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Jon's Blog
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Transcriptional adaptation |
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. https://dev.biologists.org/content/early/2020/09/12/dev.193029 |
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 |
Viltepso (Morpholino drug) approved for DMD in Japan |
Nippon Shinyaku has received marketing approval in Japan for Viltepso, a Morpholino targeting human dystrophin exon 53 for treatment of some mutations causing Duchenne muscular dystrophy. https://www.nippon-shinyaku.co.jp/file/download.php?file_id=2965 |
Wednesday, March 25, 2020 - 09:44 |
Report of compensation in mutants |
Genetic Compensation of γ CaMKII, an Evolutionarily Conserved Gene. Rothschild SC, Ingram SR, Lu FI, Thisse B, Thisse C, Parkerson JA, Tombes RM. Gene. 2020 Mar 9:144567. doi: 10.1016/j.gene.2020.144567. [Epub ahead of print] https://www.sciencedirect.com/science/article/abs/pii/S0378111920302365 |
Monday, March 16, 2020 - 09:51 |