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Elements of the C-terminal t peptide of acetylcholinesterase that determine amphiphilicity, homomeric and heteromeric associations, secretion and degradation Ste´phanie Belbeoc’h, Cinzia

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Elements of the C-terminal t peptide of acetylcholinesterase that determine amphiphilicity, homomeric and heteromeric associations, secretion and degradation

Ste´phanie Belbeoc’h, Cinzia Falasca, Jacqueline Leroy, Annick Ayon, Jean Massoulie´ and Suzanne Bon Laboratoire de Neurobiologie Cellulaire et Mole´culaire, CNRS UMR 8544, Ecole Normale Supe´rieure, Paris, France

The C-terminal t peptide (40 residues) of vertebrate

acetyl-cholinesterase (AChE) T subunits possesses a series of seven

conserved aromatic residues and forms an amphiphilic

a-helix; it allows the formation of homo-oligomers

(mono-mers, dimers and tetramers) and heteromeric associations

with the anchoringproteins, ColQ and PRiMA, which

contain a proline-rich motif (PRAD) We analyzed the

influence of mutations in the t peptide of Torpedo AChETon

oligomerization and secretion Charged residues influenced

the distribution of homo-oligomers but had little effect on

the heteromeric association with QN, a PRAD-containing

N-terminal fragment of ColQ The formation of

homo-tetramers and QN-linked tetramers required a central core of

four aromatic residues and a peptide segment extending to

residue 31; the last nine residues (32–40) were not necessary,

although the formation of disulfide bonds by cysteine C37

stabilized T4and T4–QNtetramers The last two residues of the t peptide (EL) induced a partial intracellular retention; replacement of the C-terminal CAEL tetrapeptide by KDEL did not prevent tetramerization and heteromeric association with QN, indicatingthat these associations take place in the endoplasmic reticulum Mutations that disorganize the a-helical structure of the t peptide were found to enhance degradation Co-expression with QN generally increased secretion, mostly as T4–QNcomplexes, but reduced it for some mutants Thus, mutations in this small, autonomous interaction domain bringinformation on the features that determine oligomeric associations of AChETsubunits and the choice between secretion and degradation

Keywords: acetylcholinesterase; degradation; disulfide bonds; oligomerization; secretion

In vertebrates, the acetylcholinesterase (AChE) gene

gener-ates several types of catalytic subunits through alternative

splicingin the 3¢ region of the transcripts [1–3] These

subunits possess the same common catalytic domain,

followed by distinct C-terminal peptides (r, h and t),

characterizingthe AChER, AChEH and AChET variants

[4–6] In mammals, AChERsubunits seem to be expressed

mostly duringembryogenesis and in the brain after stress

[7,8]; they correspond to a soluble, monomeric enzyme

species AChEHsubunits possess one or two cysteines and a

GPI-addition signal in their C-terminal peptide: they

generate GPI-anchored, disulfide-linked dimers, which

represent a major fraction of AChE in Torpedo electric

organs and muscles, and are expressed on the surface of blood cells in mammals [9–12] AChET subunits are expressed in muscles and in the nervous system of higher vertebrates and therefore represent the functional cholinest-erase species in the cholinergic system [3,13,14]

The C-terminal t peptide confers several characteristic properties to AChET subunits, allowingthem to form a series of homo-oligomers (monomers, dimers, tetramers and higher oligomers) when expressed in transfected COS cells [13,15]; some of these molecules are amphiphilic, i.e interact with detergent micelles [16,17] AChETsubunits also form hetero-oligomers with the collagen, ColQ, or with the transmembrane protein, PRiMA [18,19]; in mammals, these structural proteins anchor the major functional species

of cholinesterases in the basal lamina of the neuromuscular junction and in neuronal cell membranes, respectively [20,21]

In the collagen-tailed and hydrophobic-tailed forms, four catalytic AChE subunits are associated, through their C-terminal t peptides, with proline-rich attachment domains (PRAD) localized in the N-terminal regions of ColQ or PRiMA [19,22,23]

The t peptide of AChE consists of 40 residues, with a series

of seven strictly conserved aromatic residues, includingthree evenly spaced tryptophans, as well as acidic and basic residues that are conserved or semiconserved in most vertebrates [5] This peptide is necessary for the amphiphilic properties which characterize AChET subunits and some

of their oligomers (Ta, Ta, Ta), for the formation of nonamphiphilic homotetramers (Tna), as well as for the

Correspondence to S Bon, Laboratoire de Neurobiologie Cellulaire et

Mole´culaire, CNRS UMR 8544, Ecole Normale Supe´rieure,

46 rue d’Ulm, 75005 Paris, France.

Fax: + 33 1 44 32 38 87, Tel.: + 33 1 44 32 38 91,

E-mail: jean.massoulie@biologie.ens.fr

Abbreviations: AChE, acetylcholinesterase; AChE H , AChE subunit of

type H; AChE R , AChE subunit of type R; AChE T , AChE subunit

of type T; ERAD, endoplasmic reticulum associated degradation;

PRAD, proline-rich attachment domain; r, h, t, alternative C-terminal

peptides of AChE; WAT, tryptophan (W) amphiphilic tetramerization

domain.

Enzymes: acetylcholinesterase (E.C 3.1.1.7).

(Received 8 January 2004, revised 20 February 2004,

accepted 24 February 2004)

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heteromeric association of AChET subunits with QN, an

N-terminal fragment of collagen ColQ that contains a

proline-rich motif, thus producingT4–QNcomplexes [23,24]

The t peptide constitutes an autonomous interaction

domain, called the WAT [tryptophan (W) amphiphilic

tetramerization] domain, because it can associate with a

PRAD, even in the absence of the catalytic domain;

moreover, addition of a t peptide at the C-terminus of

foreign proteins, green fluorescent protein and alkaline

phosphatase, endowed them with amphiphilic properties

and enabled them to form PRAD-associated tetramers [25]

We also found that the simultaneous presence of the t

peptide and of mutations at the interface of AChE dimers –

the four helix bundle [26] – prevented the secretion of

AChET subunits [27] We recently showed that the t

peptide induces intracellular degradation through the

endoplasmic reticulum associated degradation (ERAD)/

proteasome pathway, to different extents, dependingon the

protein to which it is attached, and that aromatic residues

are necessary for this effect [28]

Recent spectroscopic studies showed that the t peptide is

organized as an amphiphilic a helix, in which aromatic

residues form a hydrophobic sector [29,30] In addition,

an analysis of intercatenary disulfide bonds in the T4–QN

complex also demonstrated that the four t peptides are

parallel and oriented in the same direction, opposite to that

of the PRAD [30] The structure of a complex formed

between synthetic peptides (four t peptides with one PRAD)

confirmed this orientation (M Harel et al., manuscript in

preparation)

In the present study, we mutated aromatic and charged

residues, suppressed the C-terminal cysteine or introduced

cysteines at other positions, and deleted more or less extended

C-terminal segments of the t peptide, in Torpedo AChET

subunits, to determine the structural basis of the

character-istic properties that the t peptide confers to AChETsubunits

Materials and methods

AChE constructs and site-directed mutagenesis

Mutagenesis was performed according to the method of

Kunkel et al [31] cDNAs encodingwild-type and mutated

Torpedo AChET, as well as the previously described

Torpedo QN protein [23], intact or deleted of its PRAD

motif (residues 70–86), were inserted into the pEFBos

vector Throughout this article, the residues of the t peptide

are numbered from 1 to 40, correspondingto positions

536–575 in the Torpedo AChETsubunit, so that the Torpedo

mutants are indicated by the modified residues, e.g W17P

Transfection of COS cells

COS cells were transfected by the DEAE-dextran method,

as described previously [24], using4 lgof DNA encoding

the AChE catalytic subunit and 4 lgof DNA encodingQN

or PRAD-deleted QN, per 100 mm dish Because Torpedo

AChE folds into its active conformation at 27C, but not at

37C, the cells were incubated for 2 days at 37 C after

transfection, then transferred to 27C and maintained at

this temperature for 3–4 days, in a medium containing10%

Nuserum (Inotech, Dottikon, Switzerland), which had been

pretreated with 10)6Msoman to inactivate serum cholin-esterases

To analyze its heteromeric interaction with an associated structural protein, AChET was coexpressed with QN[23]

By usingQN, rather than full-length ColQ, we avoid the complexity caused by the formation of the triple helical collagen and by the low salt aggregation of collagen-tailed AChE forms [32] We added a flagepitope (DYKDDDDK) at the C-terminus of QN, so that complexes containingthis protein could be characterized with the anti-flagimmunoglobulin, M2 (Kodak), as described previously [24] The effect of QNon the level of cellular and secreted activity was analysed by comparing the coexpression of AChETwith full-length QNand with a PRAD-deleted QN, to compensate for competition between the two transfected vectors

Cell extracts The cells were extracted at 20C with TMgbuffer (1% Triton X-100, 50 mM Tris/HCl, pH 7.5, 10 mM MgCl2), and then centrifuged at 10 000 g for 30 min Media were also centrifuged at 10 000 g for 30 min to remove cell debris before analysis

Enzyme assays AChE activity was determined accordingto the colorimetric method of Ellman et al [33] at room temperature As the monomeric Torpedo AChE forms produced by some mutants were inactivated by 5,5¢-dithiobis(2-nitrobenzoic acid) [34], the enzyme samples were incubated for variable periods of time, dependingon their activity, with a reaction medium containingacetylthiocholine iodide in phosphate buffer, pH 7; 5,5¢-dithiobis(2-nitrobenzoic acid) was then added and the absorbance at 414 nm was determined using

a Labsystems (Helsinki, Finland) Multiskan RC automatic plate reader Alkaline phosphatase and b-galactosidase from Escherichia coli were assayed with the chromo-genic substrates p-nitrophenyl phosphate and o-nitrophenyl galactoside, respectively

Sedimentation and electrophoretic analyses Centrifugation was performed in 5–20% sucrose gradients (50 mM Tris/HCl, pH 7.5, 50 mM MgCl2, either in the presence of 0.2% Brij-97 or in the presence of 0.2% Triton X-100) in a Beckman SW41 rotor, at 36 000 r.p.m., for 18 h

at 6C The gradients contained E coli b-galactosidase (16 S) and alkaline phosphatase (6.1 S) as internal sedi-mentation standards [24] Amphiphilic molecules generally sediment faster in the presence of Triton X-100 than in the presence of Brij-97, providingan indication of their amphiphilic character

Electrophoresis in nondenaturatingpolyacrylamide gels was performed as described by Bon et al [16], and AChE activity was revealed by the histochemical method of Karnovsky & Roots [35] In charge shift electrophoresis, the electrophoretic migration of amphiphilic molecules was accelerated in the presence of sodium deoxycholate, when compared to migration in the presence of the neutral detergent, Triton X-100, alone As an index of the degree of

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amphiphilicity, we used the ratio between migration in the

presence of DOC to migration in Triton X-100 alone, after

normalizingthese migrations to that of a nonamphiphilic

species, the wild-type tetramers T4naor T4–QN

Both sedimentation and nondenaturingelectrophoresis

provide semiquantitative information on the interaction of

AChE molecules with micelles, and are generally in

complete agreement However, in the present study, we

found that some mutations in the t peptide perturb

amphiphilic interactions in such a way that sedimentation

became essentially identical in the presence of Triton X-100

and Brij-97, while charge shift electrophoresis still showed a

marked influence of the detergent: this was the case for

dimers of aromatic mutants such as W17H or W17A In

addition, the T4–QNcomplexes formed by mutants W17F

and W17A showed an unusual retardation in sedimentation

in the presence of Triton X-100, compared with Brij-97

Results

Analyses of AChE activity and molecular forms

Figure 1 shows the sequence of the t peptide of Torpedo

AChETsubunits, and schematically illustrates its proposed

a helical structure, its association with the PRAD of

ColQ, and the various oligomers of AChETsubunits that

result from its interactions

We analyzed how mutations in the t peptide affect the

levels of cellular and secreted activity of Torpedo AChE in

transfected COS cells The activities were normalized to

those obtained for wild-type AChETin parallel transfections

Immunofluorescence of the protein produced at early stages

after transfection indicated that all mutants were expressed

in a similar manner After 2 days at 27C, a temperature

which allows the correct foldingof active Torpedo AChE

(see the Materials and methods), the level of cellular activity

reached a plateau and the rate of secretion remained

constant Maximal secretion was obtained for a truncated

mutant (I3C/stop4), which retained only the first two

residues of the t peptide, followed by a cysteine at position

3; this cysteine allowed the formation of dimers, which

lacked the aromatic residues and were therefore

nonamphi-philic The secretion of active wild-type AChET subunits

was less than 10% of the truncated mutant, showingthat a

large fraction is degraded intracellularly [27,28]

The molecular forms of AChE were identified by

electrophoresis in nondenaturingpolyacrylamide gels and

their amphiphilic character was evaluated by charge shift

electrophoresis in the presence or absence of sodium

deoxycholate [16] As Torpedo AChET monomers are

rapidly inactivated under the conditions of electrophoretic

migration, the distribution of AChE molecular forms was

analyzed by sedimentation in sucrose gradients

To analyse the capacity of Torpedo AChETsubunits to

associate with a PRAD, we coexpressed them with protein

QN(Fig 1D) This QNprotein organizes wild-type AChET

subunits into tetramers (T4–QN) that are nonamphiphilic

and efficiently secreted [24], reaching 40% of the secretion

observed with the truncated I3C/stop4 mutant The

forma-tion of QN-linked oligomers therefore rescued an important

fraction of the wild-type catalytic Torpedo AChETsubunits

from intracellular degradation

Fig 1 Structure of the t peptide and oligomeric associations of acetylcholinesterase type T subunits (AChE T ) (A) primary sequence

of the t peptide from Torpedo AChE T subunits The residues of the

t peptide, encoded by an alternatively spliced 3¢ exon, are numbered from 1 to 40 and correspond to residues 536–575 of the mature Torpedo AChE T subunit; cysteine C37, which is responsible for intercatenary disulfide bonds, is circled (B) Side view of the t peptide, with its 1–32 segment organized as an a helix The con-served aromatic residues are located in the upper sector of the helix (C) Wheel representation of the entire t peptide, putatively organ-ized as an a helix Aromatic residues, shown in shaded circles, are located in the upper sector; charged residues are in double circles (white for basic residues, grey for acidic residues) and possible salt bridges are marked by hatched bars; cysteine C37 is in a double, grey circle; arrowheads indicate residues that have been mutated to cysteines (D) Primary sequence of the proline-rich attachment domain (PRAD) motif from Torpedo ColQ The PRAD residues are shown in bold text (from cysteines 70 and 71 to phenylalanine 86), and a few adjacent residues are shown in non-bold text (E) Schematic representation of a complex between four t peptides and a PRAD The N- and C-terminal extremities (indicated N and C) and arrows show the orientations of the t peptides (black zig-zags) running opposite to the PRAD (grey line); cysteines are indicated by circles, joined by lines representingdisulfide bonds (F) Major types of homomeric and heteromeric associations analyzed in this study: T a , Ta and Ta, amphiphilic monomer, dimer and tetramer of AChE T subunits; T na

4 , nonamphiphilic tetr-amer; T 4 –Q N , tetramer associated with the N-terminal Q N fragment

of ColQ, containingthe PRAD motif The schemes of heteromeric complexes are derived from recent studies (M Harel, H Dvir,

S Bon, W Q Liu, M Vidal, C Garbay, J L Sussman,

J Massoulie´ & I Silman, unpublished results) [30].

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Mutation of charged residues of the t peptide

The t peptide contains seven acidic (D, E) and eight basic

(H, K, R) residues, which may form intracatenary salt

bridges in the helical conformation (Fig 1C) and perhaps

intercatenary salt bridges in oligomeric assemblies; we

mutated these residues to alanines, individually or in groups

Mutations D4A/E5A, E7A/R8A, K11A, E13A, R16A,

K25A, or D29A did not markedly modify the levels of

cellular and secreted activities However, other mutations

had a stronger effect, as shown in Fig 2A Both cellular and

secreted activities were increased by the point mutation,

E1A, but decreased by replacement of the first four acidic

residues (E1, D4, E5, E7) by alanines Mutation H15A

enhanced the efficiency of secretion, because it decreased the

cellular activity but increased the secreted activity

Like the wild-type AChETsubunits, all mutants produced

amphiphilic dimers (T2a) and nonamphiphilic tetramers

(T4na) However, their proportions varied, as illustrated by

the sedimentation profiles of four mutants (Fig 2B) These

profiles did not change with time after transfection They

characterize each mutant and are not simply related to the

intracellular concentration of the enzyme, as shown by the fact that the D4A/E5A and R16A mutants produced appro-ximately the same levels of cellular activity with the same proportions of molecular forms as the wild type, but differed

in the activity and molecular forms of the secreted enzyme Conversely, the secreted enzyme was quantitatively and qualitatively similar for mutants K11A and D29A, although the patterns of cellular molecular forms were different

In all cases, coexpression with QNincreased the level of secretion and produced T4–QNcomplexes, as for the wild type

Mutation of aromatic residues The three tryptophans (W10, W17, W24) and Y31 were mutated to alanines, and all seven aromatic residues were mutated to prolines These mutations had little effect on the cellular activity; the secreted activity was reduced by about half by most mutations, but significantly increased by Y31A (data not shown) and Y31P (Fig 3A) The major molecular

Fig 2 Mutations of charged residues in the t peptide (A)

Acetylcho-linesterase (AChE) activities in cell extracts and secreted into the

cul-ture medium are shown for the wild type and four mutants Grey bars

and hatched bars correspond to the AChE activities of mutants

expressed without or with Q N , respectively (Materials and methods);

the activities are normalized to those obtained for the wild type (100%)

both in the cell extracts and in the medium; the standard errors were

obtained from five independent experiments For other individual

mutations (K11A, E13A, R16A, K25A and D29A), the cellular

activities ranged from 77% to 100%, and the secreted activities

between 68% and 136% (B) Sedimentation patterns of cellular and

secreted AChE, in sucrose gradients containing 0.2% Triton X-100.

The shaded areas, as well as the total areas under the sedimentation

profiles, are proportional to the relative activities of the mutants, so

that the surface of each peak represents the actual activity of the

correspondingmolecular form: monomers (T 1 ), dimers (T 2 ) and

tetramers (T ).

Fig 3 Mutations of aromatic residues (A) Secreted activities, nor-malized to that of the wild type, for mutants of aromatic residues to prolines: the bars represent secreted activities obtained when acetyl-cholinesterase type T subunits (AChE T ) were expressed without Q N

(g rey bars) and with Q N (hatched bars); the indicated values are the means of at least three independent experiments The cellular activities ranged from 86 to 114% without Q N and from 65 to 117% with Q N (B) Amphiphilic character of dimers produced by each mutant, indicated by charge shift electrophoresis RDOC/TX is the ratio of electrophoretic migrations in the presence of Triton X-100 with sodium deoxycholate and Triton X-100 alone, normalized to those of a nonamphiphilic species (T na

4 ); the indicated values represent the means of three to six independent experiments (C) Existence of Q N -linked dimers with mu-tant W10P: electrophoretic patterns, in the presence of Triton X-100 and sodium deoxycholate; the third lane shows that a fraction of dimers and tetramers was retarded by the M2 antibody (x), indicatingthat they were associated with the Q N -flagprotein Note that the coexpression with Q N increased the secretion of dimers as well as tetramers (D) AChE molecular forms secreted by mutants of aromatic residues to prolines, expressed with and without Q N ; electrophoretic analysis in the presence of Triton X-100 and deoxycholate s, AChE T dimers; d,

Q N -linked dimers; h, tetramers; j, T 4 –Q N complexes (i.e tetramer associated with the N-terminal Q N fragment of ColQ, containing the proline-rich attachment domain motif); the origin of migration is shown

by a thin line.

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forms produced by these mutants were T2 dimers, as

illustrated in electrophoretic patterns (Fig 3D); the

pro-duction of tetramers was strongly reduced or abolished,

again with the exception of Y31 mutants The amphiphilic

character of T2 dimers was retained when individual

aromatic residues were replaced with alanines, but it was

reduced when the central aromatic residues were mutated to

prolines, in a position-dependent manner (Fig 3B)

The production of T4–QN complexes, resultingin an

increased secretion, was not affected by mutation of Y31,

but was reduced by mutations of W10 and F28, and was

essentially suppressed by mutations of F14, W17, Y20

and W24, either to alanine (not shown) or to proline

(Fig 3A,D) As illustrated in Fig 3C, we found that when

the W10P mutant was coexpressed with the flagged QN

protein, the anti-flagM2 immunoglobulin reacted with a

fraction of dimers as well as with tetramers, indicatingthe

presence of T2–QN complexes, in addition to T4–QN

complexes, in the culture medium

We replaced the central tryptophan (W17) with a

hydrophobic aromatic residue (F), an aliphatic aromatic

residue (L), a heterocyclic residue (H), as well as a proline

(P) and an alanine (A) As shown in Fig 4, these mutations

did not strongly modify the cellular activity, which remained

within the range of 83–127% of the wild type, but reduced

or suppressed the formation of homotetramers; the

amphi-philic character of the resultingdimers was similar to that of

the wild type with F, L or A, it was significantly reduced

with H and it was essentially abolished with P

Figure 4 also shows that there was no interaction with

QNin the case of W17L and W17P, a very small production

of T4–QNin the case of W17A, and a significant production

of this complex in the case of W17F and W17H For these

two mutants, coexpression with QNincreased the level of

secreted activity to about 60% of that obtained in the wild

type; in addition to the T4–QNcomplex, this coexpression

markedly increased the secretion of dimers, particularly for

W17H, but unlike those formed with the W10P mutant,

these dimers did not seem to be associated with QN, as they did not react with the M2 antibody Whereas the sedimentation of the wild-type T4–QNcomplex was abso-lutely unaffected by the presence of Triton X-100 or Brij-97

in the gradient, the sedimentation of T4–QN complexes formed with W17F and W17A was reproducibly retarded in the presence of Triton X-100 (compared to Brij-97), showingan opposite effect to that normally observed for amphiphilic enzyme species, such as T1a, T2a or T4a (see Fig 6A); the electrophoretic migration of these complexes was also slower than that of the wild-type complex This may reveal an interaction with Triton X-100 micelles, but not with Brij-97 micelles, perhaps because of an unusual exposure of the aromatic groups in these complexes

It is noteworthy that, in contrast to the wild-type Tna

4 and

T4–QN, the tetramers formed with the W17F, W17H or W17A mutants were only observed in the medium, but not

in the cell extract This indicates a significant difference in the cellular traffickingof the wild-type and mutant complexes

Perturbation of the helical organization of an aromatic cluster

To perturb the a helical organization of the aromatic-rich segment of the t peptide, we deleted residues T12 and M21, located, respectively, in its N-terminal region and near its centre (Fig 1A) Mutation M21W introduced an additional aromatic residue, which might create a steric disturbance in oligomers or in heteromeric complexes with QN

These mutations had moderate effects on the level of cellular activity, and decreased secretion to 50% of the wild type The three mutants produced mostly amphiphilic

T2a dimers in the cell extracts; in the case of M21D, the medium only contained T4natetramers (Fig 5A), in contrast

to the wild type, in which these molecular forms are present both in the cell extracts and in the medium

Figure 5A also shows that coexpression with QN increased secretion for T12D and M21W (to about 35% and 50% of the wild type, respectively), but not for M21D;

T4–QNcomplexes of T12D and M21W were characterized

in the medium by reaction with the M2 antibody, but were undetectable or barely detectable in the cell extracts, in contrast to the wild-type T4–QNcomplex

Effect of a cysteine at various positions in the t peptide The formation of intercatenary disulfide bonds between wild-type AChET subunits depends on the free cysteine residue located near the C-terminus of the t peptide, C37 Mutation of this cysteine to a serine reduced both cellular and secreted activities; it suppressed the formation of dimers and reduced cellular and secreted tetramers (Fig 6A); in the presence of QN, the secretion of T4–QN complexes was reduced to  75% of that of the wild type Thus, the presence of an intercatenary disulfide bond appears to be necessary for dimerization, but not for tetramerization, particularly in the presence of QN

To determine whether cysteines at other positions could allow dimerization and further oligomerization, we replaced residues I3, A6, T12, S19, M21, M22 or H34 with a cysteine, with or without mutation of C37 (C37S)

Fig 4 Molecular forms produced by W17 mutants; interaction with

Q N Sedimentation patterns of cellular and secreted molecular forms;

the areas under the profiles are proportional to the corresponding

activities; the top of the wild-type T 4 –Q N (tetramer associated with the

N-terminal Q N fragment of ColQ, containing the proline-rich

attach-ment domain motif) peak exceeds the frame and is shifted downwards.

Molecular forms expressed without Q N (––s––) and with Q N (- - -j- - -)

were analyzed in the presence of Triton X-100; sedimentation was also

performed in the presence of Brij-97 (Bj) for molecular forms secreted by

mutants W17F, W17H and W17A (ÆÆÆÆÆÆ) Note an unusual retardation

by Triton X-100 (Tx) for W17F and W17A.

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The relative levels of cellular and secreted activities, as

well as the distribution of molecular forms, are illustrated

in Fig 6A,B Unlike C37S, none of these mutants

produced monomers without dimers; therefore, when

two cysteines were present, they were not engaged in an

intracatenary disulfide bond, but could form intercatenary

bonds in dimers

Mutation I3C (with or without C37S) considerably

increased the cellular activity, mostly as amphiphilic dimers;

secreted activity was also increased, but to a much lesser

degree The presence of the N-terminal cysteine thus

appears to facilitate dimerization and to reduce

degrada-tion This mutation also increased the cellular activity

obtained with the W17P mutant, without restoringits

capacity to interact with QN

Mutation A6C somewhat decreased the cellular activity,

but strongly increased secretion The A6C mutant mainly

produced a nonamphiphilic 14 S species, possibly

corres-pondingto octamers This unusual oligomer dissociated

duringstorage, particularly in the presence of detergent

(Triton X-100), transiently producingamphiphilic tetramers

(T4a) (which are not usually observed in the wild type) and

amphiphilic dimers (T2a) When C37 was absent (A6C/

C37S), the 14 S species was observed in the cell extracts, but

seemed to be less stable, beingalmost entirely converted to

T2adimers in the medium

The T12C mutation introduced a cysteine in the

N-terminal part of the aromatic-rich segment (not shown)

Fig 6 Oligomeric forms obtained with cysteines at different positions in the t peptide (A) Sedimentation patterns of cellular and secreted molecular forms in gradients containing Triton X-100 (––) and Brij-97 (- - -); the shaded areas and the areas under the profiles are propor-tional to the correspondingactivities Note that dimers containing cysteines at position 21 did not sediment faster with Triton X-100 than with Brij-97, in contrast to the amphiphilic dimers (wild type, or with cysteines at positions 3, 6 or 34) Mutants A6C and A6C/C37S pro-duced a 14 S species that was progressively dissociated into amphi-philic tetramers (T a ) and ultimately amphiphilic dimers (T a ), as shown for cell extracts in an upper profile (B) Interaction of cysteine mutants with Q N , as shown by electrophoretic analysis of secreted molecular forms, in the presence of Triton X-100 and sodium deoxy-cholate (compare with Fig 5A) Note that mutant M21C (with cysteine C37) produced complexes with Q N , retarded by M2, whereas mutant M21C/C37S (without cysteine C37) did not.

Fig 5 Mutations in the aromatic-rich region; mutations of methionines

M21 and M22 (A) Electrophoretic patterns of cellular and secreted

molecular forms, in the presence of Triton X-100, with and without

deoxycholate (DOC); complexes with Q N that were retarded by M2

are indicated by x; the symbols are as in Fig 4 The secretion of

mutant M21D was not increased by coexpression with Q N , and no

complex reactingwith M2 was detected In the case of T12D and

M21W, T 4 –Q N (tetramer associated with the N-terminal Q N fragment

of ColQ, containingthe proline-rich attachment domain motif)

com-plexes were secreted, but undetectable or barely detectable in cell

extracts (B) Cellular and secreted activities (represented as in Fig 2A)

for mutants containingcysteine C37 or not (C37S) Coexpression with

Q N increased the secretion of mutants of methionine M22 and mutants

of methionine M21 which possessed cysteine C37, but not of mutants

M21A/C37S and M21S/C37S (data not shown), lackingboth M21

and C37.

Trang 7

In the absence of cysteine C37, this allowed the formation

of amphiphilic dimers, which were secreted together with

nonamphiphilic tetramers and a 14 S species This species

was, in fact, predominant in the secreted enzyme and

appeared to be much more stable than that formed with

A6C, as it was not dissociated after secretion In the

presence of cysteine C37, the T12C mutant produced mainly

nonamphiphilic tetramers, which represented the only

secreted form This suggests that tetramers may be stabilized

when disulfide bonds were formed at the two positions,

12 and 37

A cysteine at position 19, in the aromatic-rich segment

but opposite to the aromatic cluster, had very different

effects, dependingon the presence of cysteine C37 Without

C37, mutant S19C/C37S produced very low levels of

cellular or secreted activity In contrast, mutant S19C

(containingtwo cysteines at positions 19 and 37) showed a

high level of secretion, mostly as nonamphiphilic tetramers,

as observed for T12C

Mutations M21C and M22C, with or without cysteine

C37, had little effect on cellular activity (compared to the

wild-type and C37S mutant, respectively), but increased

secretion to various degrees M21C and M21C/C37S

secreted both tetramers and nonamphiphilic dimers, while

M22C and M22C/C37S secreted mostly tetramers The

dimers formed with a cysteine at position 21 appeared

nonamphiphilic, suggesting that the aromatic clusters may

be masked by an intercatenary disulfide bond in the

aromatic-rich segment

Finally, the mutants containinga cysteine at position 34

(H34C, H34C/C37S) behaved essentially like the wild type,

suggesting that the C-terminal segment of the t peptide is

flexible

Figure 6B shows that the various cysteine mutants

formed T4–QNcomplexes (reactingwith the anti-flagM2

immunoglobulin), except M21C/C37S Thus, mutation

M21C suppressed the heteromeric complex when cysteine

C37 was absent, but not when it was present: this illustrates

the importance of the C-terminal cysteine for the assembly

and/or stabilization of the T4–QN complex, in agreement

with the formation of intercaternary disulfide bonds

between the t peptide and QNcysteines

Coexpression with QNgenerally increased secretion when

T4–QNcomplexes were produced, although this effect was

marginal or absent for mutants that showed a high level of

secretion without QN(A6C, A6C/C37S, S19C) However,

coexpression induced a decrease, of 40%, in the secretion

of mutant M21C/C37S, for which complexes could not be

detected (not shown) This suggests that QN did interact

with the mutant AChET subunits, but induced their

degradation rather than the assembly of a stable, secretable

hetero-oligomer

The role of methionine 21

The fact that M21C/C37S did not associate with QN,

whereas M22C/C37S formed a T4–QN complex, may be

related to the orientation of the two adjacent methionines

relative to the aromatic cluster, and to a possible structural

role of methionine M21: this residue is conserved in

vertebrate t peptides, while M22 is replaced with other

residues in some species To examine this possibility, we

mutated M21 and M22 to alanines or serines (with or without C37S) Figure 5B shows that coexpression with QN increased the level of secretion, indicatingthe formation of

T4–QNcomplexes, for all mutants except M21A/C37S (and M21S/C37S) Thus, the presence of a methionine at position

22 is dispensable, but a methionine at position 21 contri-butes to the stability of the complex, especially when cysteine 37 is absent

The C-terminal region of the t peptide: a retention motif? The last four residues of the t peptide, CAEL, contain the cysteine involved in intercatenary disulfide bonds and also resemble the classical ER-retention signal, KDEL To determine whether its presence might induce a partial retention of AChET subunits, we introduced various mutations in this motif (Fig 7A)

It should first be noted that mutation C37S (where the cysteine was removed) did not increase secretion, but rather decreased both cellular and secreted activities; this effect may result from the fact that suppression of the cysteine prevented dimerization and reduced the level of secreted tetramers, as discussed above

Fig 7 Effects of the C-terminal cysteine (C37), of C-terminal segments and of a KDEL motif on acetylcholinesterase (AChE) molecular forms (A) Sedimentation patterns of cellular (upper row) and secreted (lower row) enzyme, in gradients containing Triton X-100 The shaded areas and the areas under the sedimentation profiles are proportional to the activities In mutant T KDEL , the C-terminal tetrapeptide (CAEL) was replaced with the canonical ER retention motif (KDEL) All mutants lackinga cysteine produced and secreted amphiphilic monomers with variable proportions of nonamphiphilic tetramers; these tetramers were secreted at a higher level for T KDEL than for C37S (SAEL) (B) Cellular and secreted activities obtained with and without Q N The cellular activities decreased with the extent of C-terminal deletions; coexpression with Q N increased secretion to a level comparable to that

of the wild type for mutants C37S, T KDEL , stop34 and stop32, but had essentially no effect for stop29.

Trang 8

Dimerization was also suppressed when the CAEL motif

was replaced with KDEL (TKDEL) because this mutation

removed the cysteine The presence of a C-terminal KDEL

tetrapeptide increased the level of cellular activity by about

threefold relative to C37S, mostly correspondingto T1a; this

increase in cellular enzyme appeared to facilitate

tetra-merization, as tetramers (T4na) were secreted at a higher

level than with C37S (Fig 7A)

We also deleted the last two residues, EL (stop39): the

mutant in which the C-terminal motif was reduced to CA

produced dimers and secreted 1.7-times more activity than

the wild type More extensive deletions, which removed the

cysteine, suppressed dimerization, so that monomers were

predominant in the cells and in the medium, and the levels

of activity were reduced in both compartments; the secretion

of homotetramers was reduced in stop34, compared to

C37S, and tetramerization was abolished in stop32

Like C37S, the TKDEL, stop39, stop34 and stop32

mutants formed T4–QNcomplexes, so that their secretion

was increased in the presence of QN(Fig 7B) In contrast,

the shorter mutant, stop29, showed no interaction with QN,

as indicated by the fact that coexpression did not affect

either the secreted activity or the molecular forms,

charac-terized by sedimentation To determine whether the

differ-ence between stop32 and stop29 could be ascribed to one of

the three residues D29, Q30 or Y31, we mutated each of

them to alanine: the level of T4–QNcomplexes was similar to

that of stop32 for Y31A/stop32 and Q30A/stop32, but

considerably reduced for D29A/stop32, suggesting a specific

influence of residue D29; the strongeffect observed by

mutation of this charged residue contrasts with the result

obtained when it was mutated in the full-length t peptide

(see above)

Progressive deletions from the C-terminus

of the I3C mutant

To analyze the effect of C-terminal deletions without

preventingdimerization, we used the I3C/C37S mutant,

which produced mostly amphiphilic dimers (Fig 6A) and

formed T4–QN complexes when coexpressed with QN

(Fig 8A) As in the wild type, the replacement of the

C-terminal tetrapeptide by KDEL increased the cellular

activity (mainly Ta) and reduced its secretion, but did not

abolish association with QN, producingT4–QNcomplexes

which were secreted Deletion of the last nine residues

(I3C/stop32) did not abolish association with QN, but

deletion of the last 12 residues (I3C/stop29) suppressed it

completely Thus, the presence of a cysteine at position 3

did not modify the requirement of residues 29–31 for

interaction with QN

The effect of deletions on the cellular and secreted

activities is illustrated in Fig 8B The cellular activity

remained approximately constant for all deletions, about

50% of the value observed with the full-length t peptide

Removal of the last two residues (EL) increased secretion, as

in the case of the wild type More extensive deletions in the

C-terminal region reduced secretion, compared to that of

I3C/C37S/stop39, but deletions within the aromatic region

progressively increased it, reaching a plateau when all

aro-matic residues were removed The dimers were amphiphilic

when they contained at least 29 residues of the t peptide

(stop29 and longer), but not if they contained 24 residues

or fewer (stop24 and shorter), i.e when they lacked some of the core aromatic residues

Fig 8 Effects of C-terminal segments and of a KDEL motif, in mutants containing an N-terminal cysteine (I3C) (A) Interaction with

Q N , indicated by electrophoretic patterns of cellular (top) and secreted (bottom) molecular forms obtained with and without Q N , in the presence of Triton X-100 and sodium deoxycholate Note that mu-tants I3C/C37S, I3C/KDEL and I3C/stop37 produced homomeric

T 2 dimers (s), homomeric T na

4 tetramers (h) and T 4 –Q N (tetramer associated with the N-terminal Q N fragment of ColQ, containing the proline-rich attachment domain motif) complexes (j), whereas I3C/ stop29 did not Homomeric tetramers of the I3C/KDEL mutant appeared to be partially retained intracellularly (B) Effect of C-terminal deletions on cellular and secreted activities; progressive deletions were made from the C-terminus of mutants containingan N-terminal cysteine (I3C) which allows an efficient dimerization; in the two longer mutants, the C-terminal cysteine was replaced with a serine (C37S); mutated residues are underlined in the sequence, shown alongthe horizontal axis Cellular (upper frame) and secreted (lower frame) activities, expressed as percentage of the wild type, are shown

as a function of the remainingleng th of the t peptide Asterisks indicate mutants that produced amphiphilic dimers; mutants stop34 and shorter produced nonamphiphilic dimers.

Trang 9

The t peptide: an elongated amphiphilic a helix

with a cluster of aromatic residues

Previous studies suggested that the amphiphilic properties

of the t peptide reflect the formation of a cluster of aromatic

residues in its a helical conformation [30] The present

mutations confirm the role of aromatic residues, but show

that they differ considerably in their importance

Amphiphilicity was not affected by mutations of charged

residues, or by deletions of the C-terminal region which

removed up to 11 residues, i.e maintained all the aromatic

residues, except Y31 The amphiphilic character was

reduced to various extents when the central residues (F14,

W17, Y20, W24, F28) were replaced with prolines, but was

not affected by mutation of the first and last residues (W10,

Y31) Replacement of the most critical residue, W17, by

other, different, amino acids (F, L, A, H, P) showed that

amphiphilicity was indifferent to their aromatic nature, that

it did not strongly depend on their hydrophobicity, but was

very sensitive to the a helical structure Thus, the

amphi-philic properties of the t peptide appear to depend

predominantly on the spatial organization of a cluster of

hydrophobic residues

In agreement with a previous study [30], we obtained no

evidence that two cysteines, introduced at several positions

in the N- and C-terminal regions of the mutated t peptides,

could form an intracaternary disulfide bond The present

results thus confirm that the t peptide forms an elongated

amphiphilic helix, rather than foldingback on itself as a

hairpin in which the N- and C-terminal ends might be joined

by a disulfide bond

Homomeric associations of AChETsubunits

In contrast to the wild-type AChET subunits, the C37S

mutant did not form stable dimers, showingthat an

intercaternary disulfide bond is necessary The position of

this bond appeared very flexible, as dimers were formed

when cysteines were introduced at various positions along

the t peptide (with or without cysteine C37): this did not

seem to depend on the orientation of the residue relative

to the helical axis, although some positions produced

higher proportions of dimers than others However,

although most dimers were amphiphilic, those formed in

the presence of a cysteine at position 21 were

nonamphi-philic, indicatingthat, in this case, the hydrophobic

patches occluded each other because of the formation of a

disulfide bond joiningthe aromatic clusters near their

centers

Dependingon the position of an added cysteine, AChET

subunits could form predominantly dimers or tetramers – or

even higher oligomers sedimenting at 14 S, possibly

octa-mers This shows that the interactions between the t peptides

may present different geometries Thus, the t peptides can

form different homomeric assemblies, which may be

stabilized by intercaternary disulfide bonds and are

influ-enced by the positions of these linkages

In contrast to dimers, tetramers can be formed in the

absence of cysteine in the t peptide [27,36], although at a

lower level than for the wild type Tetramers are generally

nonamphiphilic (T4na), but some tetramers may also be amphiphilic (T4a), particularly those resultingfrom the dissociation of the nonamphiphilic 14 S species Thus, aromatic clusters may be either occluded or at least partly exposed in tetrameric assemblies, indicatingthat they correspond to distinct quaternary organizations

Heteromeric associations with the PRAD-containing protein, QN

The major physiological role of the t peptide is clearly to allow the functional localization of AChET tetramers through their association with PRAD-containing proteins, ColQ and PRiMA In the present study, we focused our attention on the formation of quaternary associations with

an N-terminal fragment of ColQ, QN This protein assem-bles with wild-type AChET subunits to form QN-linked tetramers (T4–QN), which are nonamphiphilic Previous studies showed that in these hetero-oligomers, two catalytic subunits are disulfide-linked with QN, while the other two are disulfide-linked together However, in the absence of cysteine C37, this association still occurs, indicatingthat it does not require the formation of intercaternary disulfide bonds

The complex was formed when the t peptide carried an additional cysteine at positions 3, 6, 12, 19, 22 or 34, with or without the original cysteine C37 It was not formed with a cysteine at position 21, except when C37 was present: a cysteine instead of a methionine at position 21 therefore appears unfavorable This may be partly because of the formation of nonamphiphilic disulfide-linked dimers in which the aromatic clusters are not available for interaction with the proline-rich domain of QN(PRAD) However, the mutation of methionine 21 to an alanine or a serine also weakened the formation of the complex, which again required the presence of cysteine C37: mutants M21A/C37S and M21S/C37S did not associate with QN In contrast, similar mutations of methionine M22 did not prevent the formation of T4–QNcomplexes This demonstrates that the complex was stabilized by disulfide bonds through cysteine

37, and by the presence of methonine 21, in agreement with stronginteractions of this methionine with the PRAD in a complex of isolated peptides (WAT)4 PRAD (M Harel

et al., manuscript in preparation) Nevertheless, the fact that M21 could be replaced with a tryptophan suggests that the complex can accommodate the steric constraint owingto a more bulky residue

The role of aromatic residues in the formation of the QN -linked complex has been established previously [4,28,37] Usingdeletions of single residues, we show here that the structure of the cluster is crucial for this quaternary interaction: it was abolished by deletion of residue 21, in the middle of the aromatic-rich segment The fact that deletion of residue 12, near the N-terminal end of the aromatic cluster, had no such effect, indicates that the orientation of the aromatic cluster relative to the catalytic domain is not crucial This is consistent with the notion of a flexible junction between the catalytic domain and the amphiphilic helical region of the t peptide [30] In fact, addition of a variable number of residues between the catalytic domain and the t peptide did not prevent association with Q (N Morel & S Bon, unpublished)

Trang 10

In the present study, we assessed the importance of

aromatic residues, individually, by point mutations We

observed that mutation of the central residues (F14, W17,

Y20, W24) to proline or alanine had a much stronger effect

than mutation of W10 and F28, and that Y31 had no effect

Although not identical, the impacts of these mutations were

similar to those observed on the amphiphilic character of

AChETsubunits However, mutations of W17 to different

amino acids clearly dissociated the two properties, as

mutation W17L maintained the amphiphilic character,

but totally abolished the association with QN, like mutation

W17P This association was reduced when W17 was

replaced with a phenylalanine or a histidine, and even more

strongly when it was replaced with an alanine, emphasizing

the importance of an aromatic side-chain

With some mutants, we obtained evidence that QN

induced the formation of AChETdimers: coexpression with

QNstrongly increased the secretion of dimers that were not

covalently associated with QN(as in the case of W17F and

W17H), or were at least partially disulfide-linked with it (as

in the case of W10P) Such dimers may represent an

intermediate stage in the assembly of QN-linked tetramers,

or result from the dissociation of unstable tetramers

QN-linked tetramers are usually nonamphiphilic,

indica-tingthat the clusters of aromatic residues are occluded when

the t peptides are associated with the PRAD, in agreement

with their stronginvolvement in these quaternary

interac-tions and with the crystallographic structure of a complex of

synthetic peptides (M Harel, H Dvir, S Bon, W Q Liu,

M Vidal, C Garbay, J L Sussman, J Massoulie´ &

I Silman, unpublished results) However, the QN-linked

tetramers, found with the W17F and W17A mutants, showed

some interaction with detergents, suggesting that they were

less compact than the wild-type complexes In fact, the

formation of QN-linked complexes in the presence of cysteines

within the t peptides reveals a considerable flexibility, because

disulfide bonds between these residues do not seem to be

compatible with the distances between pairs of homologous

residues in a complex of wild-type synthetic peptides [30]

The heteromeric assocation with QNwas not suppressed

by removal of the last nine residues (followingY31),

showingthat it depends primarily on the aromatic-rich

segment and does not require the C-terminal part of the

t peptide However, removal of three additional residues

(D29, Q30, Y31) abolished the interaction, and point

mutations showed that this was mostly caused by the

deletion of D29: although mutations of charged residues in

the full-length t peptide had little effect on association with

QN, this suggests that a salt bridge contributed significantly

to its stability when the complex was weakened by a

C-terminal deletion

Similarity between nonamphiphilic homomeric

and QN-linked tetramers

We observed that the formation of homomeric T4na

tetramers was suppressed by all mutations which affected

the heterometic complex, T4–QN, suggesting that both

quaternary assemblies depend on the same interactions and

possess a similar organization, in agreement with the fact

that they are both nonamphiphilic In fact, except for

M21C/C37S, mutations that affected Q -linked tetramers

appeared to reduce homomeric tetramers more severely, indicatingthat tetramers are generally stabilized by the presence of a PRAD In T4–QNcomplexes, the four a helical

t peptides are organized as a super-helix, forming a hollow tube lined by aromatic side-chains, which is occupied by the PRAD This central space is unlikely to be filled with water molecules or to remain empty in T4natetramers; it may be reduced by a change in the pitch of the super-helix

Homo- and hetero-oligomerization occur in the ER, subcellular trafficking

The presence of an ER-retention motif (KDEL) at the C-terminus of the t peptide blocked secretion, as expected, but did not prevent dimerization when a cysteine was introduced at position 3, in the N-terminal region of the

t peptide The KDEL motif actually increased the formation

of homotetramers (T4na) in the TKDELmutant, compared to the C37S mutant which also lacked the C-terminal cysteine and was terminated by the tetrapeptide, SAEL Similarly, a C-terminal KDEL did not block the formation of hetero-meric complexes T4–QN; furthermore, the KDEL motif was sterically masked in the complexes, as the complexes were efficiently secreted The fact that retention of AChET subunits in the ER did not prevent homomeric or hetero-meric associations indicates that they occur in this com-partment

We found that the last two residues of the t peptide, EL, which are also present in the ER-retention tetrapeptide, KDEL, exert a weaker, but significant, retention effect, as their deletion increased secretion It is possible that these residues help to retain isolated AChETsubunits in the ER, and thus facilitate their physiological association with the anchoringproteins, ColQ and PRiMA

Structural differences certainly explain that complexes formed with the T12D and some aromatic mutants followed

a different cellular traffickingthan the wild type: these mutant complexes were secreted, but not detectable in cellular extracts, showingthat they did not accumulate in the secretory compartment, but were either rapidly secreted

or degraded We made a similar observation for homomeric tetramers in the case of mutant M21D

Oligomerization, secretion and degradation

In steady-state cultures, we may assume that all mutants were produced at the same rate, as they only differ in the short C-terminal t peptide, so that the rates of secretion of different mutants represent the difference between the common rate of synthesis and the rate of degradation In

a previous study, we established that the presence of the

t peptide induces a partial degradation of AChETsubunits through the ERAD pathway and that this effect depends on the presence of aromatic residues [28] Occlusion of these residues may explain that oligomerization generally facili-tates secretion: for example, the relative proportions of monomers, dimers and tetramers in cellular extracts and in the medium indicate that the secretion of wild-type AChET subunits increases with their degree of oligomerization This explains why the introduction of cysteines at certain positions increased secretion, by facilitatingthe formation

of dimers (I3C), tetramers (M22C) or 14 S oligomers (A6C)

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