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General Updates | The potential future role of genome and epigenome editing in SCD and beta-thalassemia
In this video, Letizia Fontana, PhD, Institut Imagine, Paris, France, discusses the potential future role of genome and epigenome editing in the treatment of sickle cell disease (SCD) and beta-thalassemia. Once current limitations of these approaches are overcome, possibly through the development of an in vivo strategy, Dr Fontana hopes that these treatment modalities could become available to a large proportion of patients. This interview took place virtually.
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Transcript
I think that the idea of genome and epigenome editing is becoming more and more exciting these days. And since I’m very passionate, it’s really my field of expertise, I hope that this could be an approach that can be then, I mean, applicable for all the patients that have sickle cell disease or beta-thalassemia. For the moment, I agree that there are some limitations, which are mainly due to the cost of the approaches, because they remain very expensive and especially for countries where it’s more complicated to collect the cells and modify them, and I agree that for the moment it’s not suitable like for all the patients...
I think that the idea of genome and epigenome editing is becoming more and more exciting these days. And since I’m very passionate, it’s really my field of expertise, I hope that this could be an approach that can be then, I mean, applicable for all the patients that have sickle cell disease or beta-thalassemia. For the moment, I agree that there are some limitations, which are mainly due to the cost of the approaches, because they remain very expensive and especially for countries where it’s more complicated to collect the cells and modify them, and I agree that for the moment it’s not suitable like for all the patients.
But what I’m seeing is that also we are improving the in vivo strategy because now the cost came mostly also from the fact that you have to take the cells, modify, re-inject in the patient. And so there are some also complications going from the treatment that the patient has to undergo and whatever. But since we are developing more and more the in vivo strategy so where we at a certain point we will be able to inject directly our tool in vivo in the patient so we will not need some very complicated infrastructure and we don’t have to follow the patient for a long time. I think that once we will reach this point, genome editing can be easily accessible for a lot of patients.
This transcript is AI-generated. While we strive for accuracy, please verify this copy with the video.
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