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|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|
|Mutant-morphant mismatch but no genetic compensation||
Zebrafish Chromosome 14 Gene Differential Expression in the fmr1 h u2787 Model of Fragile X Syndrome.
|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.
|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.
|Monday, May 17, 2021 - 11:33|