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

In vivo Pharmacokinetic and Biodistribution profile of Peptide-Conjugated Phosphorodiamidate Morpholino Oligonucleotides

Authors:
Burki U, Blain A, Greally E, O’Donovan L, Gait M, Laval S, Straub V
Citation:
Poster at 2015 Oligonucleotide Therapeutics Society meeting
Epub:
Not Epub
Abstract:
Antisense oligonucleotide (AON) induced exon skipping is one of the most promising strategies for treating Duchene muscular...
Link:
https://www.researchgate.net/publication/283047310_In_vivo_Pharmacokinetic_and_Biodistribution_profile_of_Peptide-Conjugated_Phosphorodiamidate_Morpholino_Oligonucleotides [accessed Sep 17 2020]
Delivery Method:
Pip6A peptide-linked; subcutaneous (s.c.) or intravenous (i.v.)
Organism or Cell Type:
mdx mice
Citation Extract:
Burki U, Blain A, Greally E, O’Donovan L, Gait M, Laval S, Straub V. In vivo Pharmacokinetic and Biodistribution profile of Peptide-Conjugated Phosphorodiamidate Morpholino Oligonucleotides. Poster at 2015 Oligonucleotide Therapeutics Society meeting.

Morpholino Oligomer-Induced Dystrophin Isoforms to Map the Functional Domains in the Dystrophin Protein

Authors:
Li D, Adams AM, Johnsen RD, Fletcher S, Wilton SD
Citation:
Molec Ther. 2020 Aug 21;22:263-272. doi:10.1016/j.omtn.2020.08.019
Epub:
Not Epub
Abstract:
Dystrophin plays a crucial role in maintaining sarcolemma stability during muscle contractions, and mutations that prevent the...
Link:
https://www.sciencedirect.com/science/article/pii/S2162253120302481
Delivery Method:
(RxR)4 peptide-linked; intraperitoneal (i.p.)
Organism or Cell Type:
cell culture: murine H2K mdx myogenic cells; mice: mdx & C57BL/10ScSn
Citation Extract:
Li D, Adams AM, Johnsen RD, Fletcher S, Wilton SD. Morpholino Oligomer-Induced Dystrophin Isoforms to Map the Functional Domains in the Dystrophin Protein. Molec Ther. 2020 Aug 21;22:263-272. doi:10.1016/j.omtn.2020.08.019.

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

Authors:
Bestle D, Heindl MR, Limburg H, Lam van TV, Pilgram O, Moulton H, Stein DA, Hardes K, Eickmann M, Dolnik O, Rohde C, Klenk H-D, Garten W, Steinmetzer T, Böttcher-Friebertshäuser E
Citation:
Life Sci Alliance. 2020;3(9):e202000786 doi:10.26508/lsa.202000786
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)...
Link:
https://www.life-science-alliance.org/content/3/9/e202000786
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: Calu-3 human airway epithelial cells
Citation Extract:
Bestle D, Heindl MR, Limburg H, Lam van TV, Pilgram O, Moulton H, Stein DA, Hardes K, Eickmann M, Dolnik O, Rohde C, Klenk H-D, 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;3(9):e202000786 doi:10.26508/lsa.202000786.

Targeted Delivery of Antisense Oligonucleotides Using Neurotensin Peptide

Authors:
Nikan M, Tanowitz M, Dwyer CA, Jackson M, Gaus H, Swayze E, Rigo F, Seth PP, Prakash TP
Citation:
J Med Chem. 2020 Jul 17. doi:10.1021/acs.jmedchem.0c00840. Online ahead of print
Epub:
Not Epub
Abstract:
Despite recent advances, targeted delivery of therapeutic oligonucleotide to extra-hepatic tissues continues to be a...
Link:
https://pubs.acs.org/doi/10.1021/acs.jmedchem.0c00840
Delivery Method:
peptide-linked, intracerebroventricular (i.c.v.) injection in mice
Organism or Cell Type:
cell culture: HEK293, mice
Citation Extract:
Nikan M, Tanowitz M, Dwyer CA, Jackson M, Gaus H, Swayze E, Rigo F, Seth PP, Prakash TP. Targeted Delivery of Antisense Oligonucleotides Using Neurotensin Peptide. J Med Chem. 2020 Jul 17. doi:10.1021/acs.jmedchem.0c00840. Online ahead of print.

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...
Link:
https://www.mdpi.com/1422-0067/21/12/4511
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.

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...
Link:
https://onlinelibrary.wiley.com/doi/abs/10.1002/ps.5877
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)...
Link:
https://www.life-science-alliance.org/content/3/9/e202000786
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...
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 tubule epithelial cells; EGFP IVS2-654 transgenic 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; intravenous (i.v.)
Organism or Cell Type:
dystrophin-utrophin double-knock out 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:
Pip9b2 peptide-linked; intravenous (i.v.)
Organism or Cell Type:
mice: mdx & mdx-XistΔhs
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.

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