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Paper: Induction of cryptic pre-mRNA splice-switching by antisense oligonucleotide

Mostly phosphorothioate work

Ham KA, Keegan NP, McIntosh CS, Aung-Htut MT, Zaw K, Greer K, Fletcher Sue, Wilton SD. Induction of cryptic pre-mRNA splice-switching by antisense oligonucleotides. Sci Rep. 2021;11(1):15137 doi:10.1038/s41598-021-94639-x

Friday, July 23, 2021 - 14:22
Genetic compensation in soybean (no Morpholino)

Genetic compensation in soybean: I am putting this in the blog as an example of an organism distant from zebrafish which is also compensating for mutation.

Friday, July 16, 2021 - 14:42
Nice introduction to splice-modification therapy from OTS

From the Oligonucleotide Therapeutics Society: "Can Oligonucleotides Target RNA Splicing to Treat Disease?"

Wednesday, June 23, 2021 - 09:41
Mutant-morphant mismatch but no genetic compensation

Zebrafish Chromosome 14 Gene Differential Expression in the fmr1 h u2787 Model of Fragile X Syndrome.
Barthelson K, Baer L, Dong Y, Hand M, Pujic Z, Newman M, Goodhill GJ, Richards RI, Pederson SM, Lardelli M. Front Genet. 2021 May 31;12:625466. doi: 10.3389/fgene.2021.625466. eCollection 2021.

Friday, June 18, 2021 - 08:14
Morphant vs mutant: "...this knockout line is deemed unsuitable for our study."

Here’s a nice description of why, in this study, morphants are preferable to mutants.

Friday, June 11, 2021 - 11:53
Review of neurodegenerative disease and splice modulation

Neurodegenerative diseases: a hotbed for splicing defects and the potential therapies.
Li D, McIntosh CS, Mastaglia FL, Wilton SD, Aung-Htut MT. Transl Neurodegener. 2021 May 20;10(1):16. doi: 10.1186/s40035-021-00240-7.

Monday, May 24, 2021 - 13:32
Therapeutic Modulation of RNA Splicing in Malignant and Non-Malignant Disease (Review)

Therapeutic Modulation of RNA Splicing in Malignant and Non-Malignant Disease.
El Marabti E, Abdel-Wahab O. Trends Mol Med. 2021 May 13:S1471-4914(21)00100-3. doi: 10.1016/j.molmed.2021.04.005.

Monday, May 17, 2021 - 11:33
SARS-CoV-2 RNA targets for steric blocking

Potantial targets for steric blocking on the SARS-CoV-2 genome and transcripts.

The SARS-CoV-2 RNA interactome.
Lee S, Lee YS, Choi Y, Son A, Park Y, Lee KM, Kim J, Kim JS, Kim VN. Mol Cell. 2021 Apr 27:S1097-2765(21)00327-0. doi: 10.1016/j.molcel.2021.04.022. Online ahead of print.

Monday, May 17, 2021 - 10:23
Positive control, human and mouse splice modification

Flynn LL, Mitrpant C, Adams A, Pitout IL, Stirnweiss A, Fletcher S, Wilton SD. Targeted SMN Exon Skipping: A Useful Control to Assess In Vitro and In Vivo Splice-Switching Studies. Biomedicines. 2021;9(5):552. doi:10.3390/biomedicines9050552

Friday, May 14, 2021 - 11:52
MO knockdown of ectopic GFP in axlotl

There are few papers reporting using a Morpholino to directly target GFP in vivo; here is one example.
Schnapp E, Tamaka EM. Quantitative evaluation of morpholino-mediated protein knockdown of GFP, MSX1, and PAX7 during tail regeneration in Ambystoma mexicanum. Dev Dyn. 2005 Jan;232(1):162-70.

Friday, April 23, 2021 - 11:21