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Jon's Blog
| Post date | ||
|---|---|---|
| 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] https://www.sciencedirect.com/science/article/abs/pii/S0378111920302365 |
Monday, March 16, 2020 - 09:51 |
| Shift Pharmaceuticals Orphan Drug: Morpholino for Spinal Muscular Atrophy |
Shift Pharmaceuticals receives an Orphan Drug designation from the FDA for their Morpholino to treat Spinal Muscular Atrophy. https://smauk.org.uk/blog/treatments-research/shift-pharmaceuticals-rece... https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm... |
Monday, March 9, 2020 - 16:28 |
| Golodirsen: First Approval. Morpholino drug for DMD (exon 53 skipper) |
Golodirsen: First Approval. This is an FDA-approved Morpholino drug that causes skipping of exon 53 of human dystrophin, used for the treatment of Duchenne muscular dystrophy. |
Friday, February 7, 2020 - 14:46 |
| Available fluorochromes attached to Morpholinos |
I like carboxyfluorescein. It is bright, it doesn’t alter how the oligo behaves, and most labs already have a fluorescein cube (filters and dichroic mirror) for their fluorescent microscopes. The down side is that fluorescein photobleaches, so you should minimize its exposure to intense light and only observe it with the microscope when you are ready to take pictures and turn off the lamp soon. Carboxyfluorescein emits green. |
Wednesday, January 8, 2020 - 07:40 |
| A reversible RNA on-switch using a Vivo-Morpholino to controls gene expression of AAV-delivered therapeutics in vivo |
Zhong G, Wang H, He W, Li Y, Mou H, Tickner ZJ, Tran MH, Ou T, Yin Y, Diao H, Farzan M. A reversible RNA on-switch that controls gene expression of AAV-delivered therapeutics in vivo. Nat Biotechnol. 2019;[Epub ahead print] doi:10.1038/s41587-019-0357-y Press release: |
Monday, December 23, 2019 - 13:27 |
| FDA approves golodirsen (a Morpholino drug) |
Sarepta Therapeutics Announces FDA Approval of VYONDYS 53™ (golodirsen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 53 |
Thursday, December 12, 2019 - 16:01 |
| Paper comparing splice-modifying efficacy of Morpholinos and 2'-O-Me phosphorothioates |
McIntosh CS, Aung-Htut MT, Fletcher S, Wilton SD. Removal of the Polyglutamine Repeat of Ataxin-3 by Redirecting pre-mRNA Processing. Int J Mol Sci. 2019;20(21):5434. doi:10.3390/ijms20215434 |
Thursday, October 31, 2019 - 10:54 |
| Protein interrelationship shown by synergy of Morpholinos to different mRNAs |
Oprişoreanu A-M, Smith HL, Arya S, Webster R, Zhong Z, Wehner D, Cardozo MJ, Becker T, Talbot K, Becker CG. Interaction of Axonal Chondrolectin with Collagen XIXa1 Is Necessary for Precise Neuromuscular Junction Formation. Cell Rep. 2019;29:1082-98. doi:10.1016/j.celrep.2019.09.033 https://www.cell.com/cell-reports/fulltext/S2211-1247(19)31221-5 |
Wednesday, October 30, 2019 - 10:33 |
| Morpholino assay in plasma: HPLC-MS/MS Coupled with Solid Phase Micro-Extraction |
Two Quantitative Assays for the Phosphorodiamidate #Morpholino Oligomer (PMO) SRP-4045 in Mouse Plasma Using HPLC-MS/MS Coupled with Solid Phase Micro-Extraction. Poster at ASMS 2017. https://www.covance.com/latest-thinking/explore-library/detail.html/cova... |
Tuesday, October 29, 2019 - 13:49 |
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