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