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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 12176 scientific papers returned from the database with the search filters currently being used below.

Splice modulation of COL4A5 reinstates collagen IV assembly in an organoid model of Alport syndrome

Authors:
Saei H, Estebe B, Goudin N, Esmailpour M, Haure J, Gribouval O, Arrondel C, Moriniere V, Tian P, Lennon R, Antignac C, Mollet G, Dorval G
Citation:
JCI Insight. 2025 Dec 18;11(3):e194759. doi: 10.1172/jci.insight.194759. PMID: 41411050; PMCID: PMC12892906
Epub:
Not Epub
Abstract:
Kidney organoids are an emerging tool for disease modeling, especially genetic diseases. Among these diseases, X-linked Alport...
Link:
https://insight.jci.org/articles/view/194759
Delivery Method:
Endo-Porter PEG
Organism or Cell Type:
kidney organoids
Citation Extract:
Saei H, Estebe B, Goudin N, Esmailpour M, Haure J, Gribouval O, Arrondel C, Moriniere V, Tian P, Lennon R, Antignac C, Mollet G, Dorval G. Splice modulation of COL4A5 reinstates collagen IV assembly in an organoid model of Alport syndrome. JCI Insight. 2025 Dec 18;11(3):e194759. doi: 10.1172/jci.insight.194759. PMID: 41411050; PMCID: PMC12892906.

Modulating alternative splicing of MECP2 is a potential therapeutic strategy for Rett syndrome

Authors:
Tirumala HP, Wang L, Li Y, Bajikar SS, Anderson AG, Wang W, Trostle AJ, Zahabiyon M, Bajic A, Kim JJ, Chen H, Liu Z, Zoghbi HY
Citation:
Sci Transl Med. 2026 Mar 4;18(839):eadq4529. doi: 10.1126/scitranslmed.adq4529. Epub 2026 Mar 4. PMID: 41779872
Epub:
Not Epub
Abstract:
Rett syndrome (RTT) is a neurological disorder caused by loss-of-function mutations in methyl-CpG-binding protein 2 (MECP2),...
Link:
https://www.science.org/doi/10.1126/scitranslmed.adq4529
Delivery Method:
Vivo-Morpholino into brain?
Organism or Cell Type:
cell culture: HEK293T; mice
Citation Extract:
Tirumala HP, Wang L, Li Y, Bajikar SS, Anderson AG, Wang W, Trostle AJ, Zahabiyon M, Bajic A, Kim JJ, Chen H, Liu Z, Zoghbi HY. Modulating alternative splicing of MECP2 is a potential therapeutic strategy for Rett syndrome. Sci Transl Med. 2026 Mar 4;18(839):eadq4529. doi: 10.1126/scitranslmed.adq4529. Epub 2026 Mar 4. PMID: 41779872.

Estrogen produced in the retina by brain aromatase plays a significant role in zebrafish eye development

Authors:
Ulhaq ZS, Kishida M
Citation:
Biochem Biophys Acta Gene Regul Mech. 2026 Mar;1869(1):195133. doi: 10.1016/j.bbagrm.2025.195133. Epub 2025 Dec 15. PMID: 41407153
Epub:
Not Epub
Abstract:
Aromatase is a key enzyme in estrogen biosynthesis, expressed in multiple tissues including the brain and retina. In teleost...
Link:
https://www.sciencedirect.com/science/article/abs/pii/S1874939925000586
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Ulhaq ZS, Kishida M. Estrogen produced in the retina by brain aromatase plays a significant role in zebrafish eye development. Biochem Biophys Acta Gene Regul Mech. 2026 Mar;1869(1):195133. doi: 10.1016/j.bbagrm.2025.195133. Epub 2025 Dec 15. PMID: 41407153.

Role of Glutamate Receptor-Related Genes in Myopia Occurrence and Visual Development

Authors:
Wang R, Tao D, Lu J, Li J, Liu X, Chen C
Citation:
Curr Eye Res. 2026 Mar 10:1-11. doi: 10.1080/02713683.2026.2637650. Epub ahead of print. PMID: 41804850
Epub:
Not Epub
Abstract:
Purpose: Abnormal expression of glutamate receptors is believed to be associated with myopia. However, it is currently unclear...
Link:
https://www.tandfonline.com/doi/full/10.1080/02713683.2026.2637650
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Wang R, Tao D, Lu J, Li J, Liu X, Chen C. Role of Glutamate Receptor-Related Genes in Myopia Occurrence and Visual Development. Curr Eye Res. 2026 Mar 10:1-11. doi: 10.1080/02713683.2026.2637650. Epub ahead of print. PMID: 41804850.

Phillyrin ameliorates sepsis via targeting microRNA-203a-mediated caspase-4/caspase-11/caspase-B downregulation to suppress endothelial pyroptosis

Authors:
Zhou X, Yang G, Tan W, Ding H, Zheng W, Liu Y, Zou L, Su X, Yu L, Li W, Yang L
Citation:
Phytomedicine. 2026 Mar;152:157872. doi: 10.1016/j.phymed.2026.157872. Epub 2026 Jan 22. PMID: 41650504
Epub:
Not Epub
Abstract:
Background: Sepsis, driven by dysregulated host inflammation, remains a leading cause of global mortality and lacks...
Link:
https://www.sciencedirect.com/science/article/abs/pii/S094471132600111X
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Zhou X, Yang G, Tan W, Ding H, Zheng W, Liu Y, Zou L, Su X, Yu L, Li W, Yang L. Phillyrin ameliorates sepsis via targeting microRNA-203a-mediated caspase-4/caspase-11/caspase-B downregulation to suppress endothelial pyroptosis. Phytomedicine. 2026 Mar;152:157872. doi: 10.1016/j.phymed.2026.157872. Epub 2026 Jan 22. PMID: 41650504.

Association between exon-skipping therapy with eteplirsen and cardiac outcomes in Duchenne muscular dystrophy

Authors:
Iff J, Desguerre I, Liu Y, Sarkozy F, Tuttle E, Muntoni F, McDonald CM, Nougues MC, Amthor H, Zhong Y, Wahbi K; Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study Investigators
Citation:
J Neuromuscul Dis. 2026 Mar;13(2):179-189. doi: 10.1177/22143602251366721. Epub 2025 Aug 19. PMID: 40831143
Epub:
Not Epub
Abstract:
Background: Duchenne muscular dystrophy (DMD) leads to dilated cardiomyopathy and heart failure during teenage years or young...
Link:
https://journals.sagepub.com/doi/10.1177/22143602251366721
Delivery Method:
intravenous (i.v.) infusion
Organism or Cell Type:
human
Citation Extract:
Iff J, Desguerre I, Liu Y, Sarkozy F, Tuttle E, Muntoni F, McDonald CM, Nougues MC, Amthor H, Zhong Y, Wahbi K; Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study Investigators. Association between exon-skipping therapy with eteplirsen and cardiac outcomes in Duchenne muscular dystrophy. J Neuromuscul Dis. 2026 Mar;13(2):179-189. doi: 10.1177/22143602251366721. Epub 2025 Aug 19. PMID: 40831143.

Bnip3lb-driven mitophagy maintains fate of the embryonic hematopoietic stem cell pool

Authors:
Meader E, Walcheck MT, Leder MR, Delaney P, Falchetti M, Jing R, Li C, Kambli NK, Wrighton PJ, Sugden WW, Najia MA, Oderberg IM, Taylor VM, LeBlanc ZC, Quenzer ED, Lim SE, Schlaeger T, Daley GQ, Goessling W, North TE
Citation:
Nat Commun. 2026 Feb 24. doi: 10.1038/s41467-026-69593-9. Epub ahead of print. PMID: 41735276
Epub:
Not Epub
Abstract:
Embryonic hematopoietic stem and progenitor cells (HSPCs) have the clinically valuable ability to undergo substantial...
Link:
https://www.nature.com/articles/s41467-026-69593-9
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Meader E, Walcheck MT, Leder MR, Delaney P, Falchetti M, Jing R, Li C, Kambli NK, Wrighton PJ, Sugden WW, Najia MA, Oderberg IM, Taylor VM, LeBlanc ZC, Quenzer ED, Lim SE, Schlaeger T, Daley GQ, Goessling W, North TE. Bnip3lb-driven mitophagy maintains fate of the embryonic hematopoietic stem cell pool. Nat Commun. 2026 Feb 24. doi: 10.1038/s41467-026-69593-9. Epub ahead of print. PMID: 41735276.

Transposable element–gene chimera cartography, origination and role in enhancing transcriptome plasticity

Authors:
Cheon Y, Alvstad EG, Torre D, Quach DT, Nguyen J, Hyun K, Zhou M, Yu T, Liu L, Yoon Y, Reese F, Faraone L, Li Y, Arnold FJ, Fstkchyan YS, Basu U, Kvon E, Valente EM, Ho JSY, Byun M, Guccione E, Shi Y, Weng Z, Seldin M, Marazzi I
Citation:
Nat Struct Mol Biol (2026)
Epub:
Not Epub
Abstract:
Transposable elements (TEs) in the human genome are the heritage of ancient parasitic infections. While most of human DNA...
Link:
https://www.nature.com/articles/s41594-026-01757-z
Delivery Method:
electroporation
Organism or Cell Type:
cell culture: mouse embryonic stem cells
Citation Extract:
Cheon Y, Alvstad EG, Torre D, Quach DT, Nguyen J, Hyun K, Zhou M, Yu T, Liu L, Yoon Y, Reese F, Faraone L, Li Y, Arnold FJ, Fstkchyan YS, Basu U, Kvon E, Valente EM, Ho JSY, Byun M, Guccione E, Shi Y, Weng Z, Seldin M, Marazzi I. Transposable element–gene chimera cartography, origination and role in enhancing transcriptome plasticity. Nat Struct Mol Biol (2026).

Patient-informed CRISPR screen identifies FLNB as a congenital heart disease and ciliopathy gene

Authors:
Arrigo A, Rao V, Ratan A, Kulkarni SS
Citation:
HGG Adv. 2026 Feb 10;7(2):100580. doi: 10.1016/j.xhgg.2026.100580. Epub ahead of print. PMID: 41674076
Epub:
Not Epub
Abstract:
Heterotaxy (HTX) is a congenital disorder characterized by abnormal left-right organ placement, often leading to severe...
Link:
https://www.cell.com/hgg-advances/fulltext/S2666-2477(26)00020-5
Delivery Method:
microinjection
Organism or Cell Type:
Xenopus tropicalis
Citation Extract:
Arrigo A, Rao V, Ratan A, Kulkarni SS. Patient-informed CRISPR screen identifies FLNB as a congenital heart disease and ciliopathy gene. HGG Adv. 2026 Feb 10;7(2):100580. doi: 10.1016/j.xhgg.2026.100580. Epub ahead of print. PMID: 41674076.

Intravenous miR-30c therapy confers dual neurovascular protection and improves long-term recovery after ischemic stroke

Authors:
Jin R, Shukla M, Zhong W, Hu Y, Li J, Wang M, Li G
Citation:
Neurotherapeutics. 2026 Feb 19:e00858. doi: 10.1016/j.neurot.2026.e00858. Epub ahead of print. PMID: 41720714
Epub:
Not Epub
Abstract:
Ischemia–reperfusion (I/R) injury is a major barrier to effective reperfusion therapy in acute ischemic stroke. Secondary...
Link:
https://www.sciencedirect.com/science/article/pii/S1878747926000280
Delivery Method:
Vivo-Morpholino; intravenous (i.v.) injection
Organism or Cell Type:
C57BL/6J mice
Citation Extract:
Jin R, Shukla M, Zhong W, Hu Y, Li J, Wang M, Li G. Intravenous miR-30c therapy confers dual neurovascular protection and improves long-term recovery after ischemic stroke. Neurotherapeutics. 2026 Feb 19:e00858. doi: 10.1016/j.neurot.2026.e00858. Epub ahead of print. PMID: 41720714.

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