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