You are here

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.

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...
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...
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...
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...
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-...
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...
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...
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...
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...
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)...
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.

Pages