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

Targeting the Expanded TCF4/Fuchs’ Endothelial Corneal Dystrophy CUG Repeat with Morpholino Peptide Conjugates

Authors:
Hu J, Shen X, Kheirabadi M, Streeter MD, Qian Z, Mootha VV, Corey DR
Citation:
ACS Omega. 2023;[Epub] doi:10.1021/acsomega.3c05634
Epub:
Yes
Abstract:
Fuchs’ corneal endothelial dystrophy (FECD) is a major cause of vision loss. Corneal transplantation is the only effective...
Link:
https://pubs.acs.org/doi/10.1021/acsomega.3c05634
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: FECD patient-derived F35T corneal endothelial cells
Citation Extract:
Hu J, Shen X, Kheirabadi M, Streeter MD, Qian Z, Mootha VV, Corey DR. Targeting the Expanded TCF4/Fuchs’ Endothelial Corneal Dystrophy CUG Repeat with Morpholino Peptide Conjugates. ACS Omega. 2023;[Epub] doi:10.1021/acsomega.3c05634.

An Induced Pluripotent Stem Cell-Derived Human Blood-Brain Barrier (BBB) Model to Test the Crossing by Adeno-Associated Virus (AAV) Vectors and Antisense Oligonucleotides

Authors:
Selvakumaran J, Ursu S, Bowerman M, Lu-Nguyen N, Wood MJ, Malerba A, Yáñez-Muñoz RJ
Citation:
Biomedicines. 2023 Oct 4;11(10):2700. doi: 10.3390/biomedicines11102700
Epub:
Not Epub
Abstract:
The blood-brain barrier (BBB) is the specialised microvasculature system that shields the central nervous system (CNS) from...
Link:
https://www.mdpi.com/2227-9059/11/10/2700
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: induced brain microvascular endothelial cells (BMECs)
Citation Extract:
Selvakumaran J, Ursu S, Bowerman M, Lu-Nguyen N, Wood MJ, Malerba A, Yáñez-Muñoz RJ. An Induced Pluripotent Stem Cell-Derived Human Blood-Brain Barrier (BBB) Model to Test the Crossing by Adeno-Associated Virus (AAV) Vectors and Antisense Oligonucleotides. Biomedicines. 2023 Oct 4;11(10):2700. doi: 10.3390/biomedicines11102700.

P23 Evaluation of safety parameters and dystrophin expression by sequential administration of exon-skipping and gene therapy in a DMDmdx mouse model

Authors:
Potter R, Cooper Olson GC, Smith L, Greve J, Haile A, Wier C, Snedeker J, Burch P, Hunter B, Malmberg A, Rodino-Klapac L
Citation:
Neuromuscul Disord. 2023;33(Supp 1):S102-3. doi:10.1016/j.nmd.2023.07.150
Epub:
Not Epub
Abstract:
There are promising treatment approaches emerging for Duchenne muscular dystrophy (DMD): exon skipping with peptide-conjugated...
Link:
https://www.nmd-journal.com/article/S0960-8966(23)00330-9/pdf
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Potter R, Cooper Olson GC, Smith L, Greve J, Haile A, Wier C, Snedeker J, Burch P, Hunter B, Malmberg A, Rodino-Klapac L. P23 Evaluation of safety parameters and dystrophin expression by sequential administration of exon-skipping and gene therapy in a DMDmdx mouse model. Neuromuscul Disord. 2023;33(Supp 1):S102-3. doi:10.1016/j.nmd.2023.07.150.

Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression

Authors:
González-Martínez I, Cerro-Herreros E, Moreno N, García-Rey A, Espinosa-Espinosa J, Carrascosa-Sàez M, Piqueras-Losilla D, Arzumanov A, Seoane-Miraz D, Jad Y, Raz R, Wood MJ, Varela MA, Llamusí B, Artero R
Citation:
Mol Ther Nucleic Acids. 2023 Sep 5;34:102024. doi: 10.1016/j.omtn.2023.09.001. eCollection 2023 Dec 12
Epub:
Not Epub
Abstract:
Myotonic dystrophy type 1 (DM1) is a rare neuromuscular disease caused by a CTG repeat expansion in the DMPK gene that...
Link:
https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(23)00237-8
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: DM1, mice
Citation Extract:
González-Martínez I, Cerro-Herreros E, Moreno N, García-Rey A, Espinosa-Espinosa J, Carrascosa-Sàez M, Piqueras-Losilla D, Arzumanov A, Seoane-Miraz D, Jad Y, Raz R, Wood MJ, Varela MA, Llamusí B, Artero R. Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression. Mol Ther Nucleic Acids. 2023 Sep 5;34:102024. doi: 10.1016/j.omtn.2023.09.001. eCollection 2023 Dec 12.

Modulation of Pro-Inflammatory IL-6 Trans-Signaling Axis by Splice Switching Oligonucleotides as a Therapeutic Modality in Inflammation

Authors:
Gupta D, Orehek S, Turunen J, O’Donovan L, Gait MJ, El-Andaloussi S, Wood MJA
Citation:
Cells. 2023;12:2285. doi:10.3390/ cells12182285
Epub:
Not Epub
Abstract:
Interleukin-6 (IL-6) is a pleiotropic cytokine that plays a crucial role in maintaining normal homeostatic processes under the...
Link:
https://www.mdpi.com/2073-4409/12/18/2285
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Gupta D, Orehek S, Turunen J, O’Donovan L, Gait MJ, El-Andaloussi S, Wood MJA. Modulation of Pro-Inflammatory IL-6 Trans-Signaling Axis by Splice Switching Oligonucleotides as a Therapeutic Modality in Inflammation. Cells. 2023;12:2285. doi:10.3390/ cells12182285.

Sequence specificity defines the effectiveness of PPMOs targeting Pseudomonas aeruginosa

Authors:
Nanayakkara AK, Moustafa DA, Pifer R, Goldberg JB, Greenberg DE
Citation:
Antimicrob Agents Chemother. 2023 Aug 23:e0024523. doi: 10.1128/aac.00245-23. Online ahead of print
Epub:
Yes
Abstract:
Development of new therapeutics against antibiotic resistant pathogenic bacteria is recognized as a priority across the globe....
Link:
https://journals.asm.org/doi/10.1128/aac.00245-23
Delivery Method:
peptide-linked
Organism or Cell Type:
Pseudomonas aeruginosa (bacterium)
Citation Extract:
Nanayakkara AK, Moustafa DA, Pifer R, Goldberg JB, Greenberg DE. Sequence specificity defines the effectiveness of PPMOs targeting Pseudomonas aeruginosa. Antimicrob Agents Chemother. 2023 Aug 23:e0024523. doi: 10.1128/aac.00245-23. Online ahead of print.

The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of neuromuscular disorders

Authors:
Li X, Kheirabadi M, Dougherty PG, Kamer KJ, Shen X, Estrella NL, Peddigari S, Pathak A, Blake SL, Sizensky E, Genio CD, Gaur AB, Dhanabal M, Girgenrath M, Sethuraman N, Qian Z
Citation:
Mol Ther Nucleic Acids. 2023 Jun 29;33:273-285. doi: 10.1016/j.omtn.2023.06.022. eCollection 2023 Sep 12
Epub:
Not Epub
Abstract:
Biological therapeutic agents are highly targeted and potent but limited in their ability to reach intracellular targets. These...
Link:
https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(23)00172-5
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Li X, Kheirabadi M, Dougherty PG, Kamer KJ, Shen X, Estrella NL, Peddigari S, Pathak A, Blake SL, Sizensky E, Genio CD, Gaur AB, Dhanabal M, Girgenrath M, Sethuraman N, Qian Z. The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of neuromuscular disorders. Mol Ther Nucleic Acids. 2023 Jun 29;33:273-285. doi: 10.1016/j.omtn.2023.06.022. eCollection 2023 Sep 12.

A comparative analysis of peptide-delivered antisense antibiotics employing diverse nucleotide mimics

Authors:
Ghosh C, Popella L, Dhamodharan V, Jung J, Barquist L, Höbartner C, Vogel J
Citation:
bioRxiv. 2023;[preprint] doi:10.1101/2023.07.11.548539
Epub:
Not Epub
Abstract:
Antisense oligomer (ASO)-based antibiotics that target mRNAs of essential bacterial genes have great potential for...
Link:
https://www.biorxiv.org/content/10.1101/2023.07.11.548539v1
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: Salmonella enterica serovar Typhimurium strain SL1344
Citation Extract:
Ghosh C, Popella L, Dhamodharan V, Jung J, Barquist L, Höbartner C, Vogel J. A comparative analysis of peptide-delivered antisense antibiotics employing diverse nucleotide mimics. bioRxiv. 2023;[preprint] doi:10.1101/2023.07.11.548539.

In-Cell Penetration Selection-Mass Spectrometry Produces Noncanonical Peptides for Antisense Delivery

Authors:
Schissel CK, Farquhar CE, Loas A, Malmberg AB, Pentelute BL
Citation:
ACS Chem Biol. 2023 Mar 1. doi: 10.1021/acschembio.2c00920. Online ahead of print
Epub:
Yes
Abstract:
Peptide-mediated delivery of macromolecules in cells has significant potential therapeutic benefits, but no therapy employing...
Link:
https://pubs.acs.org/doi/10.1021/acschembio.2c00920
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: HeLa
Citation Extract:
Schissel CK, Farquhar CE, Loas A, Malmberg AB, Pentelute BL. In-Cell Penetration Selection-Mass Spectrometry Produces Noncanonical Peptides for Antisense Delivery. ACS Chem Biol. 2023 Mar 1. doi: 10.1021/acschembio.2c00920. Online ahead of print.

DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration

Authors:
Aslesh T, Erkut E, Ren J, Lim KRQ, Woo S, Hatlevig S, Moulton HM, Gosgnach S, Greer J, Maruyama R, Yokota T
Citation:
JCI Insight. 2023;8(5):e160516. doi: 10.1172/jci.insight.160516. Online ahead of print
Epub:
Not Epub
Abstract:
Antisense oligonucleotide (AO)-mediated therapy is a promising strategy to treat several neurological diseases including spinal...
Link:
https://insight.jci.org/articles/view/160516
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Aslesh T, Erkut E, Ren J, Lim KRQ, Woo S, Hatlevig S, Moulton HM, Gosgnach S, Greer J, Maruyama R, Yokota T. DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration. JCI Insight. 2023;8(5):e160516. doi: 10.1172/jci.insight.160516. Online ahead of print.

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