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ESH Erythro 2025 | The growing role of gene editing and gene therapy in SCD & important practical considerations
Sara El Hoss, PhD, Institut Imagine, Paris, France, briefly discusses the growing importance of gene editing and gene therapy in sickle cell disease (SCD) and beta-thalassemia, highlighting their promising potential for optimizing treatment approaches. Dr El Hoss emphasizes the need to focus on in vivo gene therapy to reduce the burden on patients, hospitals, and the economy, while also continuing research on small molecules. This interview took place at the 4th ESH Translational Research Conference on Pathophysiology and Clinical Advances in Sickle Cell Disease and Thalassemia in Dublin, Ireland.
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Transcript
I think that in the past few years, we have seen gene editing and gene therapy grow in sickle cell disease and in beta-thalassemia also. And I think that this is a very promising avenue for sickle cell patients. And I think that I hope that in the next 10 years, we will really be able to optimize these kinds of therapeutic approaches for patients. I think we need to try and focus our research on performing gene therapy in vivo so as to decrease the burden of this therapy on the patients and the hospitals and also on the economy because as you know, gene therapy is quite expensive...
I think that in the past few years, we have seen gene editing and gene therapy grow in sickle cell disease and in beta-thalassemia also. And I think that this is a very promising avenue for sickle cell patients. And I think that I hope that in the next 10 years, we will really be able to optimize these kinds of therapeutic approaches for patients. I think we need to try and focus our research on performing gene therapy in vivo so as to decrease the burden of this therapy on the patients and the hospitals and also on the economy because as you know, gene therapy is quite expensive. And I do believe, I think it’s very important that we point out that even though we are evolving and developing gene therapy, we need to keep doing research on the small molecules that could be also used for treatment because I think that we need to realize that maybe gene therapy will not be possible for everyone or in every setting. So, both gene therapy and also continuing our research on trying to find small molecules that could ameliorate the disease pathology are very important to pursue in sickle cell disease research now and in the future.
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