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GENE & CELL THERAPY OF RARE DISEASES

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

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