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Review of eteplirsen for DMD

Mutation-Based Therapy for Duchenne Muscular Dystrophy: Antisense Treatment Arrives in the Clinic.
Elizabeth M. McNally, Eugene J. Wyatt.
Circulation. 2017;136:979-981.

Monday, September 11, 2017 - 14:04
BLAST homology and specificity controls

I have had many discussions about specificity with a Morpholino user who does very careful BLAST analysis of each oligo prior to ordering. I had previously suggested that about 14 bases of complementary (without significant flanking complementarity) is probably an acceptable level of stability for off-target interactions found by BLAST. However, in light of a new report I agreed that a 13-base complementarity at a splice junction is enough to raise concern. Here is an excerpt of my response to her.


Wednesday, August 2, 2017 - 08:21
Paper on Morpholino specificity and CRISPR-MO combination

Number of inadvertent RNA targets for morpholino knockdown in Danio rerio is largely underestimated: evidence from the study of Ser/Arg-rich splicing factors.

Joris M, Schloesser M, Baurain D, Hanikenne M, Muller M, Motte P.

Nucleic Acids Res. 2017;[Epub ahead of print] doi:10.1093/nar/gkx638.

Tuesday, August 1, 2017 - 14:35
Formatting sequence information for emails (splice modifying oligo designs)

To email sequence information for design of a splice-modifying Morpholino, write introns in lower case and exons in upper case and write the sense sequence 5'-3'. If you show a start codon, put parentheses around it. Here is an example:


All I need for the design are 10 exonic and 25 intronic bases, like this:

Tuesday, July 25, 2017 - 08:02
Genetic compensation: A phenomenon in search of mechanisms

Genetic compensation: A phenomenon in search of mechanisms.

El-Brolosy MA, Stainier DYR.

PLoS Genet. 2017 Jul 13;13(7):e1006780. doi: 10.1371/journal.pgen.1006780. eCollection 2017 Jul. Review.

Friday, July 14, 2017 - 10:29
Book: Morpholino Oligomers

Hong and I edited a book: Morpholino Oligomers. 2017. Methods in Molecular Biology. Volume 1565. Humana Press (Springer). doi:10.1007/978-1-4939-6817-6

Links to chapters follow.

1. Summerton JE. Invention and Early History of Morpholinos: From Pipe Dream to Practical Products. Methods Mol Biol. 2017;1565:1-15. doi: 10.1007/978-1-4939-6817-6_1. Abstract: PubMed

Tuesday, April 11, 2017 - 01:57
Review of FDA-approved antisense (2017)

FDA-Approved Oligonucleotide Therapies in 2017.
Stein CA, Castanotto D.
Mol Ther. 2017 Mar 30. pii: S1525-0016(17)30122-3. doi: 10.1016/j.ymthe.2017.03.023. [Epub ahead of print] Review.

Thursday, April 6, 2017 - 08:40
Using Morpholinos to Control Gene Expression (in CPNAC)

Wiley has published an update to my Morpholino how-to and background review.

Moulton JD. Using Morpholinos to Control Gene Expression. Curr Protoc Nucleic Acid Chem. 2017 Mar 2;68:4.30.1-4.30.29. doi: 10.1002/cpnc.21.

Saturday, March 4, 2017 - 03:26
Fructose and uptake of Morpholinos

This is about fructose and Morpholinos.

Here is the original paper being discussed.

Friday, March 3, 2017 - 08:58
nasal administration of delivery-enabled Morpholino in mice

This paper is an example of nasal administration of a delivery-enabled Morpholino in mice.

Soonthornvacharin S, Rodriguez-Frandsen A, Zhou Y, Galvez F, Huffmaster NJ, Tripathi S, Balasubramaniam VRMT, Inoue A, de Castro E, Moulton H, Stein DA, Sánchez-Aparicio MT, De Jesus PD, Nguyen Q, König R, Krogan NJ, García-Sastre A, Yoh SM, Chanda SK. Systems-based analysis of RIG-I-dependent signalling identifies KHSRP as an inhibitor of RIG-I receptor activation. Nat Microbio. 2017;2:17022. doi:10.1038/nmicrobiol.2017.22

Friday, March 3, 2017 - 06:55