Gene therapy for mucopolysaccharidosis type VI is effective in cats without pre-existing immunity to AAV8 - Ferla, Rita; O'Malley, Thomas; Calcedo, Roberto; O'Donnell, Patricia; Wang, Ping; Cotugno, Gabriella; Claudiani, Pamela; Wilson, James M; Haskins, Mark; Auricchio, Alberto (Human gene therapy, 2013)
Keywords: Animals; Antibodies, Neutralizing - immunology; Antibodies, Viral - immunology; Cats; Dependovirus - genetics; Dependovirus - immunology; Dose-Response Relationship, Drug; Enzyme Activation; Femur - anatomy & histology; Gene Transfer Techniques; Genetic Therapy - methods; Genetic Vectors - administration & dosage; Glycosaminoglycans - urine; Green Fluorescent Proteins - metabolism; Liver - enzymology; Mucopolysaccharidosis VI - immunology; Mucopolysaccharidosis VI - therapy; N-Acetylgalactosamine-4-Sulfatase - blood; N-Acetylgalactosamine-4-Sulfatase - genetics; Phenotype