Document Detail


Genetic heterogeneity in severe congenital neutropenia: how many aberrant pathways can kill a neutrophil?
MedLine Citation:
PMID:  17989524     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
PURPOSE OF REVIEW: Severe congenital neutropenia is a primary immunodeficiency in which lack of neutrophils causes inadequate innate immune host response to bacterial infections. Severe congenital neutropenia occurs with sporadic, autosomal dominant, autosomal recessive and X-linked recessive inheritance, as well as in a variety of multisystem syndromes. A principal stimulus for this review is the identification of novel genetic defects and pathophysiological insights into the role of neutrophil apoptosis.
RECENT FINDINGS: The recent findings include identification of mutations in HAX1 in autosomal recessive severe congenital neutropenia (Kostmann disease), a large epidemiological study estimating the risk of progression from severe congenital neutropenia to leukemia, a better understanding of how heterozygous mutations in neutrophil elastase (ELA2) cause severe congenital neutropenia, molecular characterization of a novel syndromic form of severe congenital neutropenia called p14 deficiency and new animal models for several syndromic forms of severe congenital neutropenia.
SUMMARY: We consider the numerous genes mutated in severe congenital neutropenia, the many attempts to make animal models of severe congenital neutropenia, and the results from both human and mouse studies investigating the molecular mechanisms of neutrophil apoptosis. Investigations of how severe congenital neutropenia genes and apoptosis pathways are connected should lead to a better understanding of the pathogenesis of neutropenia and apoptosis pathways relevant to many cell types.
Authors:
Alejandro A Schäffer; Christoph Klein
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Publication Detail:
Type:  Journal Article; Research Support, N.I.H., Intramural; Research Support, Non-U.S. Gov't; Review    
Journal Detail:
Title:  Current opinion in allergy and clinical immunology     Volume:  7     ISSN:  1528-4050     ISO Abbreviation:  Curr Opin Allergy Clin Immunol     Publication Date:  2007 Dec 
Date Detail:
Created Date:  2007-11-08     Completed Date:  2008-03-06     Revised Date:  2011-09-26    
Medline Journal Info:
Nlm Unique ID:  100936359     Medline TA:  Curr Opin Allergy Clin Immunol     Country:  United States    
Other Details:
Languages:  eng     Pagination:  481-94     Citation Subset:  IM    
Affiliation:
Computational Biology Branch, National Center for Biotechnology Information, National Institutes of Health, Department of Health and Human Services, Bethesda, Maryland, USA.
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MeSH Terms
Descriptor/Qualifier:
Adaptor Proteins, Signal Transducing
Animals
Apoptosis / genetics,  immunology*
Disease Models, Animal
Humans
Neutropenia / congenital*,  genetics*,  immunology
Neutrophils / immunology*
Pancreatic Elastase / genetics,  immunology
Proteins / genetics,  immunology
Grant Support
ID/Acronym/Agency:
Z01 LM000097-06/LM/NLM NIH HHS; Z99 LM999999/LM/NLM NIH HHS
Chemical
Reg. No./Substance:
0/Adaptor Proteins, Signal Transducing; 0/HAX1 protein, human; 0/Proteins; EC 3.4.21.36/Pancreatic Elastase
Comments/Corrections

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