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

DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration

Authors:
Aslesh T, Erkut E, Ren J, Lim KRQ, Woo S, Hatlevig S, Moulton HM, Gosgnach S, Greer J, Maruyama R, Yokota T
Citation:
JCI Insight. 2023;8(5):e160516. doi: 10.1172/jci.insight.160516. Online ahead of print
Epub:
Not Epub
Abstract:
Antisense oligonucleotide (AO)-mediated therapy is a promising strategy to treat several neurological diseases including spinal...
Link:
https://insight.jci.org/articles/view/160516
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Aslesh T, Erkut E, Ren J, Lim KRQ, Woo S, Hatlevig S, Moulton HM, Gosgnach S, Greer J, Maruyama R, Yokota T. DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration. JCI Insight. 2023;8(5):e160516. doi: 10.1172/jci.insight.160516. Online ahead of print.

PPMOs: A Case Study for Cell-Penetrating Peptide Application

Authors:
Bittner S, Moulton ZX, Moulton HM
Citation:
In Cell-Penetrating Peptides (eds M. Oba and Y. Demizu). 2023:ch 15. doi:10.1002/9783527835997.ch15
Epub:
Not Epub
Abstract:
The current state of the art for in vivo antisense morpholino technology is peptide-conjugated phosphorodiamidate morpholino...
Link:
https://onlinelibrary.wiley.com/doi/10.1002/9783527835997.ch15
Delivery Method:
peptide-linked
Citation Extract:
Bittner S, Moulton ZX, Moulton HM. PPMOs: A Case Study for Cell-Penetrating Peptide Application. In Cell-Penetrating Peptides (eds M. Oba and Y. Demizu). 2023:ch 15. doi:10.1002/9783527835997.ch15.

Peptide-Conjugated PMOs for the Treatment of Myotonic Dystrophy

Authors:
Stoodley J, Miraz DS, Jad Y, Fischer M, Wood MJA, Varela MA
Citation:
Methods Mol Biol. 2023;2587:209-237. doi: 10.1007/978-1-0716-2772-3_13
Epub:
Not Epub
Abstract:
Antisense oligonucleotides (ASOs) have shown great therapeutic potential in the treatment of many neuromuscular diseases...
Link:
https://link.springer.com/protocol/10.1007/978-1-0716-2772-3_13
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Stoodley J, Miraz DS, Jad Y, Fischer M, Wood MJA, Varela MA. Peptide-Conjugated PMOs for the Treatment of Myotonic Dystrophy. Methods Mol Biol. 2023;2587:209-237. doi: 10.1007/978-1-0716-2772-3_13.

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
Organism or Cell Type:
mice
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..

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.

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