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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 12347 scientific papers returned from the database with the search filters currently being used below.

The splicing modulator CLK1 is a candidate oncogenic dependency in pediatric high-grade gliomas

Authors:
Naqvi AS, Corbett RJ, Seghal P, Conkrite KL, Rathi KS, Ennis BM, Hayer KE, Zhang B, Brown MA, Miller DP, Kraya AA, Dybas JM, Geng Z, Blackden C, Arif S, Chroni A, Lahiri A, Hollawell ML, Storm PB, Foster JB, Koptyra M, Madsen P, Diskin SJ, Thomas-Tikhonenko A, Resnick AC, Rokita JL
Citation:
bioRxiv [preprint] 2024.08.03.606419; doi.org/10.1101/2024.08.03.606419
Epub:
Not Epub
Abstract:
Pediatric brain cancer is the leading cause of disease-related mortality in children, and many aggressive tumors still lack...
Link:
https://www.biorxiv.org/content/10.1101/2024.08.03.606419v1.full
Delivery Method:
Vivo-Morpholino
Organism or Cell Type:
cell culture: KNS-42
Citation Extract:
Naqvi AS, Corbett RJ, Seghal P, Conkrite KL, Rathi KS, Ennis BM, Hayer KE, Zhang B, Brown MA, Miller DP, Kraya AA, Dybas JM, Geng Z, Blackden C, Arif S, Chroni A, Lahiri A, Hollawell ML, Storm PB, Foster JB, Koptyra M, Madsen P, Diskin SJ, Thomas-Tikhonenko A, Resnick AC, Rokita JL. The splicing modulator CLK1 is a candidate oncogenic dependency in pediatric high-grade gliomas. bioRxiv [preprint] 2024.08.03.606419; doi.org/10.1101/2024.08.03.606419.

Efficacy, biodistribution and safety comparison of chemically modified antisense oligonucleotides in the retina

Authors:
Vázquez-Domínguez I, Anido AA, Duijkers L, Hoppenbrouwers T, Hoogendoorn ADM, Koster C, Collin RWJ, Garanto A
Citation:
Nucleic Acids Res. 2024 Sep 23;52(17):10447-10463. doi: 10.1093/nar/gkae686. PMID: 39119918
Epub:
Not Epub
Abstract:
Antisense oligonucleotides (AONs) are a versatile tool for treating inherited retinal diseases. However, little is known about...
Link:
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11417397/
Delivery Method:
Vivo-Morpholino for intravitreal injection, bare Morpholino with Endo-Porter
Organism or Cell Type:
cell culture: mIMCD3 & HEK293T; mice: C57BL/6J
Citation Extract:
Vázquez-Domínguez I, Anido AA, Duijkers L, Hoppenbrouwers T, Hoogendoorn ADM, Koster C, Collin RWJ, Garanto A. Efficacy, biodistribution and safety comparison of chemically modified antisense oligonucleotides in the retina. Nucleic Acids Res. 2024 Sep 23;52(17):10447-10463. doi: 10.1093/nar/gkae686. PMID: 39119918.

Discovery and characterization of stereodefined PMO-gapmers targeting tau

Authors:
Kanatsu K, Takahashi Y, Sakaguchi T, Kim D, Murota M, Shan M, Fukami K, Itano W, Kikuta K, Yoshimura H, Kurokawa T, Nagayama Y, Ishikawa R, Dairiki R, Zhou Z, Sanders K, Stupalski J, Yasui S, Liu D, Benayoud F, Fang H, Jing E, Ogo M, Fang FG, Wang J, Choi H
Citation:
bioRxiv [preprint] 2024.05.09.591947; doi: https://doi.org/10.1101/2024.05.09.591947
Epub:
Not Epub
Abstract:
Antisense oligonucleotides (ASOs) are an important class of therapeutics to treat genetic diseases, and expansion of this...
Link:
https://www.biorxiv.org/content/10.1101/2024.05.09.591947v1
Delivery Method:
Endo-Porter
Organism or Cell Type:
cell culture: SH-SY5Y
Citation Extract:
Kanatsu K, Takahashi Y, Sakaguchi T, Kim D, Murota M, Shan M, Fukami K, Itano W, Kikuta K, Yoshimura H, Kurokawa T, Nagayama Y, Ishikawa R, Dairiki R, Zhou Z, Sanders K, Stupalski J, Yasui S, Liu D, Benayoud F, Fang H, Jing E, Ogo M, Fang FG, Wang J, Choi H. Discovery and characterization of stereodefined PMO-gapmers targeting tau. bioRxiv [preprint] 2024.05.09.591947; doi: https://doi.org/10.1101/2024.05.09.591947.

Enhancing Peptide and PMO Delivery to Mouse Airway Epithelia by Chemical Conjugation with the Amphiphilic Peptide S10

Authors:
Auger M, Sorroza-Martinez L, Brahiti N, Huppé C-A, Faucher-Giguère L, Arbi I, Hervault M, Cheng X, Gaillet B, Couture F, Guay D, Soultan A-H
Citation:
Molecular Therapy: Nucleic Acid (2024), doi: https://doi.org/10.1016/j.omtn.2024.102290
Epub:
Not Epub
Abstract:
Delivery of antisense oligonucleotides (ASOs) to airway epithelial cells is arduous due to the physiological barriers that...
Link:
https://www.sciencedirect.com/science/article/pii/S216225312400177X
Delivery Method:
intranasal peptide-linked
Organism or Cell Type:
cell culture: HeLa EGFP-654, mice: CAG-EGFP-654
Citation Extract:
Auger M, Sorroza-Martinez L, Brahiti N, Huppé C-A, Faucher-Giguère L, Arbi I, Hervault M, Cheng X, Gaillet B, Couture F, Guay D, Soultan A-H. Enhancing Peptide and PMO Delivery to Mouse Airway Epithelia by Chemical Conjugation with the Amphiphilic Peptide S10. Molecular Therapy: Nucleic Acid (2024), doi: https://doi.org/10.1016/j.omtn.2024.102290.

Tailored antisense oligonucleotides designed to correct aberrant splicing reveal actionable groups of mutations for rare genetic disorders

Authors:
Wai HA, Svobodova E, Herrera NR, Douglas AGL, Holloway JW, Baralle FE, Baralle M, Baralle D
Citation:
Exp Mol Med (2024). https://doi.org/10.1038/s12276-024-01292-1
Epub:
Not Epub
Abstract:
Effective translation of rare disease diagnosis knowledge into therapeutic applications is achievable within a reasonable...
Link:
https://www.nature.com/articles/s12276-024-01292-1
Delivery Method:
Vivo-Morpholinos, bare Morpholinos, Endo-Porter
Organism or Cell Type:
HEK293 cells
Citation Extract:
Wai HA, Svobodova E, Herrera NR, Douglas AGL, Holloway JW, Baralle FE, Baralle M, Baralle D. Tailored antisense oligonucleotides designed to correct aberrant splicing reveal actionable groups of mutations for rare genetic disorders. Exp Mol Med (2024). https://doi.org/10.1038/s12276-024-01292-1.

Cdon is required for organ Left-Right patterning via regulating DFCs migration and the sequential ciliogenesis

Authors:
Deng Z, Chang W, Li C, Li B, Huang S, Huang J, Zhang K, Li Y, Liu X, Ran Q, Guo Z, Huang S
Citation:
bioRxiv [preprint] 2024.03.12.584572; doi: https://doi.org/10.1101/2024.03.12.584572
Epub:
Not Epub
Abstract:
Cdon and boc are members of the cell adhesion molecule subfamily III Ig/fibronectin. Although they were reported to be involved...
Link:
https://www.biorxiv.org/content/10.1101/2024.03.12.584572v1.full
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Deng Z, Chang W, Li C, Li B, Huang S, Huang J, Zhang K, Li Y, Liu X, Ran Q, Guo Z, Huang S. Cdon is required for organ Left-Right patterning via regulating DFCs migration and the sequential ciliogenesis. bioRxiv [preprint] 2024.03.12.584572; doi: https://doi.org/10.1101/2024.03.12.584572.

Somatostatin signalling coordinates energy metabolism allocation to reproduction in zebrafish

Authors:
Chen J, Zhao W, Cao L, Martins RST, Canário AVM
Citation:
BMC Biol. 2024 Jul 29;22(1):163. doi: 10.1186/s12915-024-01961-7. PMID: 39075492; PMCID: PMC11288053
Epub:
Not Epub
Abstract:
BACKGROUND: Energy allocation between growth and reproduction determines puberty onset and fertility. In mammals, peripheral...
Link:
https://bmcbiol.biomedcentral.com/articles/10.1186/s12915-024-01961-7
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Chen J, Zhao W, Cao L, Martins RST, Canário AVM. Somatostatin signalling coordinates energy metabolism allocation to reproduction in zebrafish. BMC Biol. 2024 Jul 29;22(1):163. doi: 10.1186/s12915-024-01961-7. PMID: 39075492; PMCID: PMC11288053.

Leucine-Rich Repeat Kinase-2 Controls the Differentiation and Maturation of Oligodendrocytes in Mice and Zebrafish

Authors:
Filippini A, Cannone E, Mazziotti V, Carini G, Mutti V, Ravelli C, Gennarelli M, Schiavone M, Russo I
Citation:
Biomolecules. 2024; 14(7):870. https://doi.org/10.3390/biom14070870
Epub:
Not Epub
Abstract:
Leucine-rich repeat kinase-2 (LRRK2), a gene mutated in familial and sporadic Parkinson’s disease (PD), controls multiple...
Link:
https://www.mdpi.com/2218-273X/14/7/870
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Filippini A, Cannone E, Mazziotti V, Carini G, Mutti V, Ravelli C, Gennarelli M, Schiavone M, Russo I. Leucine-Rich Repeat Kinase-2 Controls the Differentiation and Maturation of Oligodendrocytes in Mice and Zebrafish. Biomolecules. 2024; 14(7):870. https://doi.org/10.3390/biom14070870.

SCAR-6 elncRNA locus epigenetically regulates PROZ and modulates coagulation and vascular function

Authors:
Ranjan G, Sehgal P, Scaria V, Sivasubbu S
Citation:
bioRxiv [preprint] 2024.07.21.604148; doi: https://doi.org/10.1101/2024.07.21.604148
Epub:
Not Epub
Abstract:
Syntenic conservation is an effective strategy to identify evolutionarily conserved lncRNA orthologs. In this study, we...
Link:
https://www.biorxiv.org/content/10.1101/2024.07.21.604148v1.full
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Ranjan G, Sehgal P, Scaria V, Sivasubbu S. SCAR-6 elncRNA locus epigenetically regulates PROZ and modulates coagulation and vascular function. bioRxiv [preprint] 2024.07.21.604148; doi: https://doi.org/10.1101/2024.07.21.604148.

Sall4 regulates downstream patterning genes during limb regeneration

Authors:
Erickson JR, Walker SE, Arenas Gomez C, Echeverri K
Citation:
Dev Biol. 2024 Jul 25:S0012-1606(24)00195-7. doi: 10.1016/j.ydbio.2024.07.015. Epub ahead of print. PMID: 39067503.
Epub:
Not Epub
Abstract:
Many salamanders can completely regenerate a fully functional limb. Limb regeneration is a carefully coordinated process...
Link:
https://www.sciencedirect.com/science/article/pii/S0012160624001957
Delivery Method:
injection then electroporation
Organism or Cell Type:
Ambystoma mexicanum (Axolotl)
Citation Extract:
Erickson JR, Walker SE, Arenas Gomez C, Echeverri K. Sall4 regulates downstream patterning genes during limb regeneration. Dev Biol. 2024 Jul 25:S0012-1606(24)00195-7. doi: 10.1016/j.ydbio.2024.07.015. Epub ahead of print. PMID: 39067503..

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