Aix-Marseille University at ESGCT 2026
Oct 27–30, 2026 Hamburg, Germany ~2.5k attendees Website
Aix-Marseille University brings 3 presentations to ESGCT 2026 in Hamburg. Its work covers gene therapy for inherited neurological and muscle disorders, including Rett syndrome, dysferlinopathies and Charcot-Marie-Tooth type 2A. One approach opens the blood-brain barrier with focused ultrasound so a brain-targeted AAV can reach its destination.
| Organization | ESGCT 2026 Attendance |
|---|---|
Aix-Marseille University Marseille, France | |
Poster Wed Oct 28 1:30pm–3:30pm A combined gene therapy strategy for Rett syndrome using focused ultrasound-mediated BBB opening, CNS-targeted AAV.CAP-B10 and MECP2 regulation Poster Thu Oct 29 2:00pm–4:00pm Gene therapy for dysferlinopathies: from minigene strategy using muscle specific viruses to naked DNA sonodelivery Poster Thu Oct 29 2:00pm–4:00pm Restoring the interplay between endoplasmic reticulum and mitochondria by gene therapy alleviates Charcot-Marie-Tooth type 2A disease Rare and inherited disease |
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