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General Updates | The development of epigenome editing and its potential use in the treatment of SCD and thalassemia
Letizia Fontana, PhD, Institut Imagine, Paris, France, discusses the concept of epigenome editing, a technique that utilizes a modified CRISPR-Cas9 system to alter gene expression without changing the DNA sequence itself. Dr Fontana explains how this approach can be applied to the treatment of sickle cell disease (SCD) and beta-thalassemia, and highlights the potential benefits of using epigenome editing as a safer alternative to traditional gene therapy strategies. This interview took place virtually.
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Transcript
Epigenome editing is a technique based on the use of a CRISPR-Cas9 that has been modified and developed in order to not cut anymore the DNA, doesn’t touch and modify anymore the DNA, but the aim is to modify the expression of the gene by attaching to the CRISPR-Cas9 modified, so a dead Cas9 now, an epigenome editor, which is a molecule, a domain, which can modify like histone marks or DNA methylation in order to re-express or repress also the gene that we want to target...
Epigenome editing is a technique based on the use of a CRISPR-Cas9 that has been modified and developed in order to not cut anymore the DNA, doesn’t touch and modify anymore the DNA, but the aim is to modify the expression of the gene by attaching to the CRISPR-Cas9 modified, so a dead Cas9 now, an epigenome editor, which is a molecule, a domain, which can modify like histone marks or DNA methylation in order to re-express or repress also the gene that we want to target. So the idea is more or less the same as the canonical CRISPR-Cas9, but differently from that, we are not touching the DNA sequence, so avoiding inserting mutations, deletions, insertions, whatever is related to the Cas9, but modifying the epigenome editing, which is around the region that we want to target, actually.
For what concerns sickle cell disease and beta-thalassemia, a lot of approaches nowadays are based on reactivating the expression of fetal hemoglobin, because we know that when re-expressing fetal hemoglobin in patients, this can improve both the sickle phenotype, because it prevents the polymerization of sickle hemoglobin, but also the thalassemia phenotype, because we rebalance all the amount of globin chains which are necessary to produce a functional hemoglobin. So the idea in general is that re-expressing fetal hemoglobin can bring an advantage for these diseases. This is the approach also used in the clinically approved Casgevy strategy, where they use CRISPR-Cas9, in order to reactivate the expression of fetal hemoglobin. So the idea is, our idea, and in general the epigenome editing idea, is to do the same, but using an epigenome editor. So avoiding, again, to touch or cut or make insertions in the DNA sequence in order to develop a strategy that is similar to what has been used and approved so far, but from a safer point of view, let’s say, so with a safer profile in terms of cutting and modifying the DNA.
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