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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 277 scientific papers returned from the database with the search filters currently being used below.
There are 277 scientific papers returned from the database with the search filters currently being used below.
Silencing mitochondrial gene expression in living cells
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Citation:
Science. 2025 Jul 31;389(6759):eadr3498. doi: 10.1126/science.adr3498. Epub 2025 Jul 31. PMID: 40403134 Epub:
Not Epub Abstract:
Mitochondria fulfill central functions in metabolism and energy supply. They express their own genome, which encodes key... Delivery Method:
pCox41-25 peptide-linked Organism or Cell Type:
HEK293T cells & isolated mitochondria Citation Extract: Cruz-Zaragoza LD, Dahal D, Koschel M, Boshnakovska A, Zheenbekova A, Yilmaz M, Morgenstern M, Dohrke JN, Bender J, Valpadashi A, Henningfeld KA, Oeljeklaus S, Kremer LS, Breuer M, Urbach O, Dennerlein S, Lidschreiber M, Jakobs S, Warscheid B, Rehling P. Silencing mitochondrial gene expression in living cells. Science. 2025 Jul 31;389(6759):eadr3498. doi: 10.1126/science.adr3498. Epub 2025 Jul 31. PMID: 40403134. |
DG9 boosts PMO nuclear uptake and exon skipping to restore dystrophic muscle and cardiac function
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Citation:
Nat Commun. 2025 May 14;16(1):4477. doi: 10.1038/s41467-025-59494-8. PMID: 40368879; PMCID: PMC12078682 Epub:
Not Epub Abstract:
Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder caused by DMD gene mutations, leading to the loss of... Delivery Method:
Endo-Porter for cells; DG9 peptide-linked for mice delivered retro-orbitally Organism or Cell Type:
cell culture: human myoblasts with exon 45 deletion, myotubes differentiated into cardiomyocytes; mdx mice Citation Extract: Shah MNA, Wilton-Clark H, Haque F, Powell B, Sutanto LE, Maradiya R, Zhabyeyev P, Roshmi RR, Anwar S, Aslesh T, Lim KRQ, Maruyama R, Bigot A, Young CS, Bittner S, Spencer MJ, Moulton HM, Oudit GY, Yokota T. DG9 boosts PMO nuclear uptake and exon skipping to restore dystrophic muscle and cardiac function. Nat Commun. 2025 May 14;16(1):4477. doi: 10.1038/s41467-025-59494-8. PMID: 40368879; PMCID: PMC12078682. |
Cell-penetrating peptide-conjugated, splice-switching oligonucleotides mitigate the phenotype in BTK/Tec double deficient X-linked agammaglobulinemia model
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Citation:
RSC Chem Biol. 2025 Mar 31;6(5):761-771. doi: 10.1039/d4cb00312h. PMID: 40171248; PMCID: PMC11955834 Epub:
Not Epub Abstract:
Splice-switching oligonucleotides (SSOs) have been developed as a treatment for various disorders, including Duchenne muscular... Delivery Method:
Pip6a peptide-linked; intravenous (i.v.) injection for mice Organism or Cell Type:
primary B cells from BTK transgenic mice, BTK transgenic mice, BTK/Tec double-deficient mice Citation Extract: Bestas B, Estupiñán HY, Wang Q, Kharazi S, He C, K Mohammad D, Gupta D, Wiklander OPB, Lehto T, Lundin KE, Berglöf A, Karlsson MCI, Abendroth F, El Andaloussi S, Gait MJ, Wood MJA, Leumann CJ, Stetsenko DA, Månsson R, Wengel J, Zain R, Smith CIE. Cell-penetrating peptide-conjugated, splice-switching oligonucleotides mitigate the phenotype in BTK/Tec double deficient X-linked agammaglobulinemia model. RSC Chem Biol. 2025 Mar 31;6(5):761-771. doi: 10.1039/d4cb00312h. PMID: 40171248; PMCID: PMC11955834. |
An antisense oligomer conjugate with unpredicted bactericidal activity against Fusobacterium nucleatum
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Citation:
mBio. 2025 Jun 11;16(6):e0052425. doi: 10.1128/mbio.00524-25. Epub 2025 Apr 29. PMID: 40298409 Epub:
Not Epub Abstract:
Fusobacteria are commensal members of the oral microbiome that can spread from their primary niche and colonize distal sites in... Delivery Method:
(RXR)4XB peptide-linked Organism or Cell Type:
Fusobacterium nucleatum Citation Extract: Cosi V, Jung J, Popella L, Ponath F, Ghosh C, Barquist L, Vogel J. An antisense oligomer conjugate with unpredicted bactericidal activity against Fusobacterium nucleatum. mBio. 2025 Jun 11;16(6):e0052425. doi: 10.1128/mbio.00524-25. Epub 2025 Apr 29. PMID: 40298409. |
Splicing correction by peptide-conjugated morpholinos as a novel treatment for late-onset Pompe disease
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Citation:
Mol Ther Nucleic Acids. 2025 Mar 27;36(2):102524. doi: 10.1016/j.omtn.2025.102524. PMID: 40255904; PMCID: PMC12008586 Epub:
Not Epub Abstract:
Late-onset Pompe disease (LOPD) is overwhelmingly caused by a single mutation that disrupts splicing of acid-alpha glucosidase... Delivery Method:
Ac-N-Arg6-Gly-OH peptide-linked Organism or Cell Type:
IPSC-derived muscle cells and skeletal muscle tissue; Late-onset Pompe disease mouse model Citation Extract: Oliver RA, Ahern ME, Castaneda PG, Jinadasa T, Bardhan A, Morgan KY, Ha K, Adhikari K, Jungels N, Liberman N, Mitra A, Greer CD, Wright AM, Thompson EG, Garcia S, Copson E, Allu S, Tan X, Callahan AJ, Cai BZ, Guerlavais V, Kim KJ, Malmberg AB. Splicing correction by peptide-conjugated morpholinos as a novel treatment for late-onset Pompe disease. Mol Ther Nucleic Acids. 2025 Mar 27;36(2):102524. doi: 10.1016/j.omtn.2025.102524. PMID: 40255904; PMCID: PMC12008586. |
Cell-penetrating peptide-conjugated, splice-switching oligonucleotides mitigate the phenotype in BTK/Tec double deficient X-linked agammaglobulinemia model
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Citation:
RSC Chem Biol. 2025 Mar 31. doi: 10.1039/d4cb00312h. Epub ahead of print. PMID: 40171248; PMCID: PMC11955834 Epub:
Not Epub Abstract:
Splice-switching oligonucleotides (SSOs) have been developed as a treatment for various disorders, including Duchenne muscular... Delivery Method:
Pip6a peptide-linked; intravenous (i.v.) injection for mice Organism or Cell Type:
cell culture: 2OS luciferase reporter cell line containing a mutated BTK intron 4, primary B cells with mutant BTK transcript; mice: BAC transgenic mice with full-length mutated human BTK pre-mRNA Citation Extract: Bestas B, Estupiñán HY, Wang Q, Kharazi S, He C, K Mohammad D, Gupta D, Wiklander OPB, Lehto T, Lundin KE, Berglöf A, Karlsson MCI, Abendroth F, El Andaloussi S, Gait MJ, Wood MJA, Leumann CJ, Stetsenko DA, Månsson R, Wengel J, Zain R, Smith CIE. Cell-penetrating peptide-conjugated, splice-switching oligonucleotides mitigate the phenotype in BTK/Tec double deficient X-linked agammaglobulinemia model. RSC Chem Biol. 2025 Mar 31. doi: 10.1039/d4cb00312h. Epub ahead of print. PMID: 40171248; PMCID: PMC11955834. |
Long-Term Dystrophin Replacement Therapy in Duchenne Muscular Dystrophy Causes Cardiac Inflammation
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Citation:
J Am Coll Cardiol Basic Trans Science. null2025, 0 (0). https://doi.org/10.1016/j.jacbts.2024.12.015 Epub:
Not Epub Abstract:
Micro-dystrophin replacement gene therapy is currently under clinical trials in Duchenne muscular dystrophy (DMD) patients.... Delivery Method:
peptide-linked combined therapy with AAV-U7; retro-orbital & intravenous (i.v.) injection Organism or Cell Type:
mice: C57BL/6J, dystrophin−/−; utrophin−/− dKO, mdx (utrophin−/−) Citation Extract: Forand A, Moog S, Mougenot N, Lemaitre M, Sevoz-Couche C, Guesmia Z, Virtanen L, Giordani L, Muchir A, Pietri-Rouxel F. Long-Term Dystrophin Replacement Therapy in Duchenne Muscular Dystrophy Causes Cardiac Inflammation. J Am Coll Cardiol Basic Trans Science. null2025, 0 (0). https://doi.org/10.1016/j.jacbts.2024.12.015. |
Phosphorodiamidate Morpholino Oligomers-Loaded Nanobubbles for Ultrasound-Mediated Delivery to the Myocardium in Muscular Dystrophy
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Citation:
ACS Omega Article ASAP. doi: 10.1021/acsomega.4c10896 Epub:
Not Epub Abstract:
Microbubbles (MBs) and nanobubbles (NBs) can oscillate and collapse in response to ultrasound exposure, resulting in contrast... Delivery Method:
R8 peptide-linked; intravenous (i.v.) injection Organism or Cell Type:
mdx mice Citation Extract: Endo-Takahashi Y, Sakurai A, Oguri Y, Katagiri F, Akiyama S, Sashida S, Yamaguchi T, Marunouchi T, Suzuki R, Maruyama K, Tanonaka K, Nomizu M, Negishi Y. Phosphorodiamidate Morpholino Oligomers-Loaded Nanobubbles for Ultrasound-Mediated Delivery to the Myocardium in Muscular Dystrophy. ACS Omega Article ASAP. doi: 10.1021/acsomega.4c10896. |
Targeting a Novel Site in Exon 51 with Antisense Oligonucleotides Induces Enhanced Exon Skipping in a Mouse Model of Duchenne Muscular Dystrophy
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Citation:
Nucleic Acid Ther. 2025 Feb 7. doi: 10.1089/nat.2024.0049. Epub ahead of print. PMID: 39916530 Epub:
Not Epub Abstract:
Exon skipping with antisense oligonucleotides (ASOs) can correct disease-causing mutations of Duchenne muscular dystrophy (DMD... Delivery Method:
peptide-linked or bare oligo; intravenous (i.v.) injection Organism or Cell Type:
mdx mice Citation Extract: Oppeneer T, Qi Y, Henshaw J, Larimore K, Melton A, Puoliväli J, Carter C, Fant P, Brennan S, Wetzel LA, Sigg MA, Crawford BE, Magat J, Froelich S, Woloszynek JC, O'Neill CA. Targeting a Novel Site in Exon 51 with Antisense Oligonucleotides Induces Enhanced Exon Skipping in a Mouse Model of Duchenne Muscular Dystrophy. Nucleic Acid Ther. 2025 Feb 7. doi: 10.1089/nat.2024.0049. Epub ahead of print. PMID: 39916530. |
A peptide conjugate enables systemic injection of the morpholino inducer and more durable induction of T3H38 ribozyme-controlled AAV transgene in mice
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Citation:
Gene Ther. 2025 Feb 12. doi: 10.1038/s41434-025-00519-8. Epub ahead of print. PMID: 39939797 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, Vivo-Morpholino Organism or Cell Type:
mouse Citation Extract: Tang X, Wang H, Yin Y, Zhong G. A peptide conjugate enables systemic injection of the morpholino inducer and more durable induction of T3H38 ribozyme-controlled AAV transgene in mice. Gene Ther. 2025 Feb 12. doi: 10.1038/s41434-025-00519-8. Epub ahead of print. PMID: 39939797. |