Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human α-l-iduronidase gene

This publication has 46 references indexed in Scilit:

• Gene Transfer Strategies for Correction of Lysosomal Storage Disorders
  Acta Haematologica, 2003
• Gene Therapy with Virus Vectors for specific Disease of the Nervous System
  Published by Elsevier ,2003
• Murine Mucopolysaccharidosis VII: Impact of Therapies on the Phenotype, Clinical Course, and Pathology in a Model of a Lysosomal Storage Disease
  Pediatric and Developmental Pathology, 2001
• Enzyme Replacement Therapy in Feline Mucopolysaccharidosis I
  Molecular Genetics and Metabolism, 2001
• Hurler Syndrome: II. Outcome of HLA-Genotypically Identical Sibling and HLA-Haploidentical Related Donor Bone Marrow Transplantation in Fifty-Four Children
  Blood, 1998
• Long-Term and High-Dose Trials of Enzyme Replacement Therapy in the Canine Model of Mucopolysaccharidosis I
  Biochemical and Molecular Medicine, 1996
• Molecular genetics of muccpolysaccharidosis type I: Diagnostic, clinical, and biological implications
  Human Mutation, 1995
• Long‐term outcome of Hurler syndrome following bone marrow transplantation
  American Journal of Medical Genetics, 1993
• REVERSAL OF CLINICAL FEATURES OF HURLER'S DISEASE AND BIOCHEMICAL IMPROVEMENT AFTER TREATMENT BY BONE-MARROW TRANSPLANTATION
  The Lancet, 1981
• Hurler and Hunter Syndromes: Mutual Correction of the Defect in Cultured Fibroblasts
  Science, 1968