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|(paper): Deletion of morpholino binding sites (DeMOBS) to assess specificity of morphant phenotypes||
Another specificity control strategy involving mutants and Morpholinos
Cunningham CM, Bellipanni G, Habas R, Balciunas D. Deletion of morpholino binding sites (DeMOBS) to assess specificity of morphant phenotypes. Sci Rep. 2020 Sep 21;10(1):15366. doi: 10.1038/s41598-020-71708-1.
|Tuesday, September 22, 2020 - 09:36|
|Paper with links to resources for designing splice-switching oligos||
Marchalot A, Lambert J-M, Boyer F, Pollet J, Moreau J, Feuillard J, Faumont N, Delpy L. Splice switching oligonucleotide mediated gene knockdown in B cells and plasma cells. bioRxiv. 2020;[preprint] doi:10.1101/2020.09.18.302984
Links to resources for designing splice-switching oligos
|Monday, September 21, 2020 - 10:31|
On the mechanism behind many differing phenotypes of morphants and mutants.
Sztal TE, Stainier DYR. Transcriptional adaptation: a mechanism underlying genetic robustness. Development. 2020 Aug 14;147(15):dev186452. doi: 10.1242/dev.186452.
|Wednesday, September 16, 2020 - 10:48|
|Specificity test failed: Morpholino in a mutant||
This paper presents an example of a Morpholino off-target effect shown by testing the Morpholino in a CRISPR mutants.
Jiang Z, Carlantoni C, Allanki S, Ebersberger I, Stainier DYR. Tek/Tie2 is not required for cardiovascular development in zebrafish. Development. 2020 Sep 14:dev.193029. doi: 10.1242/dev.193029. Online ahead of print.
|Wednesday, September 16, 2020 - 08:44|
|(Paper) Modified nucleic acids: replication, evolution, and next-generation therapeutics||
Overview of nucleic acid analogs, including which are approved as drugs.
|Monday, September 14, 2020 - 13:10|
|Review: Therapeutic Strategies for Duchenne Muscular Dystrophy||
Sun C, Shen L, Zhang Z, Xie X. Therapeutic Strategies for Duchenne Muscular Dystrophy: An Update. Genes. 2020;11:837. doi:10.3390/genes11080837
|Thursday, July 23, 2020 - 10:49|
|Targeting alternative splicing to upregulate protein expression||
Potential application in antisense therapeutics: upregulation of targeted protein expression
Here is a new paper demonstrating techniques for targeting steric-blocking antisense oligos to alternatively-spliced transcripts undergoing NMD to alter the splicing and recover transcripts encoding useful proteins. This is a technique for antisense upregulation of a protein activity.
|Thursday, July 9, 2020 - 13:45|
|Clinical antisense review (2020)||
Clinical antisense review
|Friday, June 26, 2020 - 12:18|
|Retention of the last intron||
Here are a few publications addressing Morpholinos and retention of the last intron.
See the section: Retention of wnt11b Intron 4 Recapitulates the Failure to Form Somites in tra2b Morphants
|Wednesday, May 6, 2020 - 09:43|
|Presentations/posters from the 2020 MDA conference involving Morpholinos||
Long-term Safety & Efficacy of Golodirsen in Male Patients with Duchenne Muscular Dystrophy (DMD) Amenable to Exon 53 Skipping
PMO-based miRNA site blocking oligo (SBO) mediated utrophin upregulation in mdx mice, a therapeutic approach for Duchenne Muscular Dystrophy (DMD)
|Thursday, March 26, 2020 - 16:00|