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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 12511 scientific papers returned from the database with the search filters currently being used below.

Mdx mice dosed with antisense to CD49d and dystrophin exon skip morpholino; reduced force loss and affected gene expression pathways support ATL1102 combination therapy in DMD patients

Authors:
Padhye AS, Kiriaev L, Tiong C, Gearing LJ, Wilson T, Houweling PJ, Tachas G
Citation:
bioRxiv [preprint] 2024.11.23.625028; doi: https://doi.org/10.1101/2024.11.23.625028
Epub:
Not Epub
Abstract:
Background: VLA-4 is a disease progression biomarker in patients with Duchenne Muscular Dystrophy (DMD) including those treated...
Link:
https://www.biorxiv.org/content/10.1101/2024.11.23.625028v1
Delivery Method:
intravenous (i.v.)
Organism or Cell Type:
mdx mice
Citation Extract:
Padhye AS, Kiriaev L, Tiong C, Gearing LJ, Wilson T, Houweling PJ, Tachas G. Mdx mice dosed with antisense to CD49d and dystrophin exon skip morpholino; reduced force loss and affected gene expression pathways support ATL1102 combination therapy in DMD patients. bioRxiv [preprint] 2024.11.23.625028; doi: https://doi.org/10.1101/2024.11.23.625028.

Unveiling the Role of TMPRSS2 in the Proteolytic Activation of Pandemic and Zoonotic Influenza Viruses and Coronaviruses in Human Airway Cells

Authors:
Schwerdtner M, Schmacke LC, Nave J, Limburg H, Steinmetzer T, Stein DA, Moulton HM, Böttcher-Friebertshäuser E
Citation:
Viruses. 2024 Nov 20;16(11):1798. doi: 10.3390/v16111798. PMID: 39599912; PMCID: PMC11599139
Epub:
Not Epub
Abstract:
The zoonotic transmission of influenza A viruses (IAVs) and coronaviruses (CoVs) may result in severe disease. Cleavage of...
Link:
https://pmc.ncbi.nlm.nih.gov/articles/PMC11599139/
Delivery Method:
(RXR)4 peptide-linked
Organism or Cell Type:
cell culture: Calu-3
Citation Extract:
Schwerdtner M, Schmacke LC, Nave J, Limburg H, Steinmetzer T, Stein DA, Moulton HM, Böttcher-Friebertshäuser E. Unveiling the Role of TMPRSS2 in the Proteolytic Activation of Pandemic and Zoonotic Influenza Viruses and Coronaviruses in Human Airway Cells. Viruses. 2024 Nov 20;16(11):1798. doi: 10.3390/v16111798. PMID: 39599912; PMCID: PMC11599139.

An RNA interference approach for functional studies in the sea urchin and its use in analysis of nodal signaling gradients

Authors:
Wilson K, Manner C, Miranda E, Berrio A, Wray GA, McClay DR
Citation:
Dev Biol. 2024 Dec;516:59-70. doi: 10.1016/j.ydbio.2024.08.002. Epub 2024 Aug 3. PMID: 39098630; PMCID: PMC11425896
Epub:
Not Epub
Abstract:
Dicer substrate interfering RNAs (DsiRNAs) destroy targeted transcripts using the RNA-Induced Silencing Complex (RISC) through...
Link:
https://www.sciencedirect.com/science/article/abs/pii/S0012160624002070
Delivery Method:
microinjection
Organism or Cell Type:
Lytechinus variegatus (sea urchin)
Citation Extract:
Wilson K, Manner C, Miranda E, Berrio A, Wray GA, McClay DR. An RNA interference approach for functional studies in the sea urchin and its use in analysis of nodal signaling gradients. Dev Biol. 2024 Dec;516:59-70. doi: 10.1016/j.ydbio.2024.08.002. Epub 2024 Aug 3. PMID: 39098630; PMCID: PMC11425896.

Golodirsen restores DMD transcript imbalance in Duchenne Muscular Dystrophy patient muscle cells

Authors:
Rossi R, Torelli S, Moore M, Ala P, Morgan J, Malhotra J, Muntoni F
Citation:
Skelet Muscle. 2024 Nov 29;14(1):28. doi: 10.1186/s13395-024-00360-4. PMID: 39614336; PMCID: PMC11606086
Epub:
Not Epub
Abstract:
Background: Antisense oligonucleotides (AON) represent a promising treatment for Duchenne muscular dystrophy (DMD) carrying out...
Link:
https://skeletalmusclejournal.biomedcentral.com/articles/10.1186/s13395-024-00360-4
Delivery Method:
Endo-Porter
Organism or Cell Type:
cell culture: DMD-patient-derived primary fibroblasts
Citation Extract:
Rossi R, Torelli S, Moore M, Ala P, Morgan J, Malhotra J, Muntoni F. Golodirsen restores DMD transcript imbalance in Duchenne Muscular Dystrophy patient muscle cells. Skelet Muscle. 2024 Nov 29;14(1):28. doi: 10.1186/s13395-024-00360-4. PMID: 39614336; PMCID: PMC11606086.

Improved In Vivo Metabolic Stability and Silencing Efficacy of siRNAs with Phosphorothioate Linkage-Free, GalNAc-Conjugated Sense Strands Containing Morpholino-LNA Modifications

Authors:
Datta D, Kumar P, Kundu J, Qin J, Gilbert JA, Schofield S, Donnelly DP, Liu J, Degaonkar R, Egli M, Manoharan M
Citation:
Org Lett. 2024 Nov 29;26(47):10061-10065. doi: 10.1021/acs.orglett.4c02903. Epub 2024 Nov 11. PMID: 39528231
Epub:
Not Epub
Abstract:
To ensure specificity, loading of the sense strand of small interfering RNAs (siRNAs) into RISC must be inhibited. We show here...
Link:
https://pubs.acs.org/doi/10.1021/acs.orglett.4c02903
Delivery Method:
None
Organism or Cell Type:
None
Citation Extract:
Datta D, Kumar P, Kundu J, Qin J, Gilbert JA, Schofield S, Donnelly DP, Liu J, Degaonkar R, Egli M, Manoharan M. Improved In Vivo Metabolic Stability and Silencing Efficacy of siRNAs with Phosphorothioate Linkage-Free, GalNAc-Conjugated Sense Strands Containing Morpholino-LNA Modifications. Org Lett. 2024 Nov 29;26(47):10061-10065. doi: 10.1021/acs.orglett.4c02903. Epub 2024 Nov 11. PMID: 39528231.

Zebrafish in-vivo study reveals deleterious activity of human TBC1D24 genetic variants linked with autosomal dominant hearing loss

Authors:
Sarosiak A, Jędrychowska J, Oziębło D, Gan NS, Bałdyga N, Leja ML, Węgierski T, Cruz IA, Raible DW, Skarżyński H, Tylzanowski P, Korzh V, Ołdak M
Citation:
Biochim Biophys Acta Mol Basis Dis. 2025 Feb;1871(2):167598. doi: 10.1016/j.bbadis.2024.167598. Epub 2024 Nov 23. PMID: 39586506.
Epub:
Not Epub
Abstract:
Hearing loss is a common sensory impairment with a heterogeneous genetic etiology. Genetic variants in the TBC1D24 gene have...
Link:
https://www.sciencedirect.com/science/article/abs/pii/S0925443924005921
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Sarosiak A, Jędrychowska J, Oziębło D, Gan NS, Bałdyga N, Leja ML, Węgierski T, Cruz IA, Raible DW, Skarżyński H, Tylzanowski P, Korzh V, Ołdak M. Zebrafish in-vivo study reveals deleterious activity of human TBC1D24 genetic variants linked with autosomal dominant hearing loss. Biochim Biophys Acta Mol Basis Dis. 2025 Feb;1871(2):167598. doi: 10.1016/j.bbadis.2024.167598. Epub 2024 Nov 23. PMID: 39586506..

Genetic Evidence for Estrogenic Effects of Benzophenone-2 on Zebrafish Neurodevelopment and Its Signaling Mechanism

Authors:
Qi Z, Zhai Y, Han Y, Li K, Wang T, Li P, Li J, Zhou X, Zhao X, Song W
Citation:
Environ Sci Technol. 2024 Dec 10;58(49):21433-21449. doi: 10.1021/acs.est.4c06892. Epub 2024 Nov 23. PMID: 39579127
Epub:
Not Epub
Abstract:
Estrogens play a crucial role in regulating various biological responses during the early stages of neurodevelopment....
Link:
https://pubs.acs.org/doi/10.1021/acs.est.4c06892
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Qi Z, Zhai Y, Han Y, Li K, Wang T, Li P, Li J, Zhou X, Zhao X, Song W . Genetic Evidence for Estrogenic Effects of Benzophenone-2 on Zebrafish Neurodevelopment and Its Signaling Mechanism. Environ Sci Technol. 2024 Dec 10;58(49):21433-21449. doi: 10.1021/acs.est.4c06892. Epub 2024 Nov 23. PMID: 39579127.

Alternative splicing dysregulation across tissue and therapeutic approaches in a mouse model of myotonic dystrophy type 1

Authors:
Hicks SM, Frias JA, Mishra SK, Scotti M, Muscato DR, Valero MC, Adams LM, Cleary JD, Nakamori M, Wang E, Berglund JA
Citation:
Mol Ther Nucleic Acids. 2024 Sep 13;35(4):102338. doi: 10.1016/j.omtn.2024.102338. PMID: 39391766; PMCID: PMC11465180
Epub:
Not Epub
Abstract:
Myotonic dystrophy type 1 (DM1), the leading cause of adult-onset muscular dystrophy, is caused by a CTG repeat expansion....
Link:
https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(24)00225-7
Delivery Method:
peptide-linked; intramuscular (i.m.)
Organism or Cell Type:
HSA(LR) mice
Citation Extract:
Hicks SM, Frias JA, Mishra SK, Scotti M, Muscato DR, Valero MC, Adams LM, Cleary JD, Nakamori M, Wang E, Berglund JA. Alternative splicing dysregulation across tissue and therapeutic approaches in a mouse model of myotonic dystrophy type 1. Mol Ther Nucleic Acids. 2024 Sep 13;35(4):102338. doi: 10.1016/j.omtn.2024.102338. PMID: 39391766; PMCID: PMC11465180.

Genetic requirement of dact1/2 to regulate noncanonical Wnt signaling and calpain 8 during embryonic convergent extension and craniofacial morphogenesis

Authors:
Carroll SH, Schafer S, Kawasaki K, Tsimbal C, Jule AM, Hallett SA, Li E, Liao EC
Citation:
Elife. 2024 Nov 21;13:RP91648. doi: 10.7554/eLife.91648. PMID: 39570288; PMCID: PMC11581427
Epub:
Not Epub
Abstract:
Wnt signaling plays crucial roles in embryonic patterning including the regulation of convergent extension (CE) during...
Link:
https://elifesciences.org/articles/91648
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Carroll SH, Schafer S, Kawasaki K, Tsimbal C, Jule AM, Hallett SA, Li E, Liao EC. Genetic requirement of dact1/2 to regulate noncanonical Wnt signaling and calpain 8 during embryonic convergent extension and craniofacial morphogenesis. Elife. 2024 Nov 21;13:RP91648. doi: 10.7554/eLife.91648. PMID: 39570288; PMCID: PMC11581427.

Linking planar polarity signalling to actomyosin contractility during vertebrate neurulation

Authors:
Novotna S, Maia LA, Radaszkiewicz KA, Roudnicky P, Harnos J
Citation:
Open Biol. 2024 Nov;14(11):240251. doi: 10.1098/rsob.240251. Epub 2024 Nov 20. PMID: 39561813; PMCID: PMC11576107
Epub:
Not Epub
Abstract:
Actomyosin contractility represents an ancient feature of eukaryotic cells participating in many developmental and homeostasis...
Link:
https://royalsocietypublishing.org/doi/10.1098/rsob.240251
Delivery Method:
microinjection
Organism or Cell Type:
Xenopus laevis
Citation Extract:
Novotna S, Maia LA, Radaszkiewicz KA, Roudnicky P, Harnos J. Linking planar polarity signalling to actomyosin contractility during vertebrate neurulation. Open Biol. 2024 Nov;14(11):240251. doi: 10.1098/rsob.240251. Epub 2024 Nov 20. PMID: 39561813; PMCID: PMC11576107.

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