11:30 - 11:45am
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 - 11:45am
    Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot
    Open to all, subject to availability
    -

    Speaker(s)

    William Lostal

    Genethon, Evry

    Events

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

    Symposium
    9:30 - 10:15am
    Not recorded

    Gene Therapy of Primary Immunodeficiencies (PID)
    Alain Fischer

    Symposium
    10:15 - 11:00am
    Not recorded

    Genetic Engineering of Human Hematopoiesis for the Treatment of Inheri…
    Luigi Naldini

    Symposium
    11:00 - 11:45am
    Not recorded

    Gene Therapy of Hemophilias
    Amit Nathwani

    Symposium
    2:15 - 3:00pm
    Not recorded

    New Mutation-Specific Pharmacotherapies for Cystic Fibrosis…
    Kris de Boeck

    Symposium
    3:00 - 3:35pm
    Not recorded

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

    Symposium
    3:35 - 3:55pm
    Not recorded

    From Promising Clinical Studies to Recent Clinical Trials for the Frag…
    Vincent des Portes

    Symposium
    3:55 - 4:30pm
    Not recorded

    The Discovery and Development of Olesoxime for Spinal Muscular Atrophy…
    Rebecca Pruss

    Symposium
    5:00 - 5:35pm
    Not recorded

    From Gene Identification to Functional Analysis and Treatment of Achon…
    Laurence Legeai-Mallet

    Symposium
    5:35 - 5:55pm
    Not recorded

    Inhibition of Thromboxane Synthase, a New Mechanism to Increase Bone D…
    Valerie Cormier-Daire

    Symposium
    5:55 - 6:15pm
    Not recorded

    Selected Oral Communications: Drug Screening on Hutchinson Gilford Pro…
    Xavier Nissan

    Symposium
    6:15 - 6:30pm
    Not recorded

    Novel Approaches for Treatment of Retinal Diseases
    José-Alain Sahel

    Symposium
    9:00 - 9:45am
    Not recorded

    Gene Replacement Therapy for Myotubular Myopathy
    Anna Buj Bello

    Symposium
    9:45 - 10:15am
    Not recorded

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

    Symposium
    10:15 - 10:35am
    Not recorded

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

    Symposium
    11:00 - 11:30am
    Not recorded

    Selected Oral Communication: High Expression and Therapeutic Efficienc…
    William Lostal

    Symposium
    11:30 - 11:45am
    Not recorded

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

    Symposium
    11:45am - 12:00pm
    Not recorded

    Round Table: Can We Afford Cost of Innovative Therapies for Rare Disea…
    Franck Dufour et Serge Braun

    Symposium
    12:00 - 12:45pm
    Not recorded

    See also

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