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Oligo types, therapeutic Morpholinos, upregulation with Morpholinos

Here are some types of oligos sorted by their mechanisms.

RNase-H dependent

  • DNA
  • Phosphorothioate DNA

RNase-independent (steric blocking oligos)

  • Morpholinos
  • 2’-O-substituted oligos (often phosphorothioate linkages)
    • 2’-O methyl
    • 2’-O methoxyethyl
  • PNA (peptide nucleic acids)
  • LNA (locked nucleic acid)

Argonaute dependent

  • siRNA
  • shRNA

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

https://www.embopress.org/doi/full/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
https://www.mdpi.com/1422-0067/22/7/3472

 Monday, March 29, 2021 - 13:39
Papers describing Morpholinos used in DMD studies

Papers describing Morpholinos used in DMD studies
Note: this is not an exhaustive list. Using DMD-related keywords in the Morpholino publications database will turn up many more citations. Some key papers are marked with triple astrisks (***). The papers are divided into non-clinical (first) and clinical (following).

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

https://www.nature.com/articles/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.
Tsoumpra MK, Fukumoto S, Matsumoto T, Takeda S, Wood MJA, Aoki Y. EBioMedicine. 2019 Jul;45:630-645. doi: 10.1016/j.ebiom.2019.06.036. Epub 2019 Jun 27.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6642283/

 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.
Rogers H, Adeniyi O, Ramamoorthy A, Bailey S, Pacanowski M.
Clin Transl Sci. 2020 Dec 5. doi: 10.1111/cts.12945. Online ahead of print.
https://ascpt.onlinelibrary.wiley.com/doi/10.1111/cts.12945

 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
https://www.nature.com/articles/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.
Diofano F, Weinmann K, Schneider I, Thiessen KD, Rottbauer W, Just S.
PLoS Genet. 2020 Nov 2;16(11):e1009088. doi: 10.1371/journal.pgen.1009088. eCollection 2020 Nov.

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.
https://academic.oup.com/jac/advance-article/doi/10.1093/jac/dkaa460/596...

 Tuesday, October 6, 2020 - 10:47

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