Pathogenetic mechanisms in peroxisomal disorders

Current Opinion in Neurology

Volumn 9, Issue 6, 1996, Pages 473-476

  Moser, Hugo W ^a  

^a JOHNS HOPKINS UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADRENOLEUKODYSTROPHY; BIOGENESIS; DIET THERAPY; DISORDERS OF PEROXISOMAL FUNCTIONS; GENE MUTATION; GENETIC ANALYSIS; HUMAN; PATHOGENESIS; PEROXISOME; SHORT SURVEY; X CHROMOSOME LINKAGE;

EID: 0030467749     PISSN: 13507540     EISSN: None     Source Type: Journal    
DOI: 10.1097/00019052-199612000-00014     Document Type: Short Survey

References (41)

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37. Ho JK, Moser H, Kishimoto Y, Hamilton JA: Interaction of very long chain fatty acid with model membranes and serum albumin: implication for the pathogenesis of adrenoleukodystrophy. Clin Invest 1995, 96:1455-1463 A demonstration of possible pathogenetic mechanisms in X-ALD. Very long chain fatty acids that accumulate in X-ALD disrupt the membrane structure in model membranes and their rate of transfer to albumen is up to 10 000 times slower than the more common long chain fatty acids.
38. Moser HW, Borel J: Dietary therapy for adrenoleukodystrophy. Ann Rev Nutr 1995, 15:379-397.
39. Moser HW: Komrower lecture: adrenoleukodystrophy: natural history, treatment and outcome. J Inher Metab Dis 1995, 18:435-447.
40. Kickler TS, Zinkham WH, Moser A, Shankroff J, Borel J, Moser H: Effect of erucic acid on platelets in patients with adrenoleukodystrophy. Biochem Mol Med 1995, 57:125-133.
41. Krivit W, Lockman LA, Watkins PAS, Hirsch J, Shapiro EG: The future for treatment by bone marrow transplantation for adrenoleukodystrophy, metachromatic leukodystrophy, globoid leukodystrophy and Hurler syndrome. J Inher Metab Dis 1995, 18:398-412. An update on a promising therapeutic approach. Strategies to reduce morbidity are being developed but selection of patients, details of management, and evaluation of results require additional study.