Cobblestone lissencephaly: neuropathological subtypes and correlations with genes of dystroglycanopathies.

Details

Serval ID
serval:BIB_4F1C02702D4B
Type
Article: article from journal or magazin.
Collection
Publications
Title
Cobblestone lissencephaly: neuropathological subtypes and correlations with genes of dystroglycanopathies.
Journal
Brain : A Journal of Neurology
Author(s)
Devisme L., Bouchet C., Gonzalès M., Alanio E., Bazin A., Bessières B., Bigi N., Blanchet P., Bonneau D., Bonnières M., Bucourt M., Carles D., Clarisse B., Delahaye S., Fallet-Bianco C., Figarella-Branger D., Gaillard D., Gasser B., Delezoide A.L., Guimiot F., Joubert M., Laurent N., Laquerrière A., Liprandi A., Loget P., Marcorelles P., Martinovic J., Menez F., Patrier S., Pelluard F., Perez M.J., Rouleau C., Triau S., Attié-Bitach T., Vuillaumier-Barrot S., Seta N., Encha-Razavi F.
ISSN
1460-2156 (Electronic)
ISSN-L
0006-8950
Publication state
Published
Issued date
2012
Volume
135
Number
Pt 2
Pages
469-482
Language
english
Abstract
Cobblestone lissencephaly represents a peculiar brain malformation with characteristic radiological anomalies, defined as cortical dysplasia combined with dysmyelination, dysplastic cerebellum with cysts and brainstem hypoplasia. Cortical dysplasia results from neuroglial overmigration into the arachnoid space, forming an extracortical layer, responsible for agyria and/or 'cobblestone' brain surface and ventricular enlargement. The underlying mechanism is a disruption of the glia limitans, the outermost layer of the brain. Cobblestone lissencephaly is pathognomonic of a continuum of autosomal recessive diseases with cerebral, ocular and muscular deficits, Walker-Warburg syndrome, muscle-eye-brain and Fukuyama muscular dystrophy. Mutations in POMT1, POMT2, POMGNT1, LARGE, FKTN and FKRP genes attributed these diseases to α-dystroglycanopathies. However, studies have not been able to identify causal mutations in the majority of patients and to establish a clear phenotype/genotype correlation. Therefore, we decided to perform a detailed neuropathological survey and molecular screenings in 65 foetal cases selected on the basis of histopathological criteria. After sequencing the six genes of α-dystroglycanopathies, a causal mutation was observed in 66% of cases. On the basis of a ratio of severity, three subtypes clearly emerged. The most severe, which we called cobblestone lissencephaly A, was linked to mutations in POMT1 (34%), POMT2 (8%) and FKRP (1.5%). The least severe, cobblestone lissencephaly C, was linked to POMGNT1 mutations (18%). An intermediary type, cobblestone lissencephaly B, was linked to LARGE mutations (4.5%) identified for the first time in foetuses. We conclude that cobblestone lissencephaly encompasses three distinct subtypes of cortical malformations with different degrees of neuroglial ectopia into the arachnoid space and cortical plate disorganization regardless of gestational age. In the cerebellum, histopathological changes support the novel hypothesis that abnormal lamination arises from a deficiency in granule cells. Our studies demonstrate the positive impact of histoneuropathology on the identification of α-dystroglycanopathies found in 66% of cases, while with neuroimaging criteria and biological values, mutations are found in 32-50% of patients. Interestingly, our morphological classification was central in the orientation of genetic screening of POMT1, POMT2, POMGNT1, LARGE and FKRP. Despite intensive research, one-third of our cases remained unexplained; suggesting that other genes and/or pathways may be involved. This material offers a rich resource for studies on the affected neurodevelopmental processes of cobblestone lissencephaly and on the identification of other responsible gene(s)/pathway(s).
Keywords
Brain/metabolism, Brain/pathology, Cobblestone Lissencephaly/genetics, Cobblestone Lissencephaly/metabolism, Dystroglycans/genetics, Dystroglycans/metabolism, Female, Fetus, Humans, Infant, Newborn, Male, Mannosyltransferases/genetics, Mannosyltransferases/metabolism, Membrane Proteins/genetics, Membrane Proteins/metabolism, N-Acetylglucosaminyltransferases/genetics, N-Acetylglucosaminyltransferases/metabolism, Proteins/genetics, Proteins/metabolism
Pubmed
Open Access
Yes
Create date
14/06/2012 11:33
Last modification date
20/08/2019 15:04
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