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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 12343 scientific papers returned from the database with the search filters currently being used below.
There are 12343 scientific papers returned from the database with the search filters currently being used below.
A Population Pharmacokinetic Model to Inform Extension of the Eteplirsen Dosing Regimen Across the Broad DMD Population
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Citation:
CPT Pharmacometrics Syst Pharmacol. 2025 Feb 26. doi: 10.1002/psp4.70001. Epub ahead of print. PMID: 40009522 Epub:
Not Epub Abstract:
Duchenne muscular dystrophy (DMD) is characterized by progressive, irreversible muscle damage that usually leads to premature... Delivery Method:
intravenous (i.v.) infusion Organism or Cell Type:
human Citation Extract: Patel Y, Orogun L, Yocum N, Rodino-Klapac LR, East L. A Population Pharmacokinetic Model to Inform Extension of the Eteplirsen Dosing Regimen Across the Broad DMD Population. CPT Pharmacometrics Syst Pharmacol. 2025 Feb 26. doi: 10.1002/psp4.70001. Epub ahead of print. PMID: 40009522. |
Improved induction of ribozyme-controlled AAV transgene via peptide-conjugated morpholino oligos
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Citation:
Gene Ther. 2025 Feb 26. doi: 10.1038/s41434-025-00520-1. Epub ahead of print. PMID: 40011707 Epub:
Not Epub Abstract:
Genetic switches that allow for precise control over transgene expression timing or levels may improve the safety and expand... Delivery Method:
peptide-linked Organism or Cell Type:
mouse Citation Extract: Cheng T, Chen B, Zou W. Improved induction of ribozyme-controlled AAV transgene via peptide-conjugated morpholino oligos. Gene Ther. 2025 Feb 26. doi: 10.1038/s41434-025-00520-1. Epub ahead of print. PMID: 40011707. |
Unveiling the role of RPS17 and SLC4A1 in diamond-Blackfan Anemia: A zebrafish-based study
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Citation:
Blood Cells Mol Dis. 2025 Feb 25;112:102912. doi: 10.1016/j.bcmd.2025.102912. Epub ahead of print. PMID: 40015014 Epub:
Not Epub Abstract:
Diamond-Blackfan Anemia (DBA) is a rare congenital disorder characterized by macrocytic anemia, physical abnormalities, and... Delivery Method:
microinjection Organism or Cell Type:
zebrafish Citation Extract: Kim K, Lee H, Ahn S, Kim YH, Oh CK . Unveiling the role of RPS17 and SLC4A1 in diamond-Blackfan Anemia: A zebrafish-based study. Blood Cells Mol Dis. 2025 Feb 25;112:102912. doi: 10.1016/j.bcmd.2025.102912. Epub ahead of print. PMID: 40015014. |
TGF-β signaling controls neural crest developmental plasticity via SMAD2/3
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Citation:
Dev Cell. 2025 Feb 18:S1534-5807(25)00059-0. doi: 10.1016/j.devcel.2025.01.018. Epub ahead of print. PMID: 39983721 Epub:
Not Epub Abstract:
The neural crest is a highly plastic stem cell population that represents an exception to the germ layer theory. Despite being... Delivery Method:
electroporation Organism or Cell Type:
chick embryo Citation Extract: Rothstein M, Azambuja AP, Kanno TY, Breen C, Simoes-Costa M. TGF-β signaling controls neural crest developmental plasticity via SMAD2/3. Dev Cell. 2025 Feb 18:S1534-5807(25)00059-0. doi: 10.1016/j.devcel.2025.01.018. Epub ahead of print. PMID: 39983721. |
Dominant rhabdomyolysis linked to a recurrent ATP2A2 variant reducing SERCA2 function in muscle
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Citation:
Brain. 2025 Feb 19:awaf067. doi: 10.1093/brain/awaf067. Epub ahead of print. PMID: 39970126 Epub:
Not Epub Abstract:
Rhabdomyolysis is an acute failure of cellular homeostasis resulting in muscle breakdown, triggered by trauma, infection, drugs... Delivery Method:
microinjection Organism or Cell Type:
zebrafish Citation Extract: Malaichamy S, Idoux R, Polavarapu K, Šikić K, Holla E, Thompson R, Spendiff S, Schänzer A, Kusters B, Freeman E, Hentschel A, O'Neil D, Carmona-Martinez R, Dobelmann V, Tucht C, Schouten M, Ruck T, Schara-Schmidt U, Kamsteeg EJ, Ramadža DP, Jakovčević A, Zigman T, Čavka M, Karcagi V, Herczegfalvi A, Laurie S, Matalonga L, Beltran S, Horvath R, Voermans N, Roos A, Barić I, Lochmüller H. Dominant rhabdomyolysis linked to a recurrent ATP2A2 variant reducing SERCA2 function in muscle. Brain. 2025 Feb 19:awaf067. doi: 10.1093/brain/awaf067. Epub ahead of print. PMID: 39970126. |
De novo and inherited variants in DDX39B cause a novel neurodevelopmental syndrome
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Citation:
Brain. 2025 Feb 7:awaf035. doi: 10.1093/brain/awaf035. Epub ahead of print. PMID: 39918047 Epub:
Not Epub Abstract:
DDX39B is a conserved member of the DEAD-box family of ATP-dependent RNA helicases, critical in mRNA metabolism across... Delivery Method:
microinjection Organism or Cell Type:
zebrafish Citation Extract: Booth KTA, Jangam SV, Chui MMC, Treat K, Graziani L, Soldano A, Ruan Y, Wan-Hei Hui J, White K, Christensen CK, Lynnes T, Yamamoto S, Kanca O, Tsang MHY, Lynch SA, Mullegama SV, Batista J, Iancu D, Joss SK, Wong SYY, Mak CCY, Kwong AKY, Bellen HJ, Conboy E, Sanges R, Leung AY, Wangler MF, Chung BHY, Vetrini F. De novo and inherited variants in DDX39B cause a novel neurodevelopmental syndrome. Brain. 2025 Feb 7:awaf035. doi: 10.1093/brain/awaf035. Epub ahead of print. PMID: 39918047. |
Morpholino oligomer delivery via bath immersion for use in reverse genetic studies on the early development of eastern oysters (Crassostrea virginica)
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Citation:
Aquaculture. Available online 5 February 2025, 742261 Epub:
Not Epub Abstract:
The eastern oyster genome and expanding omics data have provided valuable insights into this species. However, the limited... Delivery Method:
Vivo-Morpholino, peptide-linked, bathing Organism or Cell Type:
Crassostrea virginica (eastern oyster) Citation Extract: Xu L, Small JM, Hood SM, Zhao M, Plough LV, Wong T-T. Morpholino oligomer delivery via bath immersion for use in reverse genetic studies on the early development of eastern oysters (Crassostrea virginica). Aquaculture. Available online 5 February 2025, 742261. |
Nup107 contributes to the maternal-to-zygotic transition by preventing the premature nuclear export of pri-miR427
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Citation:
Development. 2025 Jan 15;152(2):dev202865. doi: 10.1242/dev.202865. Epub 2025 Feb 4. PMID: 39791357 Epub:
Not Epub Abstract:
Emerging evidence suggests that the nuclear pore complex can have unique compositions and distinct nucleoporin functions in... Delivery Method:
microinjection Organism or Cell Type:
X. tropicalis Citation Extract: Kostiuk V, Kabir R, Levangie K, Empke S, Morgan K, Owens NDL, Lusk CP, Khokha MK. Nup107 contributes to the maternal-to-zygotic transition by preventing the premature nuclear export of pri-miR427. Development. 2025 Jan 15;152(2):dev202865. doi: 10.1242/dev.202865. Epub 2025 Feb 4. PMID: 39791357. |
ROS regulates circadian rhythms by modulating Ezh2 interactions with clock proteins
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Citation:
Redox Biology. 2025. 103526, ISSN 2213-2317, https://doi.org/10.1016/j.redox.2025.103526 Epub:
Not Epub Abstract:
Abstract: Redox imbalance induced by the accumulation of reactive oxygen species (ROS) accelerates age-related processes, often... Delivery Method:
microinjection Organism or Cell Type:
zebrafish Citation Extract: Zhang H-Y, Li K-Y, YWang Y-L, Wei C-J, Gao Y-X, Zhou R, Zhong Y-B, Yin Z-J, Ren D-L. ROS regulates circadian rhythms by modulating Ezh2 interactions with clock proteins. Redox Biology. 2025. 103526, ISSN 2213-2317, https://doi.org/10.1016/j.redox.2025.103526. |
Solid Phase Synthesis of Casimersen and Its 3ʹ-end Modification for Therapeutic Benefits
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Citation:
ChemRxiv [preprint]. 2025; doi:10.26434/chemrxiv-2025-mf99n Epub:
Not Epub Abstract:
Phosphorodiamidate Morpholino Oligonucleotides (PMO) have been well established in the treatment of muscular dystrophies with... Delivery Method:
None Organism or Cell Type:
None Citation Extract: Sinha S, Das A, Pratihar S, Nath Sharma S, Kundu J. Solid Phase Synthesis of Casimersen and Its 3ʹ-end Modification for Therapeutic Benefits. ChemRxiv [preprint]. 2025; doi:10.26434/chemrxiv-2025-mf99n. |