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