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Tel.: +33 3 90 24 26 79, Fax: +33 3 90 24 27 81, E-mail: npotier@chimie.u-strasbg.fr Abbreviations: ESI-MS, electrospray ionization mass spectrometry; RAR, human retinoic acid receptor a

Trang 1

Monitoring ligand-mediated nuclear receptor–coregulator interactions

by noncovalent mass spectrometry

Sarah Sanglier1, William Bourguet2,*, Pierre Germain2, Virginie Chavant2, Dino Moras2,

Hinrich Gronemeyer2, Noelle Potier1and Alain Van Dorsselaer1

1

Laboratoire de Spectrome´trie de Masse Bio-Organique, CNRS UMR 7509, ECPM, Strasbourg, France;2Institut de Ge´ne´tique et de Biologie Mole´culaire et Cellulaire, CNRS INSERM ULP Colle`ge de France, Illkirch, France

Retinoid receptors are ligand-dependent transcription

factors belonging to the nuclear receptor superfamily

Retinoic acid (RARa, b, c) and retinoid X (RXRa, b, c)

receptors mediate the retinoid/rexinoid signal to the

transcriptional machineries by interacting at the first level

with coactivators or corepressors, which leads to the

recruitment of enzymatically active noncovalent

com-plexes at target gene promoters It has been shown that

the interaction of corepressors with nuclear receptors

in-volves conserved LXXI/HIXXXI/L consensus sequences

termed corepressor nuclear receptor (CoRNR) boxes

Here we describe the use of nondenaturing electrospray

ionization mass spectrometry (ESI-MS) to determine the

characteristics of CoRNR box peptide binding to the

ligand binding domains of the RARa–RXRa

hetero-dimer The stability of the RARa–RXRa–CoRNR

tern-ary complexes was monitored in the presence of different types of agonists or antagonists for the two receptors, including inverse agonists These results show unambig-uously the differential impact of distinct retinoids on corepressor binding We show that ESI-MS is a powerful technique that complements classical methods and allows one to: (a) obtain direct evidence for the formation of noncovalent NR complexes; (b) determine ligand binding stoichiometries and (c) monitor ligand effects on these complexes

Keywords: ESI-MS; noncovalent mass spectrometry; nuc-lear receptors; protein–protein interactions; protein–ligand interactions

Retinoid receptors are ligand-dependent transcription

fac-tors belonging to the nuclear receptor (NR) superfamily [1]

They are involved in vertebrate development, cell

differen-tiation and proliferation or apoptosis [2], and display

significant promise for cancer therapy and prevention [3,4]

Retinoid X (RXRa, b, c) and retinoic acid (RARa, b, c)

receptors form heterodimers, but RXRs can also

hetero-dimerize with a great number of other nuclear receptors [5]

NRs display a modular structure with distinct functional

and structural domains [1], such as the DNA-binding

domain [6] or the ligand binding domain (LBD) [7,8], which corresponds also to the region that interacts with coactiva-tors (CoA) or corepressors (CoR)

Although the molecular mechanisms by which NRs control target gene expression are in principle understood, many aspects remain to be solved This concerns both the details of the ligand-induced early events [9], and the assembly and epigenetic action of multiprotein machineries

on target gene promoters [10] As NRs respond to small ligand molecules and correspond to potent regulators of cell function [11,12], life and death [13,14], they are particularly attractive targets for the design of novel therapeutic agents

A critical step in the drug design process is the elucidation

of the ligand function, as it can be deduced from the characterization of the molecular mechanism(s) that are modulated by ligand binding, such as the interaction with coactivator and corepressor complexes that mediate the transcriptional activity of NRs In the case of retinoid receptors, the unliganded RAR–RXR heterodimers (HD) have been shown to act as repressors of transcription through recruitment of complexes containing corepressors, such as nuclear receptor corepressor [15] or silencing mediator for RARs and thyroid hormone receptor (TRs)

2[16] Upon ligand binding, NRs undergo conformational changes inducing the dissociation of repressor complexes and the subsequent association with coactivator molecules thereby promoting transcriptional activation CoAs are like CoRs; large multidomain proteins, which bind through specific motifs (referred to as NR box or LXXLL motif) to

Correspondence to N Potier, Laboratoire de Spectrome´trie de Masse

Bio-Organique, UMR CNRS 7509, ECPM, Universite´ Louis Pasteur

de Strasbourg, 25 Rue Becquerel, 67082 Strasbourg Cedex 2, France.

Tel.: +33 3 90 24 26 79, Fax: +33 3 90 24 27 81,

E-mail: npotier@chimie.u-strasbg.fr

Abbreviations: ESI-MS, electrospray ionization mass spectrometry;

RAR, human retinoic acid receptor a; RXR, human retinoid X

receptor a; CoR, corepressor; CoA, coactivator; NR, nuclear receptor;

NcoR, nuclear corepressor protein; CoRNR, corepressor nuclear

receptor; LBD, ligand binding domain; LBP, ligand binding pocket;

Vc, accelerating voltage; HD, RAR–RXR heterodimer; TR, thyroid

hormone receptor.

*Present address: Centre de Biochimie Structurale, CNRS UMR 5048,

UM1 INSERM UMR 554, Faculte´ de Pharmacie, 15 Avenue

Flahault, 34060 Montpellier, France.

(Received 17 August 2004, revised 12 October 2004,

accepted 27 October 2004)

Trang 2

agonist-bound NRs CoRs interact with NRs through

nuclear receptor interacting domains that contain two

helical motifs referred to as corepressor nuclear receptor

boxes (CoRNR1 and CoRNR2, comprising LXXI/

HIXXXI/L sequences) [17–19]

Since 1991 [13], electrospray ionization mass

spectro-metry (ESI-MS) has been regularly used to study

noncova-lent complexes, and an increasing number of studies of

protein–protein, protein–ligand and protein–DNA

com-plexes are now reported (reviewed in [20–22]) However,

such studies are not yet straightforward because some

technical difficulties, such as the presence of nonvolatile

buffers or the fragility of the complexes, are encountered

Nevertheless, ESI-MS appears to be an attractive approach

and offers new possibilities for the study of such complexes,

providing direct evidence for their formation and an

accurate determination of their binding stoichiometry For

instance, in the field of NRs, ESI-MS was previously used

by Witkowska et al [23,24] to detect the estrogen receptor

ligand binding domain in its dimeric noncovalently bound

form Craig et al [25] analyzed by microESI-MS the

binding of the human vitamin D receptor and the retinoid

X receptor-a to the osteopontin vitamin D response

element, and assessed the influence of their endogenous

ligands 1a,25-dihydroxyvitamin D3 and 9-cis retinoic acid

The importance of structural zinc ions for the interaction

between the DNA binding domain of the human vitamin D

receptor with a double-stranded DNA containing the

vitamin D response element from the mouse osteopontin

gene was determined using ESI-MS [25,26] Bourguet et al

[27] used ESI-MS to reveal the presence of an unexpected

ligand in the mRXRaF318A complex that was apparent at

the early stages of the structure refinement and Germain

et al [28] demonstrated by ESI-MS that the RXR subunit

of the HD heterodimer

when RAR is ligand-free Potier et al [29] and other groups

[30–32] described the strength of nondenaturing mass

spectrometry to identify unexpected ligands coming from

the expression host that bind to orphan receptors More

recently, the use of ESI-MS has also been reported for the

characterization of protein–ligand complexes involving

other nuclear receptors [

In the study reported here, we used ESI-MS to

charac-terize the binding of CoRNRs to the LBDs of the HD

heterodimer In particular, the influence of ligand binding to

RAR or RXR on the stability of HD–CoRNR complexes

was investigated by nondenaturing ESI-MS We show that

supramolecular mass spectrometry is a powerful tool to

rapidly and unambiguously determine if a particular peptide

sequence can interact with LBDs of the HD heterodimer

and to directly visualize the influence of various ligands on

the stability of the corresponding ternary complexes These

results could only be obtained after precisely adjusting of the

mass spectrometer interface parameters, which are

des-cribed here in detail

Materials and methods

Materials and chemicals

Expression and purification of the HD heterodimer was

performed as described by Bourguet et al [39] Peptides

containing CoRNR1 and CoRNR2 sequences were syn-thesized and used as model peptides for the corepressor proteins CoRNR1 is a 24 amino acid peptide (THRLIT LADHICQIITQDFARNQV; molecular mass 2806.2 Da) containing the consensus sequence that specifically interacts with the RAR subunit CoRNR2 contains 14 amino acids (NLGLEDIIRKALMG; molecular mass 1542.8 Da) and interacts specifically with the RXR subunit

Ligands were chosen to interact specifically with either RAR (AM80, BMS493 and BMS614) or RXR (HX531) [28,40,41]

PSG5-based Gal-RARa and VP16-RARa expression vectors, and the (17m)5x-bG-Luc reporter gene have been described [42] Gal-NCoR corresponds to a fusion between residues 1–147 of Gal4 and residues 1629–2453 of NCoR

Cell culture and transient transfections COS cells, cultured in Dulbecco’s modified Eagle’s medium/ 5% (v/v) fetal bovine serum, were transiently transfected using the standard calcium phosphate method Results were normalized to coexpressed b-galactosidase

Electrospray ionization mass spectrometry analysis Prior to any ESI-MS analysis, samples were desalted on Centricon PM30 microconcentrators (Amicon, Millipore, Molsheim, France)

Ammonium acetate presents the advantage not only of preserving the ternary and quaternary structures of proteins

in solution but also of being compatible with ESI-MS experiments

ESI-MS measurements were performed on an electro-spray time-of-flight mass spectrometer (LCT, Waters, Manchester, UK)

receptors were verified by mass spectrometry analysis in denaturing conditions: proteins were diluted to 5 pmolÆlL)1

in a water/acetonitrile mixture (1 : 1, v/v) acidified with 1% (v/v) formic acid Mass spectra were recorded in the positive ion mode on the mass range 500–2500 m/z, after calibration with horse heart myoglobin diluted to 2 pmolÆlL)1in a water/acetonitrile mixture (1 : 1, v/v) acidified with 1% (v/v) formic acid The following molecular masses were measured: 29 930 ± 1.8 Da for RARa; 26 735 ± 2.3 Da for RXRa with deletion of the N-terminal methionine and

26 867 ± 2.1 Da corresponding to RXRa These results were in agreement with the molecular masses calculated from the known amino acid sequences

The mass measurements of the noncovalent complexes were performed in ammonium acetate (50 mM, pH 6.5) Samples were diluted to 10 pmolÆlL)1in the previous buffer and continuously infused into the ESI ion source at a flow rate of 6 lLÆmin)1through a Harvard syringe pump Great care was exercised so that noncovalent interactions survive the ionization/desorption process Particularly the acceler-ating voltage (Vc), which controls the kinetic energy communicated to the ions in the interface region of the mass spectrometer, was optimized to 50 V in order to prevent ligand dissociation in the gas phase ESI-MS data were acquired in the positive ion mode on the mass range 1000–5000 m/z Calibration of the instrument was per-formed using the multiply charged ions produced by a

Trang 3

separate injection of horse heart myoglobin diluted to

2 pmolÆlL)1 in a water/acetonitrile mixture (1 : 1, v/v)

acidified with 1% (v/v) formic acid The relative abundance

of the different species present on ESI mass spectra were

measured from their respective peak intensities, assuming

that relative intensities displayed by the different species on

the ESI mass spectrum reflect the actual distribution of these

species in solution The reproducibility of the determination

of the relative proportions of the different species was

estimated to be ± 2–3%

Results

Optimization of the ESI-MS operating conditions to

detect heterodimer–ligand–corepressor complexes

To obtain optimal sensitivity without affecting the stability

of noncovalent NR complexes, we first optimized the

operating conditions Indeed, it is critical to prevent

dissociation of weak interactions in the mass spectrometer

when transferring ions from the condensed phase to the gas

phase It has been shown by several groups that the gas

phase stability of noncovalent complexes is greatly

depend-ant on the nature of the interactions involved in the

formation of the complexes [37,43–47] It seems that the gas

phase stability strongly increases with an increased

contri-bution of electrostatic interactions within a noncovalent

complex Therefore, it is important that the Vc, which

controls the kinetic energy communicated to the ions in the interface region of the mass spectrometer, is carefully set [37,47] Operating conditions were optimized on the HD– ligand–corepressor complex with CoRNR1 and BMS493,

an inverse pan-RAR agonist [28,48] that does not interact with RXRs

Mass spectra for the ligand-bound HD–BMS493– CoRNR1 complex at different Vc voltages were generated from samples in which a threefold molar excess of CoRNR1 and a twofold molar excess of BMS493 were added to the

HD heterodimer preparation (Fig 1) At low accelerating voltages (i.e for Vc£ 30 V), the HD–BMS493–CoRNR1 noncovalent complex is detected as a major component (Fig 1A) However, the observed peak shapes are broad, which probably results from incomplete desolvation A direct consequence of this is a significant loss in mass accuracy Nevertheless, significant amounts of the HD– BMS493 complex can be detected Increasing the Vc to 50 V significantly improves the signal to noise ratio and still allows the detection of the HD–BMS493–CoRNR1 com-plex as a main component with a molecular mass of

59 884.1 ± 1.4 Da, while the HD–BMS493 complex with a molecular mass of 57 080.2 ± 0.9 Da is only a minor component (Fig 1B) Under these conditions an additional ion series displaying a molecular mass of 59 474.2 ± 0.1 Da emerges and can be attributed to the unliganded HD– CoRNR1 complex Thus, increasing Vc from 30 V to 50 V improves considerably the desolvation and the accuracy of

17+ V

17+ V

16+

18+

16+

15+

17+ V

15+

18+ 17+

16+

15+

Partial peptide dissociation

Ligand dissociation

DM = 408 Da (100%)

Slight ligand dissociation (less than 5%)

A

B

C

Fig 1 Optimization of the operating conditions: ESI-MS mass spectra of the HD–BMS493–CoRNR1 complex at different accelerating voltages (Vc) Positive ESI mass spectra of the HD–BMS493–CoRNR1 diluted to 10 pmolÆlL)1 in AcONH 4 (50 m M , pH 6.5) at (A) Vc ¼ 30 V: the HD–BMS493–CoRNR1 noncovalent complex is detected without any dissociation; (B) Vc ¼ 50 V: slight dissocitation of the ligand (less than 5%) from the HD–BMS493–CoRNR1 complex; (C) Vc ¼ 80 V: 100% dissociation of the ligand from the HD–CoRNR1 and partial dissociation of the peptide from the HD–CoRNR1 complex Peaks labeled with * correspond to the different species with the additional N-terminal methionine.

Trang 4

the mass measurement but induces concomitantly some

dissociation of the ligand-bound HD–BMS493–CoRNR1

complex into the unliganded HD–CoRNR1 complex A

further increase of the accelerating voltage to 80 V (Fig 1C)

provokes dissociation of the quaternary complex to HD–

CoRNR1 (major component) and HD (minor component)

complexes No ions corresponding to a ligand-bound species

are detected under these conditions These data indicate that

the interactions involving the ligand are less stable in the gas

phase than those involving the corepressor peptide Whether

HD originates from the gas phase dissociation of the HD–

BMS493 complex or from the HD–CoRNR1 complex

cannot be distinguished

to 120–150 V (data not shown) does not lead to any major

change in the dissociation pattern regarding the complexes,

which suggests that the HD–CoRNR1 complex is quite

stable in the gas phase

The above data reveal that the chosen operating

condi-tions (buffer and control of the Vc voltage) allow the

transfer of intact noncovalent receptor–ligand–corepressor

peptide complexes from solution to the gas phase It should

be noted that such experiments were performed for each

HD–ligand–CoRNR complex described in this paper In all

cases, at low accelerating voltages (30–50 V), HD–ligand–

CoRNR complexes remained intact after passage into the

gas phase Increasing the Vc voltage (up to 80–150 V)

resulted in a stepwise removal of the ligand first, and

subsequent dissociation of the CoR peptide Consequently,

as a compromise between sufficient desolvation, optimal

transmission of the ions and nondestructive gas-phase

collisions was found at 50 V and we retained such

experimental conditions in our further experiments

Interestingly, while various biochemical studies including

circular dichroism, proteolytic digestions or gel mobility

suggested that NRs undergo conformational changes upon

ligand binding, no charge state distribution changes were

detected upon ligand binding or CoR peptide binding

(+15 to +17)

Witkowska et al [23], that ESI-MS might also be useful for

revealing structural changes in protein conformations from

subtle changes in the charge state distribution observed on

ESI mass spectra [20,49–52] So, if conformational changes

occur, these do not sufficiently affect the surface of the

protein exposed to solvent to be detected by mass

spectro-metry This latter assumption is supported by X-ray

crystallographic studies showing no obvious evidence of

new exposure of charged amino acids The mainly local

conformational changes appear not to affect the protein

surface in a way sufficient to be revealed by ESI-MS

RAR–RXR heterodimer forms a 1 : 1 noncovalent

complex with corepressor peptides

The mass spectrum obtained at a threefold molar excess of

CoRNR1 over HD displays two main ion series at mass to

charge ratios between m/z 3000 and m/z 4000 (Fig 2A) The

first series (r) corresponds to the +15 to +17 charge states

of the HD, with a molecular mass of 56 669.4 ± 2.5 Da

The second series (d) gives rise to a molecular mass of

59 478.3 ± 0.5 Da and can be attributed to the formation

of a 1 : 1 complex between HD and CoRNR1 corepressor

peptide, with a charge state distribution varying from +16

to +18 Using identical experimental conditions for HD–CoRNR2 complexes, two series of multiply charged ions were detected corresponding to the HD (56 664.8 ± 0.3 Da) and the HD–CoRNR2 complex (58 210.2 ± 1.8 Da) (Fig 3A) All data were recorded at a cone voltage

of 50 V (see above) As ESI-MS analyses faithfully reflect solution equilibrium in the previously mentioned experi-mental conditions, we reproducibly observed that only

 50% of the detected species correspond to both HD–CoRNR complexes in the absence of ligand Titration experiments, in which increasing amounts of CoRNR peptide (up to fourfold molar excess, data not shown) were added to the HD, reveal no further saturation of HD in the absence of ligand This suggests that a twofold molar excess

of CoRNR peptide is sufficient to reach the equilibrium state under our experimental conditions This ratio (50%) was taken as a reference point in the present study to monitor the effect of various classes of ligands on this equilibrium

Ligand modulation of HD–CoRNR ternary complexes

In order to monitor the effect of different types of ligands on the recruitment of two CoRNR peptides, various ligands were added to each HD–CoRNR complex and the stability

of each ternary complex was recorded by ESI-MS The antagonist activity of the inverse agonist BMS493, the RARa-selective agonist BMS614 and the RXR antagonist HX531 were confirmed by competition of AM80-induced transactivation using the chimeric Gal-RARa as ligand-dependent activator of a cognate 17mer-luciferase reporter gene (Fig 4) These data reveal the antagonistic potential of BMS493 and BMS614 and demonstrate also that HX531 has a weak RARa antagonist activity

Effects of RAR ligand binding on the stability of the HD–CoRNR1 complex Mass spectra obtained for the

HD in presence of CoRNR1 with a twofold molar excess of either the inverse pan-RAR antagonist BMS493 (Fig 2B)

or the RARa-selective agonist AM80 (Fig 2C), revealed that all ligands bind to the HD and to the HD–CoRNR complexes More importantly, they have a dramatic effect

on the initial HD to HD–CoRNR1 ratio (Fig 2A–C) In the presence of a twofold molar excess of BMS493, two ion series are observed The most abundant series (d) has a molecular mass of 59 884.1 ± 1.4 Da corresponding to the binding of one BMS493 molecule [mass difference (DM)

408 Da] to the HD–CoRNR1 complex (Fig 2B) The minor one (r) can be attributed to the fixation of one BMS493 molecule to the free HD No significant signal corresponding to unliganded species is detected Even in the presence of a twofold molar excess of BMS493, no protein/ ligand stoichiometry higher than 1 : 1 is observed, indicating that the specific binding of BMS493 to its cognate receptor

is faithfully revealed by MS and that no nonsite-specific ligand addition resulting from any artefactual aggregation during the electrospray ionization process occurs Similar results were obtained with BMS614, a RARa-selective antagonist, which induced a mass increase of 448 Da for the binding of one BMS614 molecule to the HD and HD– CoRNR complexes (data not shown) As interface condi-tions are optimized so that neither peptides nor ligands dissociate during transfer to the gas phase, we conclude that

Trang 5

the HD–BMS493 complex is present in solution at

equilibrium and does not result from the dissociation of

the HD–BMS493–CoRNR1 complex in the interface of the

mass spectrometer

For comparison of the relative proportions of the

different species present in solution, we made the

assump-tion that ionizaassump-tion efficiencies and response factors of all

species are similar, and thus that relative intensities of the

main charge states can serve to estimate the relative

abundances in solution This assumption is supported by

several points: (a) strictly identical experimental conditions

are used for the addition of each ligand (molar excess,

buffer, pH, Vc, etc.); (b) the mass contribution of the ligand

is very small compared to the mass of the HD–CoRNR

complex, thus similar response factors are expected; (c) all

species exhibit nearly the same charge states and are

detected with the same m/z ratios, suggesting that no strong

discrimination effects of the ion species upon focalization in

the interface of the mass spectrometer should occur Table 1 summarizes the relative abundances of all species in the absence or presence of the corepressor peptide The fact that the relative abundance of the HD–CoRNR1 complex significantly increases in the presence of RAR-antagonist

or inverse agonist ligands (75% and 56% of HD–CoRNR1

in the presence of, respectively, BMS493 or BMS614 instead

of 43% in the absence of ligand) indicates that these RAR-ligands stabilize the HD–CoRNR1 ternary complex Above

60 V (data not shown), the ligand is dissociated from all species in the gas phase while the ratio between HD and HD–CoRNR1 stays unchanged

To compare these data with an analysis of the ligand-induced complex formation in living cells in vivo, we performed mammalian cell two-hybrid experiments in which we monitored the interaction of a VP16 activation domain-linked RARa with a Gal-NCoR hybrid that was capable of activating a 17mer-based luciferase reporter

17+

16+

No ligand

15+

16+

351 Da

15+

17+

3200 3250 3300 3350 3400 3450 3500 3550 3600 3650 3700 3750 3800 3850 3900

m/z

• •

+1 +2 +1

17+

+2 +1 +1

Reference

17+

DM =

DM =

408 Da

16+

Stabilizing Effect

RAR-antagonist BMS493

RAR-agonist AM80

RXR-antagonist HX531

Destabilizing Effect

No apparent Effect

*

*

*

*

*

*

*

*

*

A

B

C

D

Fig 2 Effect of ligand additions on the HD–CoRNR1 ternary complex Positive ESI mass spectra of HD–CoRNR1 The mass spectra were acquired

at Vc ¼ 50 V and at a pressure in the interface of 2.5 mbar (A) Before any ligand addition After addition of twofold molar excess

the HD–CoRNR1 complex is stabilized upon BMS493 binding; (C) AM80: the HD–CoRNR1 complex is completely destabilized upon AM80 binding; (D) HX531: no apparent effect on the stability of the HD–CoRNR1 complex.

Trang 6

(Fig 5) Transcription activation indicated NCoR–RARa

interaction strongly increased upon exposing the cells to

BMS493, while only a weak effect was seen in presence of

BMS614, and little, if any, in the presence of HX531

(Fig 5) We conclude that the MS analysis faithfully

monitors the modulation of receptor–corepressor

inter-action, as it occurs in living cells

After the addition of a twofold molar excess of the

RARa-specific agonist AM80 in the presence of CoRNR1

(Fig 2C), one single ion series (r) displaying a mass

increase of about 351 Da above the molecular mass of the

HD is then observed No ions corresponding to the

ligand-bound HD–AM80–CoRNR1 or to nonliganded HD are

detected Therefore, MS analysis faithfully reveals the

previously observed effects of RAR agonists, antagonists

and inverse agonists on corepressor–HD interaction

Effects of RAR ligand binding on the stability of the HD– CoRNR2 complex ESI mass spectra obtained after the addition of a twofold molar excess of different ligands and CoRNR2 are presented in Fig 3 for the inverse pan-RAR agonist BMS493 (Fig 3B) and the RARa agonist AM80 (Fig 3C) In the absence of ligand a mixture of HD (47%) and HD–CoRNR2 (53%) was observed (Fig 3A) As for CoRNR1, the addition of RAR-specific ligands induces a dramatic change in the relative amount of the various complexes While the agonist AM80 and the antagonist BMS614 induce a complete destabilization of the ternary HD–CoRNR2 complex, no similar effect is exerted by the inverse agonist BMS493 Indeed, 22% of all observed species correspond to the HD–BMS493–CoRNR2 com-plex In all cases, a single ligand molecule is quantitatively bound to the HD complex

3200 3250 3300 3350 3400 3450 3500 3550 3600 3650 3700 3750 3800 3850 3900m/z

16+

17+

+

DM =

DM =

408 Da

17+

15+

17+

16+

351 Da

15+

17+

+2

+2

+2

+2 +1 +1

+2

+1

*

*

*

*

*

*

*

A

B

C

D

Reference

Destabilizing Effect

Destabilizing Effect

No apparent Effect

No ligand

RAR-antagonist BMS493

RAR-agonist AM80

RXR-antagonist HX531

Fig 3 Effect of ligand additions on the HD–CoRNR2 ternary complex Positive ESI mass spectra of HD–CoRNR2 The mass spectra were acquired

at Vc ¼ 50 V and at a pressure in the interface of 2.5 mbar (A) Before any ligand addition After addition of twofold molar excess

the HD–CoRNR2 complex is destabilized upon BMS493 binding; (C) AM80: the HD–CoRNR2 complex is completely destabilized upon AM80 binding (D) HX531: no apparent effect on the stability of the HD–CoRNR2 complex.

Trang 7

Effects of RXR ligands on the stability of HD–CoRNR

complexes When a twofold molar excess of the RXR

antagonist HX531 is added (Figs 2D and 3D), the

inter-pretation of the mass spectra becomes significantly more

difficult because multiple species are detected HD and

ternary HD–CoRNR1 complexes were observed both as

liganded and unliganded species, with the liganded species

involving one or even two HX531 molecules In the presence

of this RXR ligand, no major difference concerning the initial HD to HD–CoRNR1 ratio is observed in the mass spectra The addition of a twofold molar excess of a RXR-specific ligand induces some stabilization of the HD–CoRNR1 complex (51% in the presence of HX531

vs 43% in the absence of any ligand; Table 1) Because of the presence of a second ligand molecule bound to each detected complex, no clear conclusions regarding the influence of a RXR-specific ligand on the stability of the HD–CoRNR1 complex can be drawn

Discussion

ESI-MS allows monitoring of ligand modulations

on HD–CoRNR complexes Due to its ability to detect each species present in solution, ESI-MS appears to be a powerful tool to follow coregulator association and dissociation to NR upon binding of different classes of ligands In addition to previous studies [28,37], we now show that nondenaturaing mass spectro-metry can be used, not only to monitor binding of coregulator peptides but also to assess the effect of ligand binding on the stability of the corresponding NR–coregu-lator complexes

10 The interpretation of the ESI mass spectra provides several facts

(CoRNR1 or CoRNR2) bind to the HD in the absence of any ligand, and (b) ESI-MS data allow one to directly monitor the effect of ligand binding on the association/ dissociation pattern of the heterodimer and the corepressor RAR-specific ligands display a strong influence on the stability of heterodimer–CoRNR complexes, comparing well with previous reports showing that induction of corepressor release is part of the mechanism of action of RAR agonists [1,53] Conversely, the stabilization of the corepressor complex by RAR inverse agonist enhances transcriptional silencing [28,48] These MS data are fully in keeping with the corresponding results obtained by transient transactivation studies Such conclusions are not yet routine and it is of most importance to control that the solution phase image is not distorted during the ESI-MS analysis and that observed peaks on ESI mass spectra in vacuo can

be related to species effectively present in solution

transactivation relative to Am80 10

100

125

75

50

25

0

Ligands [logM]

Gal - RARα + Am80 10–8M

5 x Gal4

Gal4

Luc βG

BMS614 HX531

Fig 4 RARa antagonist potential of synthetic retinoids Transient

transactivation assays were performed to assess the antagonist activity

of BMS493 (j), BMS614 (n), and HX531 (m) COS cells were

co-transfected with reporter (17m)5x-bG-Luc and Gal-RARa The

reporter was activated with 10 n M Am80 (100%) and increasing

con-centrations of the respective retinoid were added, as indicated.

Table 1 Influence of the addition of ligands on the stability of the noncovalent HD–CoRNR complexes The relative abundance of the species are obtained by summing the peak intensities of the three predominant charge states of the complexes Reproducibility of the values is within 2–3%.

No ligand

Relative amounts of complexes detected by ESI-MS (%)

Trang 8

If the observed complex results from specific interactions

in solution, it must be sensitive to modifications of the

experimental conditions affecting its stability The fact that

different ligands induce (a) a substantial change in the mass

spectrum and (b) distinct effects on the stability of

HD–corepressor complexes, provides a good support for

a structurally-specific interaction To establish that the

interactions detected by ESI-MS in presence of RXR

ligands are really the result of in-solution interactions rather

than artefactual nonspecific associations occurring during

the ESI process [54], rigorous control experiments were

performed In particular, separate titration experiments

performed with the RXR ligand and the monomeric RXRa

subunit revealed that, even in the presence of an excess of

ligand (a threefold molar excess), no binding of a second

HX531 molecule is observed on RXRa (data not shown)

At this level, two hypotheses may be advanced to explain

the binding of a second ligand molecule to the heterodimer:

either the second ligand molecule is anchored directly in the

free RAR-binding site [this option is strongly supported by

the antagonistic action of HX531 on AM80-induced

Gal-RARa transactivation (Fig 4)], or the second ligand

molecule is nonspecifically interacting at the protein surface

Because the single RAR subunit tends to precipitate under

ESI-MS compatible conditions, experiments with

mono-meric RAR could not be achieved

Relationship between the type of interactions involved

in the formation of HD–ligand–CoRNR complexes and their gas phase stabilities

The ESI-MS detection of complexes involving few electro-static contacts between the LBP and the ligands is not straightforward As described in the literature [37,43–46], the stability of noncovalent complexes in the ESI-MS process depends strongly on the type of interaction (elec-trostatic contacts, hydrogen bonds, Van der Waals interac-tions) involved in the formation of the complex During ion transfer from the solution to the gas phase both electrostatic interactions and hydrogen bonds are emphasized, while complexes whose formations in solution are mainly driven

by hydrophobic effects appear to be weakened [55,56] The present study shows that HD–CoRNR and HD–ligand noncovalent complexes have different gas phase stabilities The Vc, which controls the energy communicated

to the ions in the interface of the mass spectrometer, needs

to be set to quite low values (30–50 V) to detect the interaction between NRs and ligands, whereas higher Vc values can be used to observe HD–CoRNR complexes, suggesting that these latter complexes are more stable in the gas phase Such behavior is in complete agreement with crystallographic data showing that interactions between NRs and ligands are mainly hydrophobic, whereas NR– CoRNR

12 interactions involve more polar contacts Until now, it has been

contacts involved in a complex formation and its subse-quent MS detection For the receptors of retinoids, the carboxylic group of the ligand is the only moiety engaged in polar interactions with the LBP Two hypotheses can be made to explain the ESI-MS observations: (a) few electro-static contacts are sufficient to maintain the NR–ligand interactions in the gas phase, and (b) the LBP conformation that totally surrounds the ligand prevents its ejection from the pocket Whether dissociation experiments might be used

to directly describe the type of interactions involved in the formation of a given noncovalent complex constitutes a new challenge

Conclusion

In this study, we demonstrate that noncovalent mass spectrometry is a powerful technique to obtain direct evidence of the formation of NR complexes and to monitor the effect of ligand binding on the stability of the resulting complexes Thanks to its unique advantage of providing direct insights into all species present in solution, even moderate changes induced by the addition of a given substrate (ligand, peptides, metallic ions, small organic molecules, etc.) in the association/dissociation pattern can be directly revealed on the ESI mass spectra Therefore, this nondenaturing ESI-MS approach will efficiently comple-ment other structural methods such as NMR and crystal-lography ESI-MS is a technique that can be extended to a wide range of noncovalent protein–protein or protein–ligand complexes Combining rapidity, sensitivity and accuracy, it will play an increasingly important role not only for the analysis of nuclear receptor action and (orphan) receptor ligand detection, but also for transcription regulation in general, which is triggered by noncovalent interactions

10000

15000

5000

0

etOH Am80

BMS493 BMS614 HX531 Gal-NCoR + VP16 - RARα

5 x Gal4

Gal4

Luc βG

NCoR

RAR VP16

Fig 5 Enhanced interaction between RARa and NCoR induced by

synthetic retinoids Mammalian two-hybrid assay with Gal-NCoR as

bait and VP16-RARa as prey were performed to assess the influence of

indicated synthetic retinoids on interaction between RARa and NCoR

in a cellular context.

Trang 9

We are grateful to Pascal Eberling for the synthesis of the CoRNR1

corepression peptide, to Dr Lazar for the kind gift of the CoRNR2

peptide and to Drs Chris Zusi and Koichi Shudo for ligands S.S.

acknowledges the CNRS and Lilly for financial support Work in the

laboratory of H.G is supported by grants from the European

Commission (QLK3-CT2002-02029, HPRN-CT2002-00268), the

Fon-dation de France, the Association for International Cancer Research,

the Association pour la Recherche sur le Cancer, the ULP, the

INSERM, the CNRS, and Bristol-Myers Squibb.

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