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Báo cáo khoa học: Novel a-conotoxins from Conus spurius and the a-conotoxin EI share high-affinity potentiation and low-affinity inhibition of nicotinic acetylcholine receptors doc

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At high concentrations 10 lm, the peptides SrIA, SrIB and [c15E]SrIB showed weak blocking effects only on a4b2 and a1b1cd subtypes, but EI also strongly blocked a3b4 receptors.. Abbrevia

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a-conotoxin EI share high-affinity potentiation and

low-affinity inhibition of nicotinic acetylcholine receptors Estuardo Lo´pez-Vera1,*,†, Manuel B Aguilar1,*, Emanuele Schiavon2, Chiara Marinzi2,

Ernesto Ortiz3, Rita Restano Cassulini2, Cesar V F Batista3, Lourival D Possani3,

Edgar P Heimer de la Cotera1, Francesco Peri2, Baltazar Becerril3and Enzo Wanke2

1 Laboratorio de Neurofarmacologı´a Marina, Departamento de Neurobiologı´a Celular y Molecular, Instituto de Neurobiologı´a, Universidad Nacional Auto´noma de Me´xico, Campus Juriquilla, Queretaro, Me´xico

2 Dipartimento di Biotecnologie e Bioscienze, Universita` di Milano-Bicocca, Milan, Italy

3 Departamento de Medicina Molecular y Bioprocesos, Instituto de Biotecnologı´a, Universidad Nacional Auto´noma de Me´xico, Cuernavaca, Me´xico

Conotoxins are small, disulfide-rich peptides that

have been isolated from Conus, a large genus of

predatory marine snails The primary structures of

more than 100 conotoxins have been determined and classified into gene superfamilies on the basis of the amino acid sequences of the signal peptides of their

Keywords

a-conotoxin; conotoxins; Conus spurius;

nicotinic receptor; potentiation

Correspondence

E Wanke, Dipartimento di Biotecnologie e

Bioscienze, Universita` di Milano-Bicocca,

Piazza della Scienza, 2U3, 20126 Milan, Italy

Fax: +39 02 64483314

Tel: +39 02 64483303

E-mail: enzo.wanke@unimib.it

*These authors contributed equally to this

work

 Present address

Instituto de Ciencias del Mar y Limnologı´a,

Universidad Nacional Auto´noma de Mexico,

Mexico

(Received 4 April 2007, revised 3 June

2007, accepted 11 June 2007)

doi:10.1111/j.1742-4658.2007.05931.x

a-Conotoxins from marine snails are known to be selective and potent competitive antagonists of nicotinic acetylcholine receptors Here we des-cribe the purification, structural features and activity of two novel toxins, SrIA and SrIB, isolated from Conus spurius collected in the Yucatan Chan-nel, Mexico As determined by direct amino acid and cDNA nucleotide sequencing, the toxins are peptides containing 18 amino acid residues with the typical 4⁄ 7-type framework but with completely novel sequences Therefore, their actions (and that of a synthetic analog, [c15E]SrIB) were compared to those exerted by the a4⁄ 7-conotoxin EI from Conus ermineus, used as a control Their target specificity was evaluated by the patch-clamp technique in mammalian cells expressing a1b1cd, a4b2 and a3b4 nicotinic acetylcholine receptors At high concentrations (10 lm), the peptides SrIA, SrIB and [c15E]SrIB showed weak blocking effects only on a4b2 and

a1b1cd subtypes, but EI also strongly blocked a3b4 receptors In contrast

to this blocking effect, the new peptides and EI showed a remarkable potentiation of a1b1cd and a4b2 nicotinic acetylcholine receptors if briefly (2–15 s) applied at concentrations several orders of magnitude lower (EC50, 1.78 and 0.37 nm, respectively) These results suggest not only that the novel a-conotoxins and EI can operate as nicotinic acetylcholine receptor inhibitors, but also that they bind both a1b1cd and a4b2 nicotinic acetyl-choline receptors with very high affinity and increase their intrinsic cho-linergic response Their unique properties make them excellent tools for studying the toxin–receptor interaction, as well as models with which to design highly specific therapeutic drugs

Abbreviations

a1b1cd, muscular nicotinic acetylcholine receptor; a3b4, peripheral nervous system nicotinic acetylcholine receptor; a4b2, central nervous system nicotinic acetylcholine receptor; Acm, S-acetamidomethyl; ACN, acetonitrile; [c15E]SrIB, synthetic a-conotoxin from Conus spurius; nAChR, nicotinic acetylcholine receptor; PTH, phenylthiohydantoin; SrIA, a-conotoxin IA from Conus spurius; SrIB, a-conotoxin IB from Conus spurius.

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precursors In general, the members of each

super-family have a characteristic arrangement of their

cys-teine residues and a particular connectivity of their

disulfide bridges Each gene superfamily comprises

one or more pharmacologic families: the O

super-family, containing x-conotoxins, j-conotoxins,

d-conotoxins, and lO-conotoxins; the M superfamily,

containing l-conotoxins, w-conotoxins, and

jM-cono-toxins; the S superfamily, containing r-conotoxins

and aS-conotoxins; the T superfamily, containing

e-conotoxins and v-conotoxins; the P superfamily,

containing the spasmodic peptides; the I superfamily,

containing several jI-conotoxins, and the A

super-family, containing a-conotoxins, aA-conotoxins and

jA-conotoxins [1]

Competitive antagonists of the nicotinic acetylcholine

receptors (nAChRs) belong to the a and aA families

On the basis of the number of residues between the

sec-ond and third cysteines and on the spacing between the

third and fourth cysteines in the mature a-conotoxins,

these peptides have been divided into three groups: the

a4⁄ 7 subfamily, the a3 ⁄ 5 subfamily, and a

heterogene-ous group including peptides that do not belong to the

two previous groups These groups have different

degrees of antagonistic effect on distinct nAChRs: a3⁄ 5

toxins block mostly muscular nicotinic acetylcholine

receptors a1b1cd subtypes, whereas a4⁄ 7 peptides, with

one exception, block neuronal subtypes [2]

In this article, we describe the purification, amino

acid sequence determination and cloning of the cDNA

encoding two novel peptides, SrIA and SrIB, found in

the venom of Conus spurius The pattern and the

spa-cing of their cysteines indicate that they belong to the

a4⁄ 7 subfamily of conotoxins [3] We also describe a third peptide, [c15E]SrIB, synthesized by substituting glutamate for the c-carboxyglutamate residue and used for comparison together with the a-EI conotoxin from Conus ermineus We showed that results with [c15E]SrIB were not significantly different from those seen with the natural compounds, and then, owing to the limited amounts of the natural toxins SrIA and SrIB, used mainly this synthetic peptide for long-dur-ation electrophysiologic tests

The discovery of new agonists or antagonists is of the utmost importance to widen the understanding of alternative functions of nAChRs, which play a crucial role in cellular and molecular mechanisms underlying brain function

Results

Purification of SrIA and SrIB Fractionation of C spurius venom by HPLC, as des-cribed in Experimental procedures, gave the profile shown in Fig 1A The fractions indicated as SrIA and SrIB were repurified by RP-HPLC, yielding the two pure peptides SrIA and SrIB (Fig 1B,C), named follow-ing the nomenclature proposed by Olivera & Cruz [1]

Amino acid sequences and cDNA cloning Automated Edman sequencing of the native peptides SrIA and SrIB unambiguously defined 12 and 13 resi-dues, respectively Low glutamine signals at positions

12 and 15 of SrIA and at position 15 of SrIB

Fig 1 Purification of SrIA and SrIB (A)

Fractionation of the crude venom by means

of an analytical RP C18 HPLC column

Pep-tides were eluted using a linear gradient of

5–95% solution B (dashed line) at a flow

rate of 1 mLÆmin)1for 90 min Eluents

were: 0.1% v ⁄ v trifluoroacetic acid in water

(solution A), and 0.09% v ⁄ v trifluoroacetic

acid in 90% v ⁄ v ACN (solution B) (B, C)

Fractions indicated in (A) as SrIA and SrIB

were repurified using a gradient of 15–30%

buffer B (dashed line), at a flow rate of

1 mLÆmin)1for 45 min.

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suggested the presence of c-carboxyglutamate residues

at these positions Residues 3, 4, 9 and 17 of both

pep-tides were tentatively assigned as cysteine (Table 1), on

the basis of the absence of any amino acid signal at

these positions This assumption was confirmed

directly by the experiments used to determine disulfide

bridges (see below) We obtained positive results

with PCR amplification of a-conotoxin-type cDNA,

reverse transcribed from C spurius venom duct

total mRNA Two primers known to match the

con-served signal peptide-coding region and the 3¢-UTR of

the a-conotoxin family, respectively [4], were

success-fully employed Exactly the same sequence was

obtained from several colonies, which, together with

the demonstrated conservation of the signal and

pro-peptide regions, indicated that the amplification

proto-col was reliable The deduced SrIA⁄ SrIB precursor

sequence agreed with the results of direct peptide

sequencing and MS data (see below), and allowed us

to define the final unambiguous primary structure for

the mature toxins (Fig 2) From the precursor

sequence, and on the basis of earlier observations by

our group with toxic peptides [5], we were also able to

predict the amidation of the C-terminal end of the

mature toxins The primary structures of SrIA and

SrIB resemble those of previously isolated

a-conoto-xins with the cysteine framework 4⁄ 7 (Table 2)

MS

The chemical monoisotopic molecular masses of

pep-tides SrIA and SrIB determined by ESI MS are

2202.9 Da and 2158.8 Da, respectively (Table 1) The

agreement with the calculated masses (assuming two disulfide bridges and an amidated C-terminus for each peptide, plus one and two c-carboxyglutamate residues for SrIB and SrIA, respectively) supports the Edman sequence assignment for each peptide The tentative assignments of amidated C-termini, based on the struc-ture of the precursor (see ‘cDNA cloning’), were con-firmed by the ESI MS data

Determination of disulfide bridges Two major and more than 20 minor absorbing peaks were observed during the chromatography of peptide SrIA after partial reduction with Tris(2-carboxyethyl) phosphine hydrochloride and alkylation with N-ethyl-maleimide (Fig 3) This high number of derivatives of peptides alkylated with N-ethylmaleimide has been observed in several studies [6], and it is thought to reflect diastereoisomers resulting from the introduction

of a new chiral center in the maleimide ring after for-mation of the S–C bond during alkylation Another factor that could generate additional derivatives is the opening of the ring of the N-ethylsuccinimidocysteines

by hydrolysis [7] Selected peptides were sequenced to reveal the positions of the alkylated cysteines The phe-nylthiohydantoin (PTH) derivative of N-ethylsuccini-midocysteine elutes between PTH-Pro and PTH-Met

in the HPLC system of the sequencer employed The presence of alkylated cysteines at positions 4 and 17 in some peptides, and at positions 3 and 9 in other pep-tides, clearly indicated that the connectivity of the two disulfide bridges in peptide SrIA is of the type I–III, II–IV The absence of peptides with labeled cysteines

at positions 3 and 17 or 4 and 9 gives additional sup-port to the proposed disulfide connectivity

The synthetic peptide [c15E]SrIB

It has been reported recently that the c-carboxygluta-mic residues present in toxin peptides may be involved in the folding process but are not relevant for their biological activity [8] Starting from this hypothesis, a peptide sequence was designed that was analogous to those found for SrIA and SrIB, but bearing glutamic acid residues in place of the

c-carbo-Table 1 Amino acid sequences and monoisotopic molecular

mas-ses of the peptides from C spurius and of synthetic peptides

[c15E]SrIB and EI.

Peptide Sequence

Experimental mass (Da)

Calculated mass (Da) SrIA RTCCSROTCRMcYPcLCG a 2202.9 2202.8

SrIB RTCCSROTCRMEYPcLCG a 2158.8 2158.8

[c15E]SrIB RTCCSROTCRMEYPELCG a 2114.8 2115.0

a

Amidated C-terminus; O, hydroxyproline; c, c-carboxyglutamate.

Fig 2 The cloned cDNA sequence and the deduced amino acid sequence of the SrIA ⁄ SrIB conotoxin precursor The residues present in the mature toxins are underlined.

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xyglutamic residues at positions 12 and 15 (Table 1).

Testing the biological properties of such a peptide,

prepared by chemical synthesis and thus with a fully

defined chemical structure (including disulfide

pat-tern), would support the amino acid sequence and

folding of the native peptides proposed above, and

additional tests would not be limited by the

availabil-ity of the peptide, as might occur with the natural

toxins SrIA and SrIB To obtain the desired folding

pattern (see Experimental procedures), we protected

the cysteine side chains with two orthogonal

protect-ing groups that can be removed selectively under

different conditions, allowing the formation of one

disulfide bridge at a time For this purpose, Cys3 and

Cys9 were introduced as S-trityl-protected amino

acids, whereas S-(acetamidomethyl)cysteine was used

for positions 4 and 17 At the end of chain assembly

on the solid support, achieved using standard

2-(1-H-benzotriazol-1-yl)-1,1,3,3-tetramethyl uronium

hexa-fluorophosphate activation protocols for Fmoc

chemistry as previously described [9,10], the peptide

resin was treated with trifluoroacetic acid for cleavage

from the solid support and side chain deprotection,

with simultaneous liberation of the two thiol groups

in positions 3 and 9 The first disulfide bond was then formed by air oxidation Finally, the bis-aceta-midomethyl-peptide generated was treated with iodine, which caused removal of the protecting group and simultaneous oxidation to disulfide, yielding the fully folded sequence

Physiologic effects of natural conotoxins and their synthetic analogs

In order to explore the physiologic role of the novel SrIA and SrIB conotoxins, we performed a series of patch-clamp experiments on single cells from the line TE671, which expresses the human muscle receptor [11], and HEK293 lines stably transfected with the human central nervous system nicotinic acetylcholine

a4b2 and peripheral nervous system nicotinic acetyl-choline a3b4 receptor subtypes As our present perfu-sion system is not sufficiently fast to resolve fast desensitizing currents such as those produced by a7 re-ceptors, we decided not to test our peptides on these receptors, to avoid reporting putatively invalid data The experiments were done by voltage-clamping the cells at ) 60 mV and comparing the responses to brief

Table 2 Amino acid sequence of SrIA, SrIB and [c15E]SrIB,

com-pared with some members of the a3 ⁄ 5, a4 ⁄ 3 and a4 ⁄ 7 subfamilies

[16,24,48].

Peptide Amino acid sequence Target

GIA ECCNPACGRHYSCGK a a 1 b 1 cd

CnIA GRCCHPACGKYYSC a a 1 b 1 cd >> a 7

ImII ACCSDRRCR-WRC a a 7 , a 1 b 1 > a 3 b 2

AnIB GGCCSHPACAANNQDYC a a3b2>> a7

PnIA GCCSLPPCAANNPDYC a a3b2>> a7

PnIB GCCSLPPCALSNPDYC a a 7 > a 3 b 2

EpI GCCSDPRCNMNNPDYC a a3b4, a3b2; a7

AuIA GCCSYPPCFATNSDYC a a3b4

Vc1.1 GCCSDPRCNYDHPEIC a a 3 a 7 b 4 , a 3 a 5 b 4

PeIA GCCSHPACSVNHPELC a a9a10, a3b2> a3b4> a7

PIA RDPCCSNPVCTVHNPQIC a a 6 ⁄ a 3 b 2 b 3 > a 6 ⁄ a 3 b 4 >

a 6 b 4 , a 3 b 2 GIC GCCSHPACAGNNQHIC a a3b2>> a4b2, a3b4

MII GCCSNPVCHLEHSNLC a a 3 b 2 >> a 7 > a 4 b 2 , a 3 b 4

GID IR G c CCSNPACRVNNOHVC a 3 ⁄ b 2 , a 7 > a 4 ⁄ b 2

EI RDOCCYHPTCNMSNPQIC a a1b1cd, a3b4, a4b2

SrIA RTCCSROTCRMc YPcLCG a a4b2, a1b1cd

SrIB RTCCSROTCRMEYPcLCG a a 4 b 2 , a 1 b 1 cd

[c15E]SrIB RTCCSROTCRMEYPELCG a a4b2, a1b1cd

a Amidated C-terminus; O, hydroxyproline; c,c-carboxyglutamate;

Y, sulfated tyrosine.

Fig 3 Determination of the disulfide bridges of peptide SrIA Deriv-atives of peptide SrIA formed by partial reduction and alkylation under acidic conditions were separated using two analytical RP C18 HPLC columns Peptides were eluted using a linear gradient (dashed line) of 10–30% solution B at a flow rate of 1 mLÆmin)1for

120 min Eluents were: 0.1% v ⁄ v trifluoroacetic acid in water (solu-tion A), and 0.09% v ⁄ v trifluoroacetic acid in 90% v ⁄ v ACN (solu-tion B) Selected peptides were sequenced, and the posi(solu-tions at which cysteines labeled with N-ethylmaleimide were observed are displayed in the corresponding diagrams The deduced connectivity

of the two disulfide bonds is indicated in the upper right inset.

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applications of 50 lm nicotine with those obtained

immediately after pretreatment with the different

tox-ins The concentration of nicotine used during the

experiments was fixed at 50 lm, because this value is

well below the saturating region of the dose–response

curve for the a1b1cd receptor, as shown in Fig 4A,

and also for the a4b2and a3b4receptors [12–14]

The pretreatment time and the concentration of each

toxin were varied in the range 3–150 s and 0.2 nm to

10 lm, respectively A typical experiment performed on

a TE671 cell with a-conotoxin [c15E]SrIB at 1 lm is

shown in Fig 4B As indicated, the first 50 lm nicotine control pulse produced a response that was strongly reduced after 180 s of toxin perfusion After 4 min of washout, the application of an additional nicotine pulse produced a recovery that was complete As the amount

of purified toxins was limited, we did the majority of the experiments with the synthetic toxin [c15E]SrIB and used a known conotoxin [15], such as EI a-conotoxin,

as a control In the case of the inhibitory effects des-cribed in Fig 4, the results obtained using natural or synthetic peptides, at the same concentration, were not

A

D

Fig 4 Blocking properties of a-conotoxins on different types of receptors (A) Dose–response curve obtained with nicotine in TE671 cells The continuous line is the Boltzmann curve that best fits the data with the following parameters: an IC50of 99 ± 12 l M , and a Hill coefficient

of 1.98 ± 0.14 (n ¼ 12) The inset shows a representative example of the recorded currents in a single cell (B) Inward currents recorded in

a TE671 cell during successive 50 l M nicotine (nic) test pulses The first and the last pulse are control and washout, respectively; the second pulse was preceded by an 180 s pretreatment with a-conotoxin [c15E]SrIB (1 l M ) (C) Fractional blockade, at fixed toxin concentration (10 l M for 180 s), on the different subtypes of nAChR *Statistically different at P < 0.05 as compared to a4b2; the numbers of experiments are given in parentheses (D) Normalized time course of the blockade, at 10 l M EI, of the nicotinic response as a function of the toxin pre-treatment time Continuous curves are exponentials that best fit the data points with the following time constants: a1b1cd (open squares), 4.9 ± 0.25 s (n ¼ 5); a 3 b4(gray squares), 11 ± 1.9 s (n ¼ 5) Insets: superimposed traces of the nicotine responses obtained in a typical TE671 cell and in an a 3 b 4 -expressing cell during control and toxin perfusion Left inset: the traces show the block at 30 s and the recovery after 40 s Right inset: the traces show the block at 5 s and the recovery after 20 s Scale bars: 2 s, 200 pA (E) Fractional response data obtained with a-conotoxin [c15E]SrIB and EI The curves are best fitted with the following IC50 and Hill coefficient: for [c15E]SrIB,

46 ± 10 n M , 1 ± 0.1, and for EI, 187 ± 43 n M , 0.48 ± 0.06, respectively The number of experiments for each point ranged from three to 12.

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significantly different, and only the data obtained with

the synthetic toxin are displayed

Inhibitory actions

Figure 4C summarizes the data obtained at high toxin

concentrations (10 lm) It can be seen that the

frac-tional blockade obtained is both receptor-dependent

and toxin-dependent [c15E]SrIB was ineffective on

a3b4receptors (n¼ 4), and was a slightly better blocker

of the a4b2 receptors (0.56 ± 0.04, n¼ 7) than of

the a1b1cd receptors (0.39 ± 0.06, n¼ 5, not

statisti-cally significant) On the other hand, EI toxin was

able to potently block muscle (0.95 ± 0.01, n¼ 5) and

ganglionic (0.91 ± 0.03, n¼ 4) receptors, but was less

potent for the central nevous system receptor

(0.61 ± 0.02, n¼ 4) These data not only confirm that

EI is an inhibitor of muscle receptors [15], but they

also show that it is a strong inhibitor of the a3b4

receptors and a relatively weak antagonist of the a4b2

central nervous system receptors, on which it had

never been tested before

To further investigate these new EI data, we also

performed a series of kinetic experiments at a

concen-tration of 10 lm (see Fig 4D) EI toxin blocked the

a1b1cd receptors with a son of 4.9 ± 0.25 s (n¼ 3),

and the a3b4 receptor was blocked with a son of

11 ± 1.9 s (n¼ 3) Moreover, the soff values that we

observed for these receptors were 150 ± 13 s (n¼ 4)

and 122 ± 6.5 s, respectively Figure 4E shows the

dose–response curve for the [c15E]SrIB and EI

a-cono-toxins on the a1b1cd receptors The estimated IC50

and Hill coefficient obtained from these data are:

46 ± 10 nm and 1 ± 0.1 for [c15E]SrIB, and

187 ± 43 nm and 0.48 ± 0.06 for EI, respectively

Because, at a toxin concentration [T], a simple

Clark’s model receptor theory predicts son¼ soff⁄

(1 + [T]⁄ KD), this relationship can be used to confirm

the previous IC50 of Martinez et al [15] on a1b1cd

receptors, which was 280 nm (low-affinity site) for the

mouse receptors, and to predict the unknown and

novel value of KD for the a3b4 receptors Indeed, we

found an IC50 value of 187 nm for a1b1cd

recep-tors (Fig 4E), which also agrees with the fractional

response of 0.04 at 10 lm EI and a soff of 150 s For

the a3b4 receptors, the above relationship results in a

KDvalue of about 1 lm

On the whole, these experiments, designed to study

the antagonistic properties of the toxin [c15E]SrIB,

showed a narrower spectrum of specificity for nAChRs

than that of the EI a-conotoxin, owing to the null

effect of [c15E]SrIB on the a3b4 subtype In contrast,

EI was found to be a broad-spectrum a-conotoxin

Potentiating effects During the experiments designed to study inhibitory actions of the two new peptides SrIA and SrIB, we dis-covered that brief applications, at low toxin concentra-tions, resulted in increased responses that were immediately reversed after washout of the toxin A typical experiment performed on an a1b1cd-expressing cell with different concentrations of a-conotoxin [c15E]SrIB is shown in Fig 5A It can be seen that the first and the last brief control pulses of 50 lm nicotine produced very similar inward currents However, if

15 s pretreatments with toxin were immediately fol-lowed by the same brief nicotine pulses, currents increased, and then decreased as a function of the drug concentration

In order to shed light on this novel action of the a-conotoxins, we started to investigate whether the various peptides exerted different levels of potentiation

on the same a1b1cd receptor To clarify whether this novel mechanism was peculiar to the new conotoxins

or common also to other, already known, conotoxins,

we chose the EI a-conotoxin, which is considered to be

an inhibitory conotoxin [15]

At a fixed toxin concentration of 10 nm, the relative potentiation, (Itoxin) Icontrol)⁄ Icontrol, of the synthetic [c15E]SrIB, the natural SrIB and SrIA peptides, and the EI a-conotoxin, were as follows: 0.46 ± 0.09 (n¼ 10), 0.47 ± 0.08 (n¼ 10), 0.44 ± 0.15 (n ¼ 9), and 0.54 ± 0.13 (n¼ 9), respectively These results suggest that, at least for the a1b1cd receptor type and during brief periods of time (15 s), pretreatment with a con-centration of 10 nm toxin shows no clear differences among these peptides As the amount of natural toxin available for experimentation is limited, and as no significant differences were found when using the synthetic peptide as compared to the native peptides,

we continued our assays using the two synthetically prepared products, namely [c15E]SrIB and EI The results of these preliminary experiments, obtained only with very low toxin concentrations (0.2 nm to 1 lm) and brief time intervals, do not conflict with those mentioned in Fig 4, which were obtained with very long pretreatments

To investigate these mechanisms, the toxins were studied in cells expressing various receptor types Unexpectedly, we discovered that their effects were also receptor-dependent To clarify the receptor specif-icity, we used the two toxins ([c15E]SrIB and EI) on three different receptors, namely a1b1cd, a4b2, and

a3b4, and the maximally observed relative potentiation values are shown in Fig 5B Interestingly, whereas the toxins were unable to produce potentiation in the

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ganglionic a3b4 receptor (n¼ 17), the mean fractional

potentiation in a1b1cd receptors for [c15E]SrIB

(0.75 ± 0.22, n¼ 7) was higher than that obtained for

EI (0.35 ± 0.07, n¼ 22, statistically different) The

effects of both toxins were found to be similar on the

a4b2receptor subtype

Furthermore, we investigated the dose–response

curves of the maximal fractional potentiation produced

by the [c15E]SrIB and EI conotoxins on the a1b1cd

receptors These data are shown in Fig 5C, and were

fitted to dose–response curves with EC50 values of

1.78 ± 1.9 and 0.37 ± 0.23 nm, for [c15E]SrIB and

EI, respectively An example of this type of action

(15 s toxin pretreatment) is shown in Fig 5D, in one

example of an a1b1cd-expressing cell, with both toxins

at two different concentrations (10 and 100 nm) In this experiment, the two toxins were delivered alter-nately to gain insight into the differences between their sensitivities

The kinetics of the development of the potentiated response were very fast at concentrations higher than 2–5 nm, and it was almost impossible to determine its time course, given that the rate of bath exchange was < 1 s However, by reducing the toxin concentra-tion to 0.2 nm, we were able to follow, as a funcconcentra-tion of the duration of the toxin perfusion, not only the expo-nential increase in potentiation, but also the decay of the potentiation response, up to the appearance of the blockade Indeed, if the pretreatment of the toxin las-ted for more than 10–15 s, it was possible to observe

Fig 5 Potentiation effects of a-conotoxins on different types of receptor (A) Inward currents recorded in a TE671 cell during successive

50 l M nicotine test pulses The first and the last pulse are controls; the second, third, fourth and fifth pulses were each preceded by a 15 s pretreatment with different concentrations of the a-conotoxin [c15E]SrIB (B) Maximal relative potentiation [(Itox) I control ) ⁄ I control ] for different receptor types ([c15E]SrIB, line pattern; EI, gray pattern) The maximal concentration used was 100 n M , and pretreatment lasted for 15 s The number of experiments is shown in parentheses on the bars *Statistically different at P < 0.05 as compared to the EI effect (C) Dose– response relationships for potentiation, observed in a1b1cd receptors, for a-conotoxins [c15E]SrIB (open squares), and EI (gray squares) Con-tinuous lines are dose–response curves fitting the experimental data with the following values of IC 50 (n M ) (maximal): for [c15E]SrIB, 1.78 ± 1.9, 0.93 ± 0.11; for EI, 0.37 ± 0.23 n M , 0.46 ± 0.1 Each point represents a variable number of experiments from three to 11 (D) In the same cell, the two toxins were applied alternately, each for 15 s pretreatment intervals at different concentrations as indicated (E, F) The potentiation ⁄ blockade (open squares) kinetics on a 1 b 1 cd receptors, for [c15E]SrIB (E) at 0.2 n M and EI (F) at 0.2 and 1 n M Continuous curves are exponentials with the following time constants: [c15E]SrIB, s on 7.07 ± 0.1.1, s off 31 ± 2.3 s; EI, s on (0.2 n M ) 6.03 ± 0.32, s off (0.2 n M ) 16.4 ± 1.3 s; soff(1 n M ) 9.4 ± 1.5 s See text.

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an exponentially decaying depotentiation process We

show two examples obtained by using the two different

toxins on the a1b1cd receptor Figure 5E,F shows the

potentiation⁄ blockade (Itoxin⁄ Icontrol) data versus

dur-ation of toxin pretreatment obtained from experiments

done at 0.2 nm [c15E]SrIB or 0.2 and 1 nm EI,

respect-ively (n¼ 3) Note the different time scales in Fig 5E

and Fig 5F Potentiation data at 1 nm are not shown

for [c15E]SrIB, because they were too fast to be

resolved On the contrary, data at 1 nm for EI,

although fast (but not fitted to exponentials), are

shown because they illustrate the interesting

depotenti-ation with a time constant different from that observed

at 0.2 nm From these experiments, it can be seen that

both the development of potentiation and the

depoten-tiation or block are dependent on the toxin type and

concentration These data suggest a very complex

mechanism of toxin–receptor interaction that warrants

additional study Unfortunately, this was beyond the

scope of this study

On the whole, these results suggest that the

potentia-tion described here could be a property of different

clas-ses of a-conotoxins On the other hand, we do not

exclude the possibility that this effect could be confined

to the conotoxins that act on both neuronal and

muscu-lar receptor subtypes, as those used in this work are the

only ones reported to be active on both targets On

the a1b1cd receptor, the synthetic toxin [c15E]SrIB was

less potent than EI, but the latter was less efficient

Discussion

Biochemical characterization of SrIA and SrIB

The primary structures of peptides SrIA and SrIB

iso-lated from the worm-hunting snail C spurius reflect

post-translational modifications of proline and

gluta-mine residues, together with the amidation of the

C-terminus of a shared toxin precursor From analysis

of the cDNA sequence, the C-terminus, including the

last cysteine, is: CGGRR This sequence is typically

present in peptides processed post-translationally

Several rules have emerged from matching the

sequences of the mature peptides with the nucleotide

sequences of the cDNAs encoding scorpion toxins If

one or two basic residues are present at the

C-termi-nus, they are removed post-translationally If a glycine

precedes the basic residue(s), it is used to amidate the

residue preceding the glycine [5] The MS analyses of

toxins SrIA and SrIB showed that these peptides are

in fact amidated

The amino acid sequences indicate that the peptides

share structural features typical of the a-conotoxin

family The two peptides contain four and seven resi-dues between the second and the third cysteines, and between the third and the fourth cysteines, respectively (CCX4CX7C) This spacing defines the subfamily of the a4⁄ 7-conotoxins (Table 2), the most widespread category of nicotinic antagonists present in cone snail venoms [2] The a4⁄ 7-conotoxins have a conserved proline in loop I, which comprises residues between the second and the third cysteines Together with Vc1a [16], peptides SrIA and SrIB are the only known a4⁄ 7-conotoxins in which this constant proline is post-trans-lationally modified to hydroxyproline (Table 2) This derivative has been found in l-conotoxins, x-conot-oxins, j-conotx-conot-oxins, jA-conotx-conot-oxins, aA-conotx-conot-oxins, w-conotoxins, e-conotoxins, v-conotoxins, r-conotox-ins, jM-conotoxr-conotox-ins, d-conotoxr-conotox-ins, and I-conotoxins [17] It was also discovered in the a4⁄ 7-conotoxin GID [18], although not at the conserved proline of loop I Another unusual characteristic of SrIA and SrIB is the presence of c-carboxyglutamate residues This post-translational modification has been described in Conus peptides such as the conantokins, the c-conotoxins, the I-conotoxins, and the e-conotoxins [17], and in the N-terminal region of the a4⁄ 7-conotoxin GID [18] However, Vc1a and peptides SrIA and SrIB are the only a-conotoxins in which c-carboxyglutamate residues occur in loop II, which comprises residues between the third and the fourth cysteines

Peptides SrIA and SrIB have 18 amino acids and an amidated C-terminus They are predicted to have charges of 0 and + 1, respectively, at physiologic pH

It has been pointed out that a-conotoxins specific for neuronal subtypes of nAChR are neutral or negatively charged [19], whereas a-conotoxins that target muscle receptors have a net positive charge [20] Because, according to these authors, peptide SrIA could be considered a potential antagonist of neuronal nAChR, and toxin SrIB a probable antagonist of muscle nAChR, we decided to test peptides SrIA and SrIB in biological preparations separately expressing neuronal (central, a4b2, and ganglionic, a3b4) and muscle (a1b1cd) subtypes of nAChR Unexpectedly, peptides SrIA and SrIB were active on both central and muscle types of the nAChR, which constitutes a novel activity profile of the conserved a4⁄ 7-conotoxin-type scaffold Even more surprising was the finding that peptides SrIA and SrIB have nAChR-potentiating activity, in contrast to all previously studied a4⁄ 7-conotoxins

It has been postulated that divergence within a sin-gle superfamily to produce functionally different famil-ies is one of the neuropharmacologic strategies employed by the Conus genus, and may account in part for its success in nature [21]

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Structure–function relationship for SrIA, SrIB,

and EI

Peptides EI, SrIA and SrIB contain structural

elements of the two types of conotoxins that act

differentially on neuronal and muscle nAChR Toxin

EI [15] (present study) and peptides SrIA, SrIB and

[c15E]SrIB are the only conotoxins with a type I

cysteine scaffold known to act on muscle nAChR

Except for SrIA, they have positive net charges that

might contribute to their activity on muscle receptors

[20], and they (except EI) share with most of the

a3⁄ 5-conotoxins (blockers of a1b1cd nAChR) a

tyro-sine at position 4 of loop II that is not present in

any of the a4⁄ 7 conotoxins known previously

(Table 2) This tyrosine has been found to make an

important contribution to the affinity of toxin MI

for the a⁄ d subunit interface of the muscle nAChR

[22] The three peptides have threonines and

methio-nines at position 4 of loop I and position 2 of

loop II, respectively These residues are not present

at these positions in any of the other a4⁄ 7 toxins

studied to date, with the exception of Met10 in toxin

EpI (Table 2) It seems probable that these

threo-nines and methiothreo-nines are somewhat involved in the

binding and⁄ or activity with muscle nAChR

Alter-natively, the nonpolar methionine residue at position

2 of loop II might be involved in binding to

neuron-al nAChR subtypes, because neuron-all known a4⁄

7-cono-toxins have a nonpolar residue at this position

(Table 2) Peptides EI, SrIA, SrIB and [c15E]SrIB

have very similar hydrophobic aliphatic residues

occupying position 7 of loop II (isoleucine in toxin

EI; leucine in peptides SrIA, SrIB, and [c15E]SrIB);

aliphatic residues (leucine, isoleucine, or valine) also

occur at this position in toxins MII, PeIA, GIC,

Vc1.1, PIA, and GID, which target diverse neuronal

subtypes (including a3b4 and a4b2) with variable

affinities (Table 2) Thus, it is probable that

hydro-phobic aliphatic residues at position 7 of loop II

contribute to the binding and⁄ or activity of peptides

EI, SrIA, SrIB and [c15E]SrIB with a3b4 and⁄ or

a4b2 nAChRs Finally, except for toxin GID,

peptides SrIA, SrIB, and [c15E]SrIB are the only

a4⁄ 7-conotoxins known to have an arginine at

posi-tion 1 of loop II (Table 2) In GID, this residue has

been demonstrated to contribute to the block of the

a4b2 subtype [18], which is consistent with the

biolo-gical activity of peptides SrIA, SrIB and [c15E]SrIB

on a4b2 nAChRs So far, the toxin with the highest

affinity (IC50¼ 152 nm) for the a4b2 subtype is GID,

and it blocks the a3b2 and a7 subtypes with 

40-fold higher affinities [18]

The physiologic role of the SrI and EI a-conotoxins

In the present article, we have defined the weak antag-onist properties of the novel C spurius a-conotoxins, and of a synthetic analog of one of them, on three of the more important types of acetylcholine receptor Moreover, while comparing these properties with those

of the well-known a-conotoxin EI, we discovered that

it has a selectivity spectrum somewhat different from that known previously a-Conotoxin EI had been considered a specific blocker of a1b1cd nAChRs [15,17,23,24], but our results show that it also may block the a3b4and a4b2neuronal subtypes

This part of our results emphasizes the importance of testing conotoxins not only on the expected subtypes of the known molecular target (based on the toxin sequence and on the current pharmacologic knowledge

in the field), but also on other target subtypes and even

on nonrelated targets Recently, toxin ImII has been found to inhibit both a7and a1b1cd nAChRs to similar extents [25], whereas the a3⁄ 5-conotoxin CnIA not only inhibits fetal muscle nAChRs, but also blocks the neur-onal a7subtype, although with an 80-fold lower affin-ity [26] One surprising and distinct activaffin-ity associated with the same protein scaffold of the a4⁄ 7-conotoxins has been reported for toxin q-TIA from C tulipa; it inhibits the a1-adrenoreceptor, and has the same disul-fide connectivity as ‘classic’ a-conotoxins [27] Like toxin q-TIA, which has an extended N-terminal sequence, peptides SrIA and SrIB have sequence fea-tures (hydroxylated proline in loop I and c-carboxyglut-amate residues in loop II) that differ considerably from those of other a4⁄ 7-conotoxins

The second part of our results reveals a novel cono-toxin-induced functional nAChR state consisting of a potentiation of the response; the potentiation can be detected both with the new toxins and with EI It can

be observed and quantitatively characterized at extre-mely low concentrations and with brief applications Interestingly, longer applications produced either a null effect or an inhibitory effect, as expected from the kinetic data shown in Fig 5E,F and the affinity of the inhibitory process, which were evaluated with pro-longed pretreatments

The a-conotoxins described in this communication showed that they can regulate the nAChR response It

is known [28] that nAChRs are subjected to a variety

of actions, including the increase or decrease of the affinity of the receptor for nicotinic ligands, a phenom-enon that may occur in the absence of agonist, and possibly results from stabilization of the desensitized state [29] Numerous examples of positive and negative

Trang 10

allosteric effectors acting at neuronal nAChRs have

been reported, illustrating the importance of the

allos-teric nature of this protein For example, it was shown

that progesterone and 17-b-estradiol act as negative

and positive effectors, respectively, of the a4b2receptor

subtype [30,31] Atropine and zinc are reported to have

similar effects on some nAChRs [32,33], although the

required concentrations of these drugs were higher by

more than two orders of magnitude than those of our

peptides Interestingly, the same mixed partial agonist

and antagonist behavior was observed for the

well-known blocker d-tubocurarine [34] It has been

repor-ted recently [35] that a-conotoxin PnIA and a synthetic

derivative of it ([A10L]PnIA) weakly potentiate

acetyl-choline-activated currents in the wild-type a7 nAChR;

these authors also reported that on mutant (a7-L247T)

receptors, [A10L]PnIA potentiated the

acetylcholine-evoked current and acted as an agonist by itself The

mechanisms involved in these processes may be related

to previous findings that a-conotoxin MI binds to two

distinct sites on the a1b1cd nAChR, one at the ad

interface, and another at the ac (or ae) interface [36]

Concluding remarks

As it is unknown how and where the peptides studied

in this work bind the different receptor assemblies, it is

premature to suggest any hypothesis regarding the

structure–function mechanisms underlying the peptide

binding Single-channel studies are in progress using

mutagenized peptides and cells expressing specific

nAChRs

These peptides are promising tools for studies at a

detailed molecular level of the structure–activity

rela-tionship that underlies the action of the

nAChR-target-ing conotoxins Considering that nAChRs are

implicated in brain diseases such as schizophrenia,

noc-turnal frontal lobe epilepsy [37], and Alzheimer’s

dis-ease, these new peptides are also candidate models to

develop potentially therapeutic drugs of major

import-ance [38]; for example, peptides SrIA, SrIB and

[c15E]SrIB might lead to the development of a4b2

-select-ive enhancers, which are beginning to be discovered [39]

Experimental procedures

Specimen collection and venom extraction

Specimens of C spurius were collected in the Yucatan

Channel, Mexico The venom was obtained by dissection of

the venom ducts The ducts were homogenized in 10 mL of

0.1% v⁄ v trifluoroacetic acid and 40% v ⁄ v acetonitrile

(ACN) The homogenate was centrifuged at 17 000 g for

30 min at 4C using a Beckman Coulter Avanti J20 centri-fuge with JA-20 rotor The supernatant, containing the pep-tides, was subsequently processed

Peptide purification by RP-HPLC HPLC was performed on an Agilent 1100 Series LC System (G1322A Degasser, G1311A Quaternary Pump, G1315B Diode Array Detector, G1328A Manual Injector; Hewlett-Packard, Waldbronn, Germany) The venom extract was fractionated with a Vydac (Toluca, Mexico) C18 analytical reverse-phase column (218TP54, 5 lm, 4.6· 250 mm) equipped with a Vydac C18 guard column (218GK54, 5 lm, 4.6· 10 mm) Peptides were eluted with a linear gradient of 5–95% solution B

at a flow rate of 1 mLÆmin)1over 90 min, where solution A is 0.1% v⁄ v aqueous trifluoroacetic acid and solution B is 0.09%

v⁄ v trifluoroacetic acid in 90% v ⁄ v aqueous ACN The same column was also employed to repurify the components of the venom, using a linear gradient of 15–30% of solution B at a flow rate of 1 mLÆmin)1for 45 min

Amino acid sequence Peptides were adsorbed onto polybrene-treated (Biobrene Plus; Applied Biosystems, Foster City, CA) glass fiber fil-ters, and the amino acid sequence was determined by auto-mated Edman degradation using an automatic instrument (Procise 491 Protein Sequencing System; Applied Biosys-tems) by the pulsed-liquid method

MS analysis Native peptides were applied directly into a Finnigan LCQDUO ion trap mass spectrometer (Finnigan, San Jose, CA) The LCQ mass spectrometer is coupled to a Surveyor syringe pump delivery system The eluate at 20 lLÆmin)1 was split to allow only 5% of the sample to enter the nano-spray source (1.0 lLÆmin)1) The spray voltage was set to 1.6 kV, and the capillary temperature was set to 130C All spectra were obtained in the positive-ion mode The acquisition and deconvolution of data were performed with xcalibursoftware (Thermo Electron Corp., Nashville, TN)

on a Windows NT PC system

Determination of disulfide bridges The connectivity of the cysteines of toxin SrIA was deter-mined by partial reduction with Tris(2-carboxyethyl) phos-phine hydrochloride and alkylation with N-ethylmaleimide The peptide (11.8 nmol) was dissolved in 10 lL of denatur-ing buffer (0.1 m sodium citrate containdenatur-ing 6 m guanidine hydrochloride, pH 3.0), and 27 lL of 0.1 m Tris(2-carboxy-ethyl) phosphine hydrochloride in the same buffer was added The mixture was incubated for 15 min at room

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