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

Reduced expression of human metapneumovirus matrix protein impacts virus assembly and ribonucleoprotein localization

Authors:
Heim CJ, Stein DA, Moulton HM, Galperin E, Dutch RE
Citation:
J Virol. 2026 Aug 31:e0106626. doi: 10.1128/jvi.01066-26. Epub ahead of print. PMID: 42671868
Epub:
Not Epub
Abstract:
Human metapneumovirus (HMPV) causes severe respiratory tract infections in all cohorts, particularly in vulnerable groups such...
Link:
https://journals.asm.org/doi/10.1128/jvi.01066-26
Delivery Method:
(RXR)4XB peptide-linked
Organism or Cell Type:
A549 cells
Citation Extract:
Heim CJ, Stein DA, Moulton HM, Galperin E, Dutch RE. Reduced expression of human metapneumovirus matrix protein impacts virus assembly and ribonucleoprotein localization. J Virol. 2026 Aug 31:e0106626. doi: 10.1128/jvi.01066-26. Epub ahead of print. PMID: 42671868.

Peptide-phosphorodiamidate morpholino oligomer therapy for dysferlinopathy induces pseudoexon skipping and restoration of functional protein

Authors:
Gooding JE, Park G, Wagh A, Watts JK, Dominov JA, Brown RH Jr
Citation:
JCI Insight. 2026 Jul 14;11(16):e204742
Epub:
Not Epub
Abstract:
The dysferlinopathies are a spectrum of autosomal recessive muscle diseases caused by mutations in the dysferlin gene (DYSF)....
Link:
https://insight.jci.org/articles/view/204742
Delivery Method:
Endo-Porter for myotubes, Pip9b2 peptide-linked for mice, DNA-RNA hybrids with cholesterol conjugated to a nucleic acid strand that is complementary to PMO for mice; intravenous (i.v.) for mice
Organism or Cell Type:
patient-derived myotubes; PE44.1 mice
Citation Extract:
Gooding JE, Park G, Wagh A, Watts JK, Dominov JA, Brown RH Jr. Peptide-phosphorodiamidate morpholino oligomer therapy for dysferlinopathy induces pseudoexon skipping and restoration of functional protein. JCI Insight. 2026 Jul 14;11(16):e204742.

Enhanced antisense oligonucleotide delivery reveals that transcript turnover impacts apparent splicing rescue in myotonic dystrophy

Authors:
Shea EN, Olafson HR, Muscato DR, Adams LM, Valero MC, McKee KK, Shen X, Dougherty PG, Hicks AN, Liu N, Kheirabadi M, Streeter M, Li X, Mon P, Girgenrath M, Sethuraman N, Qian Z, Wang ET
Citation:
Mol Ther. 2026 May 19:S1525-0016(26)00381-3. doi: 10.1016/j.ymthe.2026.04.060. Epub ahead of print. PMID: 42163456
Epub:
Not Epub
Abstract:
Steric-blocking antisense oligonucleotides rescue myotonic dystrophy type 1 phenotypes in preclinical models and are under...
Link:
https://www.cell.com/molecular-therapy-family/molecular-therapy/abstract/S1525-0016(26)00381-3
Delivery Method:
peptide-linked, intravenous (i.v.) injection
Organism or Cell Type:
HSALR mice
Citation Extract:
Shea EN, Olafson HR, Muscato DR, Adams LM, Valero MC, McKee KK, Shen X, Dougherty PG, Hicks AN, Liu N, Kheirabadi M, Streeter M, Li X, Mon P, Girgenrath M, Sethuraman N, Qian Z, Wang ET. Enhanced antisense oligonucleotide delivery reveals that transcript turnover impacts apparent splicing rescue in myotonic dystrophy. Mol Ther. 2026 May 19:S1525-0016(26)00381-3. doi: 10.1016/j.ymthe.2026.04.060. Epub ahead of print. PMID: 42163456.

PPMO-based exon skipping therapy improves respiratory function in the mdx mouse model of Duchenne muscular dystrophy

Authors:
Biswas DD, Rodriguez MYH, El Haddad L, Hoffmann G, Copson E, Mayman S, Jose J, Lang S, Chen O, Dela Pena A, Levin E, Abdelbarr M, Slyne A, ElMallah MK
Citation:
Mol Ther Nucleic Acids. 2025 Dec 26;37(1):102810. doi: 10.1016/j.omtn.2025.102810. PMID: 41552386; PMCID: PMC12809079
Epub:
Not Epub
Abstract:
Duchenne muscular dystrophy (DMD) is a severe X-linked recessive disorder caused by a deficiency of dystrophin, leading to...
Link:
https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(25)00364-6
Delivery Method:
peptide-linked, intravenous (i.v.) injection
Organism or Cell Type:
mdx mice
Citation Extract:
Biswas DD, Rodriguez MYH, El Haddad L, Hoffmann G, Copson E, Mayman S, Jose J, Lang S, Chen O, Dela Pena A, Levin E, Abdelbarr M, Slyne A, ElMallah MK. PPMO-based exon skipping therapy improves respiratory function in the mdx mouse model of Duchenne muscular dystrophy. Mol Ther Nucleic Acids. 2025 Dec 26;37(1):102810. doi: 10.1016/j.omtn.2025.102810. PMID: 41552386; PMCID: PMC12809079.

Cardiac and skeletal muscle delivery of biotherapeutics with a blood vessel epicardial substance-targeting peptide

Authors:
Wang B, Cao J, Wu J, Zhao Y, Zhang Y, Abendroth F, Lin C, Zhong L, Yu H, Seow Y, Ou M, Vázquez O, Mei L, Yin H, Han G
Citation:
Biomaterials. 2026 Jan 4;329:123986. doi: 10.1016/j.biomaterials.2026.123986. Epub ahead of print. PMID: 41506143
Epub:
Not Epub
Abstract:
Although peptide-based delivery strategies show promise for muscle and heart diseases, delivery of biotherapeutics to both...
Link:
https://www.sciencedirect.com/science/article/abs/pii/S0142961226000104
Delivery Method:
BV2 peptide-linked, exosome; injection
Organism or Cell Type:
mdx and dystrophin/utrophin double-knockout (DKO) mice
Citation Extract:
Wang B, Cao J, Wu J, Zhao Y, Zhang Y, Abendroth F, Lin C, Zhong L, Yu H, Seow Y, Ou M, Vázquez O, Mei L, Yin H, Han G. Cardiac and skeletal muscle delivery of biotherapeutics with a blood vessel epicardial substance-targeting peptide. Biomaterials. 2026 Jan 4;329:123986. doi: 10.1016/j.biomaterials.2026.123986. Epub ahead of print. PMID: 41506143.

Targeted antisense oligonucleotide treatment rescues developmental alterations in spinal muscular atrophy organoids

Authors:
Faravelli I, Rinchetti P, Tambalo M, Simutin I, Mapelli L, Mancinelli S, Miotto M, Rizzuti M, D'Angelo A, Cordiglieri C, Forotti G, Peano C, Kunderfranco P, Calandriello L, Comi GP, Paraboschi E, Pali E, Beatrice F, D'Angelo E, Przedborski S, Nizzardo M, Lodato S, Corti S
Citation:
Nat Commun. 2025 Dec 21. doi: 10.1038/s41467-025-67725-1. Epub ahead of print. PMID: 41423447
Epub:
Not Epub
Abstract:
Spinal muscular atrophy (SMA) is a severe neurological disease caused by mutations in the SMN1 gene, characterized by early...
Link:
https://www.nature.com/articles/s41467-025-67725-1
Delivery Method:
R6 peptide-linked
Organism or Cell Type:
SMA type 1 patient-derived spinal cord and cerebral organoids
Citation Extract:
Faravelli I, Rinchetti P, Tambalo M, Simutin I, Mapelli L, Mancinelli S, Miotto M, Rizzuti M, D'Angelo A, Cordiglieri C, Forotti G, Peano C, Kunderfranco P, Calandriello L, Comi GP, Paraboschi E, Pali E, Beatrice F, D'Angelo E, Przedborski S, Nizzardo M, Lodato S, Corti S. Targeted antisense oligonucleotide treatment rescues developmental alterations in spinal muscular atrophy organoids. Nat Commun. 2025 Dec 21. doi: 10.1038/s41467-025-67725-1. Epub ahead of print. PMID: 41423447.

DG9-Conjugated Morpholino-Based Exon 51-Skipping Therapy for Duchenne Muscular Dystrophy

Authors:
Shah MNA, Sutanto LE, Yokota T
Citation:
Methods Mol Biol. 2026;2963:1-14. doi: 10.1007/978-1-0716-4738-7_1. PMID: 41028673
Epub:
Not Epub
Abstract:
Duchenne muscular dystrophy (DMD) is a severe genetic disorder caused by mutations in the DMD gene that leads to the loss of...
Link:
https://link.springer.com/protocol/10.1007/978-1-0716-4738-7_1
Delivery Method:
DG9 peptide-linked; retro-orbital
Organism or Cell Type:
mdx mice
Citation Extract:
Shah MNA, Sutanto LE, Yokota T. DG9-Conjugated Morpholino-Based Exon 51-Skipping Therapy for Duchenne Muscular Dystrophy. Methods Mol Biol. 2026;2963:1-14. doi: 10.1007/978-1-0716-4738-7_1. PMID: 41028673.

DG9-Conjugated Morpholino Rescues Phenotype in Spinal Muscular Atrophy Mice

Authors:
Haque US, Kohut M, Yokota T
Citation:
Methods Mol Biol. 2026;2963:15-40. doi: 10.1007/978-1-0716-4738-7_2. PMID: 41028674
Epub:
Not Epub
Abstract:
Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease characterized by decreased expression of the survival...
Link:
https://link.springer.com/protocol/10.1007/978-1-0716-4738-7_2
Delivery Method:
DG9 peptide-linked, subcutaneous (s.c.)
Organism or Cell Type:
SMA mice
Citation Extract:
Haque US, Kohut M, Yokota T. DG9-Conjugated Morpholino Rescues Phenotype in Spinal Muscular Atrophy Mice. Methods Mol Biol. 2026;2963:15-40. doi: 10.1007/978-1-0716-4738-7_2. PMID: 41028674.

Integrative Multi-Omics Analysis In Vivo Identifies Influenza A Virus Host Factors

Authors:
Shriver LP, Polacco B, Eckhardt M, Chang MW, Martínez-Romero C, Adkins-Travis K, Cahalane C, Haas K, Stein DA, Fourati S, Martin-Sancho L, Metreveli G, Johnson JR, Muñoz-Moreno R, Newton BW, Kaake RM, Zhou Y, Jimenez-Morales D, VonDollen J, Verschueren E, Swaney DL, Nguyen TT, Stevenson EJ, Stancliffe E, Patti GJ, Albrecht R, Moulton HM, Pache L, Sekaly RP, Benner C, Hultquist JF, Chanda SK, Garcia Sastre A, Krogan NJ
Citation:
iScience. 22 September 2025
Epub:
Not Epub
Abstract:
Influenza A virus (IAV) infection remodels cellular processes to support viral replication. Modulation of host factors by the...
Link:
https://www.sciencedirect.com/science/article/pii/S2589004225019054
Delivery Method:
intranasal peptide-linked
Organism or Cell Type:
Balb/CJ mice; mouse embryonic fibroblasts (MEFs)
Citation Extract:
Shriver LP, Polacco B, Eckhardt M, Chang MW, Martínez-Romero C, Adkins-Travis K, Cahalane C, Haas K, Stein DA, Fourati S, Martin-Sancho L, Metreveli G, Johnson JR, Muñoz-Moreno R, Newton BW, Kaake RM, Zhou Y, Jimenez-Morales D, VonDollen J, Verschueren E, Swaney DL, Nguyen TT, Stevenson EJ, Stancliffe E, Patti GJ, Albrecht R, Moulton HM, Pache L, Sekaly RP, Benner C, Hultquist JF, Chanda SK, Garcia Sastre A, Krogan NJ. Integrative Multi-Omics Analysis In Vivo Identifies Influenza A Virus Host Factors. iScience. 22 September 2025.

Antisense phosphorodiamidate morpholino oligomers retain activity in Burkholderia cepacia complex biofilm

Authors:
Mendez AR, Pybus C, Greenberg DE
Citation:
Frontiers in Microbiology. Volume 16 - 2025, 2025. doi: 10.3389/fmicb.2025.1660799
Epub:
Not Epub
Abstract:
Background: Members of the Burkholderia cepacia complex (Bcc) are known to cause severe pulmonary infections in...
Link:
https://www.frontiersin.org/journals/microbiology/articles/10.3389/fmicb.2025.1660799
Delivery Method:
peptide-linked
Organism or Cell Type:
Burkholderia cepacia
Citation Extract:
Mendez AR, Pybus C, Greenberg DE. Antisense phosphorodiamidate morpholino oligomers retain activity in Burkholderia cepacia complex biofilm. Frontiers in Microbiology. Volume 16 - 2025, 2025. doi: 10.3389/fmicb.2025.1660799.

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