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