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Antisense oligonucleotides to treat spinal muscular atrophy (Shift Pharmaceuticals)

Antisense oligonucleotides to treat spinal muscular atrophy
Shift Pharmaceuticals (University of Missouri)
Abstract for Midwest Drug Development Conference, Sept. 30-Oct. 1, 2019. Omaha NE.
https://www.mwdrugdevelopment.com/speakers/oligonucleotides-spinal-muscu...

Monday, May 6, 2019 - 10:13
Morpholinos: Research Tools and Therapeutics (Cell Press Selections)

New Cell Press Selections: Morpholinos: Research Tools and Therapeutics

Friday, April 26, 2019 - 14:07
Storage of Morpholinos: refrigerate or room temperature?

Morpholino oligos are completely chemically & biologically (enzymatically) stable sitting in water at 4C or at room temperature as long as the pH is not extreme. However, they undergo physical changes that can affect their biological activity. Chilled storage increases the tendency of the oligos to associate with container walls (this is especially a problem if the oligos undergo freeze-thaw cycles). Once a Morpholino has associated tightly with the wall of a container, it can be difficult or impossible to get the oligo back into solution at neutral pH.

Tuesday, April 23, 2019 - 08:01
Proposed mechanism underlying genetic compensation

Genetic paradox explained by nonsense. Wilkinson MF. Nature. 2019;568:179-180. doi: 10.1038/d41586-019-00823-5
https://www.nature.com/articles/d41586-019-00823-5

Genetic compensation triggered by mutant mRNA degradation. El-Brolosy MA et al. Nature. 2019;568:193-197. doi:10.1038/s41586-019-1064-z
https://www.nature.com/articles/s41586-019-1064-z

Thursday, April 11, 2019 - 13:46
Antibody validation by Morpholino

Here is an example of using Morpholinos for antibody validation.

Ratzan W, Rayaprolu V, Killian SE, Bradley R, Kohout SC. The voltage sensing phosphatase (VSP) localizes to the apical membrane of kidney tubule epithelial cells. PLoS ONE. 2019;14(4):e0209056. doi:10.1371/journal.pone.0209056

https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0209056

Wednesday, April 10, 2019 - 12:44
Paper: PTC-bearing mRNA elicits a genetic compensation response via Upf3a and COMPASS components

Another brick pulled from the wall concealing the mechanism of genetic compensation. Nonsense-mediated decay appears again.

Ma Z, Zhu P, Shi H, Guo L, Zhang Q, Chen Y, Chen S, Zhang Z, Peng J, Chen J. PTC-bearing mRNA elicits a genetic compensation response via Upf3a and COMPASS components. Nature. 2019;[Epub ahead of print] doi:10.1038/s41586-019-1057-y

https://www.nature.com/articles/s41586-019-1057-y

Wednesday, April 3, 2019 - 11:38
mutant-morphant disagreement in zebrafish - a story

Here is a story of mutant-morphant disagreement in zebrafish and its effects on one investigator's research. http://thenode.biologists.com/dead-ends-in-research-can-be-new-beginnings/research/

Tuesday, April 2, 2019 - 11:11
Antisense delivery enhancement strategies: meta-analysis

Comparison of enhancements to antisense delivery (peptides, dendrimers, etc.)

Assisted delivery of antisense therapeutics in animal models of heritable neurodegenerative and neuromuscular disorders: a systematic review and meta-analysis.

van der Bent ML, Paulino da Silva Filho O, van Luijk J, Brock R, Wansink DG.

Sci Rep. 2018 Mar 8;8(1):4181. doi: 10.1038/s41598-018-22316-7.

https://www.nature.com/articles/s41598-018-22316-7

Tuesday, March 26, 2019 - 08:54
Photo-Morpholinos: no longer a displayed product line

We are removing Photo-Morpholinos as a displayed product line. Photo-Morpholinos are hard to use - we have made many but they have led to few publications. Any Photo-Morpholino project involves far more interation and development time than a simple Morpholino knockdown; there are many parameters that need to be optimized and several Photo-Morpholino sequences will likely need to be tested. Labs have ordered them without the funding needed to purchase multiple oligos to fine-tune the system sufficiently to get good data; we end up with disgruntled customers.

Friday, February 15, 2019 - 14:45
Letter to Editor: Morpholino-Mediated Gene knockdown in Zebrafish: It's All About Dosage and Validation

Morpholino-Mediated Gene knockdown in Zebrafish: It's All About Dosage and Validation.

Van Gils M, Vanakker OM.

J Invest Dermatol. 2019 Jan 29. pii: S0022-202X(19)30042-9. doi: 10.1016/j.jid.2019.01.017. [Epub ahead of print] No abstract available.

This is a discussion of specificity and p53-mediated effects in the context of a reply to a commentary on a recent paper.

https://www.jidonline.org/article/S0022-202X(19)30042-9/pdf

Monday, February 4, 2019 - 11:03

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