MS PPMO from Wilton ... plus other uses. | SRPT Message Board Posts

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Msg  13938 of 13952  at  9/11/2019 12:42:45 PM  by

schmiggins


MS PPMO from Wilton ... plus other uses.

Kindness of Jon Moulton (posting it on twitter):

https://www.nature.com/articles/s41598-019-49385-6

Reduction of integrin alpha 4 activity through splice modulating antisense oligonucleotides

"....Peptide conjugated phosphorodiamidate morpholino antisense oligomers targeting ITGA4 were also assessed for their effect in delaying disease progression in the experimental autoimmune encephalomyelitis mouse model of multiple sclerosis. With the promising results in ameliorating disease progression, we are optimistic that the candidate oligomer may also be applicable to many other diseases associated with integrin alpha 4 mediated inflammation. This highly specific strategy to down-regulate protein expression through interfering with normal exon selection during pre-mRNA processing should be applicable to many other gene targets that undergo splicing during expression."

Will we take this from Wilton to augment our GT MS program? I would imagine we would. And notice again, this approach would work for other diseases than the MS mouse model that they tested it on. And as we saw a few days ago we've come up with new advances on the PPMO that should be used with this thing someday.


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