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Jon's Blog

		Post date
Broad review of the RNA therapeutics field (2020)	Here's a broad review of the RNA therapeutics field, hosted on two sites. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7495341/ https://pharmrev.aspetjournals.org/content/72/4/862	Tuesday, October 6, 2020 - 10:44
(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. https://www.nature.com/articles/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 https://www.biorxiv.org/content/10.1101/2020.09.18.302984v1 Links to resources for designing splice-switching oligos	Monday, September 21, 2020 - 10:31
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. https://dev.biologists.org/content/147/15/dev186452	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. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7469316/	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 Open-access review article https://www.mdpi.com/2073-4425/11/8/837	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 Dhuri K, Bechtold C, Quijano E, Pham H, Gupta A, Vikram A, Bahal R. Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development. J Clin Med. 2020;9(6):2004. doi:10.3390/jcm9062004 https://www.mdpi.com/2077-0383/9/6/2004	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 Dichmann DS, Walentek P, Harland RM. The Alternative Splicing Regulator Tra2b Is Required for Somitogenesis and Regulates Splicing of an Inhibitory Wnt11b Isoform. Cell Rep. 2015 Jan 21. pii: S2211-1247(14)01099-7. doi: 10.1016/j.celrep.2014.12.046. [Epub ahead of print]. http://www.ncbi.nlm.nih.gov/pubmed/25620705	Wednesday, May 6, 2020 - 09:43

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