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