Bone marrow transplantation correcting β-galactosidase activity does not influence neurological outcome in juvenile GM1-gangliosidosis

Journal of Inherited Metabolic Disease

Volumn 28, Issue 5, 2005, Pages 797-798

  Shield, J P H ^a   Stone, J ^a   Steward, C G ^a  

^a BRISTOL ROYAL HOSPITAL FOR CHILDREN   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

BETA GALACTOSIDASE;

ARTICLE; BONE MARROW TRANSPLANTATION; CASE REPORT; CELL DIFFERENTIATION; DEGENERATIVE DISEASE; DEMYELINATION; ELECTROENCEPHALOGRAPHY; ENZYME ACTIVITY; FEMALE; GM1 GANGLIOSIDOSIS; HLA MATCHING; HUMAN; MOTOR DEVELOPMENT; MYELIN DEFICIENCY; NEUROIMAGING; NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; SCHOOL CHILD;

ANIMALS; BETA-GALACTOSIDASE; BONE MARROW CELLS; BONE MARROW TRANSPLANTATION; BRAIN; CHILD; DISEASE MODELS, ANIMAL; DOGS; FEMALE; GANGLIOSIDOSIS, GM1; HUMANS; INFANT; MAGNETIC RESONANCE IMAGING; TREATMENT OUTCOME;

EID: 25144488143     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-005-0089-7     Document Type: Article

References (3)

1. 1 gangliosidosis. Clin Genet 38: 274-280. 2125250
2. Peters C, Shapiro EG, Anderson J, et al (1998) Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. Blood 91: 2601-2608. 9516162
3. Simard AR, Rivest S (2004) Bone marrow stem cells have the ability to populate the entire CNS into fully differentiated parenchymal microglia. FASEB J 18: 998-1000. 15084516