New Human Gene-Editing Trials
by Alyssa Butts
A group virtually presented safety trials with 52 patients. In the hempophilla trials thier method invovled using a virus carrying the factor IX gene to infect liver cells. The virus then remains active in infected cells, providing them with a copy of a functional factor IX gene that can stick around for a while. After six months of the infusion of the virus, these patients' levels of factor IX had risen to the point where they had a bleeding risk similar to the low end of the general population. That level is classified as a very mild hemophilia and doesn't require treatment. Mostly invovled side effects where an immune response to the virus, which was treated with steroids.
The second trial was smaller but therpay was more complex. The trial fouced on sickle cell anima and a form of β-thalassemia. One of the ideas they had for treating these anemias is to reactivate the fetal hemoglobin gene. It has a higher affinity for oxygen, allowing it to pick up oxygen from the adult form in the placenta. Their theory was that if you elimate BCL11A, you can reactivate the fetal hemoglobin genes. Unfortunately, getting rid of it isn't simple, because it performs essential functions in other cells. To get around this problem, the researchers behind the new work obtained blood stem cells from patients with β-thalassemia and sickle-cell disease. They were then subjected to CRISPR gene editing, which had deleted a piece of DNA that was essential for activating BCL11A in red blood cells. When these edited stem cells formed red blood cells in culture, they produced elevated levels of fetal hemoglobin.
Does this also make the person who receives it not pass on the genetic disorder, or does each generation need to receive this treatment?
Would gene- editing be the best way to get rid of or cure diseases?
I would think that each generation would need to receive the treatment for an amount of time, What I'm worried about is the genetic disorder could change, just remain dormant in some or the treatment could not work for some people so the genetic disorder may always be there.
ReplyDelete-Angeline Upchurch
It sounds like the person who receives this treatment will still pass on these genes as the therapies don't modify the genes, they treat the symptoms. Gene editing could potentially cure diseases that result from mutated genes, however, we would need more research on this. Additionally, once we start modifying human genes we must maintain a fine line between editing for a medical purpose and editing to give rise to desired traits.
ReplyDeleteCarissa Bersche
While gene editing is helpful in curing diseases, I would not consider it the best way. It is incredibly expensive and can become unethical. Because it is so expensive, it is safe to assume only people with higher incomes would have access to this type of technology, meaning the socioeconomic gap between the rich and the poor becomes even bigger. In theory, it's easy to say that gene editing would be only be strictly used for things such as curing diseases. However, I believe it will be challenging to regulate and to make sure it doesn't fall into the 'wrong hands'.
ReplyDeleteTrinity Mathis
I feel like this treatment could work but you would still pass it on as its still technically in your DNA. But seeing the cost of it and going every generation it can get very out of hand with pricing. I don't think gene editing is the best way to "cure" diseases yet but it could possibly work.
ReplyDelete-Tyler A.
Yes, I totally agree with you on the cost. It will be hard for some people to keep doing this treatment if the gene passes down. I did a little more research on it and they said the gene would not pass down.
DeleteAlyssa Butts
I think that gene editing is a good way to treat many diseases but it still needs time and research before it becomes an essential therapy at hospitals. For now, most diseases can be cured without the use of gene editing and we should stick to that until more research has been conducted. -Shirin Saha
ReplyDeleteI don't think gene editing is the best way to treat this. 1) Doesn't get rid of the genes that cause the disease, it can still be passed down 2) Too costly to treat the people that are mostly affected by this disease.
ReplyDelete-Ilori Tankpinou