Gene therapies: what prospects for researchers and patients?

2024-02-14 05:00:03

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”.

This research in the field of gene therapies aims to develop treatments for certain hereditary disorders or certain diseases: which ones? At what stage is this research? What are the prospects for recovery and what is the number of patients affected?

• 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

Watch the entire show here: What to expect from Crispr technology in gene therapy?