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Jon's Blog

		Post date
N=1 clinical trials (paper)	Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives. Synofzik M, van Roon-Mom WMC, Marckmann G, van Duyvenvoorde HA, Graessner H, Schüle R, Aartsma-Rus A. Nucleic Acid Ther. 2021 Sep 29. doi: 10.1089/nat.2021.0039. Online ahead of print. https://pubmed.ncbi.nlm.nih.gov/34591693/	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 https://www.mdpi.com/1420-3049/26/17/5420	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. Jakutis G, Stainier DYR. Annu Rev Genet. 2021 Jul 27. doi: 10.1146/annurev-genet-071719-020342. Online ahead of print. https://www.annualreviews.org/doi/pdf/10.1146/annurev-genet-071719-020342	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 https://www.nature.com/articles/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?" https://www.oligotherapeutics.org/can-oligonucleotides-target-rna-splici...	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. https://www.frontiersin.org/articles/10.3389/fgene.2021.625466/full	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. https://translationalneurodegeneration.biomedcentral.com/articles/10.118...	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. https://www.cell.com/trends/molecular-medicine/fulltext/S1471-4914(21)00100-3	Monday, May 17, 2021 - 11:33

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