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|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|
|Example of specificity control using a Morpholino in its mutant||
This paper is a nice example of Morpholino specificity control by using a Morpholino in its corresponding mutant.
Chambers BE, Gerlach GF, Clark EG, Chen KH, Levesque AE, Leshchiner I, Goessling W, Wingert RA. Tfap2a is a novel gatekeeper of nephron differentiation during kidney development. Development. 2019 Jun 3. pii: dev.172387. doi: 10.1242/dev.172387. [Epub ahead of print]
|Wednesday, July 10, 2019 - 11:53|
|Targeting mitochondrially-encoded circular RNAs in the cytosol: mitochondrial protein uptake suppression||
Here is an interesting report of Morpholinos targeting mitochondrially-encoded circular RNAs in the cytosol to prevent RNA-chaperoned protein uptake into the mitochondria.
Liu X, Wang X, Li J, Hu S, Deng Y, Yin H, Bao X, Zhang QC, Wang G, Wang B, Shi Q, Shan G. The identification of mecciRNAs and their roles in mitochondrial entry of proteins. bioRxiv. 2019;[preprint] doi:10.1101/668665
|Friday, July 5, 2019 - 12:32|
|Dosing mama mouse i.v. for transplacental delivery of Vivo-Morpholinos to pups||
Touma M, Kang X, Gao F, Zhao Y, Cass AA, Biniwale R, Xiao X, Eghbali M, Coppola G, Reemtsen B, Wang Y. Wnt11 regulates cardiac chamber development and disease during perinatal maturation. JCI Insight. 2017 Sep 7;2(17). pii: 94904. doi: 10.1172/jci.insight.94904. eCollection 2017 Sep 7.
|Friday, May 31, 2019 - 12:00|
|Review - Antisense-mediated splice intervention to treat human disease: the odyssey continues||
Pitout I, Flynn LL, Wilton SD, Fletcher S.
Antisense-mediated splice intervention to treat human disease: the odyssey continues.
F1000Research 2019,8(F1000 Faculty Rev):710 doi:10.12688/f1000research.18466.1
|Wednesday, May 22, 2019 - 13:51|
|Innate immune stimulation by Morpholinos in Xenopus?||
The discussion of innate immune stimulation by Morpholinos in Xenopus continues.
Paraiso KD, Blitz IL, Zhou JJ, Cho KWY. Morpholinos Do Not Elicit an Innate Immune Response during Early Xenopus Embryogenesis. Dev Cell. 2019;49(4):643-50. doi:10.1016/j.devcel.2019.04.019
|Monday, May 20, 2019 - 14:37|