I had a friend with Fukutin-related limb-girdle muscular dystrophy R13 and am wondering if CRISPR could be the solution for her.
I had a friend with Fukutin-related limb-girdle muscular dystrophy R13 and am wondering if CRISPR could be the solution for her.
I'm not saying your attitude is obviously wrong, but there must be a lot of people whose imagination is captured by making tools and the potential of that to change the world. The development of a tool like optogenetics, for instance, makes me feel like amazing things can be accomplished. It's leverage.
Maybe you and I have different definitions of 'platform' company? I define as a way to iterate to get new products, like Regeneron uses their humanized mice to crank-out mAbs, or Genetech/Amgen use recombinant molecular biology to make mAbs, or Vertex uses their in vitro ion channel conductance screening method to make new structure correctors for CFTR...
Was money a problem in general along the way?
I agree that you should give serious pause. Probably the most serious pause of your life, but still. I wouldn't shy away too quickly. When you know, you know.
So you have a much larger pool of genetic and non-genetic diseases that can be cured now. For example if someone had low lung function due to lung scarring, this might be an interesting treatment to look at.