e-School Series 5
25 years of experience of gene therapy for ADA-SCID: brining a medicinal product to approval Alessandro Aiuti, San Raffaele Telethon Institute for Gene Therapy IRCCS Ospedale San Raffaele and Vita Salute San Raffaele University, Milan
VIDEO FOR ESGCT MEMBERS ONLY
Beta Thal – the long way to cure
Giuliana Ferrari, San Raffaele Telethon Institute for Gene Therapy
Bone marrow failure syndromes: Fanconi anemia, a Stem Cell Disease
Juan Bueren, División de Terapias Innovadoras en el Sistema Hematopoyético
VIDEO NO LONGER AVAILABLE
The CNS - gene therapy for Alzheimer's disease and beyond
Nathalie Cartier, ICM, Inserm, Paris
The liver - main target in gene therapy
Gloria Gonzalez-Aseguinolaza, Cima, Pamplona
Gene and cell therapies for Epidermolysis Bullosa
Matthias Titeux, Institut Imagine
Lysosomal storage diseases - gene therapy as treatment option
Fatima Bosch, Univesitat Autonoma de Barcelona
Gene therapy for inherited retinopathies
Stylianos Michalakis, Ludwig-Maximilians-Universität München
Video no longer available
Gene Editing - from experimental to clinical trials
Matthew Porteus, Stanford University