You are here
Jon's Blog
| Post date | ||
|---|---|---|
| Vivo-Morpholinos as switching molecules for virally-delivered targets in mice |
Vivo-Morpholinos as switching molecules for virally-delivered targets in mice. Cripe TP, Hutzen B, Currier MA, Chen CY, Glaspell AM, Sullivan GC, Hurley JM, Deighen MR, Venkataramany AS, Mo X, Stanek JR, Miller AR, Wijeratne S, Magrini V, Mardis ER, Mendell JR, Chandler DS, Wang PY. Leveraging gene therapy to achieve long-term continuous or controllable expression of biotherapeutics. Sci Adv. 2022 Jul 15;8(28):eabm1890. doi: 10.1126/sciadv.abm1890. Epub 2022 Jul 13. |
Wednesday, August 10, 2022 - 12:14 |
| Head-to-head comparisons, Morpholinos and Phosphorothioates |
Single Stranded Fully Modified-Phosphorothioate Oligonucleotides can Induce Structured Nuclear Inclusions, Alter Nuclear Protein Localization and Disturb the Transcriptome In Vitro. Flynn LL, Li R, Pitout IL, Aung-Htut MT, Larcher LM, Cooper JAL, Greer KL, Hubbard A, Griffiths L, Bond CS, Wilton SD, Fox AH, Fletcher S. Front Genet. 2022;13:791416. doi:10.3389/fgene.2022.791416 https://www.frontiersin.org/articles/10.3389/fgene.2022.791416/full |
Tuesday, April 19, 2022 - 10:15 |
| Adverse Drug Reactions and Toxicity of the FDA-approved Antisense Oligonucleotide Drugs |
Adverse Drug Reactions and Toxicity of the FDA-approved Antisense Oligonucleotide Drugs. https://dmd.aspetjournals.org/content/early/2022/02/27/dmd.121.000418.long |
Thursday, March 24, 2022 - 14:08 |
| 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 |
Pages
