May. 31 at 3:34 PM
$KRRO I find it very interesting for RNA to be edited to fix a deficiency in a gene. I have positions in VERV and ARCT. ARCT-032 supply entire length of mRNA for fixing deficient cftr grne by tecnically doing protein replacement. VERV-301 is gene editing to LP(a) to lower genetically driven high cholesterol... it's a one time DNA efitor. They all use LNP.
Because i am closely following DMD gene therapy. I was thinking helthy mRNA Dystrophin gene supplied to muscle cell was way to go. But if you can find locations of mutant genes then just edit those RNA genes prior to translation it will be great too...No risks to dna mishandling and upside to do combination with protein replacement gene therapy. Just getting interested in RNA editing tech.
