Animals Discriminant analysis Engineering Gene therapy Cell–virus interaction Exon skipping Accelerometry Animal model Analytical ultracentrifugation Air tamponade Duchenne DMD Human-monocyte-derived dendritic cells Cone photoreceptors Intravitreal Congenital amaurosis Centronuclear myopathy Capsid Childhood blindness Innate immunity GC-content Cell therapy Click chemistry Age Identity Tolerance Calcium Genes Skeletal muscle ABCA4 High-throughput sequencing Capsid assembly AAV vectors DMDmdx rat CMC Impurities Capsid thermostability Clinical trials Treg Exercise Fluid-air exchange Ectopic expression Baculovirus Immune responses In vivo GRMD Immunomodulation Clinical trial Infectivity Immunogenicity Acute treatment Intrathymic gene transfer Homopolymers Capsid thermal stability Immunogenicity inflammation gene therapy AAV Gene Therapy Gene-therapy High-throughput technology GRMD dog Beta-subunit Gene transfer Humoral immunity Eye surgery Homologous recombination Insect cells Antibodies/immunology/metabolism Inflammation Inherited retinal dystrophies Mice Quality control Cerebellum Dystrophin Antibody responses Intra-cerebrospinal fluid injection AAV vector Manufacturing Crigler–Najjar Syndrome Adenoassociated viral vector Adeno-associated virus Identity assay Vector Dose study Gene Transfer Adenoviridae/immunology CNS AAV Bioconjugates Assembly-activating protein Duchenne muscular dystrophy AUC CD8 + T lymphocytes Dynamin 2 Gait assessment AAP Homogeneity Immune response Baculovirus expression vector Immunosuppression Bioorthogonality B-Lymphocytes/immunology/metabolism