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Blocking a cryptic splice site activiated in a mutant to restore normal splicing

Bergsma AJ, in ’t Groen SLM, Catalano F, Yamanaka M, Takahashi S, Okumiya T, van der Ploeg AT, Pijnappel WWMP. A generic assay for the identification of splicing variants that induce nonsense-mediated decay in Pompe disease. Eur J Hum Genet. 2020;{Epub ahead of print]. doi:10.1038/s41431-020-00751-3
https://www.nature.com/articles/s41431-020-00751-3

Monday, November 9, 2020 - 11:16
Morpholinos used to probe genetic compensation in mutants

Genetic compensation prevents myopathy and heart failure in an in vivo model of Bag3 deficiency.
Diofano F, Weinmann K, Schneider I, Thiessen KD, Rottbauer W, Just S.
PLoS Genet. 2020 Nov 2;16(11):e1009088. doi: 10.1371/journal.pgen.1009088. eCollection 2020 Nov.

https://journals.plos.org/plosgenetics/article?id=10.1371/journal.pgen.1...

Tuesday, November 3, 2020 - 09:10
Inhibition of SARS-CoV-2 in Vero cell cultures by peptide-conjugated morpholino-oligomers

Rosenke K, Leventhal S, Moulton H, Hatlevig S, Hawman D, Feldmann H, Stein DA. Inhibition of SARS-CoV-2 in Vero cell cultures by peptide-conjugated morpholino-oligomers. J Antimicrob Chemother. 2020;[Epub ahead of print]:dkaa460 doi:10.1093/jac/dkaa460.
https://academic.oup.com/jac/advance-article/doi/10.1093/jac/dkaa460/596...

Tuesday, October 6, 2020 - 10:47
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

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