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Jon's Blog
| Post date | ||
|---|---|---|
| Pharmacol & Tox of FDA-approved antisense |
Absorption, distribution, metabolism, and excretion of FDA-approved antisense oligonucleotide drugs. |
Tuesday, March 1, 2022 - 14:34 |
| Electroporation protocol: Amaxa 4D X unit nucleofector system |
Goossens R, Aartsma-Rus A. In Vitro Delivery of PMOs in Myoblasts by Electroporation. Methods Mol Biol. 2022;2434:191-205. doi: 10.1007/978-1-0716-2010-6_12. |
Tuesday, March 1, 2022 - 08:50 |
| Analysis of an apparent mutant/morphant disagreement |
Barthelson K, Baer L, Dong Y, Hand M, Pujic Z, Newman M, Goodhill GJ, Richards RI, Pederson SM, Lardelli M. Zebrafish Chromosome 14 Gene Differential Expression in the fmr1 (h u2787) Model of Fragile X Syndrome. Front Genet. 2021 May 31;12:625466. doi: 10.3389/fgene.2021.625466. eCollection 2021. https://www.frontiersin.org/articles/10.3389/fgene.2021.625466/full Interesting analysis of an apparent mutant/morphant disagreement finding no genetic compensation experimentally. |
Tuesday, February 8, 2022 - 14:24 |
| Generating Zebrafish RNA-Less Mutant Alleles by Deleting Gene Promoters with CRISPR/Cas9 |
A frameshift mutation did not phenocopy the corresponding Morpholino. To avoid transcriptional adaptation (genetic compensation), the authors made mutants with deleted promoters, eliminating the NMD transcript that would trigger transcriptional adaptation. |
Thursday, December 16, 2021 - 10:11 |
| N=1 clinical trials (paper) |
Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives. |
Friday, October 1, 2021 - 09:29 |
| Chemistry of Peptide-Oligonucleotide Conjugates: A Review |
It appears CPP are becoming a hot topic for reviews. This is a nice open-access example. Klabenkova K, Fokina A, Stetsenko D. Chemistry of Peptide-Oligonucleotide Conjugates: A Review. Molecules. 2021; 26(17):5420. https://doi.org/10.3390/molecules26175420 |
Friday, September 10, 2021 - 10:43 |
| Review: Genotype-Phenotype Relationships in the Context of Transcriptional Adaptation and Genetic Robustness |
Genotype-Phenotype Relationships in the Context of Transcriptional Adaptation and Genetic Robustness. |
Wednesday, July 28, 2021 - 09:40 |
| 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 |
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