Sofia Benkhelifa-Ziyyat
Research scientist , Scientific manager of MyoVector, PhD
Titles and positions
- Research scientist , Scientific manager of MyoVector, PhD
- Association Institute of Myology
Expertises
- Gene therapy for muscle diseases
- Biology of viral vectors
- AAV (Adeno-Associated Virus) vector production
Presentation
While clinical trials for therapies using AAV (Adeno-Associated Virus) vectors for neuromuscular diseases are ongoing, it appears crucial to study the optimal conditions for the effectiveness of these vectors. The injection of large doses involves a technological challenge for their production, a very high cost, but also risks of immune responses. The development and optimization of these vectors necessarily involves the identification of their potential intracellular barriers.
I’m interested in the intracellular transport of AAV vectors and their efficiency in models of muscle pathologies treated with gene therapy, in this case Duchenne muscular dystrophy and Autosomal Dominant Centronuclear Myopathy.
In addition to my research activity, I am scientific manager of the technical platform for the production of AAV MyoVector vectors which I manage within the Center of Research in Myology.