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Báo cáo khoa học: SOX10, in combination with Sp1, regulates the endothelin receptor type B gene in human melanocyte lineage cells pptx

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Here, we provide evi-dence that SOX10 regulates the expression of EDNRB gene in human melanocyte-lineage cells, as judged by RNA interference and chromatin im-munoprecipitation analyses.

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receptor type B gene in human melanocyte lineage cells Satoru Yokoyama, Kazuhisa Takeda and Shigeki Shibahara

Department of Molecular Biology and Applied Physiology, Tohoku University School of Medicine, Seiryo-machi, Aoba-ku, Sendai, Miyagi, Japan

Waardenburg syndrome (WS) is an

auditory–pigmen-tary disorder, which is characterized by varying

combi-nations of sensorineural hearing loss, heterochromia

iridis, and patchy abnormal pigmentation of the hair

and skin [1] WS is associated with the deficiency of

neural crest-derived melanocytes, and is classified into

four types, depending on the presence or absence of

additional symptoms [2–9] WS type1 (WS1) and type

2 (WS2) are distinguished by the presence or absence

of dystopia canthorum, respectively The presence of

limb abnormalities distinguishes WS type 3 (WS3)

from WS2 WS type 4 (WS4), referred to as Hirsch-sprung’s disease type 2 or Shaa–Waardenburg syn-drome, is characterized by the presence of the aganglionic megacolon WS1 and WS3 are caused by mutations in the PAX3 gene [2], and some cases of WS2 are associated with mutations in the microphthal-mia-associated transcription factor (MITF) gene [3] or SLUG (SNAI2) gene [4] WS4 is due to mutations in the endothelin receptor type B (EDNRB) gene [5,6], the endothelin 3 (EDN3) gene [7,8], or the Sry-box 10 (SOX10) gene [9]

Keywords

endothelin receptor type B; melanocytes;

SOX10; Sp1; Waardenburg syndrome

Correspondence

K Takeda, Department of Molecular Biology

and Applied Physiology, Tohoku University

School of Medicine, 2–1 Seiryo-machi,

Aoba-ku, Sendai, Miyagi 980–8575, Japan

Fax: +81 22 7178118

Tel: +81 22 7178114

E-mail: ktakeda@mail.tains.tohoku.ac.jp

(Received 15 November 2005, revised 20

February 2006, accepted 27 February 2006)

doi:10.1111/j.1742-4658.2006.05200.x

Waardenburg syndrome (WS) is an auditory–pigmentary disorder that exhibits varying combinations of sensorineural hearing loss and abnormal pigmentation of the hair and skin WS type 4 (WS4), a subtype of WS, is characterized by the presence of the aganglionic megacolon and is associ-ated with mutations in the gene encoding either endothelin 3, endothelin receptor type B (EDNRB), or Sry-box 10 (SOX10) Here, we provide evi-dence that SOX10 regulates the expression of EDNRB gene in human melanocyte-lineage cells, as judged by RNA interference and chromatin im-munoprecipitation analyses Human melanocytes preferentially express the EDNRB transcripts derived from the conventional EDNRB promoter SOX10 transactivates the EDNRB promoter through the cis-acting ele-ments, the two CA-rich sequences and the GC box Moreover, a transcrip-tion factor Sp1 enhances the degree of the SOX10-mediated transactivatranscrip-tion

of the EDNRB promoter through these cis-acting elements Furthermore,

we have shown that the EDNRB promoter is heavily methylated in HeLa human cervical cancer cells, lacking EDNRB expression, but not in mel-anocytes and HMV-II melanoma cells The expression of EDNRB became detectable in HeLa cells after treatment with a demethylating reagent, 5¢-aza-2¢-deoxycytidine, which was further enhanced in the transformed cells over-expressing SOX10 We therefore suggest that SOX10, alone or in combination with Sp1, regulates transcription of the EDNRB gene, thereby ensuring appropriate expression level of EDNRB in human melanocytes

Abbreviations

ChIP, chromatin immunoprecipitaion; EDN, endothelin; EDNRA, endothelin receptor type A; EDNRB, endothelin receptor type B; EMSA, electrophoretic mobility shift assay(s); ENS, enteric nervous system; HMG, high mobility group; MITF, microphthalmia-associated

transcription factor; PAX3, paired box gene 3; siRNA, small interfering RNA; SOX10, Sry-box 10; WS, Waardenburg syndrome; YDBS, Yemenite deaf–blind hypopigmentation syndrome.

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neural tube and migrated along the dorsolateral

path-way [11,12] PAX3 affects the development of

melano-cytes in culture by regulating MITF expression [13]

Likewise, SOX10 activates the MITF gene promoter

[14–17]

Recently, Sox10 has been shown to regulate Ednrb

expression in the precursors of the enteric nervous

sys-tem (ENS) through the ENS enhancers, which contain

the Sox10-binding sites, in the mouse Ednrb gene [18]

In fact, Sox10 mRNA and Ednrb mRNA exhibit

over-lapping expression patterns in neural crest derivatives

in wild-type mice [19], whereas the Ednrb expression

is reduced in the dominant megacolon (Dom) mouse,

which carries the truncated mutation of Sox10 gene

[19] The Dom mouse represents a model for human

congenital megacolon [19,20]

The SOX genes encode transcription factors with a

high-mobility group box (HMG box) as a

DNA-bind-ing motif [21] SOX10 is defective in some cases of

WS4 [9,22,23] and in patients with Yemenite deaf–

blind hypopigmentation syndrome (YDBS) [24] YDBS

is a rare disorder characterized by severe early hearing

loss, microcornea and colobomata, and cutaneous

pig-mentation abnormalities These two syndromes exhibit

a remarkable difference in the phenotype of the enteric

nervous system; namely, aganglionic megacolon is

associated with WS4, but not with YDBS

The mutations in the EDNRB gene are associated

with WS4, which is inherited in a dominant [6] or

recessive [5] mode EDNRB belongs to a superfamily

of G protein-coupled receptors [25] Its ligand,

endo-thelin (EDN), is a highly potent vasoconstricting

pep-tide of 21 amino acid residues and consists of three

subtypes EDN1, EDN2, and EDN3 [26] EDNRB has

a high affinity for all three EDNs, whereas endothelin

receptor type A (EDNRA), a subtype of EDNR,

pos-sesses a higher affinity for EDN1 and EDN2 than

EDN3 [25,27,28] The Ednrb gene is expressed

postnat-aly in various tissues, including the myenteric plexus,

mucosal layer, ganglia, and blood vessels of the

sub-mucosa of the colon [29,30] Importantly, the

Edn3-Ednrb signaling is required for the terminal migration

of melanoblasts and the precursors of the ENS [31,32]

However, little is known about the regulatory

mechan-ism of Ednrb expression in melanocytes

We have investigated the hierarchy between SOX10

and EDNRB in melanocyte-lineage cells EDNRB

while the EDNRBD3-encoded protein contains addi-tional N-terminal amino acids (89 residues), followed

by the EDNRB protein [33] Here, we show that the conventional EDNRB mRNA is preferentially expressed in melanocytes, and that SOX10 activates the conventional EDNRB promoter, alone or in com-bination with a transcription factor Sp1 Furthermore, the EDNRB promoter is heavily methylated in HeLa human cervical cancer cells, which do not express the EDNRB gene, but not methylated in melanocytes Notably, enforced expression of SOX10 induces the expression of EDNRB in HeLa cells only when HeLa cells were treated with a demethylating reagent, 5¢-aza-2¢-deoxycytidine The present study suggests that SOX10 is responsible for appropriate expression of the EDNRBgene in human melanocyte-lineage cells

Results and discussion

SOX10 is required for the expression

of EDNRB gene

To investigate the expression profiles of SOX10 and EDNRB mRNAs in normal human epidermal melano-cytes (NHEM) and human melanoma cells, we carried out northern blot analysis (Fig 1A) SOX10 and EDNRB mRNAs are coexpressed in NHEM and four human melanoma cell lines EDNRB mRNAs were detected as two major bands of about 4300 and 1800 nucleotides, which are generated by the use of alternat-ive polyadenylation sites [34] To explore the hierarchy among SOX10, EDNRB, and MITF, we carried out the RNA interference analysis against SOX10 in HMV-II melanoma cells (Fig 1B) We initially con-firmed that the expression of SOX10 protein was reduced by the small interfering RNA (siRNA) against SOX10 (65% reduction), but not by the LacZ siRNA (Fig 1B) Because SOX10 has been known as a trans-activator for MITF gene [14–17], MITF could be used

as a positive control for the SOX10 siRNA The expression of MITF protein was reduced by the SOX10 siRNA (69% reduction), but not changed by the LacZ siRNA These results confirm the regulatory role of SOX10 in the expression of MITF, thereby indicating that the SOX10 siRNA worked properly in HMV-II cells Likewise, EDNRB protein was reduced

by the SOX10 siRNA (46% reduction) Subsequently,

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we examined the expression of EDNRB mRNA in

those HMV-II cells by northern blot analysis, showing

that the expression of EDNRB mRNA was reduced in

HMV-II cells when the SOX10 siRNA was transfected

(upper band 15% and lower band 35% reduction)

(Fig 1C) We repeated a similar experiment with the

SOX10 siRNA and confirmed the reduced expression

of EDNRB mRNA (upper band 10%, lower band

24% reduction) (data not shown) These results

sug-gest that SOX10 is required for the expression of

EDNRB gene in human melanocyte-lineage cells

Under the conditions used, there were no noticeable

changes in the viability of the cells transfected with the

SOX10 siRNA, despite that SOX10 is required for

melanocyte survival It is conceivable that the reduced

level of SOX10 in the experiment does not affect cell

survival Alternatively, certain genes may compensate

for the down-regulation of SOX10 expression

Identification of a major species of EDNRB gene

transcripts

EDNRB mRNA consists of at least four transcripts

with different 5¢-ends (conventional EDNRB,

EDNRBD1, EDNRBD2, and EDNRBD3), which are

derived from three promoters of the human EDNRB

gene [33] (Fig 2A) Thus, EDNRB mRNA may consist

of eight isoforms, because each EDNRB transcript

may have the two different 3¢-ends (see Fig 1),

gener-ated by the use of alternative polyadenylation sites

[34] However, because of small differences in the

size of each transcript, it is practically impossible to

identify the four transcripts by northern blot analysis

We therefore performed S1 nuclease mapping analysis

to identify EDNRB transcripts expressed in NHEM and HMV-II melanoma cells (Fig 2B) Three protec-ted fragments of 436, 407, and 228 bases were detecprotec-ted

in NHEM and HMV-II cells but not in HeLa cervical cancer cells The two fragments of 436 and 407 bases are preferentially detected and consistent with the con-ventional EDNRB mRNA, transcribed from the two adjacent transcriptional initiation sites [35] The faint signal of 228 bases represents the expression of EDNRBD2 mRNA or EDNRBD3 mRNA In contrast, the signal for the EDNRBD1 transcript of 997 bases was undetectable These results indicate that the con-ventional EDNRB mRNA is abundantly expressed in human melanocytes Furthermore, the alternative pro-moters have not been reported in the mouse Ednrb gene In the present study, we thus focused on the conventional promoter of the EDNRB gene, which

is termed, the EDNRB promoter, unless otherwise specified

Functional analysis of the EDNRB gene promoter

in melanocyte-lineage cells

We first analyzed the promoter activity of the EDNRB gene by transient transfection assays in HMV-II mel-anoma cells and HeLa cervical cancer cells (Fig 3A) The deletion study showed that the promoter activities

of the EDNRB reporter constructs were higher in HMV-II cells than in HeLa cells by about twofold, except for a construct pGL3-E ()12), carrying the

Fig 1 MITF and EDNRB genes are regulated by SOX10 (A) Northern blot analysis of SOX10 and EDNRB mRNA Total RNA was prepared from the indicated cell lines at the top of the panel Autoradiograms of the RNA blots hybridized with the indicated32P-labeled cDNA probes are shown The bottom panel shows the expression of 28S ribosomal RNA visualized by ultraviolet transilluminator (internal control) EDNRB mRNA is detected as two bands, about 4300 nucleotides and 1800 nucleotides as described previously [34] (B) Effects of siRNA against SOX10 on the expression of SOX10, MITF and EDNRB protein Whole cell extracts were prepared from HMV-II human melanoma cells transfected with each siRNA against SOX10 (siSOX10) or LacZ (siLacZ), or untransfected cells (–) The bottom panel shows a-tubulin as an internal control (C) The effect of siRNA against SOX10 on the expression of EDNRB mRNA Total RNA was prepared from the indicated cells same as (B) b-actin mRNA was used as an internal control.

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12-base pairs promoter region Likewise, the promoter

activity of the EDNRB promoter was higher in normal

human epidermal melanocytes and other human

melanoma cell lines, 624mel (Fig 3B), G361 and

SK-MEL-28 (data not shown) than that detected in

HeLa cells (Fig 3B) Thus, the 5¢-flanking region

between)105 and )12 is required for the basal

promo-ter activity of the EDNRB gene in melanocytes-lineage

cells and may confer the marginal cell specificity on

the ENDRB promoter There was noticeable difference

in the promoter activities in melanoma cells between

pGL3-E ()3002) and pGL3-E ()105) (Fig 3A), which

suggests the presence of the negative elements for the

promoter activity in the deleted region Such a

differ-ence in the promoter activity was also detected in

HeLa cells On the other hand, the EDNRB promoter

contains the three potential SOX10 sites (Fig 3A),

which correspond to the enteric nervous system (ENS)

enhancer in the mouse Ednrb gene [18] The expression

levels of pGL3-Em, containing the mutations at the three potential SOX10 sites, were lower than those of

a wild-type construct, pGL3-E ()3022), but the expres-sion level of pGL3-Em is significantly higher in HMV-II cells than that in HeLa cells This observation was also seen in normal human epidermal melano-cytes, and 624mel human melanoma cell lines exam-ined (Fig 3B), and other human melanoma cell lines, G361 and SK-MEL-28 (data not shown) These results suggest that the potential SOX10 sites are dispensable for melanocytes lineage-specific expression of EDNRB, which is consistent in part with the report in the trans-genic mouse analysis of the Ednrb gene [18]

Transactivation of the EDNRB gene promoter

by SOX10 and Sp1

We then analyzed the effect of SOX10 on the promoter function of the EDNRB gene by transient cotransfection

Fig 2 Identification of the major transcripts of the EDNRB gene in human melanocytes (A) Schematic representation of the promoters of the EDNRB gene The EDNRB gene is shown as a line and four arrows show the transcriptional initiation sites of each transcript The open boxes of each transcript show the 5¢-untranslated region The shadow boxes and dotted boxes indicate the protein-coding region of each transcript The transcripts derived from the conventional EDNRB, EDNRBD1 and the EDNRBD2 promoters code for the same protein The protein encoded by EDNRBD3 mRNA has D3-specific N-terminal amino acids (dotted boxes), followed by the common coding region (shaded boxes) The splicing sites are shown by a gap The number shown on the transcripts represents the position from the transcriptional initi-ation site (+1) of the conventional EDNRB mRNA The conventional EDNRB mRNA has two transcriptional initiiniti-ation sites (+1 and +30) [35] The S1 probe, shown at the bottom, contains the EDNRB gene sequences (line) and the vector sequence (dotted line) The end-labeled site

of S1 probe is the XcmI site (position +436 in the antisense strand of EDNRB cDNA), and is indicated with an asterisk The predicted frag-ments are shown below the transcripts (boxes), together with each predicted size (B) S1 nuclease mapping analysis of EDNRB transcripts.

An autoradiogram of the S1 nuclease mapping analysis is shown Total RNA was prepared from the indicated cell lines at the top of panel The S1 probe and four predicted fragments are indicated as arrows Size markers were end-labeled with 1-kb DNA Ladder and 100-bp DNA Ladder (New England Biolabs, Beverly, MA, USA).

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assays (Fig 4A) SOX10 significantly increased the

expression levels of reporter constructs, containing the

promoter region between)105 and )12 The localized

promoter region ()105 and )12), which is also

respon-sible for the marginal melanocyte-lineage specificity of

the EDNRB promoter, contains the GC box, a

consen-sus sequence of the binding site for Sp1 (Fig 4B)

Moreover, Sp1 has been reported to interact with

SOX10 [36,37] We also confirmed the interaction of

SOX10 and Sp1 by in vitro pull-down assay (data not shown) We therefore analyzed whether Sp1 influences the function of the EDNRB promoter An Sp1 expres-sion plasmid was coexpressed with SOX10 expresexpres-sion plasmid in HeLa cells, which endogenously express Sp1 protein [38] We thus confirmed the over-expression of Sp1 protein in HeLa cells, when transfected with Sp1 expression plasmid (Fig 4C) Sp1 or SOX10 alone increased the promoter activity of pGL3-E ()3022)

A

B

Fig 3 The EDNRB gene promoter shows the melanocyte lineage cell-specific activity (A) Functional analysis of the EDNRB gene promoter

in melanoma cells The left panel shows the reporter plasmids used The number shown at the 5¢- or 3¢-end of each construct represents the position from the transcriptional initiation site (+1) The open circles on the EDNRB gene show the potential SOX10 sites, which correspond

to the enteric nervous system (ENS) enhancers in mice [18] The internal deletion site is shown as a gap X shows the mutation of each potential SOX10 site Relative luciferase activity in transient transfection assays is shown on the right The cell lines used are HeLa cells (open bars) and HMV-II cells (filled bars) Luciferase activity was normalized by each internal control activity (pRL-TK) Relative luciferase activ-ity is shown as the ratio to the normalized luciferase activactiv-ity obtained with pGL3-E ( )3022) in HeLa cells Data are mean ± SD of at least three independent experiments The activity with * or ** is significantly higher in HMV-II cells than the value obtained with each reporter plasmid in HeLa cells, P < 0.01 or P < 0.05 (B) Functional analysis of the EDNRB gene promoter in melanocytes-lineage cells The left panel shows the reporter plasmids used Relative luciferase activity in transient transfection assays is shown on the right The cell lines used are HeLa cells, HMV-II and 624mel melanoma cells (dotted bars), and normal human epidermal melanocytes (NHEM) (shadow bars) Luciferase activity was normalized by each internal control activity (pRL-TK) Relative luciferase activity is shown as the ratio to the normalized luciferase activity obtained with pGL3-E ( )3022) in HeLa cells Data are mean ± SD of at least three independent experiments The activity with * is significantly higher in melanoma cells and NHEM than the value obtained with each reporter plasmid in HeLa cells, P < 0.01 or P < 0.05.

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1.9- or 3.9-fold, respectively (Fig 3C) The combination

of SOX10 and Sp1 led to an 8.0-fold increase,

suggest-ing that SOX10 and Sp1 synergistically transactivate

the EDNRB promoter Furthermore, SOX10, alone or

in combination with Sp1, significantly increased the

expression of pGL3-Em, containing the mutations at

the three potential SOX10 sites in the putative ENS enhancer It is therefore conceivable that these potential SOX10 sites may be dispensable for the SOX10-medi-ated transactivation Taken together, the localized promoter region ()105 and )12) is responsible not only for the marginal melanocyte-lineage specificity of the

B

C

Fig 4 SOX10 and Sp1 synergistically trans-activate the EDNRB promoter (A) Effects of SOX10 on the EDNRB promoter The right panel shows the result of the transient transfection assay in HeLa cells The co-transfection with empty vector (–) or SOX10 expression vector (SOX10) is shown as a white bar or a shadow bar, respectively Rel-ative luciferase activity is shown as the ratio

to the normalized luciferase activity obtained with cotransfection of pGL3-E ( )3002) and empty vector Data are mean ± SD of at least three independent experiments The activity with * is significantly higher than the value obtained with cotransfection of each reporter plasmid and empty vector,

P < 0.01 (B) Effects of Sp1 on the SOX10-mediated transactivation of the EDNRB pro-moter Shown is the nucleotide sequence of the localized promoter region, in which a GC box is underlined HeLa cells were cotrans-fected with each reporter shown to the left, and an empty vector (open bars), Sp1 expression vector (dotted bars), SOX10 expression vector (shadow bars), or both of Sp1 and SOX10 expression vectors (filled bars) Relative luciferase activity is shown

as the ratio to the normalized luciferase activity obtained with cotransfection of pGL3-E ( )3002) and the empty vector Data are mean ± SD of at least three independent experiments The activity with * is signifi-cantly higher than the value obtained with each reporter plasmid and empty vector,

P < 0.01 (C) Over-expression of Sp1 and SOX10 in HeLa cells Enforced-expression

of Sp1 and ⁄ or SOX10 was assessed in HeLa cells transfected with Sp1 and ⁄ or SOX10 expression vector by western blot analysis Note that the amount of Sp1 pro-tein was increased in the cells transfected with Sp1 expression vector compared to that in mock-transfected HeLa cells shown

as (–).

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EDNRB promoter but also for the SOX10-mediated

transactivation

Identification of the cis-acting elements

responsible for the SOX10-mediated

transactivation of the EDNRB promoter

Within the region between)105 and )12 of the EDNRB

promoter, there is no consensus sequence, 5¢-(A ⁄ T)

(A⁄ T)CAA(A ⁄ T)-3¢, for SOX10 binding Instead, there

are the two CA-rich sequences (CA1 and CA2) and

the GC box (5¢-CCGCCC-3¢) (GC1) Notably, the

CA2 is overlapping with the GC box (Fig 5A) It has

been reported that SOX10 binds the CA-rich sequence

in the neuronal nicotinic acetylcholine receptor b four

subunit gene promoter [39] We therefore examined

whether CA1, GC1, or CA2 is involved in the basal

promoter activity and⁄ or the SOX10-mediated

transac-tivation of the EDNRB promoter (Fig 5B,C) Each

base change at CA1, GC1 or CA2 resulted in the

signi-ficant decrease in the promoter activity in normal

mel-anocytes and HMV-II melanoma cells (Fig 5B) The

GC box is especially important for the basal promoter

activity Likewise, each base change abolished the

SOX10-mediated transactivation of the EDNRB

pro-moter (Fig 5C), indicating that the three elements,

CA1, GC1 and CA2, are responsible for the

transacti-vation of the EDNRB promoter by SOX10

SOX10 binds to the CA-rich sequences and the

GC box of the EDNRB promoter in vitro

We carried out electrophoretic mobility shift assay

(EMSA) using the labeled probes, which include CA1

(CA1 probe) or GC1 and CA2 (GC1⁄ CA2 probe)

(Fig 5A,D) These oligonucleotide probes were

incu-bated with the lysates containing SOX10 protein

syn-thesized by in vitro transcription⁄ translation system

The CA1 probe was specifically bound by recombinant

SOX10 (Fig 5D, left panel) The formation of the

SOX10–DNA complexes was inhibited by CA1 probe,

but the degree of inhibition with a competitor

contain-ing mutated CA1 (mCA1) was lower than that of CA1

probe When we used the consensus SOX10-binding

site (cSOX10) as a competitor, which contains the

SOX10-binding site in human MITF gene promoter

[14–17], the formation of the SOX10-DNA complexes

was reduced The SOX10–DNA complexes were not

detected with the lysates in the case of an empty vector

as a negative control Thus, SOX10 binds to CA1 in

the EDNRB promoter Unexpectedly, the degree of

competition with the mutated CA1 was similar to that

with cSox10 competitor, which may be due to

addi-tional SOX10 binding sites in the region of CA1 probe Likewise, we showed that SOX10 specifically bound to GC1 and CA2, as the SOX10–DNA com-plexes were competed by the GC1⁄ CA2 probe or the cSOX10 probe, but not by a mutated GC1 and⁄ or CA2 (mGC1⁄ CA2, GC1 ⁄ mCA2, or mGC1 ⁄ mCA2) (Fig 5D, right panel) Taken together, these results suggest that SOX10 binds to the three cis-acting ele-ments, CA1, GC1, and CA2, in the EDNRB promoter, which is consistent with the results of the cotransfec-tion assays

Synergistic activation of the EDNRB promoter

by SOX10 and Sp1 through the two CA-rich sequences and the GC box

We analyzed the functional significance of CA1, GC1, and⁄ or CA2 in the EDNRB promoter activity (Fig 6A) Each base change at CA1 and⁄ or GC1 sig-nificantly reduced the degree of activation caused by SOX10 and Sp1, compared to the parent reporter plas-mid, pGL3-E ()3022) The base change at CA2 showed 35% reduction compared to E ()3022) (P ¼ 0.006) These results suggest that SOX10 and Sp1 synergistically transactivate the EDNRB promoter through the two CA-rich sequences and the GC box

To examine whether Sp1 binds to GC1, we performed EMSA using the labeled GC1⁄ CA2 probe and recom-binant human Sp1 protein (Fig 6B) The Sp1-DNA complexes were detected, and their formation was completely competed by a wild-type GC1⁄ CA2 probe, mutated CA2 probe (GC1⁄ mCA2), or a consensus Sp1-binding sequence (cSp1) Interestingly the complex formation was competed by mGC1⁄ CA2 probe, but its competition ability was lower than that of the GC1⁄ CA2 probe Furthermore, mGC1 ⁄ mCA2 probe did not inhibit the complex formation These results suggest that Sp1 recognizes the region containing GC1 and CA2 The binding of Sp1 to GC1 may be influ-enced by the overlapping CA2

We then examined the simultaneous binding of SOX10 and Sp1 proteins to the GC1⁄ CA2, but were unable to detect the complexes, containing both SOX10 and Sp1 proteins (data not shown) It is con-ceivable that the in vitro binding conditions are not suitable for simultaneous binding of the two proteins

to the GC1⁄ CA2

SOX10 and Sp1 bind to the EDNRB promoter

in vivo

To investigate whether SOX10 and⁄ or Sp1 bind to the EDNRB gene in vivo, chromatin immunoprecipitaion

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C

D

Fig 5 Identification of the cis-acting ele-ments responsible for the SOX10-mediated transactivation of the EDNRB promoter (A) Schematic representation of the EDNRB promoter The number represents the posit-ion from the transcriptposit-ional initiatposit-ion site (+ 1) of the EDNRB gene The two CA-rich sequences and the GC box are marked The CA1 probe (line) and the GC1 ⁄ CA2 probe (dotted line) are used in electrophoretic mobility shift assay of Fig 4(D) (B) Require-ment of CA1, GC1, and CA2 for the melano-cyte lineage cell-specific activity Base changes were introduced into CA1, GC1,

or CA2 of a parent plasmid, pGL3-E ( )3022) reporter vector X shows the muta-tion introduced into CA1, GC1, or CA2 HeLa cells (open bars), HMV-II cells (filled bars), or normal human epidermal melano-cytes (NHEM) (shaded bars) were transfected with each reporter shown to the left Other conditions are represented as indicated in Fig 3(A,C) Effects of the base change at CA1, GC1, or CA2 The transactivation of the EDNRB promoter by SOX10 was assessed in the case of each mutation shown as X Other conditions are

represent-ed as indicatrepresent-ed in Fig 3(B) (D) Electro-phoretic mobility shift assays (EMSA) for binding of SOX10 to the EDNRB promoter Shown are the autoradiographs of EMSA with the CA1 probe (left) or the GC1 ⁄ CA2 probe (right) Lanes shown as (–) indicate no protein or no competitor The mock shows the lysate containing a parent vector, pIVEX3.2-MCS SOX10 shows the lysate containing SOX10 protein synthesized by the in vitro transcription ⁄ translation Escherichia coli lysate system The compe-titors used are wild-type probe or the mutated probe (shown as Fig 5A) The consensus SOX10-binding site of human MITF gene promoter is used as a compet-itor (cSOX10) The arrows show the SOX10–DNA complexes.

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(ChIP) assay was performed in HMV-II cells

(Fig 7A) The PCR primer sets were designed to

amplify the DNA segments containing CA1, GC1, and

CA2 of the EDNRB promoter, which is responsible for

the SOX10-mediated transactivation ChIP assay

revealed that the DNA segments of the EDNRB

pro-moter were amplified when precipitated with rabbit

anti-SOX10 IgG or goat anti-Sp1 IgG in HMV-II

cells, but not noticeably amplified when precipitated

with normal rabbit IgG (negative control) or normal

goat IgG (negative control) (Fig 7B) These results

indicate that SOX10 and Sp1 bind to the region

con-taining CA1, GC1, and CA2 in the EDNRB promoter

in vivo We could not detect the amplified fragments of GAPDH gene as a negative control, when precipitated with rabbit anti-SOX10 IgG or goat anti-Sp1 IgG

SOX10 activates the EDNRB promoter in the demethylation status

It has been reported that the EDNRB promoter is located in the CpG islands, which are target sites of DNA methylation [40–42] In fact, the EDNRB pro-moter is DNA-methylated in several types of tumor

A

B

Fig 6 Sp1 is involved in the

SOX10-medi-ated transactivation of the EDNRB

promot-er (A) Effects of Sp1 on the transactivation

of the EDNRB promoter HeLa cells were

cotransfected with each reporter shown on

the left, and an empty vector (open bars),

Sp1 expression vector (dotted bars), SOX10

expression vector (shadow bars), or both of

Sp1 and SOX10 expression vectors (filled

bars) Relative luciferase activity is shown

as the ratio to the normalized luciferase

activity obtained with cotransfection of

pGL3-E ( )3002) and the empty vector Data

are mean ± SD of at least three independent

experiments The activity with # or ### is

significantly lower than the value obtained

with cotransfection of pGL3-E ( )3002),

SOX10, and Sp1, #P < 0.01 or

###P < 0.001 (B) EMSA for the in vitro

binding of Sp1 to the EDNRB promoter.

Shown is the autoradiograph of the EMSA

by using the GC1 ⁄ CA2 probe The

recomb-inant Sp1 was used The competitors used

are wild-type probe or the mutated probe

(shown as Fig 4A) The consensus Sp1

binding site is used as a competitor (cSp1)

[56] The arrows show the Sp1–DNA

com-plexes.

Trang 10

cells, leading to gene silencing [40–43] Moreover, the

GC1, identified as one of the key regulatory elements,

includes the CpG dideoxynucleotides, which are

poten-tial targets of DNA methylation (Fig 8A) We

there-fore investigated the DNA methylation status of the

EDNRB promoter in normal human epidermal

mel-anocytes (NHEM), HMV-II melanoma cells, and

HeLa cells The EDNRB promoter is heavily

methyla-ted in HeLa cells, but not in NHEM and HMV-II

cells (Fig 8A) Subsequently, we examined whether

enforced expression of SOX10 induces the endogenous

EDNRB expression in HeLa cells We established

FLAG-tagged SOX10 (F⁄ SOX10)-expressing stable

transformants from HeLa cells, and then chose the

sta-ble transformants #6 and #8, which appear to express

F⁄ SOX10 more abundantly than other transformants

(Fig 8B) Expression of EDNRB mRNA was

unde-tectable in cells transformed with the empty vector

(Mock) and the F⁄ SOX10-expressing cells, as judged

by RT-PCR analysis (Fig 8C) However, in the

mock-transformed cells, expression of EDNRB mRNA

became detectable after treatment with a demethylating

reagent, 5¢-aza-2¢-deoxycytidine (5¢-aza-dC) (Fig 8C)

Importantly, the expression levels of EDNRB mRNA

expressed in HeLa cells [38]

Thus, the degree of the methylation in the EDNRB promoter determines the transcription levels of the EDNRB gene However, we were unable to assess the contribution of the methylation status to the promoter activity by the transient expression assay, which could account in part for a small difference in the melano-cyte lineage-specific promoter activity Alternatively, the human EDNRB gene contains an additional mel-anocyte-enhancer, which is however, not carried by the reporter constructs used, containing the 3-kb length of human EDNRB gene promoter, although the 1.2-kb length of mouse Ednrb gene promoter is sufficient for melanocyte-specific activity [18]

Implications SOX10 interacts with Sp1 and activates the EDNRB promoter Sox10 also interacts with Pax3 and activates the c-RET promoter [44,45] The mutation in the c-RET gene is responsible for the pathogenesis of aganglionic megacolon [46] Ubiquitously expressed Sp1 may affect the pathogenesis of WS4 by cooper-ating directly with SOX10 or by influencing the inter-action of SOX10 with PAX3

It should be noted that WS4 shows the phenotypic variability [9,22] In one family case of WS4, for exam-ple, the proband and his sister are heterozygous for the Q377X mutation of SOX10, but only the proband has an aganglionic megacolon [22] These observations suggest the presence of modifier genes for the EDNRB gene, and the expression of the modifier genes may be influenced by the environmental factors, thereby lead-ing to the phenotypic variability of WS4 One of such modifiers might be Sp1, the function of which is modulated growth factors [47] or metals [48]

In summary, we have provided evidence that SOX10, alone or in combination with Sp1, may acti-vate transcription of the human EDNRB gene, which contributes to melanocyte lineage cell-specific expres-sion, and that the regulation of EDNRB expression by SOX10 requires the demethylation status of its promo-ter The regulatory network involving SOX10 and Sp1 may ensure the fine-tuning of EDNRB expression, which contributes the homeostasis of human melano-cytes Future research on the genetic network of WS genes will help clarify the pathogenesis of WS

B

Fig 7 Binding of SOX10 and Sp1 to the EDNRB promoter in vivo.

(A) Strategy for chromatin immunoprecipitation assay Arrows

indic-ate the PCR primers used for the EDNRB gene promoter (B)

In vivo binding of SOX10 and Sp1 to the EDNRB promoter HMV-II

cells were fixed with formaldehyde, precipitated with anti-SOX10,

anti-Sp1, normal rabbit IgG, or normal goat IgG, and subjected to

PCR for the EDNRB promoter (top panel) or GAPDH gene (bottom

panel) as a negative control The amplified DNA segments were

visualized with ultraviolet transilluminator.

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