Novel Therapies for Monogenic Diseases

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

Gene Therapy of Primary Immunodeficiencies (PID)

Genetic Engineering of Human Hematopoiesis for the Treatment of Inherited Diseases and Cancer

Gene Therapy of Hemophilias

New Mutation-Specific Pharmacotherapies for Cystic Fibrosis

Added Value of New Genomics Technologies in Undiagnosed Patients Who May Benefit from Known Therapies

From Promising Clinical Studies to Recent Clinical Trials for the Fragile X Mental Retardation Syndrome: The Challenge of Judging Efficacy

The Discovery and Development of Olesoxime for Spinal Muscular Atrophy Therapy

From Gene Identification to Functional Analysis and Treatment of Achondroplasia

Inhibition of Thromboxane Synthase, a New Mechanism to Increase Bone Density?

Selected oral communications: Drug Screening on Hutchinson Gilford Progeria Using Pluripotent Stem Cells Reveals New Pharmacological Modulators of Prelamin a Farnesylation

Novel Approaches for Treatment of Retinal Diseases

Gene Replacement Therapy for Myotubular Myopathy

REPS1 Is a Novel Gene of Neurodegeneration with Brain Iron Accumulation

Efficient AAV Gene Therapy in Cardiac and Neurological Murine Models for Friedreich Ataxia

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

Selected oral communication: Gene Therapy for Alzheimer's Disease: The Cholesterol Connection

Round table: Can We Afford Cost of Innovative Therapies for Rare Diseases?

Conclusions