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|Mechanism of uptake of Morpholino oligos in dystrophic (DMD) muscle||
This work elucidates the mechanism of uptake of Morpholino oligos in dystrophic (DMD) muscle, including roles for muscle satellite cells and macrophages.
Novak JS, Hogarth MW, Boehler JF, Nearing M, Vila MC, Heredia R, Fiorillo AA, Zhang A, Hathout Y, Hoffman EP, Jaiswal JK, Nagaraju K, Cirak S, Partridge TA.
Nat Commun. 2017 Oct 16;8(1):941. doi: 10.1038/s41467-017-00924-7.
|Thursday, October 19, 2017 - 10:36|
|Time for some music! Evo-devo||Wednesday, September 27, 2017 - 11:47|
There are several commonly-used strategies for confirming specificity of a Morpholino oligo. These are the five-mispair experiment, the second non-overlapping 5'-UTR oligo experiment (and the similar two oligos against one mRNA experiments), the mRNA rescue experiment, and my current favorite, the Morpholino-in-a-null-mutant experiment.
|Wednesday, September 27, 2017 - 08:46|
|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.
|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.
|Thursday, April 6, 2017 - 08:40|