We have talked about spinal muscular atrophy as a likely disease...

We have talked about spinal muscular atrophy as a likely disease indication. In reference to my previous post what is the likelihood it could be cystic fibrosis?

 

Here's a paper that is quite interesting. It brings together researchers from a number of academic institutions including Sue Fletcher and Steve Wilton from Murdoch as well as researchers who hold positions with the Telethon Kids Institute; The potential of antisense oligonucleotide therapies for inherited childhood lung diseases, published February 2018.

Researchers have identified key issues with the use of AOs to treat lung disease with genetic conditions. Here is a summary of problems in the delivery of antisense oligonucleotide therapy;

• therapeutic effect depends upon reaching both the target cell as well as intracellular molecular targets
• intracellular delivery will be ineffective if the AO cannot escape the endosome.

In fact, the paper concludes that the endosomal escape barrier is one of the most significant obstacles standing in the way of the effective use of AOs in therapeutics. Ring some bells.

Its a small world in the technological advancement of AO therapeutics and Perth appears to be at its epicentre.