Document Detail


Malformation syndromes caused by disorders of cholesterol synthesis.
MedLine Citation:
PMID:  20929975     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
Cholesterol homeostasis is critical for normal growth and development. In addition to being a major membrane lipid, cholesterol has multiple biological functions. These roles include being a precursor molecule for the synthesis of steroid hormones, neuroactive steroids, oxysterols, and bile acids. Cholesterol is also essential for the proper maturation and signaling of hedgehog proteins, and thus cholesterol is critical for embryonic development. After birth, most tissues can obtain cholesterol from either endogenous synthesis or exogenous dietary sources, but prior to birth, the human fetal tissues are dependent on endogenous synthesis. Due to the blood-brain barrier, brain tissue cannot utilize dietary or peripherally produced cholesterol. Generally, inborn errors of cholesterol synthesis lead to both a deficiency of cholesterol and increased levels of potentially bioactive or toxic precursor sterols. Over the past couple of decades, a number of human malformation syndromes have been shown to be due to inborn errors of cholesterol synthesis. Herein, we will review clinical and basic science aspects of Smith-Lemli-Opitz syndrome, desmosterolosis, lathosterolosis, HEM dysplasia, X-linked dominant chondrodysplasia punctata, Congenital Hemidysplasia with Ichthyosiform erythroderma and Limb Defects Syndrome, sterol-C-4 methyloxidase-like deficiency, and Antley-Bixler syndrome.
Authors:
Forbes D Porter; Gail E Herman
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Publication Detail:
Type:  Journal Article; Research Support, N.I.H., Extramural; Research Support, N.I.H., Intramural; Review     Date:  2010-10-07
Journal Detail:
Title:  Journal of lipid research     Volume:  52     ISSN:  0022-2275     ISO Abbreviation:  J. Lipid Res.     Publication Date:  2011 Jan 
Date Detail:
Created Date:  2010-12-14     Completed Date:  2011-06-21     Revised Date:  2012-01-02    
Medline Journal Info:
Nlm Unique ID:  0376606     Medline TA:  J Lipid Res     Country:  United States    
Other Details:
Languages:  eng     Pagination:  6-34     Citation Subset:  IM    
Affiliation:
Program in Developmental Genetics and Endocrinology, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Department of Health and Human Services, Bethesda, MD 20892, USA. fdporter@mail.nih.gov
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MeSH Terms
Descriptor/Qualifier:
Abnormalities, Multiple / etiology,  genetics,  metabolism
Animals
Cholesterol / biosynthesis*
Chondrodysplasia Punctata / etiology,  genetics,  metabolism
Congenital Abnormalities / etiology*
Humans
Lipid Metabolism, Inborn Errors / complications*,  etiology,  genetics,  metabolism
Oxidoreductases Acting on CH-CH Group Donors / deficiency,  genetics,  metabolism
Smith-Lemli-Opitz Syndrome / etiology,  genetics,  metabolism
Steroid Metabolism, Inborn Errors
Syndrome
Grant Support
ID/Acronym/Agency:
HD38572/HD/NICHD NIH HHS
Chemical
Reg. No./Substance:
57-88-5/Cholesterol; EC 1.3.-/Oxidoreductases Acting on CH-CH Group Donors

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine


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