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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 12237 scientific papers returned from the database with the search filters currently being used below.

SafeSense: An open-access safety atlas for antisense oligonucleotides adverse events in human

Authors:
Vaknin N, Ziv A, Chernobylsky T, Avkin-Nachum S, Rechavi G, Dominissini D, Mor N, N=1 Collaborative
Citation:
Mol Ther Nucleic Acids. 2026 Jul 23;37(3):103035
Epub:
Not Epub
Abstract:
Antisense oligonucleotides (ASOs) therapeutics are a powerful class of RNA-targeted therapy, with applications ranging from...
Link:
https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(26)00206-4
Delivery Method:
intravenous (i.v.) infusion
Organism or Cell Type:
human
Citation Extract:
Vaknin N, Ziv A, Chernobylsky T, Avkin-Nachum S, Rechavi G, Dominissini D, Mor N, N=1 Collaborative. SafeSense: An open-access safety atlas for antisense oligonucleotides adverse events in human. Mol Ther Nucleic Acids. 2026 Jul 23;37(3):103035.

Genetic evidence and cross-species functional characterization implicate CNN2 in age-related macular degeneration susceptibility

Authors:
Cheng FF, Mou H, Liu Z, Zhang CJ, Zhuang YY, Wu Z, Wen XR, Xue A, Zhang X, Yang J, Jin ZB
Citation:
Proc Natl Acad Sci U S A. 2026 Sep 8;123(36):e2533682123. doi: 10.1073/pnas.2533682123. Epub 2026 Sep 2. PMID: 42685076
Epub:
Not Epub
Abstract:
Age-related macular degeneration (AMD) is a leading cause of irreversible visual impairment in the aging population globally....
Link:
https://www.pnas.org/doi/10.1073/pnas.2533682123
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Cheng FF, Mou H, Liu Z, Zhang CJ, Zhuang YY, Wu Z, Wen XR, Xue A, Zhang X, Yang J, Jin ZB. Genetic evidence and cross-species functional characterization implicate CNN2 in age-related macular degeneration susceptibility. Proc Natl Acad Sci U S A. 2026 Sep 8;123(36):e2533682123. doi: 10.1073/pnas.2533682123. Epub 2026 Sep 2. PMID: 42685076.

Tissue flow acts as a guidance cue for immune cell polarization and directional migration

Authors:
Le HA, Hartmann J, Alert R, Mayor R
Citation:
Nat Cell Biol. 2026 Sep 7. doi: 10.1038/s41556-026-02058-9. Epub ahead of print. PMID: 42706384
Epub:
Not Epub
Abstract:
Embryonic myeloid (EM) cells are the first immune-cell population to emerge during development and must disperse throughout the...
Link:
https://www.nature.com/articles/s41556-026-02058-9
Delivery Method:
microinjection
Organism or Cell Type:
Xenopus laevis
Citation Extract:
Le HA, Hartmann J, Alert R, Mayor R. Tissue flow acts as a guidance cue for immune cell polarization and directional migration. Nat Cell Biol. 2026 Sep 7. doi: 10.1038/s41556-026-02058-9. Epub ahead of print. PMID: 42706384.

NF2 lacking exon 11 induced by antisense gene therapy is able to partially recover merlin deficiency in NF2-SWN iPSC-derived spheroid model

Authors:
Casals-Sendra G, Jarne-Sanz I, Catasús N, Boluda-Luis I, Quer A, Amilibia E, Plotkin SR, Lázaro C, Serra E, Blanco I, Castellanos E
Citation:
Mol Ther Nucleic Acids. 2026 Aug 3;37(3):103046. doi: 10.1016/j.omtn.2026.103046. PMID: 42662719; PMCID: PMC13520096
Epub:
Not Epub
Abstract:
NF2-related schwannomatosis (NF2-SWN) is an inherited autosomal dominant disorder resulting from loss-of-function mutations in...
Link:
https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(26)00217-9
Delivery Method:
Endo-Porter, Vivo-Morpholino
Organism or Cell Type:
NF2-SWN iPSC-derived spheroids
Citation Extract:
Casals-Sendra G, Jarne-Sanz I, Catasús N, Boluda-Luis I, Quer A, Amilibia E, Plotkin SR, Lázaro C, Serra E, Blanco I, Castellanos E. NF2 lacking exon 11 induced by antisense gene therapy is able to partially recover merlin deficiency in NF2-SWN iPSC-derived spheroid model. Mol Ther Nucleic Acids. 2026 Aug 3;37(3):103046. doi: 10.1016/j.omtn.2026.103046. PMID: 42662719; PMCID: PMC13520096.

Kinetics, thermodynamics, and mechanisms of PMO interactions from computational molecular modeling

Authors:
Chou Y, Novikov I, Pierson D, Marx KA, Chanda A, Barsegov V
Citation:
Mol Ther Nucleic Acids. 2026 Aug 12;37(3):103056. doi: 10.1016/j.omtn.2026.103056. PMID: 42668701; PMCID: PMC13524873
Epub:
Not Epub
Abstract:
Phosphorodiamidate morpholino oligonucleotides (PMOs) are a class of antisense oligonucleotides. While PMOs have enabled...
Link:
https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(26)00227-1
Delivery Method:
none
Organism or Cell Type:
none
Citation Extract:
Chou Y, Novikov I, Pierson D, Marx KA, Chanda A, Barsegov V. Kinetics, thermodynamics, and mechanisms of PMO interactions from computational molecular modeling. Mol Ther Nucleic Acids. 2026 Aug 12;37(3):103056. doi: 10.1016/j.omtn.2026.103056. PMID: 42668701; PMCID: PMC13524873.

Timing matters: Exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy

Authors:
Stenler S, Huang J, van Westering TLE, Coenen-Stass AML, Jad Y, Krjutškov K, El Andaloussi S, Wood MJA, Roberts TC
Citation:
Mol Ther Nucleic Acids. 2026 Jul 17;37(3):103017. doi: 10.1016/j.omtn.2026.103017. PMID: 42568847; PMCID: PMC13448210
Epub:
Not Epub
Abstract:
Exon skipping is a leading therapeutic approach for Duchenne muscular dystrophy (DMD), whereby modulation of pre-mRNA splicing...
Link:
https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(26)00188-5
Delivery Method:
peptide-linked; intravenous (i.v.) injection
Organism or Cell Type:
mdx and C57BL/10 wild type mice
Citation Extract:
Stenler S, Huang J, van Westering TLE, Coenen-Stass AML, Jad Y, Krjutškov K, El Andaloussi S, Wood MJA, Roberts TC. Timing matters: Exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy. Mol Ther Nucleic Acids. 2026 Jul 17;37(3):103017. doi: 10.1016/j.omtn.2026.103017. PMID: 42568847; PMCID: PMC13448210.

De novo E-cadherin/catenin complex formation controls basal epithelial mechanics and force transmission for apoptotic cell clearance

Authors:
Häkkinen HM, Batet M, Bianchi LF, Jiménez-Delgado S, Pezzano F, Wieser S, Ciampa L, Hoijman E, Vibe C, Wijma S, Ruprecht V
Citation:
Nat Commun. 2026 Aug 27;17(1):8900. doi: 10.1038/s41467-026-76710-1. PMID: 42660948; PMCID: PMC13522565
Epub:
Not Epub
Abstract:
Beyond serving as cohesive barriers, epithelia clear apoptotic cells to regulate development, homeostasis and inflammation. How...
Link:
https://www.nature.com/articles/s41467-026-76710-1
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Häkkinen HM, Batet M, Bianchi LF, Jiménez-Delgado S, Pezzano F, Wieser S, Ciampa L, Hoijman E, Vibe C, Wijma S, Ruprecht V. De novo E-cadherin/catenin complex formation controls basal epithelial mechanics and force transmission for apoptotic cell clearance. Nat Commun. 2026 Aug 27;17(1):8900. doi: 10.1038/s41467-026-76710-1. PMID: 42660948; PMCID: PMC13522565.

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.

Ptprz1b phosphatase binds Prickle2 to promote its membrane localization

Authors:
Le Y, Novotna S, Maia LA, Tolwinski NS, Winkler C, Harnos J
Citation:
iScience. 2026 Jul 13;29(8):116707
Epub:
Not Epub
Abstract:
The Wnt/planar cell polarity (PCP) pathway plays a critical role in the development and homeostasis of multicellular organisms...
Link:
https://www.cell.com/iscience/fulltext/S2589-0042(26)02083-3
Delivery Method:
microinjection
Organism or Cell Type:
Xenopus laevis
Citation Extract:
Le Y, Novotna S, Maia LA, Tolwinski NS, Winkler C, Harnos J. Ptprz1b phosphatase binds Prickle2 to promote its membrane localization. iScience. 2026 Jul 13;29(8):116707.

Four new Duchenne muscular dystrophy mouse models with clinically relevant exon deletions in the human DMD gene

Authors:
van Putten M, Linssen M, de Winter CT, Brouwers CM, Claassens JWC, Verwey N, Walsh M, Stan TL, Aartsma-Rus A, Hohenstein P
Citation:
Dis Model Mech. 2026 Aug 21:dmm.052875
Epub:
Not Epub
Abstract:
Mutation specific therapeutic approaches, like exon skipping or gene-editing, hold promise for the treatment of Duchenne...
Link:
https://journals.biologists.com/dmm/article/doi/10.1242/dmm.052875/372612/Four-new-Duchenne-muscular-dystrophy-mouse-models
Delivery Method:
Vivo-Morpholino, intramuscular (i.m.) injection
Organism or Cell Type:
humanized DMD mouse models with either a deletion of exon 44, 45, 51 or 53
Citation Extract:
van Putten M, Linssen M, de Winter CT, Brouwers CM, Claassens JWC, Verwey N, Walsh M, Stan TL, Aartsma-Rus A, Hohenstein P. Four new Duchenne muscular dystrophy mouse models with clinically relevant exon deletions in the human DMD gene. Dis Model Mech. 2026 Aug 21:dmm.052875.

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