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Jon's Blog
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(Review) Recent Trends in Antisense Therapies for Duchenne Muscular Dystrophy |
Recent Trends in Antisense Therapies for Duchenne Muscular Dystrophy. |
Thursday, March 30, 2023 - 14:23 |
Morpholinos shorter than 25 bases |
A Morpholino user, accustomed to using 25-base Morpholinos in Xenopus laevis, was concerned about a design she received for a 20-base Morpholino. She wrote to ask if we had designed many short oligos and if they worked well. Here is my response. ------------------------------------- |
Wednesday, February 15, 2023 - 11:13 |
Molecular dynamics of aqueous Morpholino oligos |
Maksudov F, Kliuchnikov E, Pierson D, Ujwal ML, Marx KA, Chanda A, Barsegov V. Therapeutic Phosphorodiamidate Morpholino Oligonucleotides, an RNA mimic: Physical Properties, Solution Structures, and Folding Thermodynamics. Mol Ther Nucleic Acids. 2023;[Epub] doi:10.1016/j.omtn.2023.02.007 https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S21...(23)00027-6 |
Wednesday, February 15, 2023 - 10:15 |
Discussion of RNA change on adding translation blocking Morpholino |
From: **** Hello Jon, |
Wednesday, January 18, 2023 - 12:35 |
History: exon skipping |
Lim, K.R.Q., Yokota, T. (2018). Invention and Early History of Exon Skipping and Splice Modulation. In: Yokota, T., Maruyama, R. (eds) Exon Skipping and Inclusion Therapies. Methods in Molecular Biology, vol 1828. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-8651-4_1 |
Tuesday, January 17, 2023 - 11:03 |
Master’s thesis based on microarray analysis of WT, Morphant and rescue fish |
Master’s thesis based on microarray analysis of WT, Morphant and rescue fish |
Monday, January 16, 2023 - 11:32 |
Vivo-Morpholino uptake by zebrafish larvae at 4dpf |
Liang H, Li Y, Li M, Zhou W, Chen J, Zhang Z, Yang Y, Ran C, Zhou Z. The effect and underlying mechanism of yeast β-glucan on antiviral resistance of zebrafish against spring viremia of carp virus infection. Front Immunol. 2022 Nov 3;13:1031962. doi: 10.3389/fimmu.2022.1031962. eCollection 2022. |
Monday, November 21, 2022 - 09:48 |
Vivo-Morpholinos as switching molecules for virally-delivered targets in mice |
The authors of this new paper use an engineered viral vector (AAV) to express a secreted antibody. They make a version where the antibody is out-of-frame and a Morpholino is used to skip an exon, bring the antibody in-frame and switch on its functional expression; the target is a payload of the AAV and the switching molecule is the Morpholino. This use of Morpholinos as switching molecules in engineered systems has great therapeutic potential. In the same paper, they controlled the expression of their AAV cargo with a Vivo-Morpholino after delivery into a mouse. |
Wednesday, August 10, 2022 - 12:14 |
Head-to-head comparisons, Morpholinos and Phosphorothioates |
Single Stranded Fully Modified-Phosphorothioate Oligonucleotides can Induce Structured Nuclear Inclusions, Alter Nuclear Protein Localization and Disturb the Transcriptome In Vitro. Flynn LL, Li R, Pitout IL, Aung-Htut MT, Larcher LM, Cooper JAL, Greer KL, Hubbard A, Griffiths L, Bond CS, Wilton SD, Fox AH, Fletcher S. Front Genet. 2022;13:791416. doi:10.3389/fgene.2022.791416 https://www.frontiersin.org/articles/10.3389/fgene.2022.791416/full |
Tuesday, April 19, 2022 - 10:15 |
Adverse Drug Reactions and Toxicity of the FDA-approved Antisense Oligonucleotide Drugs |
Adverse Drug Reactions and Toxicity of the FDA-approved Antisense Oligonucleotide Drugs. https://dmd.aspetjournals.org/content/early/2022/02/27/dmd.121.000418.long |
Thursday, March 24, 2022 - 14:08 |