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Broad review of the RNA therapeutics field (2020)

Here's a broad review of the RNA therapeutics field, hosted on two sites.

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.

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
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.

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
Open-access review article

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

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].

Wednesday, May 6, 2020 - 09:43