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Symposium

Novel Therapies for Monogenic Diseases

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    Program

    Genome Engineering with CRISPR-Cas9: Biology, Mechanisms and Applications in Medicine
    Emmanuelle Charpentier

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    Not recorded
    Symposium
    Novel Therapies for Monogenic Diseases
    Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot
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    Gene Therapy of Primary Immunodeficiencies (PID)
    Alain Fischer

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    Not recorded
    Symposium
    Novel Therapies for Monogenic Diseases
    Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot
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    Genetic Engineering of Human Hematopoiesis for the Treatment of Inherited Diseases and Cancer
    Luigi Naldini

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    Not recorded
    Symposium
    Novel Therapies for Monogenic Diseases
    Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot
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    Gene Therapy of Hemophilias
    Amit Nathwani

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    Not recorded
    Symposium
    Novel Therapies for Monogenic Diseases
    Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot
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    New Mutation-Specific Pharmacotherapies for Cystic Fibrosis
    Kris de Boeck

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    Not recorded
    Symposium
    Novel Therapies for Monogenic Diseases
    Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot
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    Added Value of New Genomics Technologies in Undiagnosed Patients Who May Benefit from Known Therapies
    Judith Melki

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    Not recorded
    Symposium
    Novel Therapies for Monogenic Diseases
    Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot
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    From Promising Clinical Studies to Recent Clinical Trials for the Fragile X Mental Retardation Syndrome: The Challenge of Judging Efficacy
    Vincent des Portes

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    Not recorded
    Symposium
    Novel Therapies for Monogenic Diseases
    Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot
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    The Discovery and Development of Olesoxime for Spinal Muscular Atrophy Therapy
    Rebecca Pruss

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    Not recorded
    Symposium
    Novel Therapies for Monogenic Diseases
    Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot
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    From Gene Identification to Functional Analysis and Treatment of Achondroplasia
    Laurence Legeai-Mallet

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    Not recorded
    Symposium
    Novel Therapies for Monogenic Diseases
    Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot
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    Inhibition of Thromboxane Synthase, a New Mechanism to Increase Bone Density?
    Valerie Cormier-Daire

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    Not recorded
    Symposium
    Novel Therapies for Monogenic Diseases
    Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot
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    Selected Oral Communications: Drug Screening on Hutchinson Gilford Progeria Using Pluripotent Stem Cells Reveals New Pharmacological Modulators of Prelamin a Farnesylation
    Xavier Nissan

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    Not recorded
    Symposium
    Novel Therapies for Monogenic Diseases
    Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot
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    Novel Approaches for Treatment of Retinal Diseases
    José-Alain Sahel

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    Not recorded
    Symposium
    Novel Therapies for Monogenic Diseases
    Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot
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    Gene Replacement Therapy for Myotubular Myopathy
    Anna Buj Bello

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    Not recorded
    Symposium
    Novel Therapies for Monogenic Diseases
    Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot
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    REPS1 Is a Novel Gene of Neurodegeneration with Brain Iron Accumulation
    Arnold Munnich

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    Not recorded
    Symposium
    Novel Therapies for Monogenic Diseases
    Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot
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    Efficient AAV Gene Therapy in Cardiac and Neurological Murine Models for Friedreich Ataxia
    Hélène Puccio

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    Not recorded
    Symposium
    Novel Therapies for Monogenic Diseases
    Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot
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    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

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    Not recorded
    Symposium
    Novel Therapies for Monogenic Diseases
    Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot
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    Selected Oral Communication: Gene Therapy for Alzheimer's Disease: The Cholesterol Connection
    Nathalie Cartier

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    Not recorded
    Symposium
    Novel Therapies for Monogenic Diseases
    Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot
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    Round Table: Can We Afford Cost of Innovative Therapies for Rare Diseases?
    Franck Dufour et Serge Braun

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    Not recorded
    Symposium
    Novel Therapies for Monogenic Diseases
    Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot
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    See also

    Alain Fischer, chair Experimental medicine
    Jean-Louis Mandel, chair Human genetics