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A sub-strate molecule first binds to the peripheral site PAS at the entrance of the gorge [3] and slides down to the acylation site CAS, where it is hydrolyzed and the products escape the

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Drosophila melanogaster acetylcholinesterase active site gorge by enlarging a back channel

Florian Nachon1, Jure Stojan2and Didier Fournier3

1 De´partement de Toxicologie, CRSSA, Grenoble, France

2 Institute of Biochemistry, Medical Faculty, Ljubljana, Slovenia

3 IPBS, Universite´ Paul Sabatier ⁄ CNRS, Toulouse, France

Acetylcholinesterase (EC 3.1.1.7) is a serine hydrolase

that catalyzes the cleavage of acetylcholine Structural

studies have revealed that its active site is buried in a

20 A˚ deep gorge with a bottleneck [1] According to

a recently developed kinetic model, substrate and

product molecules follow the same path [2] A

sub-strate molecule first binds to the peripheral site (PAS)

at the entrance of the gorge [3] and slides down to

the acylation site (CAS), where it is hydrolyzed and

the products escape the gorge via the entrance The

active site gorge is too narrow to allow the crossing

between a substrate molecule en route to the CAS

and a product molecule en route to the exit

Conse-quently, at very high substrate concentrations, there

is a traffic jam preventing the exit of the reaction

product through the main entrance, resulting in

inhi-bition [4]

However, molecular dynamics experiments have pro-vided evidence for a loop movement leading to the for-mation of a back door suitable for product exit [5] Locking the loop with salt or disulfide bridges [6,7] had no significant effect on the kinetics parameters, indicating that exit through the back door is not the main exit route for the product However, residual activity upon fasciculin binding suggests that the back door route might become the most important route when the main entrance is blocked [8,9] Recent kinetic crystallography studies provide some structural insights regarding the putative backdoor Conformation changes of Trp84, which belongs to the backdoor region of Torpedo californica acetylcholinesterase, sug-gest that this residue might behave like a revolving door [10] In addition, Nachon et al [11] reported that the back door region of the Drosophila

acetylcholines-Keywords

acetycholinesterase; back door; inhibition;

substrate; traffic

Correspondence

F Nachon, Unite´ d’enzymologie,

De´partement de Toxicologie, Centre de

Recherches du Service de Sante´ des

Arme´es (CRSSA), 24 Avenue des Maquis

du Gre´sivaudan, 38700 La Tronche, France

Fax: +33 476636962

Tel: +33 476639765

E-mail: fnachon@crssa.net

(Received 18 December 2007, revised 14

March 2008, accepted 18 March 2008)

doi:10.1111/j.1742-4658.2008.06413.x

To test a product exit differing from the substrate entrance in the active site of acetylcholinesterase (EC 3.1.1.7), we enlarged a channel located at the bottom of the active site gorge in the Drosophila enzyme Mutation of Trp83 to Ala or Glu widens the channel from 5 A˚ to 9 A˚ The kinetics of substrate hydrolysis and the effect of ligands that close the main entrance suggest that the mutations facilitate both product exit and substrate entrance Thus, in the wild-type, the channel is so narrow that the ‘back door’ is used by at most 5% of the traffic, with the majority of traffic pass-ing through the main entrance In mutants Trp83Ala and Trp83Glu, ligands that close the main entrance do not inhibit substrate hydrolysis because the traffic can pass via an alternative route, presumably the enlarged back channel

Abbreviations

CAS, acylation site; DmAChE, Drosophila acetylcholinesterase; PAS, peripheral site.

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terase (DmAChE) is much less stabilized than that of

other cholinesterases, such as Torpedo californica

acetylcholinesterase Indeed, two key residues for the

stabilization of Trp84 are not conserved in DmAChE:

Met83, which stabilizes Trp84 in the Torpedo enzyme

through sulfur-p interactions, is replaced by an

isoleu-cine; Tyr442, which hydroxyl bridges Trp84 to Trp432

and Gly80 via hydrogen bonding, is replaced by an

aspartate that is also much less bulky (Fig 1) In the

absence of these stabilizing elements, Trp83 of

DmAChE is prone to oscillations between two

alter-nate conformations, as shown by the crystal structures

(protein databank codes 1DX4 and 1QO9) One of these conformations results in the formation of a chan-nel approximately 5 A˚ in diameter, connecting the gorge to the bulk solvent (Fig 2A)

The present study aimed to progressively enlarge this channel by mutating Trp83 to Tyr, Glu or Ala to test

Fig 1 View of the back door region from the outside of DmAChE

(residues and labels in green) and Torpedo californica

acetylcholin-esterase (residues and labels in fushia) Residues are represented

by sticks The hydrogen bonds involving the hydroxyl of Tyr442 are

indicated by a yellow dash.

A

B

Fig 2 View of the back channel from the active site gorge of wild-type DmAChE (A) and Trp83Ala mutant (B) The protein databank code for wild-type DmAChE is 1DX4 Residues delimiting the hole are represented by sticks The solvent accessible surface is repre-sented by a mesh.

E

K p

SpE

SpE

K L

EAS

K p

k 2

b k 2

k 3

E

a k 3

K p

K L L

Choline

Choline

SpEAS

K p

Acetate

Acetate

S

Scheme 1 Reaction scheme for the hydro-lysis of acetylthiocholine by DmAChE S, acetylthiocholine; E, free enzyme; EA, acetylated enzyme All other intermediates represent enzyme–substrate complexes and the subscript ‘p’ denotes the substrate bound to the peripheral anionic site.

Trang 3

the effect of an open back door on the kinetics for

substrate hydrolysis

Results and Discussion

The effect of substrate concentration on

acetylthiocho-line hydrolysis for the four proteins is shown in Fig 3

These data were fitted using Scheme 1, which permits

the description of substrate activation and inhibition

in a manner consistent with the structural data

[4,12] The values of the parameters for hydrolysis of

acetylthiocholine by wild-type DmAChE (Table 1) are

strongly restrained because they were deduced from

analysis of inhibition of substrate hydrolysis and

accel-eration of decarbamoylation by substrate analogue [2],

inhibition of substrate hydrolysis by reversible

inhibi-tors [13–17], hydrolysis of substrate at different

tem-peratures [18] and hydrolysis of different substrates

[19] For the purposes of alternative substrate traffic in

and out of the active site of DmAChE, the mutation

to Tyr had no significant effect The pS curves (i.e

curves showing enzyme activity at different substrate

concentrations) for acetylthiocholine hydrolysis by Glu

and Ala mutants, however, were shifted to higher

concentrations of substrate and became symmetric

Consequently, the best fits (Fig 3) were obtained by

assuming that mutations did not affect binding of

substrate at the peripheral site (Kp), the rate constant

for deacetylation (k3) and the acceleration of

deacety-lation (a) Any other assumption resulted in an

unsta-ble fitting Consistently, it appears that substitution of

Trp83 by smaller side chains did not affect

deacetyla-tion parameters k3 and a because the amino acid at

position 83 is too far away from the activated water

molecule during deacetylation As expected, the main

difference is the affinity for the catalytic site Kc

(= Kp· KL) because Trp83 is the main component of

substrate stabilization at the catalytic site via cation-p

interaction with the quaternary ammonium moiety of

acetylthiocholine This is consistent with the high

apparent Kmreported for the same mutation in human

acetylcholinesterase [20,21], although the difference appears at different magnitudes In addition, mutation

of this Trp to Glu or Ala decreased acylation (k2),

as reported for human butyrylcholinesterase [22] Acet-ylation may be subdivided into three steps: accommo-dation of substrate at the CAS, chemical transesterification and choline exit In regard to the effect on affinity, we can hypothesize that mutations modify accommodation of the substrate

Another striking difference is parameter b, which represents the effect of substrate bound at the periph-eral site on acylation and choline exit (Scheme 1) Parameter b for the wild-type enzyme is estimated at 0.050 ± 0.025 (i.e acylation step is reduced to 5% when a substrate molecule is bound to the PAS) The traffic of choline outside the gorge is blocked when the PAS is occupied and choline stays inside the active site, resulting in inhibition [9] Parameter b is signifi-cantly different from zero, and no combination of parameters leading to a satisfactory fit can be obtained

if b is restrained to zero This suggests that an alterna-tive exit for choline may exist when the PAS is occu-pied by a substrate molecule but would account for approximately 5% of choline traffic Factor b for the Trp83Ala mutant is estimated at 1.05 (Table 1) The acetylation step is not reduced in this mutant, suggest-ing that choline can freely exit despite the entrance of the gorge being occupied by a substrate molecule This

is expected because mutation Trp83Ala enlarges the channel by up to 9 A˚, thus facilitating the passage of choline (Fig 2B) In the case of the Glu mutation, the

b value is linked to the k2 value and thus both cannot

be estimated independently However, if b is set to 1 (i.e the symmetry of the pS curve supporting it), the

Table 1 Kinetics parameters obtained for the various mutants.

k2(s)1) 52 000 ± 26 000 1818 ± 130 689 ± 30

a

Parameters restrained in the simulation.

10 100 1000 10 000 100 000 0

200 400 600 800

1000

W Y E A

ATCh (µ M )

Fig 3 Activity of the wild-type (W) and mutated DmAChEs (Y, E, A) at different acetylthiocholine concentrations (pS curves) Theoret-ical curves were calculated according to the Scheme 1 specific rate equation, using the corresponding kinetic parameters from Table 1.

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fit is satisfactory This suggests that choline can exit

freely through the back channel as in the alanine

mutant

According to Scheme 1, inhibition by excess of

substrate does not only originate from inhibition

of choline exit (b < 1), but also from inhibition of

deacetylation following the sliding of a molecule of

substrate inside the acetylated active site and

occupa-tion of the peripheral site by a second substrate

mole-cule (complex SpEAS does not deacetylate in

Scheme 1) This mechanism was suggested by excess

substrate inhibition of decarbamoylation and the

crys-tal structure of the SpEAS obtained by soaking crystals

in a solution containing a high substrate concentration

[2,4] This second mechanism remains active in the Glu

and Ala mutants because inhibition was observed at a

high substrate concentration (Fig 3) We observed a

shift of the pS curve towards higher substrate

concen-trations due to the lower affinity of both the free and

acetylated mutated active sites

If choline could leave the active site by the back

channel, we might also hypothesize that acetylcholine

enters using the same path To test this hypothesis, we

used two inhibitors specific for the peripheral site that

bind to Trp321 close to the entrance: propidium and

aflatoxin B1 In the wild-type DmAChE, the affinity of

propidium for the peripheral site is estimated to be

80 pm, and the affinity for aflatoxin to be 3.5 lm,

when considering competition between the substrate

and inhibitor only at the PAS (Fig 4A) However,

inhibition is completely abolished by the substitution

of Trp83 by Ala or Glu It should be strongly

empha-sized at this point that, according to the proposed

reaction scheme (Scheme 1) enlarged by the binding of

inhibitor to the PAS, inhibition at low substrate

con-centrations should always be observed Therefore, the

complete absence of inhibition by peripheral ligands

does not originate from changes in substrate hydrolysis

parameters (Table 1), and the simulation can readily

confirm this The loss of inhibition following

muta-tions of Trp83 might be interpreted as a strong

decrease in affinity of ligand for the peripheral site,

resulting from a hypothetic allosteric interaction

[23,24] However, binding to the peripheral site was

not affected by mutations, as demonstrated by changes

in fluorescence Furthermore, considering that

inhibi-tion arises because inhibitors bound to the PAS hinder

the entrance of acetylcholine to the CAS in the

wild-type enzyme, it appears that, in the two mutants

(Trp83Ala⁄ Glu), inhibitors that bind to the PAS did

not prevent the entrance of substrate into the active

site At this point, a plausible explanation is that the

substrate may enter by an alternative route (i.e the

back channel at the bottom of active site) This hypothesis is in accordance with reported results observed with Trp83Ala mutants: the strong decrease

of inhibition of propidium [21] and the increase of remaining activity upon peripheral site saturation by fasciculin [24]

Finally, minor deviations of pS curves upon binding

of inhibitors on the PAS (Fig 4B,C), may be assigned

10 100 1000 10 000 100 000 0

200 400 600 800

1000

A

B

C

prop10 µ M

Afl50 µ M

ref prop1 µ M

prop10 µ M

Afla10 µ M

Afla50 µ M

prop1 µ M

prop10 µ M

Afla10 µ M

Afla50 µ M

ref

10 100 1000 10 000 100 000

0 100 200 300 400

0 200 400 600

ref

–1 )

–1 )

–1 )

Fig 4 Effect of closing the entrance of the active site with ligands (propidium or aflatoxin) on activity of wild-type DmAChE (A), Trp83Ala (B) and Trp83Glu (C) mutants.

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to an allosteric interaction between PAS and CAS

[8,21,25], to a lower efficiency of the alternative route

compared to the main entrance, or to a partial overlap

of the side chain of propidium and aflatoxin with the

active site as it may span into the gorge

Experimental procedures

Protein production and purification

Mutations were introduced by site-directed mutagenesis

using the QuickChange XL kit following the

manufac-turer’s instructions (Stratagene, La Jolla, CA, USA) The

cDNA encoding DmAChE and mutants were expressed

with the baculovirus system [26] We expressed a soluble

dimeric form deprived of a hydrophobic peptide at the

C-terminal and with a 3· histidine tag replacing the loop

from amino acids 103–136 This external loop is at the

opposite side of the molecule with respect to the active

site entrance and its deletion does not affect the activity

or the stability of the enzyme Secreted

acetylcholinesteras-es were purified to homogeneity using the following steps:

ammonium sulfate precipitation, ultrafiltration with a

10 kDa cut-off membrane, affinity chromatography with

procainamide as a ligand, nitrilotriacetic acid-nickel

chro-matography and anion exchange chrochro-matography [27]

Residue numbering follows that of the mature protein

The concentrations of the enzymes were determined by

active site titration using high affinity irreversible

inhibi-tors [28]

Enzyme activity

Data acquisition and kinetics were performed with the

sub-strate acetylthiocholine as previously described [18] Briefly,

the enzymatic and non-enzymatic hydrolysis of

acetylthi-ocholine by the wild-type DmAChE and its three W83

mutants was followed using Ellman’s method [29] The

initial rate measurements were performed at

acetylthiocho-line concentrations from 2 lm to 500 mm in the absence

and presence of two ligands known to close the entrance of

the active site We used 1 and 10 lm propidium and 10 and

50 lm aflatoxin The activity was followed for 1 min after

the addition of acetylcholinesterase to the mixture, and the

spontaneous hydrolysis of the substrate was subtracted, if

present Each measurement was repeated at least four

times The experiments were carried out at 25C in 25 mm

phosphate buffer (pH 7.0) without ionic strength

compensa-tion to avoid interference with electrostatic components of

binding and chemical steps of the reaction Analysis of the

kinetics data were performed using gosa-fit, software that

is based on a simulated annealing algorithm (BioLog,

Toulouse, France; http://www.bio-log.biz) For analysis of

initial rate data in the absence of inhibitors, we used the

specific equation in Scheme 1 The effect of two ligands on the activity of the wild-type DmAChE was evaluated by the equation in Scheme 1 enlarged by the intermediates, repre-senting the competition between the substrate and inhibitor

at the peripheral site [2]

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