You are here
|A reversible RNA on-switch using a Vivo-Morpholino to controls gene expression of AAV-delivered therapeutics in vivo||
Zhong G, Wang H, He W, Li Y, Mou H, Tickner ZJ, Tran MH, Ou T, Yin Y, Diao H, Farzan M. A reversible RNA on-switch that controls gene expression of AAV-delivered therapeutics in vivo. Nat Biotechnol. 2019;[Epub ahead print] doi:10.1038/s41587-019-0357-y
|Monday, December 23, 2019 - 13:27|
|FDA approves golodirsen (a Morpholino drug)||
Sarepta Therapeutics Announces FDA Approval of VYONDYS 53™ (golodirsen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 53
|Thursday, December 12, 2019 - 16:01|
|Paper comparing splice-modifying efficacy of Morpholinos and 2'-O-Me phosphorothioates||
McIntosh CS, Aung-Htut MT, Fletcher S, Wilton SD. Removal of the Polyglutamine Repeat of Ataxin-3 by Redirecting pre-mRNA Processing. Int J Mol Sci. 2019;20(21):5434. doi:10.3390/ijms20215434
|Thursday, October 31, 2019 - 10:54|
|Protein interrelationship shown by synergy of Morpholinos to different mRNAs||
Oprişoreanu A-M, Smith HL, Arya S, Webster R, Zhong Z, Wehner D, Cardozo MJ, Becker T, Talbot K, Becker CG. Interaction of Axonal Chondrolectin with Collagen XIXa1 Is Necessary for Precise Neuromuscular Junction Formation. Cell Rep. 2019;29:1082-98. doi:10.1016/j.celrep.2019.09.033
|Wednesday, October 30, 2019 - 10:33|
|Morpholino assay in plasma: HPLC-MS/MS Coupled with Solid Phase Micro-Extraction||
Two Quantitative Assays for the Phosphorodiamidate #Morpholino Oligomer (PMO) SRP-4045 in Mouse Plasma Using HPLC-MS/MS Coupled with Solid Phase Micro-Extraction. Poster at ASMS 2017.
|Tuesday, October 29, 2019 - 13:49|
|A broader view: Approved Gene Therapy Products 2019||
Development and Clinical Translation of Approved Gene Therapy Products for Genetic Disorders.
Shahryari1 A, Jazi MS, Mohammadi S, Nikoo HR, Nazari Z, Hosseini ES, Burtscher I, Mowla SJ, Lickert H.
Front Genet. 25 September 2019. doi:10.3389/fgene.2019.00868
|Thursday, September 26, 2019 - 14:51|
|Paper: Site Selective Antibody-Oligonucleotide Conjugation via Microbial Transglutaminase.||
This paper describes using a microbial transglutamase to put an azide group in a peptide onto the terminal carboxylic acid of an antibody protein, then using Cu-free click chemistry to affix an antisense oligo at that site. They didn't use Morpholinos, but in principle one could.
|Friday, September 13, 2019 - 12:05|
|Paper on delivery in cultures: In Vitro Validation of Phosphorodiamidate Morpholino Oligomers.||
Aung-Htut MT, McIntosh CS, West KA, Fletcher S, Wilton SD. In Vitro Validation of Phosphorodiamidate Morpholino Oligomers. Molecules. 2019;24(6):2922 doi:0.3390/molecules24162922
cell culture: human dermal fibroblasts, human myoblasts
|Monday, August 12, 2019 - 13:13|
|Review on compensation: mutant & morphant||
Gene redundancy and gene compensation: An updated view.
J Genet Genomics. 2019 Jul 19. pii: S1673-8527(19)30115-8. doi: 10.1016/j.jgg.2019.07.001. [Epub ahead of print] Review.
|Tuesday, August 6, 2019 - 09:18|
|Delay in development and behavioural abnormalities in the absence of p53 in zebrafish||
Delay in development and behavioural abnormalities in the absence of p53 in zebrafish.
|Wednesday, July 31, 2019 - 09:30|