Leukodystrophy and gene therapy with a dimmer switch

New England Journal of Medicine

Volumn 364, Issue 6, 2011, Pages 572-573

  Orchard, Paul J ^a   Wagner, John E ^a  

^a UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DNA; GALACTOSYLCERAMIDASE; MICRORNA;

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ENZYME REPLACEMENT; GENE EXPRESSION REGULATION; GENE THERAPY; GLOBOID CELL LEUKODYSTROPHY; HUMAN; MICROGLIA; NONHUMAN; PATHOPHYSIOLOGY; PRIORITY JOURNAL; SHORT SURVEY;

EID: 79851502456     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMcibr1013082     Document Type: Short Survey

References (5)

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3. Krivit W, Shapiro EG, Peters C, et al. Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy. N Engl J Med 1998;338:1119-26. (Pubitemid 28227482)
4. Escolar ML, Poe MD, Provenzale JM, et al. Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease. N Engl J Med 2005;352:2069-81. (Pubitemid 40664308)
5. Krivit W, Sung JH, Shapiro EG, Lockman LA. Microglia: the effector cell for reconstitution of the central nervous system following bone marrow transplantation for lysosomal and peroxisomal storage diseases. Cell Transplant 1995;4:385-92.