UK approves gene-editing drug

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Amity Island

Amity Island

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DNA is the blueprint of life - and genes contain the instructions for how every cell in our body works.

Gene-editing allows the precise manipulation of DNA. The treatment involves removing bone marrow stem cells from a patient's blood.
In a laboratory, the gene-editing tool Crispr uses molecular scissors to make precise cuts in the DNA of the cells, thus disabling the faulty gene. The modified cells are infused back, allowing the body to start producing functioning haemoglobin.

www.bbc.co.uk

Casgevy: UK approves gene-editing drug for sickle cell

Medical regulators approve a gene therapy that aims to cure sickle cell disease and beta thalassemia.
www.bbc.co.uk
 
Such good news if it works! I’ve been reading about Crispr for a while. Hopefully there will be a lot else it can help with.
 
It sounds promising that’s for sure. That’s a really high success rate from the trials as well. Hopefully they’ll be able to advance it to help manage, cure and potentially prevent genetic conditions
 
Does anyone know if a potential parent had cured (gene editing) their genetic problem, this fix would be passed through on to their children, or would their children also need to undergo similar gene editing should they inherit same problem?
 
Just a guess here but I think the fix would pass to the children because they couldn’t inherit something that neither parent has. Crispr severs the gene that causes the condition and therefore I don’t think that gene would be able to pass on
 
Amity Island said:
Does anyone know if a potential parent had cured (gene editing) their genetic problem, this fix would be passed through on to their children, or would their children also need to undergo similar gene editing should they inherit same problem?
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I think the kind of germ-line editing which could lead to heritable changes is widely regarded as unethical, at least on the basis of current knowledge.

As I understand it, the recently approved treatment operates on stem cells & the changes are not heritable.

But that's based only on my very skimpy knowledge of the field.
 
Lily123 said:
Just a guess here but I think the fix would pass to the children because they couldn’t inherit something that neither parent has.
Click to expand...
I think that would be true if all cells were modified, but I'm not sure if anyone knows how to even attempt that. The approved process involves removing bone marrow stem cells and modifying and returning those. So just a small fraction of the cells, but the important ones for creating red blood cells.

(As I understand it for germline treatment the only idea is to treat an early embryo (just a few cells) where you could modify the whole organism. I don't think that's even close to ethical approval and I'm not sure whether anyone thinks it's technically doable.)
 
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