Document Detail


White matter lesions in spastic paraplegia with mutations in SPG5/CYP7B1.
MedLine Citation:
PMID:  19187859     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
Hereditary spastic paraplegias (HSPs) are relatively frequent disorders presenting great genetic heterogeneity. The recent identification of mutations in SPG5/CYP7B1 in six autosomal recessive kindred linked to the SPG5 locus on chromosome 8q prompted us to test the relative frequency of SPG5/CYP7B1 variants in 12 families and in sporadic HSP patients by high-resolution melting screening combined with direct sequencing. We present two patients who harbored three mutations (including two novel variants) in SPG5/CYP7B1 and white matter involvement evidenced at brain MRI. In HSP patients in whom no other genes were mutated, screening of SPG5/CYP7B1 seems to have a low diagnostic yield in autosomal recessive (8%) and sporadic (<1%) cases, even in those with complicated clinical features.
Authors:
Roberta Biancheri; Marianna Ciccolella; Andrea Rossi; Alessandra Tessa; Denise Cassandrini; Carlo Minetti; Filippo M Santorelli
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Publication Detail:
Type:  Case Reports; Journal Article; Research Support, Non-U.S. Gov't    
Journal Detail:
Title:  Neuromuscular disorders : NMD     Volume:  19     ISSN:  0960-8966     ISO Abbreviation:  Neuromuscul. Disord.     Publication Date:  2009 Jan 
Date Detail:
Created Date:  2009-02-03     Completed Date:  2009-05-20     Revised Date:  2011-11-24    
Medline Journal Info:
Nlm Unique ID:  9111470     Medline TA:  Neuromuscul Disord     Country:  England    
Other Details:
Languages:  eng     Pagination:  62-5     Citation Subset:  IM    
Affiliation:
IRCCS G. Gaslini Pediatric Institute and University of Genoa, Unit of Muscular and Neurodegenerative Diseases, IRCCS Bambino Gesù Children's Hospital, Piazza S. Onofrio 4, 00165 Rome, Italy.
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MeSH Terms
Descriptor/Qualifier:
Adolescent
Adult
Brain / metabolism,  pathology*,  physiopathology
Child
Child, Preschool
Chromosomes, Human, Pair 8 / genetics
DNA Mutational Analysis
Female
Genetic Predisposition to Disease / genetics
Genetic Testing
Humans
Magnetic Resonance Imaging
Male
Mutation / genetics*
Nerve Fibers, Myelinated / metabolism,  pathology*
Pedigree
Predictive Value of Tests
Spastic Paraplegia, Hereditary / genetics*,  pathology*,  physiopathology
Steroid Hydroxylases / genetics*
Young Adult
Grant Support
ID/Acronym/Agency:
GGP06188//Telethon
Chemical
Reg. No./Substance:
EC 1.14.-/Steroid Hydroxylases; EC 1.14.13.-/CYP7B1 protein, human
Comments/Corrections
Comment In:
Neuromuscul Disord. 2009 Jul;19(7):507; author reply 507-8   [PMID:  19556130 ]

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


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