2024-02-13 09:00:04
On December 15, 2023, the European Medicines Agency (EMA) approved the first treatment using Crispr genome modification technology. This medical advance aims to cut out a DNA sequence and replace it with another, using a pair of “molecular scissors”. The first treatment using this method should be to relieve patients suffering from sickle cell anemia or beta thalassemia, two genetic hemoglobin diseases.
How does this new medical approach work? What perspectives does CRISPR technology open up for medicine? Finally, is gene therapy safe for humans?
Professor Marina Cavazzanapediatrician, professor of Hematology, director of the Department of Biotherapy of the Hospital Necker-Sick childrenParis Cité University and director of the Center for Clinical Investigation in Biotherapy at the Imagine Institute Dr Giulia Hardouin, post-doctoral student in the team of Annarita Miccio, team leader and Inserm researcher, in the field of stem cells and the treatment of hemoglobin diseases, at theInstitut Imaginein Paris Dr Laure Josephhematologist atNecker Hospitalspecialist in sickle cell disease for adults.
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► Steve Brunache – L’union.
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