I was drifting around Amazon Prime last night and came across a documentary called "Special Blood". The movie is about a disease called H.A.E. for short.
Hereditary Angioedema, is a pretty horrid deal. It's very rare, inherited, and way too often, kills kids. The lucky people that it doesn't kill get to get to suffer attacks for three or four days every week or two.
The quick version: sufferers have a genetic variant. If you've read my other posts you know about variants. This particular variant (in simplest terms) reduces or eliminates the creation of a protein in their bodies. The protein is called the C1 Inhibitor, and what it does inside the body is it tells the body to stop swelling. Often the swelling attacks the throat. Ergo, the fatalities.
Funding and research to do something for these people is lacking. It's pretty rare, and therefor insufficient return on investment is de-motivating established "normal" efforts at solutions.
It's exactly this situation where I think our project can make a difference. The community we are trying to form would have the ability to "give a nod" to enable work on this disease. And there's good reason to believe something can be done in short order.
First we check to see what AddGene has on the C1 Inhibitor (Caution: Deep Science Content. Feel free to skip it)
Now, Have a look at this:
It's a CRISPR vector available from a company called OriGene and what it does is "knocks-In" the genetic code responsible for creating the C1 Inhibitor Protein in mamals.
What I wanted you to notice is the price.
Now I'm not saying that it's as easy as ordering up this CRISPR juice, spinning it around a few times in a centrifuge to extract the little rings of DNA (plasmid) from the test tube, mixing in some polyethylenimine to help the CRISPR pass though cell walls and do it's repair work on little Eva's DNA.
Or am I?
I apologize for having to bring this up again. Many of you know about EOS. EOS has the resources to keep this project alive and they are willing to give them away to worthy projects (we hope like ours). It doesn't happen just because. We need to get their attention or we likely won't be around much longer. They've setup a way for us to get their attention called #EOSHackathon. We are competing with other projects. To get there, and get EOS to notice us, we need you and all your friends to Like this Video. Currently, we're in 10th place. We need to stay in the top 10 for 6 more days so the judges will have a chance to look at our project & we desperately need your LIKE
As Always, Grateful for the Support You've Already Shown Us,
~Greg
I hope they get the attention of EOS since it really is a great project that will help many
I do CRISPR every day and have made dozens of genetically modified cell lines. I can easily do this, but your article is overly simplified. Having said that, I haven't done any reading on this disease or the mutation that causes it, but I'm certain I could fix it... And that you wouldn't want to use PEI for the transfection ;)