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Jon's Blog
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Oligo types, therapeutic Morpholinos, upregulation with Morpholinos |
Here are some types of oligos sorted by their mechanisms. RNase-H dependent
RNase-independent (steric blocking oligos)
Argonaute dependent
About the Morpholino drugs |
Thursday, April 15, 2021 - 09:24 |
Review of oligo delivery for therapeutics |
Hammond SH et al. Delivery of oligonucleotide-based therapeutics: challenges and opportunities. Embo Mol Med. 2021:e13243. doi: 10.15252/emmm.202013243 |
Wednesday, April 7, 2021 - 09:48 |
Review of mutant-morphant discrepency & genetic compensation |
Salanga CM, Salanga MC. Genotype to Phenotype: CRISPR Gene Editing Reveals Genetic Compensation as a Mechanism for Phenotypic Disjunction of Morphants and Mutants. Int J Mol Sci. 2021;22(7),3472. doi:10.3390/ijms22073472 |
Monday, March 29, 2021 - 13:39 |
Papers describing Morpholinos used in DMD studies |
Papers describing Morpholinos used in DMD studies NON-CLINICAL PAPERS |
Thursday, March 4, 2021 - 15:01 |
A description of using Morpholinos to block splice-regulatory protein binding sites |
Biayna J, Mazuelas H, Gel B, Terribas E, Dumbovic G, Rosas I, Fernández-Rodriguez J, Blanco I, Castellanos E, Carrió M, Lazaro C, Serra E. Using antisense oligonucleotides for the physiological modulation of the alternative splicing of NF1 exon 23a during PC12 neuronal differentiation. Sci Rep. 2021;11(1):3661. doi:10.1038/s41598-021-83152-w |
Thursday, February 11, 2021 - 10:44 |
Review: Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases |
Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases. |
Thursday, December 10, 2020 - 13:45 |
Clinical Pharmacology Studies Supporting Oligonucleotide Therapy Development: An Assessment of Therapies Approved and in Development Between 2012-2018 |
Clinical Pharmacology Studies Supporting Oligonucleotide Therapy Development: An Assessment of Therapies Approved and in Development Between 2012-2018. |
Monday, December 7, 2020 - 10:08 |
Blocking a cryptic splice site activiated in a mutant to restore normal splicing |
Bergsma AJ, in ’t Groen SLM, Catalano F, Yamanaka M, Takahashi S, Okumiya T, van der Ploeg AT, Pijnappel WWMP. A generic assay for the identification of splicing variants that induce nonsense-mediated decay in Pompe disease. Eur J Hum Genet. 2020;{Epub ahead of print]. doi:10.1038/s41431-020-00751-3 |
Monday, November 9, 2020 - 11:16 |
Morpholinos used to probe genetic compensation in mutants |
Genetic compensation prevents myopathy and heart failure in an in vivo model of Bag3 deficiency. https://journals.plos.org/plosgenetics/article?id=10.1371/journal.pgen.1... |
Tuesday, November 3, 2020 - 09:10 |
Inhibition of SARS-CoV-2 in Vero cell cultures by peptide-conjugated morpholino-oligomers |
Rosenke K, Leventhal S, Moulton H, Hatlevig S, Hawman D, Feldmann H, Stein DA. Inhibition of SARS-CoV-2 in Vero cell cultures by peptide-conjugated morpholino-oligomers. J Antimicrob Chemother. 2020;[Epub ahead of print]:dkaa460 doi:10.1093/jac/dkaa460. |
Tuesday, October 6, 2020 - 10:47 |