A therapeutic strategy in which short RNAs work as chaperones that bind to TDP-43

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The journal Science published an interesting article a few days ago. It concerns a fragment of RNA with promising properties. enter link description here

On the one hand, it appears to properly fold the TDP-43 proteins involved in ALS (it's a kind of chaperone molecule), and on the other hand, it could relocate these proteins to the cell nucleus. enter image description here

If these results could be translated to humans, this would be the first drug capable of treating a large proportion of ALS patients. The approach is conceptually similar to that of antisense therapies, which provided the first truly effective drug against ALS, but which is only suitable for one or two percent of these patients.

This result stems from years of work involving numerous people and testing thousand candidate molecules.

A patent application has been filed, which, given the significant investment involved, demonstrates that the researchers have good hopes of being able to commercialize their expertise. Nevertheless, caution is advised.



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