You are here
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|
|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]
|Monday, March 16, 2020 - 09:51|