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

Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy

Authors:
Gushchina LV, Vetter TA, Frair EC, Bradley AJ, Grounds KM, Lay JW, Huang N, Suhaiba A, Schnell FJ, Hanson G, Simmons TR, Wein N, Flanigan KM
Citation:
Mol Ther Nucleic Acids. 2022 Nov 9;30:479-492. doi: 10.1016/j.omtn.2022.10.025. eCollection 2022 Dec 13.
Epub:
Not Epub
Abstract:
Duchenne muscular dystrophy (DMD) is a devastating muscle-wasting disease that arises due to the loss of dystrophin expression...
Link:
https://www.cell.com/molecular-therapy-family/nucleic-acids/pdf/S2162-2531(22)00291-8.pdf
Delivery Method:
peptide-linked; intravenous (i.v.)
Organism or Cell Type:
mice: C57Bl/6 & Dup2
Citation Extract:
Gushchina LV, Vetter TA, Frair EC, Bradley AJ, Grounds KM, Lay JW, Huang N, Suhaiba A, Schnell FJ, Hanson G, Simmons TR, Wein N, Flanigan KM. Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy. Mol Ther Nucleic Acids. 2022 Nov 9;30:479-492. doi: 10.1016/j.omtn.2022.10.025. eCollection 2022 Dec 13..

Common and species-specific molecular signatures, networks, and regulators of influenza virus infection in mice, ferrets, and humans

Authors:
Forst CV, Martin-Sancho L, Tripathi S, Wang G, Dos Anjos Borges LG, Wang M, Geber A, Lashua L, Ding T, Zhou X, Carter CE, Metreveli G, Rodriguez-Frandsen A, Urbanowski MD, White KM, Stein DA, Moulton H, Chanda SK, Pache L, Shaw ML, Ross TM, Ghedin E, García-Sastre A, Zhang B
Citation:
Sci Adv. 2022 Oct 7;8(40):eabm5859. doi: 10.1126/sciadv.abm5859. Epub 2022 Oct 5
Epub:
Not Epub
Abstract:
Molecular responses to influenza A virus (IAV) infections vary between mammalian species. To identify conserved and species-...
Link:
https://www.science.org/doi/10.1126/sciadv.abm5859
Delivery Method:
intranasal peptide-linked
Organism or Cell Type:
BALB/c mice
Citation Extract:
Forst CV, Martin-Sancho L, Tripathi S, Wang G, Dos Anjos Borges LG, Wang M, Geber A, Lashua L, Ding T, Zhou X, Carter CE, Metreveli G, Rodriguez-Frandsen A, Urbanowski MD, White KM, Stein DA, Moulton H, Chanda SK, Pache L, Shaw ML, Ross TM, Ghedin E, García-Sastre A, Zhang B. Common and species-specific molecular signatures, networks, and regulators of influenza virus infection in mice, ferrets, and humans. Sci Adv. 2022 Oct 7;8(40):eabm5859. doi: 10.1126/sciadv.abm5859. Epub 2022 Oct 5.

Exon skipping induces uniform dystrophin rescue with dose-dependent restoration of serum miRNA biomarkers and muscle biophysical properties

Authors:
Chwalenia K, Oieni J, Zemła J, Lekka M, Ahlskog N, Coenen-Stass AML, McClorey G, Wood MJA, Lomonosova Y, Roberts TC
Citation:
Mol Ther Nucleic Acids. 2022 Aug 25;29:955-968. doi: 10.1016/j.omtn.2022.08.033. eCollection 2022
Epub:
Not Epub
Abstract:
Therapies that restore dystrophin expression are presumed to correct Duchenne muscular dystrophy (DMD), with antisense-mediated...
Link:
https://pmc.ncbi.nlm.nih.gov/articles/PMC9464767/
Delivery Method:
peptide-linked; intravenous (i.v.)
Organism or Cell Type:
mdx mice
Citation Extract:
Chwalenia K, Oieni J, Zemła J, Lekka M, Ahlskog N, Coenen-Stass AML, McClorey G, Wood MJA, Lomonosova Y, Roberts TC. Exon skipping induces uniform dystrophin rescue with dose-dependent restoration of serum miRNA biomarkers and muscle biophysical properties. Mol Ther Nucleic Acids. 2022 Aug 25;29:955-968. doi: 10.1016/j.omtn.2022.08.033. eCollection 2022.

Helix-Stabilized Cell-Penetrating Peptides for Delivery of Antisense Morpholino Oligomers: Relationships among Helicity, Cellular Uptake, and Antisense Activity

Authors:
Takada H, Tsuchiya K, Demizu Y
Citation:
Bioconjug Chem. 2022 Jun 23. doi: 10.1021/acs.bioconjchem.2c00199. Online ahead of print
Epub:
Yes
Abstract:
The secondary structures of cell-penetrating peptides (CPPs) influence their properties including their cell-membrane...
Link:
https://pubs.acs.org/doi/10.1021/acs.bioconjchem.2c00199
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: HeLa 705
Citation Extract:
Takada H, Tsuchiya K, Demizu Y. Helix-Stabilized Cell-Penetrating Peptides for Delivery of Antisense Morpholino Oligomers: Relationships among Helicity, Cellular Uptake, and Antisense Activity. Bioconjug Chem. 2022 Jun 23. doi: 10.1021/acs.bioconjchem.2c00199. Online ahead of print.

In-Cell Penetration Selection-Mass Spectrometry Produces Noncanonical Peptides for Antisense Delivery

Authors:
Schissel CK, Farquhar CE, Loas A, Malmberg AB, Pentelute BL
Citation:
bioRxiv. 2022;[preprint] doi:10.1101/2022.04.13.488231
Epub:
Not Epub
Abstract:
Peptide-mediated delivery of macromolecules in cells has significant potential therapeutic benefits, but no therapy employing...
Link:
https://www.biorxiv.org/content/10.1101/2022.04.13.488231v1.full
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: HeLa 654
Citation Extract:
Schissel CK, Farquhar CE, Loas A, Malmberg AB, Pentelute BL. In-Cell Penetration Selection-Mass Spectrometry Produces Noncanonical Peptides for Antisense Delivery. bioRxiv. 2022;[preprint] doi:10.1101/2022.04.13.488231.

The RNA helicase DHX16 recognizes specific viral RNA to trigger RIG-I-dependent innate antiviral immunity

Authors:
Hage A, Bharaj P, van Tol S, Giraldo MI, Gonzalez-Orozco M, Valerdi KM, Warren AN, Aguilera-Aguirre L, Xie X, Widen SG, Moulton HM, Lee B, Johnson JR, Krogan NJ, García-Sastre A, Shi PY, Freiberg AN, Rajsbaum R
Citation:
Cell Rep. 2022 Mar 8;38(10):110434. doi: 10.1016/j.celrep.2022.110434
Epub:
Not Epub
Abstract:
Type I interferons (IFN-I) are essential to establish antiviral innate immunity. Unanchored (or free) polyubiquitin (poly-Ub)...
Link:
https://www.cell.com/cell-reports/fulltext/S2211-1247(22)00161-9
Delivery Method:
intranasal peptide-linked
Organism or Cell Type:
mice: C57BL/6 & BALB/c
Citation Extract:
Hage A, Bharaj P, van Tol S, Giraldo MI, Gonzalez-Orozco M, Valerdi KM, Warren AN, Aguilera-Aguirre L, Xie X, Widen SG, Moulton HM, Lee B, Johnson JR, Krogan NJ, García-Sastre A, Shi PY, Freiberg AN, Rajsbaum R. The RNA helicase DHX16 recognizes specific viral RNA to trigger RIG-I-dependent innate antiviral immunity. Cell Rep. 2022 Mar 8;38(10):110434. doi: 10.1016/j.celrep.2022.110434.

Proof of concept of peptide-linked blockmiR-induced MBNL functional rescue in myotonic dystrophy type 1 mouse model

Authors:
Overby SJ, Cerro-Herreros E, González-Martínez I, Varela MA, Seoane-Miraz D, Jad Y, Raz R, Møller T, Pérez-Alonso M, Wood MJ, Llamusí B, Artero R
Citation:
Mol Ther Nucleic Acids. 2022:[Epub ahead of print] doi:10.1016/j.omtn.2022.02.003
Epub:
Yes
Abstract:
Myotonic dystrophy type 1 is a debilitating neuromuscular disease causing muscle weakness, myotonia, and cardiac dysfunction....
Link:
https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(22)00031-2
Delivery Method:
Pip9b2 peptide-linked; intravenous (i.v.)
Organism or Cell Type:
DM1 cells; HSALR mice
Citation Extract:
Overby SJ, Cerro-Herreros E, González-Martínez I, Varela MA, Seoane-Miraz D, Jad Y, Raz R, Møller T, Pérez-Alonso M, Wood MJ, Llamusí B, Artero R. Proof of concept of peptide-linked blockmiR-induced MBNL functional rescue in myotonic dystrophy type 1 mouse model. Mol Ther Nucleic Acids. 2022:[Epub ahead of print] doi:10.1016/j.omtn.2022.02.003.

Cell-Penetrating d-Peptides Retain Antisense Morpholino Oligomer Delivery Activity

Authors:
Schissel CK, Farquhar CE, Malmberg AB, Loas A, Pentelute BL
Citation:
ACS Bio Med Chem Au. 2022 Feb 16;2(2):150-160. doi: 10.1021/acsbiomedchemau.1c00053. PMID: 37101743; PMCID: PMC10114648
Epub:
Yes
Abstract:
Cell-penetrating peptides (CPPs) can cross the cell membrane to enter the cytosol and deliver otherwise nonpenetrant...
Link:
https://pmc.ncbi.nlm.nih.gov/articles/PMC10114648/
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: HeLa 654
Citation Extract:
Schissel CK, Farquhar CE, Malmberg AB, Loas A, Pentelute BL. Cell-Penetrating d-Peptides Retain Antisense Morpholino Oligomer Delivery Activity. ACS Bio Med Chem Au. 2022 Feb 16;2(2):150-160. doi: 10.1021/acsbiomedchemau.1c00053. PMID: 37101743; PMCID: PMC10114648.

Generation of Protein-Phosphorodiamidate Morpholino Oligomer Conjugates for Efficient Cellular Delivery via Anthrax Protective Antigen

Authors:
Palacio-Castañeda V, Brock R, Verdurmen WPR
Citation:
Methods Mol Biol. 2022;2434:129-141. Humana, New York, NY. doi:10.1007/978-1-0716-2010-6_8
Epub:
Not Epub
Abstract:
Phosphorodiamidate morpholino oligomers (PMOs) offer great promise as therapeutic agents for translation blocking or splice...
Link:
https://link.springer.com/protocol/10.1007/978-1-0716-2010-6_8
Delivery Method:
peptide-linked
Citation Extract:
Palacio-Castañeda V, Brock R, Verdurmen WPR. Generation of Protein-Phosphorodiamidate Morpholino Oligomer Conjugates for Efficient Cellular Delivery via Anthrax Protective Antigen. Methods Mol Biol. 2022;2434:129-141. Humana, New York, NY. doi:10.1007/978-1-0716-2010-6_8.

Development of DG9 peptide-conjugated single- and multi-exon skipping therapies for the treatment of Duchenne muscular dystrophy

Authors:
Lim KRQ, Woo S, Melo D, Huang Y, Dzierlega K, Shah MNA, Aslesh T, Roshmi RR, Echigoya Y, Maruyama R, Moulton HM, Yokota T
Citation:
Proc Natl Acad Sci U S A. 2022 Mar 1;119(9):e2112546119. doi: 10.1073/pnas.2112546119
Epub:
Not Epub
Abstract:
Duchenne muscular dystrophy (DMD) is primarily caused by out-of-frame deletions in the dystrophin gene. Exon skipping using...
Link:
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8892351/
Delivery Method:
DG9 peptide-linked; retro-orbital
Organism or Cell Type:
mdx mice
Citation Extract:
Lim KRQ, Woo S, Melo D, Huang Y, Dzierlega K, Shah MNA, Aslesh T, Roshmi RR, Echigoya Y, Maruyama R, Moulton HM, Yokota T. Development of DG9 peptide-conjugated single- and multi-exon skipping therapies for the treatment of Duchenne muscular dystrophy. Proc Natl Acad Sci U S A. 2022 Mar 1;119(9):e2112546119. doi: 10.1073/pnas.2112546119.

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