Skip to main content

You are here

Jon's Blog

		Post date
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
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

Pages