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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
Vivo-Morpholino used to control activity of an engineered ribozyme

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

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.

Tuesday, October 29, 2019 - 13:49
A broader view: Approved Gene Therapy Products 2019

Development and Clinical Translation of Approved Gene Therapy Products for Genetic Disorders.

Shahryari1 A, Jazi MS, Mohammadi S, Nikoo HR, Nazari Z, Hosseini ES, Burtscher I, Mowla SJ, Lickert H.

Front Genet. 25 September 2019. doi:10.3389/fgene.2019.00868

Thursday, September 26, 2019 - 14:51
Paper: Site Selective Antibody-Oligonucleotide Conjugation via Microbial Transglutaminase.

This paper describes using a microbial transglutamase to put an azide group in a peptide onto the terminal carboxylic acid of an antibody protein, then using Cu-free click chemistry to affix an antisense oligo at that site. They didn't use Morpholinos, but in principle one could.

Friday, September 13, 2019 - 12:05
Paper on delivery in cultures: In Vitro Validation of Phosphorodiamidate Morpholino Oligomers.

Aung-Htut MT, McIntosh CS, West KA, Fletcher S, Wilton SD. In Vitro Validation of Phosphorodiamidate Morpholino Oligomers. Molecules. 2019;24(6):2922 doi:0.3390/molecules24162922

cell culture: human dermal fibroblasts, human myoblasts
electroporation (nucelofection), Lipofectin, Lipofectamine, Endo-Porter

Monday, August 12, 2019 - 13:13
Review on compensation: mutant & morphant

Gene redundancy and gene compensation: An updated view.

Peng J.

J Genet Genomics. 2019 Jul 19. pii: S1673-8527(19)30115-8. doi: 10.1016/j.jgg.2019.07.001. [Epub ahead of print] Review.

Tuesday, August 6, 2019 - 09:18