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|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||
|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
|Wednesday, February 15, 2023 - 10:15|