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Combined use of clinical and MRI Keywords angiogenesis; CCM1; CCM2; CCM3; cerebral cavernous malformations; cerebral hemorrhage; KRIT1; PDCD10; stroke; vascular malformations Corresponde

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Recent insights into cerebral cavernous malformations: the molecular genetics of CCM

Florence Riant1,2, Francoise Bergametti2, Xavier Ayrignac2, Gwenola Boulday2and Elisabeth

Tournier-Lasserve1,2

1 AP-HP, Hoˆpital Lariboisie`re, Laboratoire de Ge´ne´tique, Paris, France

2 INSERM UMR-S 740, Universite´ Paris 7 Diderot, France

Introduction

Cerebral cavernous malformations (CCM⁄ OMIM

116860) are vascular lesions histologically

character-ized by abnormally enlarged capillary cavities

with-out intervening brain parenchyma From large series

based on necropsy and⁄ or magnetic resonance

imag-ing (MRI) studies, their prevalence in the general

population has been estimated to be close to 0.1–

0.5% Most CCMs are located within the central

nervous system but they sometimes affect either the

retina or the skin [1]

CCM occur both sporadically and in a familial

context The proportion of familial cases has been

esti-mated to be as high as 50% in Hispano-American

CCM patients [2] and close to 10–40% in Caucasian

patients [1] The CCM pattern of inheritance is

autosomal dominant with incomplete clinical and neuroradiological penetrance The presence of multiple lesions on cerebral MRI is one of the main features of familial CCM which is an evolutive condition as assessed by the strong correlation between patient age and the number of lesions (Fig 1) [1–3] The average age-of-onset is around 30 years but symptoms can start in early infancy or in old age The main symptoms include seizures and cerebral hemorrhages Sporadic cases most often have a single lesion on MRI, are not inherited and do not carry a CCM gene germline mutation However, some CCM patients who have multiple MRI lesions do not have any known clinically affected relative and therefore present as sporadic cases Combined use of clinical and MRI

Keywords

angiogenesis; CCM1; CCM2; CCM3;

cerebral cavernous malformations; cerebral

hemorrhage; KRIT1; PDCD10; stroke;

vascular malformations

Correspondence

E Tournier-Lasserve, INSERM UMR-S 740;

Universite´ Paris7 Diderot, 10 Avenue du

Verdun, 75010 Paris, France

Fax: +33 157278594

Tel: +33 157278593

E-mail: tournier-lasserve@univ-paris-diderot.fr

(Received 1 August 2009, revised 4

November 2009, accepted 4 December

2009)

doi:10.1111/j.1742-4658.2009.07535.x

Cerebral cavernous malformations (CCM) are vascular lesions which can occur as a sporadic (80% of the cases) or familial autosomal dominant form (20%) Three CCM genes have been identified: CCM1⁄ KRIT1, CCM2⁄ MGC4607 and CCM3 ⁄ PDCD10 Almost 80% of CCM patients affected with a genetic form of the disease harbor a heterozygous germline mutation in one of these three genes Recent work has shown that a two-hit mechanism is involved in CCM pathogenesis which is caused by a com-plete loss of any of the three CCM proteins within endothelial cells lining the cavernous capillary cavities These data were an important step towards the elucidation of the mechanisms of this condition

Abbreviations

CCM, cerebral cavernous malformations; MRI, magnetic resonance imaging; PDCD10, Programmed Cell Death 10.

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screening with molecular testing has helped to clarify

what might have first seemed confusing [1]

Three CCM genes have been mapped and identified

in the recent years These molecular genetics data have

provided useful information for clinical care of the

patients and were an important step towards the

understanding of the mechanisms of this disorder This

minireview summarizes the advances in CCM

molecu-lar genetics and the remaining gaps in this field In

addition to the identification of CCM genes, a number

of recent biochemical in vitro studies and in vivo CCM

animal model studies have helped to unravel the

func-tional roles of these proteins and are be the focus of

the two accompanying minireviews by Faurobert and

Albiges-Rizo [4] and Chan et al [5]

CCM genes germline mutations Genetic linkage analyses mapped three CCM loci to chromosome 7q (CCM1), 7p (CCM2) and 3q (CCM3) [6,7] A strong founder effect has been oberved in His-pano-American CCM patients with most families linked to the CCM1 locus [8] In Caucasian families, the proportions of families linked to each CCM locus were 40% (CCM1), 20% (CCM2) and 40% (CCM3) [7] The three genes located at these loci have now been identified (Fig 2) [9–13]

The CCM1 gene contains 16 coding exons which encode for Krit1, a 736-amino acid protein containing three ankyrin domains and one band 4.1 ezrin radixin moesin (FERM) domain CCM2, a 10-exon gene, encodes for the MGC4607 protein, also called malc-avernin, which contains a phosphotyrosin binding domain CCM3 includes seven exons which encode for Programmed Cell Death 10 (PDCD10), a protein with-out any known conserved functional domain, which may be involved in apoptosis Considerable progress has been made recently in understanding the biochemi-cal pathways in which those proteins might be involved (see Faurobert and Albiges-Rizo [4])

More than 150 distinct CCM1⁄ CCM2 ⁄ CCM3 germ-line mutations have been published to date [9–23] Those mutations were highly stereotyped because almost all led to a premature termination codon through different mechanisms including nonsense, splice-site and frameshift mutational events, as well as large genomic rearrangements These data strongly suggest that a loss of function, through mRNA decay

of the mutated allele, is the most likely pathophysio-logical mechanism involved in CCM patients Only four ‘missense’ mutations within CCM1 have been reported to date; interestingly, all of them actually activated cryptic splice sites and led to an aberrant splicing of CCM1 mRNA and a frameshift with a

Fig 1 Cerebral magnetic resonance imaging of a 4-year-old familial

CCM patient Multiple CCM lesions are shown (arrowheads) as

well as a cerebral hemorrhage (arrow).

Fig 2 CCM loci and genes Three CCM

genes have been mapped and identified to

date, CCM1 ⁄ KRIT1, CCM2 ⁄ MGC4607 and

CCM3 ⁄ PDCD10 In 22% of CCM cases

with multiple lesions, no mutation is

detected in these three genes using

currently available technologies.

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premature stop codon [10,24] The only known

mis-sense mutation which did not affect splicing has been

located within the C-terminal part of the

phosphotyro-sin binding domain of CCM2 [12] This mutation has

been shown to abolish the interaction of CCM2 and

CCM1, strongly suggesting its causality [25] Only four

inframe deletions have been reported: two affect

ex-ons 17 and 18 of CCM1, one deletes exon 2 of CCM2

and one deletes exon 5 of CCM3 The last two

dele-tions have been used to map potential relevant

interac-tion domains of CCM2 and CCM3 [23,26] However,

it is not known if these putative truncated proteins

were indeed produced and stable in vivo

Sequencing of all coding exons of the three CCM

genes and search for genomic rearrangements using

cDNA and⁄ or quantitative multiplex PCR such as

multiplex ligation-dependent probe amplification in

Caucasian non-Hispano-American CCM multiplex

families led to the identification of the causative

muta-tion in 95% of the families [23,27] Approximately

72% of multiplex families harbored a mutation in

CCM1, 18% in CCM2 and 10% in CCM3 The

CCM3 proportion was much lower than expected,

based on previous linkage data which suggested that

40% of CCM families were linked to the CCM3 locus

The mutation detection rate was lower in sporadic

cases with multiple lesions, ranging from 45% to 67%

[22,23,27] Most of these sporadic cases with multiple

lesions had either inherited their mutation from one of

their asymptomatic parents because of incomplete

pen-etrance or had a de novo mutation Sporadic cases with

multiple lesions in whom no mutation was detected are

nevertheless most likely affected by a genetic form of

the disease Several hypotheses may be raised to

explain the absence of any detected mutation,

includ-ing a somatic mosaicism of a de novo mutation which

occured during gestation and is not detectable in DNA

extracted from peripheral blood cells It will be

impor-tant to solve this in the future because it is of interest

for genetic counseling [1] With regard to sporadic

CCM cases with a unique lesion on cerebral MRI, no

mutation was detected in reported series [16,17]

Com-bination of these data with those obtained in familial

CCM strongly suggests that sporadic cases with a

unique lesion who would harbor a germline mutation

are most likely very rare

Haplotyping data strongly suggested a founder effect

in the Hispano-American CCM population; this was

confirmed by the detection of a Q455X stop codon

mutation in CCM1 in most families with this ethnic

background [10] Recurrent mutations have also been

identified in a few additional populations [21,28]

How-ever, in most cases, despite their highly stereotyped

consequences, germline CCM mutations are ‘private’ mutations present in only one or very few families

Biallelic somatic and germline mutations in CCM lesions Based on the autosomal dominant pattern of inheri-tance of CCM and the presence of multiple lesions in familial CCM, contrasting with the detection of a sin-gle lesion in nonhereditary cavernous angiomas, it has been proposed that a second hit affecting the wild-type allele might be involved in CCM lesions pathophysiol-ogy, as reported previously in retinoblastoma or other vascular malformations [29,30] According to this hypothesis, CCM formation would be caused by a complete loss, within affected cells, of the two alleles

of a given CCM gene Loss of one of the alleles (first hit) would be the result of a germline mutation and loss of the second allele (second hit) will occur somati-cally

This hypothesis is not easy to test because of the heterogeneous cellular nature of CCM lesions and the very limited number of endothelial cells lining the cap-illary cavities Indeed, direct sequencing of the DNA extracted from a heterogeneous lesion may not detect the mutation depending of the proportion of the cells which harbor this mutation within the lesion This approach was initially used to screen CCM lesions from both sporadic and a few familial patients and did not detect any somatic mutation except in one spo-radic case [31] In this latter case, two CCM1 missense mutations, F97S and K569E, were detected in the CCM lesion and were shown to be absent in the blood

of the patient However, the data were difficult to interprete because of the nature of the mutations which were not truncating mutations (a possible aber-rant splicing effect of these two mutations was not investigated) and the fact that the biallelism of these mutations was not explored

In 2005, Gault et al reported the first biallelic CCM1 germline and somatic truncating mutation in a CCM lesion, strongly supporting this ‘two-hit’ mecha-nism in the formation of lesions, at least in CCM1 patients; they demonstrated recently that this second hit occurred within the endothelial cells [32,33]

Biallelic somatic and germline mutations in each of the three CCM genes were recently reported by Akers

et al [34] These authors amplified and sequenced a large number of clones from 10 CCM lesions resected from patients harboring a heterozygous germline muta-tion in either CCM1 (two patients), CCM2 (five patients) and CCM3 (two patients) One CCM lesion was analyzed for each patient They were able to

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convincingly establish the presence of a biallelic

somatic and germline deleterious mutation in four of

these lesions from two CCM1 patients, one CCM2

patient and one CCM3 patient The proportion of

amplicons carrying the somatic mutation ranged from

4% to 16% None of these mutations was detected

through direct sequencing of lesion DNA, emphasizing

the lack of sensitivity of direct sequencing of lesion

DNA These data established the existence of biallelic

somatic and germline mutations, whatever the nature

of the CCM gene involved, at least in some lesions

No mutation was detected in the six remaining lesions

Several hypotheses may be raised to explain this

absence of mutation including the incomplete

sensitiv-ity of this type of approach which would miss a second

hit consisting in either large genomic deletions and⁄ or

epigenetic silencing mechanisms

Interestingly, the authors showed, using laser

cap-ture, that the somatic mutation occurred in endothelial

cells and not in the intervening neural tissue The

pro-portion of endothelial cells which harbor the somatic

mutation was estimated in one lesion and shown to be

close to 30%, suggesting the mosaicism of this somatic

mutation These data are in agreement with those

obtained very recently with an

immunohistochemistry-based approach which showed a mosaic loss of

expres-sion of CCM proteins in endothelial cells lining CCM

caverns [35] This question would, however, require

additional investigations It would also be important

to analyze several lesions from a given patient to test

for the presence of the same mutation in multiple

lesions A unique somatic mutation has indeed been

detected in multifocal lesions in another hereditary

vascular condition suggesting a common origin for

abnormal endothelial cells lying in distant sites [36]

Altogether these data strongly suggest that CCM, as

several other hereditary vascular conditions, show a

paradominant inheritance It remains to determine

when do occur the somatic, second hit, events

Are there additional CCM genes?

Previous linkage data obtained on 20 large North

American families suggested that the three CCM loci

on 7p, 7q and 3q would most likely account for all

CCM families [7] However, despite extensive screening

of exonic sequences for point mutations and deletions,

no mutation was detected in 5% of familial CCM

cases and a larger proportion of sporadic cases wth

multiple lesions [27] In addition, the proportion of

families showing a mutation within PDCD10 (10%) at

the CCM3 locus on chromosome 3q25, was much

lower than expected based on linkage data (40%)

Several mutually nonexclusive hypotheses may explain these data such as: (a) the existence of muta-tions affecting cis-regulatory elements located at long distances from known CCM transcription units; (b) epigenetic silencing of these three genes; and (c) the existence of additional nonidentified CCM genes, one

of which is possibly located close to PDCD10

Recently, an additional gene, Zona Pellucida-like Domain containing 1(ZPLD1), has been reported to be disrupted in a CCM patient harboring a balanced translocation between chromosome X and chromo-some 3q [37] ZPLD1 is located on chromochromo-some three centromeric to PDCD10 The expression of the mRNA

in lymphoblastoid cell lines of the patient was shown

to be significantly decreased suggesting that the inter-ruption of this gene may be causal However, the same authors screened this gene in 20 additional CCM patients without any mutation in CCM1⁄ CCM2 ⁄ CCM3 and did not detect any mutation These data suggest that either this gene is involved in very rare CCM patients or its interruption does not cause CCM but that the translocation present in this patient deregulated the expression of a gene unidentified yet Additional work currently conducted in several teams should help in the next future to identify the molecular anomalies of CCM patients ‘without’ mutations

Conclusions and future The recent identification of the three CCM genes is an important step towards the elucidation of the mecha-nisms of this condition It helped to clarify several fea-tures of this condition including its incomplete clinical and MRI penetrance as well as the molecular basis of sporadic cases with multiple lesions Additional large series studies are needed to evaluate genotype–pheno-type correlations (particularly the prognosis) depending

of the nature of the mutated gene Several additional questions, however, have to be adressed What is the nature of the molecular anomaly in familial CCM cases in whom no mutation has been detected? Are sporadic cases CCM patients with multiple lesions showing a mosaicism for a germline mutation? Are there modifying genes that may explain the intrafamil-ial clinical variability? In addition to these questions, one main challenge is to understand the mechanisms

of this condition The recent identification of several of the biochemical pathways involving CCM proteins as well as the analysis of several fish and mouse CCM animal models has already provided a number of clues

to this goal (see Faurobert and Albiges-Rizo [4] and Chan et al [5])

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