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

Scavenger receptor class A1 mediates uptake of morpholino antisense oligonucleotide into dystrophic skeletal muscle

Authors:
Miyatake S, Mizobe Y, Tsoumpra M, Lim KRQ, Hara Y, Shabanpoor F, Yokota T, Takeda S, Aoki Y
Citation:
Mol Ther Nucl Acids. 2019 Jan 25;14:520-535. doi: 10.1016/j.omtn.2019.01.008.
Epub:
Not Epub
Abstract:
Exon-skipping using phosphorodiamidate morpholino oligomers (PMOs) is a promising treatment strategy for Duchenne muscular...
Link:
https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(19)30012-5
Delivery Method:
peptide-linked, cell bathing
Organism or Cell Type:
mice mdx62, cell culture: H2K-mdx 52 myoblasts
Citation Extract:
Miyatake S, Mizobe Y, Tsoumpra M, Lim KRQ, Hara Y, Shabanpoor F, Yokota T, Takeda S, Aoki Y. Scavenger receptor class A1 mediates uptake of morpholino antisense oligonucleotide into dystrophic skeletal muscle. Mol Ther Nucl Acids. 2019 Jan 25;14:520-535. doi: 10.1016/j.omtn.2019.01.008. .

Comprehensive RNA-Sequencing Analysis in Serum and Muscle Reveals Novel Small RNA Signatures with Biomarker Potential for DMD

Authors:
Coenen-Stass AML, Sork H, Gatto S, Godfrey C, Bhomra A, Krjutškov K, Hart JR, Westholm JO, O'Donovan L, Roos A, Lochmüller H, Puri PL, El Andaloussi S, Wood MJA, Roberts TC
Citation:
Mol Ther Nucleic Acids. 2018 Dec 7;13:1-15. doi: 10.1016/j.omtn.2018.08.005. Epub 2018 Aug 17
Epub:
Not Epub
Abstract:
Extracellular small RNAs (sRNAs), including microRNAs (miRNAs), are promising biomarkers for diseases such as Duchenne muscular...
Link:
https://www.sciencedirect.com/science/article/pii/S2162253118302105
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Coenen-Stass AML, Sork H, Gatto S, Godfrey C, Bhomra A, Krjutškov K, Hart JR, Westholm JO, O'Donovan L, Roos A, Lochmüller H, Puri PL, El Andaloussi S, Wood MJA, Roberts TC. Comprehensive RNA-Sequencing Analysis in Serum and Muscle Reveals Novel Small RNA Signatures with Biomarker Potential for DMD. Mol Ther Nucleic Acids. 2018 Dec 7;13:1-15. doi: 10.1016/j.omtn.2018.08.005. Epub 2018 Aug 17.

Embryonic myosin is a regeneration marker to monitor utrophin based therapies for DMD

Authors:
Guiraud S, Edwards B, Squire SE, Moir L, Berg A, Babbs A, Ramadan N, Wood MJ, Davies KE
Citation:
Hum Mol Genet. 2018;ddy353 doi:10.1093/hmg/ddy353
Epub:
Yes
Abstract:
Duchenne muscular dystrophy (DMD) is a lethal, X-linked muscle-wasting disease caused by lack of the cytoskeletal protein...
Link:
https://academic.oup.com/hmg/advance-article/doi/10.1093/hmg/ddy353/5124574
Delivery Method:
peptide-linked
Organism or Cell Type:
mice mdx
Citation Extract:
Guiraud S, Edwards B, Squire SE, Moir L, Berg A, Babbs A, Ramadan N, Wood MJ, Davies KE. Embryonic myosin is a regeneration marker to monitor utrophin based therapies for DMD. Hum Mol Genet. 2018;ddy353 doi:10.1093/hmg/ddy353 .

Cmah-dystrophin deficient mdx mice display an accelerated cardiac phenotype that is improved following peptide-PMO exon skipping treatment

Authors:
Betts CA, McClorey G, Healicon R, Hammond SM, Manzano R, Muses S, Ball V, Godfrey C, Merritt TM, Westering T, O'Donovan L, Wells KE, Gait MJ, Wells DJ, Tyler D, Wood MJ
Citation:
Hum Mol Genet. 2018 Oct 2. doi: 10.1093/hmg/ddy346. [Epub ahead of print]
Epub:
Yes
Abstract:
Duchenne muscular dystrophy (DMD) is caused by loss of dystrophin protein, leading to progressive muscle weakness and premature...
Link:
https://academic.oup.com/hmg/advance-article/doi/10.1093/hmg/ddy346/5114290
Delivery Method:
peptide-linked
Organism or Cell Type:
mice mdx
Citation Extract:
Betts CA, McClorey G, Healicon R, Hammond SM, Manzano R, Muses S, Ball V, Godfrey C, Merritt TM, Westering T, O'Donovan L, Wells KE, Gait MJ, Wells DJ, Tyler D, Wood MJ. Cmah-dystrophin deficient mdx mice display an accelerated cardiac phenotype that is improved following peptide-PMO exon skipping treatment. Hum Mol Genet. 2018 Oct 2. doi: 10.1093/hmg/ddy346. [Epub ahead of print].

Phase Transitions Drive the Formation of Vesicular Stomatitis Virus Replication Compartments

Authors:
Heinrich BS, Maliga Z, Stein DA, Hyman AA, Whelan SPJ
Citation:
mBio. 2018;9:e02290-17. doi:10.1128/mBio.02290-17
Epub:
Not Epub
Abstract:
RNA viruses that replicate in the cell cytoplasm typically concentrate their replication machinery within specialized...
Link:
https://mbio.asm.org/content/9/5/e02290-17
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: Vero
Citation Extract:
Heinrich BS, Maliga Z, Stein DA, Hyman AA, Whelan SPJ. Phase Transitions Drive the Formation of Vesicular Stomatitis Virus Replication Compartments. mBio. 2018;9:e02290-17. doi:10.1128/mBio.02290-17.

In Vivo Evaluation of Multiple Exon Skipping with Peptide-PMOs in Cardiac and Skeletal Muscles in Dystrophic Dogs

Authors:
Maruyama R, Aoki Y, Takeda S, Yokota T
Citation:
Methods Mol Biol. 2018;1828:365-379. doi: 10.1007/978-1-4939-8651-4_23
Epub:
Not Epub
Abstract:
Exon skipping is an emerging approach to treating Duchenne muscular dystrophy (DMD), one of the most common lethal genetic...
Link:
https://link.springer.com/protocol/10.1007%2F978-1-4939-8651-4_23
Delivery Method:
peptide-linked
Organism or Cell Type:
Canis familiaris (dog)
Citation Extract:
Maruyama R, Aoki Y, Takeda S, Yokota T. In Vivo Evaluation of Multiple Exon Skipping with Peptide-PMOs in Cardiac and Skeletal Muscles in Dystrophic Dogs. Methods Mol Biol. 2018;1828:365-379. doi: 10.1007/978-1-4939-8651-4_23.

A Novel Zebrafish Model for Assessing In Vivo Delivery of Morpholino Oligomers

Authors:
Kim J, Clark K, Barton C, Tanguay R, Moulton H
Citation:
Methods Mol Biol. 2018;1828:293-306. doi: 10.1007/978-1-4939-8651-4_18
Epub:
Not Epub
Abstract:
Morpholino oligomers have great therapeutic potential for treatment of a broad range of human diseases, including viral,...
Link:
https://link.springer.com/protocol/10.1007%2F978-1-4939-8651-4_18
Delivery Method:
peptide-linked
Organism or Cell Type:
zebrafish
Citation Extract:
Kim J, Clark K, Barton C, Tanguay R, Moulton H. A Novel Zebrafish Model for Assessing In Vivo Delivery of Morpholino Oligomers. Methods Mol Biol. 2018;1828:293-306. doi: 10.1007/978-1-4939-8651-4_18.

Skipping of Duplicated Dystrophin Exons: In Vitro Induction and Assessment

Authors:
Greer K, Fletcher S, Wilton SD
Citation:
Methods Mol Biol. 2018;1828:219-228. doi: 10.1007/978-1-4939-8651-4_13
Epub:
Not Epub
Abstract:
Duplications of one or more dystrophin exons that disrupt the reading frame account for about 15% of all Duchenne cases, and...
Link:
https://link.springer.com/protocol/10.1007%2F978-1-4939-8651-4_13
Delivery Method:
peptide-linked
Citation Extract:
Greer K, Fletcher S, Wilton SD. Skipping of Duplicated Dystrophin Exons: In Vitro Induction and Assessment. Methods Mol Biol. 2018;1828:219-228. doi: 10.1007/978-1-4939-8651-4_13.

A platform for discovery of functional cell-penetrating peptides for efficient multi-cargo intracellular delivery

Authors:
Hoffmann K, Milech N, Juraja SM, Cunningham PT, Stone SR, Francis RW, Anastasas M, Hall CM, Heinrich T, Bogdawa HM, Winslow S, Scobie MN, Dewhurst RE, Florez L, Ong F, Kerfoot M, Champain D, Adams AM, Fletcher S, Viola HM, Hool LC, Connor T, Longville BAC, Tan Y-F, Kroeger K, Morath V, Weiss GA, Skerra A, Hopkins RM, Watt PM
Citation:
Sci Rep. 2018;8:12538 doi:10.1038/s41598-018-30790-2
Epub:
Not Epub
Abstract:
Cell penetrating peptides (CPPs) offer great potential to deliver therapeutic molecules to previously inaccessible...
Link:
https://www.nature.com/articles/s41598-018-30790-2
Delivery Method:
peptide-linked
Organism or Cell Type:
mice mdx
Citation Extract:
Hoffmann K, Milech N, Juraja SM, Cunningham PT, Stone SR, Francis RW, Anastasas M, Hall CM, Heinrich T, Bogdawa HM, Winslow S, Scobie MN, Dewhurst RE, Florez L, Ong F, Kerfoot M, Champain D, Adams AM, Fletcher S, Viola HM, Hool LC, Connor T, Longville BAC, Tan Y-F, Kroeger K, Morath V, Weiss GA, Skerra A, Hopkins RM, Watt PM. A platform for discovery of functional cell-penetrating peptides for efficient multi-cargo intracellular delivery. Sci Rep. 2018;8:12538 doi:10.1038/s41598-018-30790-2.

Inhibiting neutral amino acid transport for the treatment of phenylketonuria

Authors:
Belanger AM, Przybylska M, Gefteas E, Furgerson M, Geller S, Kloss A, Cheng SH, Zhu Y, Yew NS
Citation:
JCI Insight. 2018 Jul 26;3(14). pii: 121762. doi: 10.1172/jci.insight.121762. [Epub ahead of print]
Epub:
Not Epub
Abstract:
The neuropathological effects of phenylketonuria (PKU) stem from the inability of the body to metabolize excess phenylalanine (...
Link:
https://insight.jci.org/articles/view/121762
Delivery Method:
Endo-Porter in culture, peptide-linked in mice
Organism or Cell Type:
cell culture: primary mouse renal tubular epithelial cells, mice
Citation Extract:
Belanger AM, Przybylska M, Gefteas E, Furgerson M, Geller S, Kloss A, Cheng SH, Zhu Y, Yew NS. Inhibiting neutral amino acid transport for the treatment of phenylketonuria. JCI Insight. 2018 Jul 26;3(14). pii: 121762. doi: 10.1172/jci.insight.121762. [Epub ahead of print].

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