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Jon's Blog
| Post date | ||
|---|---|---|
| Review of eteplirsen for DMD |
Mutation-Based Therapy for Duchenne Muscular Dystrophy: Antisense Treatment Arrives in the Clinic. |
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. https://academic.oup.com/nar/article/doi/10.1093/nar/gkx638/3984534/Numb... |
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: GGTGATGTAGTACGC(ATG)GTAGACCATTTTGACAGgtgtacgttattgccgctcgcgcgcgtagcagtgacagtgacgagacgtcagGTTTTGTGCATGCGGCGTAGTAAACAAGgtgccctgcatacgcgctcgtcgaatagctctagatcccaatagcgcaagGGTGGCCCTCGAT 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. http://journals.plos.org/plosgenetics/article?id=10.1371/journal.pgen.10... |
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. |
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. http://oregonstate.edu/ua/ncs/archives/2017/mar/often-villain-fructose-m... 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 |
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