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Jon's Blog
| Post date | ||
|---|---|---|
| Review: Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases |
Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases. |
Thursday, December 10, 2020 - 13:45 |
| Clinical Pharmacology Studies Supporting Oligonucleotide Therapy Development: An Assessment of Therapies Approved and in Development Between 2012-2018 |
Clinical Pharmacology Studies Supporting Oligonucleotide Therapy Development: An Assessment of Therapies Approved and in Development Between 2012-2018. |
Monday, December 7, 2020 - 10:08 |
| 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 |
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. 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. |
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. |
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 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. |
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 |
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