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

Interpretable Deep Learning for De Novo Design of Cell-Penetrating Abiotic Polymers

Authors:
Schissel CK, Mohapatra S, Wolfe JM, Fadzen CM, Bellovoda K, Wu C-L, Wood JA, Malmberg AB, Loas A, Gomez-Bombarelli R, Pentelute BL.
Citation:
bioRxiv. 2020;[preprint] doi:10.1101/2020.04.10.036566
Epub:
Not Epub
Abstract:
There are more amino acid permutations within a 40-residue sequence than atoms on Earth. This vast chemical search space...
Link:
https://www.biorxiv.org/content/10.1101/2020.04.10.036566v1
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: HeLa, human renal proximal tublule epithelial cells, mice
Citation Extract:
Schissel CK, Mohapatra S, Wolfe JM, Fadzen CM, Bellovoda K, Wu C-L, Wood JA, Malmberg AB, Loas A, Gomez-Bombarelli R, Pentelute BL.. Interpretable Deep Learning for De Novo Design of Cell-Penetrating Abiotic Polymers. bioRxiv. 2020;[preprint] doi:10.1101/2020.04.10.036566.

Combined treatment with peptide-conjugated phosphorodiamidate morpholino oligomer-PPMO and AAV-U7 rescues the severe DMD phenotype in mice

Authors:
Forand A, Muchir A, Mougenot N, Sevoz-Couche C, Peccate C, Lemaitre M, Izabelle C, Wood MJA, Lorain SL, Piétri-Rouxel F
Citation:
Molec Ther Methods Clin Devel. 2020;17:695-708. doi:10.1016/j.omtm.2020.03.011
Epub:
Not Epub
Abstract:
Duchenne muscular dystrophy (DMD) is a devastating neuromuscular disease caused by an absence of the dystrophin protein, which...
Link:
https://www.cell.com/molecular-therapy-family/methods/fulltext/S2329-0501(20)30041-3
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Forand A, Muchir A, Mougenot N, Sevoz-Couche C, Peccate C, Lemaitre M, Izabelle C, Wood MJA, Lorain SL, Piétri-Rouxel F. Combined treatment with peptide-conjugated phosphorodiamidate morpholino oligomer-PPMO and AAV-U7 rescues the severe DMD phenotype in mice. Molec Ther Methods Clin Devel. 2020;17:695-708. doi:10.1016/j.omtm.2020.03.011.

Uniform sarcolemmal dystrophin expression is required to prevent extracellular microRNA release and improve dystrophic pathology

Authors:
van Westering TLE, Lomonosova Y, Coenen‐Stass AML, Betts CA, Bhomra A, Hulsker M, Clark LE, McClorey G, Aartsma‐Rus A, van Putten M, Wood MJA, Roberts TC
Citation:
J Cachexia Sarcopenia Musc. 2019;[Epub] doi:10.1002/jcsm.12506
Epub:
Yes
Abstract:
Background: Duchenne muscular dystrophy (DMD) is a fatal muscle‐wasting disorder caused by genetic loss of dystrophin protein....
Link:
https://onlinelibrary.wiley.com/doi/full/10.1002/jcsm.12506
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
van Westering TLE, Lomonosova Y, Coenen‐Stass AML, Betts CA, Bhomra A, Hulsker M, Clark LE, McClorey G, Aartsma‐Rus A, van Putten M, Wood MJA, Roberts TC. Uniform sarcolemmal dystrophin expression is required to prevent extracellular microRNA release and improve dystrophic pathology. J Cachexia Sarcopenia Musc. 2019;[Epub] doi:10.1002/jcsm.12506.

The ETS transcription factor ELF1 regulates a broadly antiviral program distinct from the type I interferon response

Authors:
Seifert LL, Si C, Saha D, Sadic M, de Vries M, Ballentine S, Briley A, Wang G, Valero-Jimenez AM, Mohamed A, Schaefer U, Moulton HM, García-Sastre A, Tripathi S, Rosenberg BR, Dittmann M
Citation:
PLoS Pathog. 2019 Nov 4;15(11):e1007634. doi: 10.1371/journal.ppat.1007634. PMID: 31682641; PMCID: PMC6932815
Epub:
Not Epub
Abstract:
Induction of vast transcriptional programs is a central event of innate host responses to viral infections. Here we report a...
Link:
https://pmc.ncbi.nlm.nih.gov/articles/PMC6932815/
Delivery Method:
intranasal peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Seifert LL, Si C, Saha D, Sadic M, de Vries M, Ballentine S, Briley A, Wang G, Valero-Jimenez AM, Mohamed A, Schaefer U, Moulton HM, García-Sastre A, Tripathi S, Rosenberg BR, Dittmann M. The ETS transcription factor ELF1 regulates a broadly antiviral program distinct from the type I interferon response. PLoS Pathog. 2019 Nov 4;15(11):e1007634. doi: 10.1371/journal.ppat.1007634. PMID: 31682641; PMCID: PMC6932815.

Multiplex in situ hybridization within a single transcript: RNAscope reveals dystrophin mRNA dynamics

Authors:
Hildyard JCW, Rawson F, Wells DJ, Piercy RJ
Citation:
bioRxiv. 2019;[preprint] doi:10.1101/791780
Epub:
Not Epub
Abstract:
Dystrophin plays a vital role in maintaining muscle health, yet low mRNA expression, lengthy transcription time and the...
Link:
https://www.biorxiv.org/content/10.1101/791780v1
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Hildyard JCW, Rawson F, Wells DJ, Piercy RJ. Multiplex in situ hybridization within a single transcript: RNAscope reveals dystrophin mRNA dynamics. bioRxiv. 2019;[preprint] doi:10.1101/791780 .

Hexose potentiates peptide-conjugated morpholino oligomer efficacy in cardiac muscles of dystrophic mice in an age-dependent manner

Authors:
Han G, Gu B, Lin C, Ning H, Song J, Gao X, Moulton HM, Yin HF
Citation:
Mol Ther Nucl Acids. 2019 Sep 23;18:341-350. doi: 10.1016/j.omtn.2019.09.012. [Epub ahead of print]
Epub:
Not Epub
Abstract:
Insufficient delivery of oligonucleotides to muscle and heart remains a barrier for clinical implementation of antisense...
Link:
http://www.sciencedirect.com/science/article/pii/S2162253119302598
Delivery Method:
peptide-linked
Organism or Cell Type:
mice mdx
Citation Extract:
Han G, Gu B, Lin C, Ning H, Song J, Gao X, Moulton HM, Yin HF. Hexose potentiates peptide-conjugated morpholino oligomer efficacy in cardiac muscles of dystrophic mice in an age-dependent manner. Mol Ther Nucl Acids. 2019 Sep 23;18:341-350. doi: 10.1016/j.omtn.2019.09.012. [Epub ahead of print].

Reduction of integrin alpha 4 activity through splice modulating antisense oligonucleotides

Authors:
Aung-Htut MT, Comerford I, Johnsen R, Foyle K, Fletcher S, Wilton SD
Citation:
Sci Rep. 2019;9(1):12994. doi:10.1038/s41598-019-49385-6
Epub:
Not Epub
Abstract:
With recent approvals of antisense oligonucleotides as therapeutics, there is an increasing interest in expanding the...
Link:
https://www.nature.com/articles/s41598-019-49385-6
Delivery Method:
in vitro lipofectamine (fibroblasts) electroporation (Jurkat) , in vivo peptide-linked
Organism or Cell Type:
cell culture: primary dermal fibroblasts, Jurkat; autoimmune encephalomyelitis mouse model of multiple sclerosis
Citation Extract:
Aung-Htut MT, Comerford I, Johnsen R, Foyle K, Fletcher S, Wilton SD. Reduction of integrin alpha 4 activity through splice modulating antisense oligonucleotides. Sci Rep. 2019;9(1):12994. doi:10.1038/s41598-019-49385-6.

Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice

Authors:
Klein AF, Varela MA, Arandel L, Holland A, Naouar N, Arzumanov A, Seoane D, Revillod L, Bassez G, Ferry A, Jauvin D, Gourdon G, Puymirat J, Gait MJ, Furling D, Wood MJA
Citation:
J Clin Invest. 2019 Nov 1;129(11):4739-4744. doi: 10.1172/JCI128205
Epub:
Not Epub
Abstract:
Antisense oligonucleotides (ASOs) targeting pathologic RNAs have shown promising therapeutic corrections for many genetic...
Link:
https://www.jci.org/articles/view/128205
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: patient-derived muscle DM1 mice
Citation Extract:
Klein AF, Varela MA, Arandel L, Holland A, Naouar N, Arzumanov A, Seoane D, Revillod L, Bassez G, Ferry A, Jauvin D, Gourdon G, Puymirat J, Gait MJ, Furling D, Wood MJA. Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice. J Clin Invest. 2019 Nov 1;129(11):4739-4744. doi: 10.1172/JCI128205.

Chimeras of Cell-Penetrating Peptides Demonstrate Synergistic Improvement in Antisense Efficacy

Authors:
Fadzen CM, Holden RL, Wolfe JM, Choo ZN, Schissel CK, Yao M, Hanson GJ, Pentelute BL
Citation:
Biochemistry. 2019 Sep 24;58(38):3980-3989. doi: 10.1021/acs.biochem.9b00413. Epub 2019 Sep 6. PMID: 31450889
Epub:
Yes
Abstract:
Phosphorodiamidate morpholino oligonucleotides (PMOs) make up a promising class of therapeutics for genetic disease. PMOs...
Link:
https://pubs.acs.org/doi/pdf/10.1021/acs.biochem.9b00413
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: HeLa-654
Citation Extract:
Fadzen CM, Holden RL, Wolfe JM, Choo ZN, Schissel CK, Yao M, Hanson GJ, Pentelute BL. Chimeras of Cell-Penetrating Peptides Demonstrate Synergistic Improvement in Antisense Efficacy. Biochemistry. 2019 Sep 24;58(38):3980-3989. doi: 10.1021/acs.biochem.9b00413. Epub 2019 Sep 6. PMID: 31450889.

TMPRSS2 is the major activating protease of influenza A virus in primary human airway cells and influenza B virus in human type II pneumocytes

Authors:
Limburg H, Harbig A, Bestle D, Stein DA, Moulton HM, Jaeger J, Janga H, Hardes K, Koepke J, Schulte L, Koczulla AR, Schmeck B, Klenk HD, Böttcher-Friebertshäuser E
Citation:
J Virol. 2019 Oct 15;93(21):e00649-19. doi: 10.1128/JVI.00649-19. PMID: 31391268; PMCID: PMC6803253
Epub:
Yes
Abstract:
Cleavage of influenza virus hemagglutinin (HA) by host cell proteases is essential for virus infectivity and spread. We...
Link:
https://journals.asm.org/doi/10.1128/jvi.00649-19
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: primary human bronchial epithelial cells (HBEC), primary type II alveolar epithelial cells (AECII) and Calu-3 cells
Citation Extract:
Limburg H, Harbig A, Bestle D, Stein DA, Moulton HM, Jaeger J, Janga H, Hardes K, Koepke J, Schulte L, Koczulla AR, Schmeck B, Klenk HD, Böttcher-Friebertshäuser E. TMPRSS2 is the major activating protease of influenza A virus in primary human airway cells and influenza B virus in human type II pneumocytes. J Virol. 2019 Oct 15;93(21):e00649-19. doi: 10.1128/JVI.00649-19. PMID: 31391268; PMCID: PMC6803253.

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