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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

https://www.mdpi.com/1422-0067/20/21/5434/htm

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
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

https://www.frontiersin.org/articles/10.3389/fgene.2019.00868/full

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

https://www.mdpi.com/1420-3049/24/18/3287

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 in 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

https://www.mdpi.com/1420-3049/24/16/2922

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.

https://www.sciencedirect.com/science/article/pii/S1673852719301158

Tuesday, August 6, 2019 - 09:18
Delay in development and behavioural abnormalities in the absence of p53 in zebrafish

Delay in development and behavioural abnormalities in the absence of p53 in zebrafish.
Elabd S, Jabeen NA, Gerber V, Peravali R, Bourdon JC, Kancherla S, Vallone D, Blattner C.
PLoS One. 2019 Jul 19;14(7):e0220069. doi: 10.1371/journal.pone.0220069. eCollection 2019.
https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0220069

Wednesday, July 31, 2019 - 09:30
Example of specificity control using a Morpholino in its mutant

This paper is a nice example of Morpholino specificity control by using a Morpholino in its corresponding mutant.

Chambers BE, Gerlach GF, Clark EG, Chen KH, Levesque AE, Leshchiner I, Goessling W, Wingert RA. Tfap2a is a novel gatekeeper of nephron differentiation during kidney development. Development. 2019 Jun 3. pii: dev.172387. doi: 10.1242/dev.172387. [Epub ahead of print]

https://dev.biologists.org/content/146/13/dev172387

Wednesday, July 10, 2019 - 11:53
Targeting mitochondrially-encoded circular RNAs in the cytosol: mitochondrial protein uptake suppression

Here is an interesting report of Morpholinos targeting mitochondrially-encoded circular RNAs in the cytosol to prevent RNA-chaperoned protein uptake into the mitochondria.

Liu X, Wang X, Li J, Hu S, Deng Y, Yin H, Bao X, Zhang QC, Wang G, Wang B, Shi Q, Shan G. The identification of mecciRNAs and their roles in mitochondrial entry of proteins. bioRxiv. 2019;[preprint] doi:10.1101/668665

Friday, July 5, 2019 - 12:32

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