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Jon's Blog

		Post date
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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