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

P.120 Unlocking the potential of oligonucleotide therapeutics for Duchenne muscular dystrophy through enhanced delivery

Authors:
Mellion M, McArthur J, Holland A, Gunnoo S, Ching S, Johnson R, Irwin C, Lonkar P, Bracegirdle S, Svenstrup N, Goyal J, Godfrey C, Larkindale J
Citation:
Neuromuscul Disord. 2022;32(Suppl 1):S99. doi:10.1016/j.nmd.2022.07.236
Epub:
Not Epub
Abstract:
PGN-EDO51 is PepGen's clinical candidate to treat individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51...
Link:
https://www.sciencedirect.com/science/article/pii/S0960896622004382
Delivery Method:
peptide-linked
Organism or Cell Type:
mice, non-human primates
Citation Extract:
Mellion M, McArthur J, Holland A, Gunnoo S, Ching S, Johnson R, Irwin C, Lonkar P, Bracegirdle S, Svenstrup N, Goyal J, Godfrey C, Larkindale J. P.120 Unlocking the potential of oligonucleotide therapeutics for Duchenne muscular dystrophy through enhanced delivery. Neuromuscul Disord. 2022;32(Suppl 1):S99. doi:10.1016/j.nmd.2022.07.236.

O.14 EEV-Conjugated PMO results in nuclear foci reduction and aberrant splicing correction in myotonic dystrophy cell and animal models

Authors:
Grigenrath M, Estrella N, Hicks A, Shen X, Wysk M, Kheirabadi M, Streeter M, Lian W, Liu N, Blake S, Brennan C, Li N, Batagui V, Oye K, Gao N, Wang D, Qian X, Sethuraman N
Citation:
Neuromuscul Disord. 2022;32(Suppl 1):S96. doi:/10.1016/j.nmd.2022.07.224
Epub:
Not Epub
Abstract:
Myotonic dystrophy type 1 (DM1) is the most common adult-onset muscular dystrophy, manifesting in multisystemic effects...
Link:
https://www.sciencedirect.com/science/article/pii/S0960896622004266
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: patient-derived muytoubes, mice
Citation Extract:
Grigenrath M, Estrella N, Hicks A, Shen X, Wysk M, Kheirabadi M, Streeter M, Lian W, Liu N, Blake S, Brennan C, Li N, Batagui V, Oye K, Gao N, Wang D, Qian X, Sethuraman N. O.14 EEV-Conjugated PMO results in nuclear foci reduction and aberrant splicing correction in myotonic dystrophy cell and animal models. Neuromuscul Disord. 2022;32(Suppl 1):S96. doi:/10.1016/j.nmd.2022.07.224.

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:
peptide-linked
Organism or Cell Type:
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://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(22)00232-3
Delivery Method:
peptide-linked
Organism or Cell Type:
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.

Anti-cancer effect of hUC-MSC-derived exosome-mediated delivery of PMO-miR-146b-5p in colorectal cancer

Authors:
Yu S, Liao R, Bai L, Guo M, Zhang Y, Zhang Y, Yang Q, Song Y, Li Z, Meng Q, Wang S, Huang X
Citation:
Research Square 2022:[preprint] doi:10.21203/rs.3.rs-1704679/v1
Epub:
Not Epub
Abstract:
Background: Antisense oligonucleotide (ASO) is a novel therapeutic platform for targeted cancer therapy. Colorectal cancer (CRC...
Link:
https://www.researchsquare.com/article/rs-1704679/v1
Delivery Method:
peptide-linked exosomal
Organism or Cell Type:
cell culture: CRC
Citation Extract:
Yu S, Liao R, Bai L, Guo M, Zhang Y, Zhang Y, Yang Q, Song Y, Li Z, Meng Q, Wang S, Huang X. Anti-cancer effect of hUC-MSC-derived exosome-mediated delivery of PMO-miR-146b-5p in colorectal cancer. Research Square 2022:[preprint] doi:10.21203/rs.3.rs-1704679/v1.

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:
C57BL/6 or BALB/c mice
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:
peptide-linked
Organism or Cell Type:
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.

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