Re - Intronn, they are partly owned by Proteome Sciences (PRM.L) which are quoted in the UK.
Also, FWIW, I pulled this off a UK message board -
First, what is it exactly that Intronn achieved? The following is a fair summary:
“The UI team used an engineered, defective common cold virus as a carrier to deliver the genetic information needed to produce a short piece of RNA that contained the CF genetic correction. This RNA molecule attaches to the defective RNA at the site of the mistake. The spliceosome complex of the target cells is then "tricked" into skipping across the two RNA molecules to produce a full-length, error-free RNA. This resulting molecule is the template for a normal CFTR protein.”
sciencedaily.com\2001\12\011224082410.htm
The problem with the above is that it requires a virus vector to work. Virus vectors have a lot of limitations at present which may or may not be solved to such a level that this kind of gene therapy becomes routine in the clinic. Now, a few months ago (November) Intronn also announced that they had managed to achieve gene correction by using fairly small oligonucleotides: small enough that they believed one day could bypass the need for delivery via a virus vector. The following is a summary of this from New Scientist:
“As with conventional gene therapy, though, delivering the PTMs (pre-therapeutic mRNAs),
is the biggest obstacle. At present, Intronn is using viruses to deliver DNA-from which cells make lots of PTMs-but it believes that PTMs could be small and versatile enough to be packaged and given as pills.”
From New Scientist magazine, vol 172 issue 2317, 17/11/2001, page 19
Putting together the above you have a very exciting possibility: On the one hand the Intronn approach to gene therapy is truly unique since it aims to correct rather than replace defective genes. On the other hand their potential to minimise the size of the delivered agent means that they may be able to by-pass the need for a virus-based delivery system. The two together could revolutionise gene therapy, and this was the point of my earliest post... |
