11:30 to 11:45
Symposium
Not recorded

Selected Oral Communication: High Expression and Therapeutic Efficiency of Systemic Delivery of a Dual AAV Strategy in a Murine Model for Dysferlin Deficiencies

William Lostal
Novel Therapies for Monogenic Diseases
11:30 to 11:45
    Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot
    Open to all, subject to availability
    -

    Speaker(s)

    William Lostal

    Genethon, Evry

    Events

    Symposium
    09:30 to 10:15
    Emmanuelle Charpentier

    Genome Engineering with CRISPR-Cas9: Biology, Mechanisms and Applicati…

    Not recorded
    Symposium
    10:15 to 11:00
    Alain Fischer

    Gene Therapy of Primary Immunodeficiencies (PID)

    Not recorded
    Symposium
    11:00 to 11:45
    Luigi Naldini

    Genetic Engineering of Human Hematopoiesis for the Treatment of Inheri…

    Not recorded
    Symposium
    14:15 to 15:00
    Amit Nathwani

    Gene Therapy of Hemophilias

    Not recorded
    Symposium
    15:00 to 15:35
    Kris de Boeck

    New Mutation-Specific Pharmacotherapies for Cystic Fibrosis…

    Not recorded
    Symposium
    15:35 to 15:55
    Judith Melki

    Added Value of New Genomics Technologies in Undiagnosed Patients Who M…

    Not recorded
    Symposium
    15:55 to 16:30
    Vincent des Portes

    From Promising Clinical Studies to Recent Clinical Trials for the Frag…

    Not recorded
    Symposium
    17:00 to 17:35
    Rebecca Pruss

    The Discovery and Development of Olesoxime for Spinal Muscular Atrophy…

    Not recorded
    Symposium
    17:35 to 17:55
    Laurence Legeai-Mallet

    From Gene Identification to Functional Analysis and Treatment of Achon…

    Not recorded
    Symposium
    17:55 to 18:15
    Valerie Cormier-Daire

    Inhibition of Thromboxane Synthase, a New Mechanism to Increase Bone D…

    Not recorded
    Symposium
    18:15 to 18:30
    Xavier Nissan

    Selected Oral Communications: Drug Screening on Hutchinson Gilford Pro…

    Not recorded
    Symposium
    09:00 to 09:45
    José-Alain Sahel

    Novel Approaches for Treatment of Retinal Diseases

    Not recorded
    Symposium
    09:45 to 10:15
    Anna Buj Bello

    Gene Replacement Therapy for Myotubular Myopathy

    Not recorded
    Symposium
    10:15 to 10:35
    Arnold Munnich

    REPS1 Is a Novel Gene of Neurodegeneration with Brain Iron Accumulatio…

    Not recorded
    Symposium
    11:00 to 11:30
    Hélène Puccio

    Efficient AAV Gene Therapy in Cardiac and Neurological Murine Models f…

    Not recorded
    Symposium
    11:30 to 11:45
    William Lostal

    Selected Oral Communication: High Expression and Therapeutic Efficienc…

    Not recorded
    Symposium
    11:45 to 12:00
    Nathalie Cartier

    Selected Oral Communication: Gene Therapy for Alzheimer's Disease: The…

    Not recorded
    Symposium
    12:00 to 12:45
    Franck Dufour et Serge Braun

    Round Table: Can We Afford Cost of Innovative Therapies for Rare Disea…

    Not recorded

    See also

    Alain Fischer, chair Experimental medicine
    Jean-Louis Mandel, chair Human genetics
    Novel Therapies for Monogenic Diseases