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Acute RNAi versus knockout: mutation triggers compensation

No Morpholino work in this one, but it explores a difference between targeting RNA versus DNA.

Cell-Intrinsic Adaptation Arising from Chronic Ablation of a Key Rho GTPase Regulator.
Cerikan B, Shaheen R, Colo GP, Gläßer C, Hata S, Knobeloch KP, Alkuraya FS, Fässler R, Schiebel E.
Dev Cell. 2016 Sep 28. pii: S1534-5807(16)30595-0. doi: 10.1016/j.devcel.2016.08.020.

http://www.sciencedirect.com/science/article/pii/S1534580716305950

Tuesday, October 4, 2016 - 11:35
Morpholino drug approved by FDA

The US Food and Drug Administration today (19 Sep 2016) granted Accelerated Approval to eteplirsen (EXONDYS 51), a Morpholino oligo-based treatment for some forms of Duchenne muscular dystrophy (DMD). This is the first approval of a Morpholino drug.

Monday, September 19, 2016 - 15:31
miRNA targeting and second oligo specificity controls

Once again, a conversation with a new Morpholino user led to a discussion others might find useful.

For the two non-overlapping oligo specificity control, you do two separate sets of injections of the two oligos targeting the same miRNA. If the embryos treated with the oligo targeting the 5' end of the miRNA produces the same phenotype as the oligo targeting the 3' end of the miRNA, then that is good: it supports the idea that the phenotype you are seeing is caused by knocking down the activity of the miRNA you intend to target, and not caused by binding to an unexpected RNA.

Friday, September 9, 2016 - 09:59
Vivo-Morpholino use in rodent livers: annotated citations

In response to a question about the history of Vivo-Morpholino use in rodent livers, I assembled this annotated citation list.

Wednesday, August 31, 2016 - 10:47
phase 1 clinical trial with a Morpholino

Here's a report of a phase 1 clinical trial with a Morpholino oligo.

Komaki H, Nagata T, Saito T, Masuda S, Takeshita E, Tachimori H, Sasaki M, Takeda S. Exon 53 skipping of the dystrophin gene in patients with Duchenne muscular dystrophy by systemic administration of NS-065/NCNP-01: A phase 1, dose escalation, first-in-human study. Neuromuscular Disord. 2015;26(2)S261-2. doi:10.1016/j.nmd.2015.06.276

Tuesday, July 19, 2016 - 13:57
J David Lambert's notes on oligo handling, autoclaving

From the paper:
Lambert JD, Johnson AB, Hudson CN, Chan A. Dpp/BMP2-4 Mediates Signaling from the D-Quadrant Organizer in a Spiralian Embryo. Curr Biol. 2016;[Epub ahead of print] doi:10.1016/j.cub.2016.05.059
http://www.cell.com/current-biology/abstract/S0960-9822(16)30556-5

Friday, July 8, 2016 - 09:13
Morpholino special issue: Journal of Drug DDD

There's a Morpholino special issue of the Austin Journal of Drug Discovery, Development and Delivery.
You may have to click “Antisense Drug Research and Development” to open the list of the papers.

Thursday, July 7, 2016 - 07:29
Link to an antimicrobial antisense review (Sully & Geller)

Antisense antimicrobial therapeutics.

Sully EK, Geller BL.

Curr Opin Microbiol. 2016 Jun 29;33:47-55. doi: 10.1016/j.mib.2016.05.017. [Epub ahead of print] Review.

http://www.sciencedirect.com/science/article/pii/S1369527416300807

(open-access)

Wednesday, July 6, 2016 - 14:19
Tm of a Morpholino - Effective? Specific?

A Morpholino user asked me about his oligo design, which I had marked with a Tm of 82.5°C. He wondered about efficacy and specificity of such an oligo. Here's my response.

Tuesday, June 14, 2016 - 10:58
Review: Splice-switching antisense oligonucleotides as therapeutic drugs

Here's a review I'd like to keep track of.

Splice-switching antisense oligonucleotides as therapeutic drugs.

Havens MA, Hastings ML.

Nucleic Acids Res. 2016 Jun 10. pii: gkw533. [Epub ahead of print]

http://nar.oxfordjournals.org/content/early/2016/06/10/nar.gkw533.long

Monday, June 13, 2016 - 14:20

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