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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 275 scientific papers returned from the database with the search filters currently being used below.
There are 275 scientific papers returned from the database with the search filters currently being used below.
Peptide-Conjugated PMOs for the Treatment of Myotonic Dystrophy
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... 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
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... 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
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... 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
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... 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
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-... 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
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... 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
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... 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
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... 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
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... 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
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)... 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. |