CRISPR Gene-Editing Being Offered To British Blood Disorder Patients

The first therapy that uses gene-editing is to be offered on the NHS in a "revolutionary breakthrough" for patients. From a report: It will be used as a potential cure for the blood disorder beta thalassaemia. Stem cells which make blood will be extracted, reprogrammed to correct the condition and returned to the patient's body. It could spare them needing a blood transfusion, every three to five weeks, for life. People with beta thalassaemia struggle to produce enough haemoglobin, which is the protein in red blood cells that carries oxygen around the body. It is a genetic disease that is passed down through families and caused by defects in the body's instructions for manufacturing haemoglobin. It can leave people severely tired, weak, and short of breath and also cuts life expectancy.

"Nope... no notes. I just enjoyed it."

[Pseudonymous Powers]

This is great news!

I know someone with this

[Baron_Yam]

You can tell when they're in need of a blood transfusion because they get tired and depressed. And after the transfusion, they're giddy and a bit manic for a week.

It's got to be rough having your blood doing that to you, and given the package includes a significantly reduced life expectancy, I think I'd go for the experimental gene therapy if it were me.

Re: I know someone with this

[St.Creed]

Yeah me too. And I'd also do gene checking on any offspring before it hatches.

Re:

[Baron_Yam]

This is why I think an outright prohibition on germline genetic engineering is a bad thing. Once we have confidence it can be done with accuracy and precision, we should start to edit genes in heritable ways so we don't need to worry about each new generation of children.

Re:

[Whateverthisis]

Except it edits different people in different ways. And it edits in other places too, because we can't yet control the chemistry with real precision. And the way the conduct this therapy because of those risks is they remove the patient's bone marrow cells, edit them in lab, culture up millions of cells, sort through to find the ones that are successful, then once they get those cells they put the patient in a medically induced coma for several weeks, kill all of their remaining bone marrow with chemo the

Re:

[gef7]

For clarity, you should also name chimeric every blood recipient and that's a bit too many. Blood is an organ after all.

More to the point, the situation with this class of blood diseases is so desperate that in certain countries (India, I am looking at you) they can cause a quite literal run on the blood bank. That's on top of the serious complications coming with years of blood transfusions with iron overloading and the accumulation of other risks which are even still being discovered. What's more: the