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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
SARS-CoV-2 RNA targets for steric blocking

Potantial targets for steric blocking on the SARS-CoV-2 genome and transcripts.

The SARS-CoV-2 RNA interactome.
Lee S, Lee YS, Choi Y, Son A, Park Y, Lee KM, Kim J, Kim JS, Kim VN. Mol Cell. 2021 Apr 27:S1097-2765(21)00327-0. doi: 10.1016/j.molcel.2021.04.022. Online ahead of print.

https://www.sciencedirect.com/science/article/pii/S1097276521003270

Monday, May 17, 2021 - 10:23
Positive control, human and mouse splice modification

Flynn LL, Mitrpant C, Adams A, Pitout IL, Stirnweiss A, Fletcher S, Wilton SD. Targeted SMN Exon Skipping: A Useful Control to Assess In Vitro and In Vivo Splice-Switching Studies. Biomedicines. 2021;9(5):552. doi:10.3390/biomedicines9050552

https://www.mdpi.com/2227-9059/9/5/552/htm

Friday, May 14, 2021 - 11:52
MO knockdown of ectopic GFP in axlotl

There are few papers reporting using a Morpholino to directly target GFP in vivo; here is one example.
Schnapp E, Tamaka EM. Quantitative evaluation of morpholino-mediated protein knockdown of GFP, MSX1, and PAX7 during tail regeneration in Ambystoma mexicanum. Dev Dyn. 2005 Jan;232(1):162-70.
https://anatomypubs.onlinelibrary.wiley.com/doi/full/10.1002/dvdy.20203

Friday, April 23, 2021 - 11:21
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

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