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