotype in BTK/Tec double deficient X-linked agammaglobulinemia model

Authors:
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
Citation:
RSC Chem Biol. 2025 Mar 31. doi: 10.1039/d4cb00312h. Epub ahead of print. PMID: 40171248; PMCID: PMC11955834
Epub:
Not Epub
Abstract:
Splice-switching oligonucleotides (SSOs) have been developed as a treatment for various disorders, including Duchenne muscular...
Link:
https://pmc.ncbi.nlm.nih.gov/articles/PMC11955834/
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. RSC Chem Biol. 2025 Mar 31. doi: 10.1039/d4cb00312h. Epub ahead of print. PMID: 40171248; PMCID: PMC11955834.

Wdr5-mediated H3K4 methylation facilitates HSPC development via maintenance of genomic stability in zebrafish

Authors:
Wang X, Liu M, Zhang Y, Ma D, Wang L, Liu F
Citation:
Proc Natl Acad Sci U S A. 2025 Mar 25;122(12):e2420534122. doi: 10.1073/pnas.2420534122. Epub 2025 Mar 20. PMID: 40112113
Epub:
Not Epub
Abstract:
During fetal stage, hematopoietic stem and progenitor cells (HSPCs) undergo rapid proliferation with a tight control of genomic...
Link:
https://www.pnas.org/doi/10.1073/pnas.2420534122
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Wang X, Liu M, Zhang Y, Ma D, Wang L, Liu F. Wdr5-mediated H3K4 methylation facilitates HSPC development via maintenance of genomic stability in zebrafish. Proc Natl Acad Sci U S A. 2025 Mar 25;122(12):e2420534122. doi: 10.1073/pnas.2420534122. Epub 2025 Mar 20. PMID: 40112113.

Elevated body temperature exacerbates arrhythmia and seizure-like activity in a zebrafish model of Timothy syndrome

Authors:
Semenova SA, Sams AM, Nammi D, Menold M, Margolin G, Sinclair JL, Robertson KA, Larkin K, Kelly S, Golden A, Feldman B, Burgess HA
Citation:
bioRxiv [preprint] 2025.03.11.642683; doi: https://doi.org/10.1101/2025.03.11.642683
Epub:
Not Epub
Abstract:
Timothy syndrome (TS) is a multisystem disorder with autistic-like features, seizures and arrhythmia as the main symptoms. Most...
Link:
https://www.biorxiv.org/content/10.1101/2025.03.11.642683v1
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Semenova SA, Sams AM, Nammi D, Menold M, Margolin G, Sinclair JL, Robertson KA, Larkin K, Kelly S, Golden A, Feldman B, Burgess HA. Elevated body temperature exacerbates arrhythmia and seizure-like activity in a zebrafish model of Timothy syndrome. bioRxiv [preprint] 2025.03.11.642683; doi: https://doi.org/10.1101/2025.03.11.642683.

A Robust Strategy for Introducing Amino-Modifiers in Nucleic Acids: Enabling Novel Amino Tandem Oligonucleotide Synthesis in DNA and RNA

Authors:
Saraya JS, Horton NG, Sammons SR, O'Flaherty DK
Citation:
Chemistry. 2025 Mar 24:e202500448. doi: 10.1002/chem.202500448. Epub ahead of print. PMID: 40127302
Epub:
Not Epub
Abstract:
Amino-modifiers are pivotal chemical modifications in nucleic acid scaffolds, serving applications ranging from (bio)...
Link:
https://chemistry-europe.onlinelibrary.wiley.com/doi/10.1002/chem.202500448
Delivery Method:
None
Organism or Cell Type:
None
Citation Extract:
Saraya JS, Horton NG, Sammons SR, O'Flaherty DK. A Robust Strategy for Introducing Amino-Modifiers in Nucleic Acids: Enabling Novel Amino Tandem Oligonucleotide Synthesis in DNA and RNA. Chemistry. 2025 Mar 24:e202500448. doi: 10.1002/chem.202500448. Epub ahead of print. PMID: 40127302.

Catalytic activity of KMT5B promotes cilia tuft formation without affecting chromatin accessibility of ciliary genes

Authors:
Tait J, Marthen C, Hoelscher B, Straub T, Rupp R
Citation:
bioRxiv 2025.03.18.644017; doi: https://doi.org/10.1101/2025.03.18.644017
Epub:
Not Epub
Abstract:
Multiciliated cells are a specialized cell type found in the brain, reproductive tract and respiratory tract of mammals....
Link:
https://www.biorxiv.org/content/10.1101/2025.03.18.644017v1
Delivery Method:
microinjection
Organism or Cell Type:
Xenopus laevis
Citation Extract:
Tait J, Marthen C, Hoelscher B, Straub T, Rupp R. Catalytic activity of KMT5B promotes cilia tuft formation without affecting chromatin accessibility of ciliary genes. bioRxiv 2025.03.18.644017; doi: https://doi.org/10.1101/2025.03.18.644017.

Long-Term Dystrophin Replacement Therapy in Duchenne Muscular Dystrophy Causes Cardiac Inflammation

Authors:
Forand A, Moog S, Mougenot N, Lemaitre M, Sevoz-Couche C, Guesmia Z, Virtanen L, Giordani L, Muchir A, Pietri-Rouxel F
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....
Link:
https://www.jacc.org/doi/10.1016/j.jacbts.2024.12.015
Delivery Method:
peptide-linked combined therapy with AAV-U7; retro-orbital & intravenous (i.v.) injection
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.

A nucleus-encoded dynamin-like protein controls endosymbiont division in the trypanosomatid Angomonas deanei

Authors:
Maurya AK, Kröninger L, Ehret G, Bäumers M, Marson M, Scheu S, Nowack ECM
Citation:
Sci Adv. 2025 Mar 21;11(12):eadp8518. doi: 10.1126/sciadv.adp8518. Epub 2025 Mar 19. PMID: 40106558
Epub:
Not Epub
Abstract:
Angomonas deanei is a trypanosomatid of the Strigomonadinae. All members of this subfamily contain a single β-proteobacterial...
Link:
https://www.science.org/doi/10.1126/sciadv.adp8518
Delivery Method:
electroporation
Organism or Cell Type:
Angomonas deanei (trypanosoma)
Citation Extract:
Maurya AK, Kröninger L, Ehret G, Bäumers M, Marson M, Scheu S, Nowack ECM. A nucleus-encoded dynamin-like protein controls endosymbiont division in the trypanosomatid Angomonas deanei. Sci Adv. 2025 Mar 21;11(12):eadp8518. doi: 10.1126/sciadv.adp8518. Epub 2025 Mar 19. PMID: 40106558.

Modeling epilepsy by loss-of-function of the CUG-binding protein Elav-like family member 2 in zebrafish with multi-omics analysis

Authors:
Wang X, Zhang J, Rao X, Liu Y, Lin Z, Chen F, Luo R, Sun H, Gan J
Citation:
Chin Med J (Engl). 2025 Mar 11. doi: 10.1097/CM9.0000000000003398. Epub ahead of print. PMID: 40066778
Epub:
Not Epub
Abstract:
Background: The CUG-binding protein Elav-like family member 2 (CELF2) gene has been linked to the pathogenesis of epilepsy, but...
Link:
https://journals.lww.com/cmj/fulltext/9900/modeling_epilepsy_by_loss_of_function_of_the.1466.aspx
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Wang X, Zhang J, Rao X, Liu Y, Lin Z, Chen F, Luo R, Sun H, Gan J. Modeling epilepsy by loss-of-function of the CUG-binding protein Elav-like family member 2 in zebrafish with multi-omics analysis. Chin Med J (Engl). 2025 Mar 11. doi: 10.1097/CM9.0000000000003398. Epub ahead of print. PMID: 40066778.

Zinc finger protein Znf296 is a cardiac-specific splicing regulator required for cardiomyocyte formation

Authors:
Li X, Yang S, Wang L, Zhang X, Zhang A, Wang Y, Shi DL, Li H
Citation:
Am J Pathol. 2025 Mar 21:S0002-9440(25)00078-1. doi: 10.1016/j.ajpath.2025.02.006. Epub ahead of print. PMID: 40122456
Epub:
Not Epub
Abstract:
Heart formation and function are tightly regulated at transcriptional and post-transcriptional levels. The dysfunction of...
Link:
https://ajp.amjpathol.org/article/S0002-9440(25)00078-1/abstract
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Li X, Yang S, Wang L, Zhang X, Zhang A, Wang Y, Shi DL, Li H. Zinc finger protein Znf296 is a cardiac-specific splicing regulator required for cardiomyocyte formation. Am J Pathol. 2025 Mar 21:S0002-9440(25)00078-1. doi: 10.1016/j.ajpath.2025.02.006. Epub ahead of print. PMID: 40122456.

The Interaction Between the asb5a and asb5b Subtypes Jointly Regulates the L-R Asymmetrical Development of the Heart in Zebrafish

Authors:
Zhou W, Cai W, Li Y, Gao L, Liu X, Liu S, Lei J, Zhang J, Wang Y, Jiang Z, Wu X, Fan X, Li F, Zheng L, Yuan W
Citation:
Int. J. Mol. Sci. 2025, 26, 2765. https://doi.org/10.3390/ijms26062765
Epub:
Not Epub
Abstract:
The asb5 gene, a member of the Asb protein subfamily characterized by six ankyrin repeat domains, is highly conserved and...
Link:
https://www.mdpi.com/1422-0067/26/6/2765
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Zhou W, Cai W, Li Y, Gao L, Liu X, Liu S, Lei J, Zhang J, Wang Y, Jiang Z, Wu X, Fan X, Li F, Zheng L, Yuan W. The Interaction Between the asb5a and asb5b Subtypes Jointly Regulates the L-R Asymmetrical Development of the Heart in Zebrafish. Int. J. Mol. Sci. 2025, 26, 2765. https://doi.org/10.3390/ijms26062765.

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