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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.

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.

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....
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:[Epub ahead of print] doi:10.1021/acsbiomedchemau.1c00053
Epub:
Yes
Abstract:
Cell-penetrating peptides (CPPs) can cross the cell membrane to enter the cytosol and deliver otherwise nonpenetrant...
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:[Epub ahead of print] doi:10.1021/acsbiomedchemau.1c00053.

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...
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...
Delivery Method:
peptide-linked
Organism or Cell Type:
mice hDMDdel52;mdx
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.

A Dystrophin Exon-52 Deleted Miniature Pig Model of Duchenne Muscular Dystrophy and Evaluation of Exon Skipping

Authors:
Echigoya Y, Trieu N, Duddy W, Moulton HM, Yin H, Partridge TA, Hoffman EP, Kornegay JN, Rohret FA, Rogers CS, Yokota T
Citation:
Int J Mol Sci. 2021;22(23):13065. doi:10.3390/ijms222313065
Epub:
Not Epub
Abstract:
Duchenne muscular dystrophy (DMD) is a lethal X-linked recessive disorder caused by mutations in the DMD gene and the...
Delivery Method:
peptide-linked, Endo-Porter
Organism or Cell Type:
Sus scrofa domestica (pig)
Citation Extract:
Echigoya Y, Trieu N, Duddy W, Moulton HM, Yin H, Partridge TA, Hoffman EP, Kornegay JN, Rohret FA, Rogers CS, Yokota T. A Dystrophin Exon-52 Deleted Miniature Pig Model of Duchenne Muscular Dystrophy and Evaluation of Exon Skipping. Int J Mol Sci. 2021;22(23):13065. doi:10.3390/ijms222313065 .

Targeting IgE polyadenylation signal with antisense oligonucleotides decreases IgE secretion and plasma cell viability

Authors:
Marchalot A, Horiot C, Lambert J-M, Carrion C, Oblet C, Pollet J, Cogné M, Moreau J, Laffleur B, Delpy L
Citation:
J Allergy Clin Immunol. 2021 Nov 2;S0091-6749(21)01680-8. doi: 10.1016/j.jaci.2021.09.039.
Epub:
Not Epub
Abstract:
Background: Allergy regroups numerous complex and various diseases classified as IgE-dependent or non-dependent...
Delivery Method:
Vivo-Morpholino, peptide-linked
Organism or Cell Type:
cell culture: B cells, myeloma cells; InEps mice
Citation Extract:
Marchalot A, Horiot C, Lambert J-M, Carrion C, Oblet C, Pollet J, Cogné M, Moreau J, Laffleur B, Delpy L. Targeting IgE polyadenylation signal with antisense oligonucleotides decreases IgE secretion and plasma cell viability. J Allergy Clin Immunol. 2021 Nov 2;S0091-6749(21)01680-8. doi: 10.1016/j.jaci.2021.09.039..

Deep Learning Enables Discovery of a Short Nuclear Targeting Peptide for Efficient Delivery of Antisense Oligomers

Authors:
López-Vidal EM, Schissel CK, Mohapatra S, Bellovoda K, Wu C-L, Wood JA, Malmberg AB, Loas A, Gómez-Bombarelli R, Pentelute BL
Citation:
JACS Au. 2021;[Epub] doi:10.1021/jacsau.1c00327
Epub:
Not Epub
Abstract:
Therapeutic macromolecules such as proteins and oligonucleotides can be highly efficacious but are often limited to...
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: HeLa, mice
Citation Extract:
López-Vidal EM, Schissel CK, Mohapatra S, Bellovoda K, Wu C-L, Wood JA, Malmberg AB, Loas A, Gómez-Bombarelli R, Pentelute BL. Deep Learning Enables Discovery of a Short Nuclear Targeting Peptide for Efficient Delivery of Antisense Oligomers. JACS Au. 2021;[Epub] doi:10.1021/jacsau.1c00327.

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

Authors:
Schissel CK, Farquhar CE, Malmberg AB, Loas A, Pentelute BL
Citation:
bioRxiv. 2021;[preprint] doi:10.1101/2021.09.30.462617
Epub:
Not Epub
Abstract:
Cell-penetrating peptides (CPPs) can cross the cell membrane to enter the cytosol and deliver otherwise non-penetrant...
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. bioRxiv. 2021;[preprint] doi:10.1101/2021.09.30.462617.

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