Manufacturing CRISPR-Cas9 DNA damage response Next generation sequencing Dose study Analytical ultracentrifugation Animal model Chemistry Vector Dmd zebrafish Calcium Dystrophin Crigler–Najjar Syndrome Cell therapy Adenoassociated viral vector Chimie des sucres Treg Bioconjuguaison DMDmdx rat Dynamin 2 Duchenne Bioconjugates Childhood blindness Capsid engineering Bioorthogonality Air tamponade Capsid thermal stability AAV vectors Retina Clinical trial DMD mdx rat Capsid Assembly-activating protein Cell–virus interaction ABCA4 AAP Antibodies/immunology/metabolism Immune response AUC Capsid assembly Mice Autophagy Gene therapy Adeno-associated virus Skeletal muscle Inflammation Cellular immune response Immunosuppression Bioconjugaison Ciblage Baculovirus expression vector Cornea Tolerance Quality control Cerebellum Clinical trials Canaux transient receptor potential DMD Eye surgery Age CMC Bioconjugation Beta-subunit Adeno-associated vector Adenoviridae/immunology Click chemistry B-Lymphocytes/immunology/metabolism Exercise Bioorthogonal click chemistry Antibody responses CNS Cone photoreceptors DNA double-strand breaks Ectopic expression Acute treatment Fluid-air exchange Alpha-1 antitrypsin deficiency AAV vector Autophagie Discriminant analysis Exon skipping Animal models for DMD Insect cells Carbohydrate Chemistry AAV Capsid thermostability CD8 + T lymphocytes Accelerometry Centronuclear myopathy Baculovirus Targeting AAV gene therapy Anti-AAV T cells and anti-AAV IgG Tyrosine Biodistribution Duchenne muscular dystrophy AAV3B Congenital amaurosis Animals Engineering