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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. 10.3390/ijms241914862

Wednesday, October 11, 2023 - 12:41
Delivery of Morpholinos by irradiation

Morpholino oligonucleotides useful in cancer treatment
Includes 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.
Wilton-Clark H, Yokota T. Pharmaceutics. 2023; 15(3):778.

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