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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 275 scientific papers returned from the database with the search filters currently being used below.
There are 275 scientific papers returned from the database with the search filters currently being used below.
Using muscle homing peptide CyPep10 to deliver phosphorodiamidate morpholino oligomers in the mdx mouse
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Citation:
Molecular Therapy - Nucleic Acids (2025), doi: https://doi.org/10.1016/j.omtn.2025.102625 Epub:
Not Epub Abstract:
The severe muscle wasting disorder Duchenne muscular dystrophy (DMD) is characterized by the absence of dystrophin, a protein... Delivery Method:
CP10 and acetyl-R6Gly peptide-linked; intravenous (i.v.) Organism or Cell Type:
mdx mice Citation Extract: Schneider A-FE, Tanganyika-de Winter CL, Jirka SMG, Tan X, Thompson EG, Ha K, Mitra A, Garcia S, Luimes M, Oliver RA, Guerlavais V, Aartsma-Rus A. Using muscle homing peptide CyPep10 to deliver phosphorodiamidate morpholino oligomers in the mdx mouse. Molecular Therapy - Nucleic Acids (2025), doi: https://doi.org/10.1016/j.omtn.2025.102625. |
Exon skipping peptide-conjugated morpholinos downregulate dynamin 2 to rescue centronuclear myopathy
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Citation:
Brain. 2025 Jul 3:awaf249. doi: 10.1093/brain/awaf249. Epub ahead of print. PMID: 40608957 Epub:
Not Epub Abstract:
Centronuclear myopathies (CNM) are rare congenital disorders characterized by muscle weakness and disorganization of myofibers... Delivery Method:
Pip8b2 peptide-linked; mice: intravenous (i.v.) and intramuscular (i.m.) in the tibialis anterior (TA) Organism or Cell Type:
cell culture: C2C12 mouse myoblasts; mice: Dnm2R369W/+ Citation Extract: Moschovaki-Filippidou F, de Carvalho Neves J, Diedhiou N, Jad Y, Böhm J, Wood MJA, Varela MA, Laporte J. Exon skipping peptide-conjugated morpholinos downregulate dynamin 2 to rescue centronuclear myopathy. Brain. 2025 Jul 3:awaf249. doi: 10.1093/brain/awaf249. Epub ahead of print. PMID: 40608957. |
Regulatory T Cell Function Is Not Affected by Antisense Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer (PPMO)-Mediated TMPRSS2 Truncation
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Citation:
Drugs and Drug Candidates. 2025; 4(2):25. https://doi.org/10.3390/ddc4020025 Epub:
Not Epub Abstract:
Background: TMPRSS2 plays an important role in the viral entry mechanisms of influenza viruses and coronaviruses. Therefore,... Delivery Method:
peptide-linked Organism or Cell Type:
Human regulatory T cells (Treg) and responder T cells Citation Extract: Gunne S, Sailer FK, Keutmann L, Schwerdtner M, Moulton HM, Böttcher-Friebertshäuser E, Schiffmann S. Regulatory T Cell Function Is Not Affected by Antisense Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer (PPMO)-Mediated TMPRSS2 Truncation. Drugs and Drug Candidates. 2025; 4(2):25. https://doi.org/10.3390/ddc4020025. |
Silencing mitochondrial gene expression in living cells
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Citation:
Science. 2025 May 22:eadr3498. doi: 10.1126/science.adr3498. Epub ahead of print. 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:
peptide-linked (pCox41-25) 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 May 22:eadr3498. doi: 10.1126/science.adr3498. Epub ahead of print. 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 Organism or Cell Type:
cell culture: human myoblasts with exon 45 deletion, myotubes differentiated into cardiomyocytes; mice: hDMDdel52/mdx 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 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 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. |