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Jon's Blog
| Post date | ||
|---|---|---|
| E1v1.11: a Morpholino antisense oligo for Spinal Muscular Atrophy |
Optimization and Safety Results of E1v1.11: a morpholino antisense oligonucleotide therapeutic for Spinal Muscular Atrophy. Oconnor S, Hansen H, Osman E, Kaifer KD, Morcos P, Kacher M, Beatty D, Lorson C. MDA Conference Abstracts. 2024;Poster 85 |
Tuesday, November 14, 2023 - 13:29 |
| Video interview: Jon on splice modification with Morpholinos |
Here's an interview with Jon Moulton on Pharma's Almanac discussing using Morpholinos to alter splicing. |
Tuesday, November 14, 2023 - 10:41 |
| E1i1 oligo activates cryptic site in exon 1 |
Here's a report of an unusual splice response to a Morpholino targeting the e1i1 junction. We'd expect the common outcome, insertion of intron 1. Instead, the splice was redirected to a cryptic splice site in exon 1, resulting in a shortened mature transcript. The new splice site knocked the downstream sequence out of frame, so this produced a good knockdown. |
Monday, November 13, 2023 - 11:00 |
| in silico Morpholino sequence design |
Here's another computer-based oligo sequence picker. Pacelli C, Rossi A, Milella M, Colomb T, Le Pera L. RNA-Based Strategies for Cancer Therapy: In Silico Design and Evaluation of ASOs for Targeted Exon Skipping. Int. J. Mol. Sci. 2023, 24, 14862. |
Wednesday, October 11, 2023 - 12:41 |
| Delivery of Morpholinos by irradiation |
Patent: |
Thursday, July 13, 2023 - 15:33 |
| (Methods Cell Biol):Tools to characterize genetic mutants and assess transcriptional adaptation in zebrafish |
Cardenas-Rodriguez M, Drummond IA. The challenge of dissecting gene function in model organisms: Tools to characterize genetic mutants and assess transcriptional adaptation in zebrafish. Methods Cell Biol. 2023;176:1-25. doi: 10.1016/bs.mcb.2022.12.019. Epub 2023 Jan 27. |
Thursday, May 11, 2023 - 15:29 |
| I have an article in Pharma's Almanac - Morpholino stories |
Morpholino article in Pharma's Almanac |
Friday, May 5, 2023 - 15:34 |
| (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 |
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