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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 12236 scientific papers returned from the database with the search filters currently being used below.

Deciphering the impact of PROM1 alternative splicing on human photoreceptor development and maturation

Authors:
Moya-Molina M, Dorgau B, Flood E, Letteboer SJF, Lorentzen E, Coxhead J, Smith G, Roepman R, Nagaraja Grellscheid S, Armstrong L, Lako M
Citation:
Cell Death Dis. 2024 Oct 1;15(10):721. doi: 10.1038/s41419-024-07105-7. PMID: 39353897
Epub:
Not Epub
Abstract:
Alternative splicing (AS) is a crucial mechanism contributing to proteomic diversity, which is highly regulated in tissue- and...
Link:
https://www.nature.com/articles/s41419-024-07105-7
Delivery Method:
Endo-Porter PEG
Organism or Cell Type:
retinal organoids derived from hiPSCs
Citation Extract:
Moya-Molina M, Dorgau B, Flood E, Letteboer SJF, Lorentzen E, Coxhead J, Smith G, Roepman R, Nagaraja Grellscheid S, Armstrong L, Lako M. Deciphering the impact of PROM1 alternative splicing on human photoreceptor development and maturation. Cell Death Dis. 2024 Oct 1;15(10):721. doi: 10.1038/s41419-024-07105-7. PMID: 39353897.

Prox1a promotes liver growth and differentiation by repressing cdx1b expression and intestinal fate transition in zebrafish

Authors:
Hu Y, Luo Z, Wang M, Wu Z, Liu Y, Cheng Z, Sun Y, Xiong JW, Tong X, Zhu Z, Zhang B
Citation:
J Genet Genomics. 2024 Sep 27:S1673-8527(24)00248-0. doi: 10.1016/j.jgg.2024.09.010. Online ahead of print. PMID: 39343095
Epub:
Not Epub
Abstract:
The liver is a key endoderm-derived multifunctional organ within the digestive system. Prospero homeobox 1 (Prox1) is an...
Link:
https://www.sciencedirect.com/science/article/abs/pii/S1673852724002480
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Hu Y, Luo Z, Wang M, Wu Z, Liu Y, Cheng Z, Sun Y, Xiong JW, Tong X, Zhu Z, Zhang B. Prox1a promotes liver growth and differentiation by repressing cdx1b expression and intestinal fate transition in zebrafish. J Genet Genomics. 2024 Sep 27:S1673-8527(24)00248-0. doi: 10.1016/j.jgg.2024.09.010. Online ahead of print. PMID: 39343095.

Deoxyhypusine synthase deficiency syndrome zebrafish model: aberrant morphology, epileptiform activity, and reduced arborization of inhibitory interneurons

Authors:
Shojaeinia E, Mastracci TL, Soliman R, Devinsky O, Esguerra CV, Crawford AD
Citation:
Mol Brain. 2024 Sep 27;17(1):68. doi: 10.1186/s13041-024-01139-w. PMID: 39334388
Epub:
Not Epub
Abstract:
DHPS deficiency syndrome is an ultra-rare neurodevelopmental disorder (NDD) which results from biallelic mutations in the gene...
Link:
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11429087/
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Shojaeinia E, Mastracci TL, Soliman R, Devinsky O, Esguerra CV, Crawford AD. Deoxyhypusine synthase deficiency syndrome zebrafish model: aberrant morphology, epileptiform activity, and reduced arborization of inhibitory interneurons. Mol Brain. 2024 Sep 27;17(1):68. doi: 10.1186/s13041-024-01139-w. PMID: 39334388.

Targeting KIT by frameshifting mRNA transcripts as a therapeutic strategy for aggressive mast cell neoplasms

Authors:
Snider DB, Arthur GK, Falduto GH, Olivera A, Ehrhardt-Humbert LC, Smith E, Smith C, Metcalfe DD, Cruse G
Citation:
Mol Ther. 2022 Jan 5;30(1):295-310. doi: 10.1016/j.ymthe.2021.08.009. Epub 2021 Aug 8. PMID: 34371183; PMCID: PMC8753370
Epub:
Not Epub
Abstract:
Activating mutations in c-KIT are associated with the mast cell (MC) clonal disorders cutaneous mastocytosis and systemic...
Link:
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8753370/
Delivery Method:
cell culture: electroporation; mice: Vivo-Morpholino injection, intravenous (i.v.) & intratumoral
Organism or Cell Type:
cell culture: HMC-1.2 & LAD2; mice: DBA/2J
Citation Extract:
Snider DB, Arthur GK, Falduto GH, Olivera A, Ehrhardt-Humbert LC, Smith E, Smith C, Metcalfe DD, Cruse G. Targeting KIT by frameshifting mRNA transcripts as a therapeutic strategy for aggressive mast cell neoplasms. Mol Ther. 2022 Jan 5;30(1):295-310. doi: 10.1016/j.ymthe.2021.08.009. Epub 2021 Aug 8. PMID: 34371183; PMCID: PMC8753370.

Fgf8 contributes to the pathogenesis of Nager syndrome

Authors:
Ulhaq ZS, You M-S, Yabe T, Takada S, Chen J-K, Ogino Y, Jiang Y-J, Tse WKF
Citation:
International Journal of Biological Macromolecules. Preprint. Available online 15 September 2024, 135692
Epub:
Not Epub
Abstract:
Nager syndrome (NS, OMIM 154400) is a rare disease characterized by craniofacial and limb malformations due to variants in...
Link:
https://www.sciencedirect.com/science/article/abs/pii/S0141813024065000
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Ulhaq ZS, You M-S, Yabe T, Takada S, Chen J-K, Ogino Y, Jiang Y-J, Tse WKF. Fgf8 contributes to the pathogenesis of Nager syndrome. International Journal of Biological Macromolecules. Preprint. Available online 15 September 2024, 135692.

Redirecting full-length FLT1 expression towards its soluble isoform promotes postischemic angiogenesis

Authors:
Bundalo M, Vorlova S, Ulrich J, Barbieri R, Richter L, Höna L, Egg M, Bock J, Schäfer S, Amézaga Solé N, Rosa A, Rizzo G, Cochain C, Kastenmüller W, Henke E, Skryabin BV, Rozhdestvensky TS, Wildgruber M, Lorenz K, Kuhn M, Zernecke A
Citation:
bioRxiv [preprint] 2024.09.19.613989; doi: https://doi.org/10.1101/2024.09.19.613989
Epub:
Not Epub
Abstract:
Vascular endothelial growth factors and their tyrosine kinase receptors are key mediators of vasculogenesis and angiogenesis...
Link:
https://www.biorxiv.org/content/10.1101/2024.09.19.613989v2.full
Delivery Method:
Endo-Porter
Organism or Cell Type:
cell culture: mouse SVEC
Citation Extract:
Bundalo M, Vorlova S, Ulrich J, Barbieri R, Richter L, Höna L, Egg M, Bock J, Schäfer S, Amézaga Solé N, Rosa A, Rizzo G, Cochain C, Kastenmüller W, Henke E, Skryabin BV, Rozhdestvensky TS, Wildgruber M, Lorenz K, Kuhn M, Zernecke A. Redirecting full-length FLT1 expression towards its soluble isoform promotes postischemic angiogenesis. bioRxiv [preprint] 2024.09.19.613989; doi: https://doi.org/10.1101/2024.09.19.613989.

Poly-GP accumulation due to C9orf72 loss of function induces motor neuron apoptosis through autophagy and mitophagy defects

Authors:
de Calbiac H, Renault S, Haouy G, Jung V, Roger K, Zhou Q, Campanari ML, Chentout L, Demy DL, Marian A, Goudin N, Edbauer D, Guerrera C, Ciura S, Kabashi E
Citation:
Autophagy. 2024 Oct;20(10):2164-2185. doi: 10.1080/15548627.2024.2358736. Epub 2024 Sep 24. PMID: 39316747; PMCID: PMC11423671
Epub:
Not Epub
Abstract:
degeneration. C9orf72 HRE is associated with lowered levels of C9orf72 expression and its translation results in the production...
Link:
https://www.tandfonline.com/doi/full/10.1080/15548627.2024.2358736
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
de Calbiac H, Renault S, Haouy G, Jung V, Roger K, Zhou Q, Campanari ML, Chentout L, Demy DL, Marian A, Goudin N, Edbauer D, Guerrera C, Ciura S, Kabashi E. Poly-GP accumulation due to C9orf72 loss of function induces motor neuron apoptosis through autophagy and mitophagy defects. Autophagy. 2024 Oct;20(10):2164-2185. doi: 10.1080/15548627.2024.2358736. Epub 2024 Sep 24. PMID: 39316747; PMCID: PMC11423671.

Tau accumulation is cleared by the induced expression of VCP via autophagy

Authors:
Giong HK, Hyeon SJ, Lee JG, Cho HJ, Park U, Stein TD, Lee J, Yu K, Ryu H, Lee JS
Citation:
Acta Neuropathol. 2024 Sep 24;148(1):46. doi: 10.1007/s00401-024-02804-z. PMID: 39316141; PMCID: PMC11422276
Epub:
Not Epub
Abstract:
Tauopathy, including frontotemporal lobar dementia and Alzheimer's disease, describes a class of neurodegenerative...
Link:
https://link.springer.com/article/10.1007/s00401-024-02804-z
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Giong HK, Hyeon SJ, Lee JG, Cho HJ, Park U, Stein TD, Lee J, Yu K, Ryu H, Lee JS. Tau accumulation is cleared by the induced expression of VCP via autophagy. Acta Neuropathol. 2024 Sep 24;148(1):46. doi: 10.1007/s00401-024-02804-z. PMID: 39316141; PMCID: PMC11422276.

Bnip3lb-driven mitophagy sustains expansion of the embryonic hematopoietic stem cell pool

Authors:
Meader E, Walcheck MT, Leder MR, Jing R, Wrighton PJ, Sugden WW, Najia MA, Oderberg IM, Taylor VM, LeBlanc ZC, Quenzer ED, Lim S-E, Daley GQ, Goessling W, North TE
Citation:
bioRxiv [preprint] 2024.09.23.614531; doi: https://doi.org/10.1101/2024.09.23.614531
Epub:
Not Epub
Abstract:
Embryonic hematopoietic stem and progenitor cells (HSPCs) have the unique ability to undergo rapid proliferation while...
Link:
https://www.biorxiv.org/content/10.1101/2024.09.23.614531v1
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Meader E, Walcheck MT, Leder MR, Jing R, Wrighton PJ, Sugden WW, Najia MA, Oderberg IM, Taylor VM, LeBlanc ZC, Quenzer ED, Lim S-E, Daley GQ, Goessling W, North TE. Bnip3lb-driven mitophagy sustains expansion of the embryonic hematopoietic stem cell pool. bioRxiv [preprint] 2024.09.23.614531; doi: https://doi.org/10.1101/2024.09.23.614531.

Heteroduplex oligonucleotide technology boosts oligonucleotide splice switching activity of morpholino oligomers in a Duchenne muscular dystrophy mouse model

Authors:
Hasegawa J, Nagata T, Ihara K, Tanihata J, Ebihara S, Yoshida-Tanaka K, Yanagidaira M, Ohara M, Sasaki A, Nakayama M, Yamamoto S, Ishii T, Iwata-Hara R, Naito M, Miyata K, Sakaue F, Yokota T
Citation:
Nat Commun. 2024 Sep 26;15(1):7530. doi: 10.1038/s41467-024-48204-5. PMID: 39327422; PMCID: PMC11427662
Epub:
Not Epub
Abstract:
The approval of splice-switching oligonucleotides with phosphorodiamidate morpholino oligomers (PMOs) for treating Duchenne...
Link:
https://www.nature.com/articles/s41467-024-48204-5
Delivery Method:
intravenous (i.v.)
Organism or Cell Type:
mdx mice
Citation Extract:
Hasegawa J, Nagata T, Ihara K, Tanihata J, Ebihara S, Yoshida-Tanaka K, Yanagidaira M, Ohara M, Sasaki A, Nakayama M, Yamamoto S, Ishii T, Iwata-Hara R, Naito M, Miyata K, Sakaue F, Yokota T. Heteroduplex oligonucleotide technology boosts oligonucleotide splice switching activity of morpholino oligomers in a Duchenne muscular dystrophy mouse model. Nat Commun. 2024 Sep 26;15(1):7530. doi: 10.1038/s41467-024-48204-5. PMID: 39327422; PMCID: PMC11427662.

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