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

Single Exon Skipping Can Address a Multi-Exon Duplication in the Dystrophin Gene

Authors:
Greer K, Johnsen R, Nevo Y, Fellig Y, Fletcher S, Wilton SD
Citation:
Int J Molec Sci. 2020;[Epub] doi:10.3390/ijms21124511
Epub:
Yes
Abstract:
Duchenne muscular dystrophy (DMD) is a severe muscle wasting disease typically caused by protein-truncating mutations that...
Delivery Method:
cell bathing, peptide-linked
Organism or Cell Type:
cell culture: primary human myoblasts
Citation Extract:
Greer K, Johnsen R, Nevo Y, Fellig Y, Fletcher S, Wilton SD. Single Exon Skipping Can Address a Multi-Exon Duplication in the Dystrophin Gene. Int J Molec Sci. 2020;[Epub] doi:10.3390/ijms21124511.

Development of a Minimized Exons 45-55 Skipping Cocktail for the Treatment of Duchenne Muscular Dystrophy

Authors:
Lim KRQ, Huang Y, Maruyama R, Woo S, Dzierlega K, Moulton H, Yokota T
Citation:
Molec Ther. 2020;28(4S1):113 abstract 237. doi:10.1016/j.ymthe.2020.04.019
Epub:
Not Epub
Abstract:
Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive neuromuscular disorder characterized by...
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Lim KRQ, Huang Y, Maruyama R, Woo S, Dzierlega K, Moulton H, Yokota T. Development of a Minimized Exons 45-55 Skipping Cocktail for the Treatment of Duchenne Muscular Dystrophy. Molec Ther. 2020;28(4S1):113 abstract 237. doi:10.1016/j.ymthe.2020.04.019.

Restoration of Full-Length Dystrophin Expression in the Dup2 Mouse Induced by Systemic Delivery of a Peptide-Conjugated Morpholino Oligomer

Authors:
Gushchina LV, Grounds KM, Huang N, Frair E, Schnell FJ, Hanson GJ, Simmons T, Wein N, Flanigan KM
Citation:
Molec Ther. 2020;28(4S1):20 abstract 39. doi:10.1016/j.ymthe.2020.04.019
Epub:
Not Epub
Abstract:
Duchenne muscular dystrophy (DMD) is an x-linked recessive genetic disorder caused by mutations that disrupt the...
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Gushchina LV, Grounds KM, Huang N, Frair E, Schnell FJ, Hanson GJ, Simmons T, Wein N, Flanigan KM. Restoration of Full-Length Dystrophin Expression in the Dup2 Mouse Induced by Systemic Delivery of a Peptide-Conjugated Morpholino Oligomer. Molec Ther. 2020;28(4S1):20 abstract 39. doi:10.1016/j.ymthe.2020.04.019.

Peptide Conjugated Morpholinos for Management of The Huanglongbing Pathosystem

Authors:
Sandoval-Mojica AF, Altman S, Hunter WB, Pelz-Stelinski KS
Citation:
Pest Manag Sci. 2020 May 1. doi: 10.1002/ps.5877. [Epub ahead of print]
Epub:
Yes
Abstract:
BACKGROUND: 'Candidatus Liberibacter asiaticus' (CLas) is the causal agent of the devastating citrus disease...
Delivery Method:
peptide-linked
Organism or Cell Type:
Candidatus Liberibacter asiaticus, Wolbachia
Citation Extract:
Sandoval-Mojica AF, Altman S, Hunter WB, Pelz-Stelinski KS. Peptide Conjugated Morpholinos for Management of The Huanglongbing Pathosystem. Pest Manag Sci. 2020 May 1. doi: 10.1002/ps.5877. [Epub ahead of print].

TMPRSS2 and furin are both essential for proteolytic activation of SARS-CoV-2 in human airway cells

Authors:
Bestle D, Heindl MR, Limburg H, Van Lam van T, Pilgram O, Moulton H, Stein DA, Hardes K, Eickmann M, Dolnik O, Rohde C, Klenk HD, Garten W, Steinmetzer T, Böttcher-Friebertshäuser E
Citation:
Life Sci Alliance. 2020 Jul 23;3(9):e202000786. doi: 10.26508/lsa.202000786. PMID: 32703818; PMCID: PMC7383062
Epub:
Not Epub
Abstract:
The novel emerged SARS-CoV-2 has rapidly spread around the world causing acute infection of the respiratory tract (COVID-19)...
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: Calu-3
Citation Extract:
Bestle D, Heindl MR, Limburg H, Van Lam van T, Pilgram O, Moulton H, Stein DA, Hardes K, Eickmann M, Dolnik O, Rohde C, Klenk HD, Garten W, Steinmetzer T, Böttcher-Friebertshäuser E. TMPRSS2 and furin are both essential for proteolytic activation of SARS-CoV-2 in human airway cells. Life Sci Alliance. 2020 Jul 23;3(9):e202000786. doi: 10.26508/lsa.202000786. PMID: 32703818; PMCID: PMC7383062.

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...
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...
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....
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...
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...
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 .

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