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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.
There are 12236 scientific papers returned from the database with the search filters currently being used below.
A JAGN1-associated severe congenital neutropenia zebrafish model revealed an altered G-CSFR signaling and UPR activation
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Citation:
Blood Adv. 2024 Aug 13;8(15):4050-4065. doi: 10.1182/bloodadvances.2023011656. PMID: 38739706 Epub:
Not Epub Abstract:
A variety of autosomal recessive mutations in the JAGN1 gene cause severe congenital neutropenia (CN). However, the underlying... Delivery Method:
microinjection Organism or Cell Type:
zebrafish Citation Extract: Doll L, Welte K, Skokowa J, Bajoghli B. A JAGN1-associated severe congenital neutropenia zebrafish model revealed an altered G-CSFR signaling and UPR activation. Blood Adv. 2024 Aug 13;8(15):4050-4065. doi: 10.1182/bloodadvances.2023011656. PMID: 38739706. |
Multiantigen T-Cell Hybridizers: A Two-Component T-Cell-Activating Therapy
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Citation:
ACS Nano. 2024 Aug 16. doi: 10.1021/acsnano.4c06500. Online ahead of print. PMID: 39149859 Epub:
Not Epub Abstract:
Multispecific T-cell-engaging scaffolds have emerged as effective anticancer therapies for the treatment of hematological... Delivery Method:
Fab-linked; mice: intravenous (i.v.) Organism or Cell Type:
B-cells, T-cells, chronic lymphocytic leukemia (CLL) cells; human non-Hodgkin's lymphoma xenograft mice Citation Extract: Gambles MT, Li S, Kendell I, Li J, Sborov D, Shami P, Yang J, Kopeček J. Multiantigen T-Cell Hybridizers: A Two-Component T-Cell-Activating Therapy. ACS Nano. 2024 Aug 16. doi: 10.1021/acsnano.4c06500. Online ahead of print. PMID: 39149859. |
Lysosomal membrane protein TMEM106B modulates hematopoietic stem and progenitor cell proliferation and differentiation by regulating LAMP2A stability
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Citation:
FASEB J. 2024 Aug 15;38(15):e23870. doi: 10.1096/fj.202400727R. PMID: 39120151 Epub:
Not Epub Abstract:
Hematopoietic stem and progenitor cells (HSPCs) are successfully employed for hematological transplantations, and impaired HSPC... Delivery Method:
microinjection Organism or Cell Type:
zebrafish Citation Extract: Guo D, Xiong H, Yang Z, Zhang R, Shi P, Yao Y, Liu M, Xu C, Wang QK. Lysosomal membrane protein TMEM106B modulates hematopoietic stem and progenitor cell proliferation and differentiation by regulating LAMP2A stability. FASEB J. 2024 Aug 15;38(15):e23870. doi: 10.1096/fj.202400727R. PMID: 39120151. |
Exome-wide association study identifies KDELR3 mutations in extreme myopia
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Citation:
Nat Commun. 2024 Aug 7;15(1):6703. doi: 10.1038/s41467-024-50580-x. PMID: 39112444; PMCID: PMC11306401. Epub:
Not Epub Abstract:
Extreme myopia (EM), defined as a spherical equivalent (SE) ≤ -10.00 diopters (D), is one of the leading causes of sight... Delivery Method:
microinjection Organism or Cell Type:
zebrafish Citation Extract: Yuan J, Zhuang YY, Liu X, Zhang Y, Li K, Chen ZJ, Li D, Chen H, Liang J, Yao Y, Yu X, Zhuo R, Zhao F, Zhou X; Myopia Associated Genetics and Intervention Consortium; Yu X, Qu J, Su J. Exome-wide association study identifies KDELR3 mutations in extreme myopia. Nat Commun. 2024 Aug 7;15(1):6703. doi: 10.1038/s41467-024-50580-x. PMID: 39112444; PMCID: PMC11306401.. |
Integrated single-cell RNA-seq analysis reveals mitochondrial calcium signaling as a modulator of endothelial-to-mesenchymal transition
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Citation:
Sci Adv. 2024 Aug 9;10(32):eadp6182. doi: 10.1126/sciadv.adp6182. Epub 2024 Aug 9. PMID: 39121218; PMCID: PMC11313856 Epub:
Not Epub Abstract:
Endothelial cells (ECs) are highly plastic, capable of differentiating into various cell types. Endothelial-to-mesenchymal... Delivery Method:
microinjection Organism or Cell Type:
zebrafish Citation Extract: Lebas M, Chinigò G, Courmont E, Bettaieb L, Machmouchi A, Goveia J, Beatovic A, Van Kerckhove J, Robil C, Angulo FS, Vedelago M, Errerd A, Treps L, Gao V, Delgado De la Herrán HC, Mayeuf-Louchart A, L'homme L, Chamlali M, Dejos C, Gouyer V, Garikipati VNS, Tomar D, Yin H, Fukui H, Vinckier S, Stolte A, Conradi LC, Infanti F, Lemonnier L, Zeisberg E, Luo Y, Lin L, Desseyn JL, Pickering J, Kishore R, Madesh M, Dombrowicz D, Perocchi F, Staels B, Pla AF, Gkika D, Cantelmo AR. Integrated single-cell RNA-seq analysis reveals mitochondrial calcium signaling as a modulator of endothelial-to-mesenchymal transition. Sci Adv. 2024 Aug 9;10(32):eadp6182. doi: 10.1126/sciadv.adp6182. Epub 2024 Aug 9. PMID: 39121218; PMCID: PMC11313856. |
The splicing modulator CLK1 is a candidate oncogenic dependency in pediatric high-grade gliomas
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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... 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
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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... 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
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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... 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
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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... 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. |
CRISPR-Cas13d as a molecular tool to achieve targeted gene expression knockdown in chick embryos
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Citation:
bioRxiv [preprint] 2024.08.03.606488; doi: https://doi.org/10.1101/2024.08.03.606488 Epub:
Not Epub Abstract:
The chick embryo is a classical model system commonly used in developmental biology due to its amenability to gene perturbation... Delivery Method:
electroporation Organism or Cell Type:
chick embryo Citation Extract: Kim M & Hutchins EJ. CRISPR-Cas13d as a molecular tool to achieve targeted gene expression knockdown in chick embryos. bioRxiv [preprint] 2024.08.03.606488; doi: https://doi.org/10.1101/2024.08.03.606488. |