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The impact of mutations on the protein structure, in the context of generating a gp41 based vaccine antigen that resembles a fusion intermediate state, is dis-cussed.. The fusogenic stat

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HIV-1 gp41 and gp160 are hyperthermostable proteins

in a mesophilic environment

Characterization of gp41 mutants

Tino Krell1, Fre´de´ric Greco1, Olivier Engel1, Jean Dubayle1, Joseline Dubayle1, Audrey Kennel1,

Benoit Charloteaux2, Robert Brasseur2, Michel Chevalier1, Regis Sodoyer1and Raphae¨lle El Habib1

1 Aventis Pasteur, Marcy l’Etoile, France; 2 Centre de Biophysique Mole´culaire Nume´rique, Faculte´ Universitaire des Sciences Agronomiques de Gembloux, Belgium

HIV gp41(24–157) unfolds cooperatively over the pH range

of 1.0–4.0 with Tmvalues of > 100C At pH 2.8, protein

unfolding was 80% reversible and the DHvH/DHcalratio of

3.7 is indicative of gp41 being trimeric No evidence for a

monomer–trimer equilibrium in the concentration range of

0.3–36 lM was obtained by DSC and tryptophan

fluores-cence Glycosylation of gp41 was found to have only a

marginal impact on the thermal stability Reduction of the

disulfide bond or mutation of both cysteine residues had

only a marginal impact on protein stability There was no

cooperative unfolding event in the DSC thermogram of

gp160 in NaCl/Pi, pH 7.4, over a temperature range of

8–129C When the pH was lowered to 5.5–3.4, a single

unfolding event at around 120C was noted, and three

unfolding events at 93.3, 106.4 and 111.8C w ere observed

at pH 2.8 Differences between gp41 and gp160, and hyperthermostable proteins from thermophile organisms are discussed A series of gp41 mutants containing single, dou-ble, triple or quadruple point mutations were analysed by DSC and CD The impact of mutations on the protein structure, in the context of generating a gp41 based vaccine antigen that resembles a fusion intermediate state, is dis-cussed A gp41 mutant, in which three hydrophobic amino acids in the gp41 loop were replaced with charged residues, showed an increased solubility at neutral pH

Keywords: gp160; gp41; HIV; hyperthermostability; site-directed mutagenesis

HIV entry is mediated by the viral envelope proteins gp41

and gp120 Both proteins are derived from the gp160

precursor following proteolytic cleavage [1] After cleavage,

both proteins remain associated [2] and are trimeric in their

prefusogenic state [3] gp41 anchors the protein complex to

the viral membrane [4], whereas gp120 binds to the human

cell-surface receptor CD4 and other receptors on the target

cell This interaction leads to a dissociation of gp120 from

gp41 [5], which induces a conformational change in gp41 [2],

resulting in the fusion of viral and cellular membranes

Both envelope proteins are vaccine candidates against

HIV However, the results of the world’s first phase III

efficacy trial using gp120 were relatively modest [6] and

efforts are nowbeing made to explore the vaccine potential

of gp41 The sequence of gp41 contains four functional

regions, as follows: the N-terminal fusion peptide (which

is thought to insert into the host cell membrane) is followed

by an ectodomain, a transmembrane region and a

cyto-plasmic domain

The ectodomain of gp41 is thought to adopt at least two

different tertiary structures [7]: a prefusogenic state, and a

lower energy fusion-active (fusogenic) state The decrease in free energy during the transition into the latter state is thought to account for the energy necessary for the fusion of viral and cellular membranes There is nowevidence that the recombinantly produced gp41 ectodomain adopts sponta-neously this fusogenic state, and 3D structural information

on gp41 is entirely on this lower energy state [8–11] The fusogenic state of the gp41 ectodomain is a trimeric coiled-coil protein in which the three helices C pack in an antiparallel manner against the central trimer of parallel helices N

It has been shown that gp41 produced in Escherichia coli forms insoluble aggregates at neutral pH [12], and aggre-gation is proposed to occur at the loop region connecting helices N and C [13] This is consistent with the fact that recombinant constructs of gp41, which have this loop replaced with a small flexible linker (gp41 models), are soluble at neutral pH Therefore, a large proportion of biochemical and biophysical studies on gp41 have been carried out using either a gp41 model [14–16] or a stoichiometric mixture of peptides corresponding to the N- and C-terminal helices, which assemble to hetero-hexamers in a native-like manner [17,18] However, recom-binant gp41 was shown to be soluble at a pH of < 3.5, and several studies have been carried out at acidic pH [19,20] The NMR structure of gp41, at pH 3.0 [9], and its X-ray structure, using crystals grown at pH 4.25 [21], are identical

to structures of gp41 fragments determined at pH values of 4.6–8.0 [8,11,16,22] This demonstrates that the structure of gp41 is not altered by acidic pH

Correspondence to T Krell, Aventis Pasteur, 1541 avenue Marcel

Me´rieux, 69280 Marcy l’Etoile, France Fax: + 33 4 37 37 31 80,

Tel.: + 33 4 37 37 90 12, E-mail: tino.krell@aventis.com

Abbreviations: SIV, simian immunodeficiency virus; TCEP,

Tris(2-carboxyethyl)phosphine.

(Received 8 January 2004, revised 1 March 2004,

accepted 3 March 2004)

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Based on the evidence that the structure of gp41 is not

altered by acidic pH, and the functional importance of the

loop region [23] with its conserved cysteine residues [24],

we have carried out our analyses at acidic pH using

recombinant gp41(24–157) containing the cysteine

resi-dues

The potent HIV entry inhibitor 5-helix [25], a

recombin-ant gp41 trimer lacking a helix C, has been shown to bind

tightly to individual C peptides It has been proposed that

the mechanism of action of 5-helix is based on the binding to

a fusion intermediate of gp41, which is characterized by an

accessible and exposed helix C [26] These data can be

regarded as evidence that a protein is capable of blocking

fusion intermediates of gp41, which consequently leads to

the inhibition of virus entry Our vaccine approach is based

on the generation of gp41 mutants, which resemble the

fusion intermediate state It is generally accepted that this

intermediate state is trimeric and that helices N and C do

not interact Here we explore ways to stabilize the

interaction between helices N, to favour a trimeric state

and, on the other hand, to destabilize the interaction

between helices N and C in order to prevent helix contact

The study of recombinant gp41, either as separate

glycosylated or nonglycosylated protein, or as part of

gp160, by biophysical techniques, forms the first part of this

article In the second part, site-directed mutagenesis data of

gp41 are presented with the aim of assessing the influence of

amino acid replacements on protein stability and solubility

Materials and methods

Materials

Gp41(24–157) Recombinant ectodomain of gp41

corres-ponding to amino acids 537–669 of the envelope protein of

the LAI isolate (p03375) with a C-terminal extension of

amino acids GGGGSHHHHHH For details of protein

expression and purification see below

Gp41(34–170) Glycosylated recombinant ectodomain of

gp41 purchased from Tebu-bio (Le Perray en Yvelines

Cedex, France) corresponding to amino acids 546–682 of

the envelope protein of the HxB2 isolate (p04578) The

protein has been expressed in Pichia pastoris The gp41

domains of isolates BH10 and HxB2 share 99% sequence

identity

Gp160 Fusion of amino acids 30–500 of the envelope

protein of the isolate MN (comprising gp120) with amino

acids 501–744 (comprising a large part of gp41) of the LAI

isolate The amino acid sequence of the gp120–gp41

cleavage site KAKRRVVQREKR (502–513 in the LAI

sequence) has been altered to KAQNHVVQNEHQ This

change prevents cleavage of gp160 into gp120 and gp41

This protein has been expressed in vaccinia virus grown on

BHK21 cells and was purified by affinity chromatography

on immobilized antibodies Further details on the

construc-tion and biochemical characterizaconstruc-tion of this protein are

found in Kieny et al [27] This protein has been used for

several HIV vaccine trials [28,29]

TCEP/HCl [Tris(2-carboxyethyl)phosphine

hydrochlo-ride] was purchased from Pierce

Cloning procedures

A 0.6 kb DNA fragment, containing the sequence encoding the ectodomain of gp41 of HIV isolate LAI (amino acids 537–669), was obtained by PCR amplification using, as template, a plasmid containing the gp120 sequence of MN and the gp41 sequence of the LAI isolate Restriction sites for BspHI and XhoI (shown in italics) were, respectively, included in the forward and reverse primers, as follows: forward primer: 5¢-CTCTTTCATGACGCTGACGGTA CAGGCC-3¢; reverse primer: 5¢-CCGCTCGAGCTAATG GTGATGGTGATGGTGTGACCCTCCCCCTCCACT TGCCCATTTATCTAA-3¢ The start codon in the for-ward primer (in bold) is a naturally occurring methionine residue A stop codon (in bold) and the DNA sequence encoding the extension GGGGSHHHHHH (underlined) were added to the reverse primer Platinum HF polymerase (Gibco Invitrogen Corp.) was used, according to the manufacturer’s instructions, for PCR amplification The PCR amplified fragment was cloned directly into the vector pM1800 using the restriction sites NcoI and XhoI The expression vector pM1800 is a derivate of pET28c (Novagen), in which the F1 origin of replication has been deleted and replaced with the Cer fragment, allowing for multimer resolution

Site-directed mutagenesis: single and multiple point mutations

The various point mutations were created using the Quick-change site-directed mutagenesis kit (Stratagene), using the instructions provided by the manufacturer Mutations were carried out directly on the expression vector containing the sequence of native gp41, and multiple mutations were introduced in a sequential manner

NC and CN fusion mutants The NC and CN gp41 fusion constructs were generated using three consecutive PCRs In the first PCR (for the NC constructs) the following two partially overlapping DNA fragments were generated: a fragment corresponding to helix N containing a C-terminal extension encoding the first six amino acids of the helix C and a fragment corresponding

to helix C containing an N-terminal extension coding for the last six amino acids of helix N In the second reaction, a stoichiometric mix of both partially overlapping fragments (no primers added) was submitted to 10 PCR cycles The third reaction was carried out with the product of the second reaction in the presence of primers containing NcoI or XhoI restriction sites and which are complementary to the 5¢ end

of helix N and to the 3¢ end of helix C An equivalent approach was used to generate the CN fusion constructs Expression

For protein expression, E coli BL21(DE3) was transformed with the corresponding plasmids Typically, 1 L cultures were grown at 37C on LB (Luria–Bertani) broth supple-mented with kanamycin (25 lgÆmL)1) Protein expression was induced at an attenuance (D) of 0.6 at 600 nm, by the addition of isopropyl thio-b- -galactoside (Q-BIOgene,

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Illkirch, France) to a final concentration of 1 mM Bacteria

were harvested, 4 h after protein induction, by

centrifuga-tion For certain mutants, protein expression was optimized

by the replacement of LB medium with Terrific Broth

medium, induction with 0.02–0.1 mM isopropyl

thio-b-D-galactoside and a growth temperature of 30C

Protein purification

The bacterial pellet resulting from a 1 L culture was

resuspended at room temperature in 95 mL of 50 mM

Tris/HCl containing 100 lMCompleteTMEDTA-free

pro-tease inhibitor cocktail (Roche Molecular Biochemicals) and

100 lgÆmL)1lysozyme (Sigma-Aldrich), pH 8.0, and gently

agitated for 30 min The bacterial suspension was then

placed on ice and cell lysis was achieved by ultrasound

treatment (4· 2 min) using a Branson-Sonifer 450

After-wards, MgCl2and Benzonase (Merck) were added to final

concentrations of 1 mM and 1 UÆmL)1, respectively The

resulting solution was then centrifuged (20 000 g, 30 min,

4C) Aliquots of the resulting supernatant and pellet were

analysed by SDS/PAGE and recombinant proteins were

detected by Western blot analysis using a monoclonal

antibody raised against poly-histidine (Novagen)

Recom-binant protein was found to be almost exclusively present in

the pellet The pellet was resuspended in 100 mL of buffer A

(50 mMTris/HCl, 8Murea, 500 mMNaCl, 10 mM

imida-zole, pH 8.0) and agitated at 4C for 30 min After filtration

using a 0.45 lm cut-off filter, protein was loaded onto a 5 ml

Hi-Trap Chelating column (Amersham Pharmacia Biotech),

previously equilibrated in buffer A After washing with

50 mL of buffer A, protein elution was achieved using a

buffer comprising 50 mMTris/HCl, 8Murea, 500 mMNaCl

and 500 mM imidazole, pH 8.0 Protein refolding was

achieved by dialysis with 50 mMformate, pH 2.8 Protein

was then sterile filtered and stored at)45 C

MALDI-TOF MS

Mass spectrometric analyses were carried out on a Biflex III

MALDI-TOF mass spectrometer (Bruker Daltonics,

Wiss-embourg, France) Samples of native and mutant gp41

(1–3 mgÆmL)1) in 50 mM formate, pH 2.8, were diluted

with 30% acetonitrile (v/v) containing 0.07% trifluoroacetic

acid to a final concentration of 0.4 mgÆmL)1 (protein

solution) A saturated solution of sinapic acid

(Sigma-Aldrich) in 70% acetonitrile (v/v) containing 0.1%

trifluoro-acetic acid was prepared and subsequently diluted

four-fold with the same solvent (matrix solution) Droplets

(1 lL) of a 1 : 1 (v/v) mixture of protein and matrix solution

were deposited on the sample slide and allowed to dry at

room temperature Positive ion mass spectra were acquired

in the linear mode with pulsed ion extraction Mass

assignments were based on an external calibration of the

instrument

DSC

DSC experiments were performed on a MicroCal VP-DSC

apparatus (MicroCal, Northampton, MA, USA) Prior to

analysis, proteins were exhaustively dialyzed against the

buffer stated in the legend of each figure, and degassed The

dialysis buffer was used for baseline scans and was present

as a reference buffer for the protein scans The system was allowed to equilibrate at 5C for 15 min, and temperatures from 5 to 129C were scanned at a rate of 85 C/h Thermograms obtained were analysed using the MicroCal version of ORIGIN The standard deviation indicated for each parameter corresponds to the error of curve fitting Details concerning the calculation of thermodynamic parameters and instrumentation have been published pre-viously [30,31]

CD Far-UV CD measurements were made at 25C in a Jasco J-810 spectropolarimeter (Tokyo, Japan) using cuvettes with a pathlength of 0.1 mm Proteins were exhaustively dialyzed against 50 mMformate, pH 2.8 All proteins were analysed at a concentration of 60 lM and spectra were corrected using the spectra of the dialysis buffer

Fluorescence spectroscopy Measurements of the intrinsic tryptophan fluorescence were carried out at 25C using a Kontron SFM 25 spectrofluorimeter (Kontron, Zurich, Switzerland) Unless stated otherwise, proteins were present at a concentration

of 1 lM in 50 mM formate, pH 2.8 Emission spectra between 300 and 400 nm were collected after excitation at

295 nm Spectra were corrected using the spectra of the buffer

Results

Protein expression of native and mutant gp41(24–157) Histidine tagged gp41(24–157), and a substantial number of mutants, have been expressed in E coli After centrifugation

of the cell lysate, all proteins were present in the pellet, which was solubilized in buffer containing 8Murea Purification was also carried out in the presence of chaotropic agents and refolding was achieved by a simple dialysis into 50 mM

formate, pH 2.8 The DSC analysis of gp41(24–157) did not provide evidence of partially or wrongly folded protein (see below)

Typically, a yield of 60 mg of pure protein per litre of cell culture was observed for native gp41(24–157) This yield appeared to be lower for certain gp41 mutants Multiple point mutations to the loop region, e.g as in the triple mutant L91K/I92K/W103D (see below), did not change the protein yield

Analysis of gp41(24–157) by CD, DSC and MALDI-TOF MS The far-UV CD spectrum of gp41(24–157) in 50 mM

formate, pH 2.8, is shown in Fig 1 The spectrum is literally superimposable to the CD spectrum of glycosylated gp41(21–166) at pH 7.5, as reported by Weissenhorn et al [32] Both spectra showminima at around 208 and 222 nm,

a crossover in sign at 202 nm, and a maximum at 193 nm, which are typical characteristics of a protein largely dominated by a-helix From the molecular ellipticity at

222 nm, an a-helix content of 75% has been calculated [33],

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which is consistent with the a-helix content of  80%

determined by Weissenhorn et al for glycosylated gp41(21–

166) at pH 7.5 [32]

Figure 2A (upper trace) shows the DSC thermogram of

gp41(24–157) after the renaturation process by dialysis into

50 mM formate, pH 2.8 (see the Materials and methods)

Two unfolding transitions at 110.4 and 119.5C are seen

(Table 1), demonstrating that this protein is

hyperther-mostable at lowpH The thermal unfolding was highly

cooperative with an DHvH/DHcalratio of 3.7 for the major

unfolding event, which is consistent with a cooperative

unfolding of a gp41 trimer Evidence that gp41 is trimeric

at pH 2.5–3 has previously been demonstrated using gel

filtration [13], analytical ultracentrifugation [19] and NMR

[9]

A major peak corresponding to the monomer, and two

smaller peaks corresponding to covalently linked dimeric

and trimeric forms of gp41(24–157), are seen in the

MALDI-TOF spectrum (Fig 2B) of the same sample It

is generally accepted that MALDI-TOF analysis results in

the disruption of all noncovalent interactions and that the

observed multimers correspond to covalently linked

multi-mers gp41(24–157) contains two cysteine residues which are

involved in an intrasubunit disulfide bond in the native

protein [34] The reaction of this protein sample with

Ellman’s reagent [35] showed that the two cysteine residues

are engaged in disulfide bonds (data not shown) This is

consistent with the monomer, observed by MS, having an

intrasubunit disulfide bond, whereas the dimer and trimer

peaks indicate the presence of intersubunit disulfide bonds

Fig 1 Far UV CD spectra of native gp41(24–157) (––) and of the

quadruple mutant W6OA/I124D/I131D/Q142N (- - - -) Proteins were

analyzed at a concentration of 60 l M in 50 m M formate, pH 2.8.

Fig 2 Analysis of native gp41(24–157) by DSC and MALDI-TOF

MS (A) DSC thermograms of gp41(24–157) before and after

reduc-tion with tris(2-carboxyethyl)phosphine (TCEP) Derived

thermo-dynamic parameters are shown in Table 1 (B) MALDI-TOF mass

spectra of gp41(24–157) before and after reduction with TCEP In

panels (A) and (B), the same samples were used for analysis The

sequence-derived mass of gp41(24–157) is 16801.5 l Spectra indicate

that the N-terminal methionine residue has been processed (C) Study

of the reversibility of the thermal unfolding of gp41(24–157) Shown

are segments of two consecutive DSC up-scans from 5 to 129 C.

Reversibility was defined as: % reversibility ¼ (DH cal2 /DH cal1 ) ·

100%, with DH cal2 being the change of enthalpy from the second

up-scan and DH cal1 the change of enthalpy from the first up-scan of the

same protein sample Reversibility data are given in Table 1 For

clarity reasons, DSC thermograms and mass spectra are moved

arbitrarily on the y-axis Proteins were analyzed in 50 m M formate,

pH 2.8.

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which have been described previously for glycosylated

gp41(21–166) [32] To verify the hypothesis of the presence

of intersubunit disulfide bonds, a gp41(24–157) sample was

analysed after reduction with TCEP, a reagent known to

reduce disulfides selectively over a pH range of 1.5–8.5 [36]

After reduction, only a single unfolding transition was

observed by DSC, which was characterized by a downshift in

Tmof 1.3C, with respect to the major peak before reduction

(Fig 2A, Table 1) This event was equally very cooperative

(DHvH/DHcal¼ 3.3), demonstrating that reduction does not

alter the trimeric state of gp41 The peaks corresponding

to covalent dimers and trimers in the mass spectrum of this

sample were significantly decreased as compared to the

sample analysed before reduction (Fig 2B) These minor

peaks can probably be attributed to the formation of

covalent multimers during ionization in the instrument, a

well-known phenomena of this technique, because multimer

peaks of similar size are observed in the spectrum of the

cysteine-free double mutant, C87S/C93S (data not shown)

This implies that the single transition seen in DSC after

reduction corresponds to the unfolding of noncovalently

associated trimers with reduced disulfide bonds, whereas

the major unfolding event before reduction represents the

unfolding of noncovalently associated trimers with

intra-subunit disulfide bonds The effect of disulfide reduction on

the thermal stability of gp41 trimers is thus relatively modest

(downshift in Tm of 1.3C) The absence of the second

unfolding transition after reduction demonstrates that this

event represents the unfolding of gp41 trimers characterized

by one or several intersubunit disulfide bonds

In summary, gp41(24–157) is, after renaturation, mainly

present in its native conformation, defined by noncovalently

associated trimers with intrasubunit disulfide bonds Based

on this result, all subsequent analyses were carried out using

protein taken after renaturation (nonreduced) The

param-eters of the major unfolding transition were used for data

analysis Furthermore, thermal unfolding of gp41(24–157)

is highly reversible, as calculated from two consecutive scans

of the protein (Fig 2C)

pH dependence of the thermal unfolding

of gp41(24–157)

gp41(24–157) has been shown to be hyperthermostable in

50 m formate, pH 2.8 To study the pH dependence of

protein unfolding, samples were analysed by DSC, CD and fluorescence spectroscopy over a pH range of 0.5–5.0 Protein was dialyzed into 50 mMformate and adjusted to the pH indicated by the addition of concentrated HCl or NaOH

The DSC scans of gp41(24–157) at different pH values are show n in Fig 3A A plot of Tmand DHvH/DHcal, as a function of pH, is shown in Fig 3B Cooperative unfolding events at temperatures of > 100C are seen in the pH range

of 1.0–4.0 The variation of Tmas a function of pH is of a slight valley shape, with the minimum at pH 2.25 (102C) The DHvH/DHcal ratio at pH 1.0–2.5 was  1 and increased to 3–4 at pH 2.8–3.5 This sudden increase was accompanied by an increase in Tm(Fig 3B) To evaluate whether the increase in DHvH/DHcal from  1 to  3 represents an association of monomers to trimers, or whether this increase corresponds to an increase in coop-erativity of the unfolding of gp41 trimers, 14 samples of gp41 at different pH values (1.0–4.0) were analysed by fluorescence spectroscopy A gp41 monomer contains eight tryptophan residues Three are involved in a buried tryptophan cluster [11], characterized by the tight packing

of W60 of helix N between W117 and W120 located at helix C of a neighbouring monomer Trimer dissociation into monomers disrupts this cluster, leading to an exposure

of the three buried tryptophan residues to the solvent, giving rise to a shift in the maximum of the emission spectrum, as shown for the gp41 model [14] The maximum of the fluorescence emission spectrum of gp41 at pH 2.8 was

349 nm (data not shown) This maximum of fluorescence emission for the analysis of gp41 at pH between 1.0 and 4.0 (data not shown) was unchanged (349 ± 1 nm), suggesting

no disruption of the tryptophan cluster and no trimer dissociation at this pH range

Samples of gp41 (all at 60 lM) at this pH range have also been analysed by CD Over the range of pH 1.0–4.0, spectra are closely superimposable and no shift in the minima or maxima is observed (data not shown) Differences in molecular ellipticity are in the range of the error associated with determination of the protein concentration

Monomer–trimer equilibrium

A monomer–trimer equilibrium has been described for the cysteine double mutant of simian immunodeficiency virus

Table 1 Thermodynamic parameters derived from the DSC analysis of gp41(24–157), gp41(34–170) and gp160 (Figs 2 and 5) ND, not determined.

Sample

T m

(C)

DH cal

(kcalÆmol)1)

DH vH

(kcalÆmol)1) DH vH /DH cal

Reversibility (%) gp41(24–157) nonreduceda 110.4 61 ± 0.4 228 ± 2 3.7 80

119.5 21 ± 0.5 165 ± 5 7.8 85 gp41(24–157) reduced (36 l M )b 109.1 94 ± 0.4 308 ± 2 3.3 85 gp41(24–157) reduced (1 l M )b 109.1 91 ± 1.0 303 ± 4 3.3 ND gp41(34–170), glycosylated c 112.6 38 ± 0.5 201 ± 4 5.3 25

106.4 32 ± 1.5 218 ± 6 6.8 100 111.8 41 ± 1.5 191 ± 7 4.6 0

a Non-reduced protein corresponds to gp41(24–157) after the dialysis step into 50 m M formate at pH 2.8 (Materials and methods).

b

Reduction was carried out by overnight dialysis of the protein into 50 m M sodium formate, 150 l M Tris(2-carboxyethyl)phosphine (TCEP)

at 4 C c See the Materials and methods for a description of the protein.

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(SIV) gp41 and the data obtained have been extrapolated to

HIV gp41 [19,37,38] Serial dilutions of gp41(24–157), at

concentrations between 36 lM and 0.3 lM, have been

prepared in 50 mMformate, pH 2.8, incubated for 5 h to

achieve equilibration, and then analysed by DSC (down to

1 l ) and intrinsic tryptophan fluorescence spectroscopy

The thermodynamic parameters of unfolding of the mono-mer are expected to be different from those of the trimono-mer Trimer dissociation is bound to alter the ratio of DHvH/

DHcal, indicative of the content of the cooperatively unfolding unit All DSC scans of serial dilutions of gp41(24–157) were very similar, providing no evidence for trimer dissociation Figure 4 shows a superimposition of the scans obtained with 36 and 1 lM of protein Derived thermodynamic parameters for these thermograms are given in Table 1

The same samples were analysed by fluorescence spectroscopy, and the disruption of the tryptophan cluster (see above) as a result of trimer dissociation is expected to change the maximum of the fluorescence emission spectrum However, the emission spectra of all samples in the concentration range between 36 and 0.3 lM were very similar (data not shown) and the maxima of all tryptophan fluorescence emission spectra were 349 ± 1 nm, indicating

no trimer dissociation

Analysis of glycosylated gp41 and gp160

To evaluate the influence of glycosylation on the thermal stability, experiments were carried out with glycosylated gp41 Furthermore, experiments were also carried out with glycosylated gp160 (see the Materials and methods for a detailed description of both proteins) to study the thermo-dynamics of protein unfolding of the entire surface protein Interestingly, the DSC scan of gp160 at 0.3 mgÆmL)1in NaCl/Pi, pH 7.4, showed no cooperative unfolding event (data not shown) A rescan of this sample showed no significant difference from the first scan However, a cooperative unfolding event at 121.2, 120.6 and 118.3C was seen when the pH was lowered to 5.5, 4.0 and 3.4, respectively (Fig 5) The DSC scan of gp160 at pH 2.8 (using the same conditions as for gp41, see above) showed three unfolding transitions at 93.3, 106.4 and 111.8C

Fig 3 Study of the pH dependence of the thermal denaturation of

gp41(24–157) (A) DSC thermograms of gp41(24–157) in the pH range

of 0.5–5.0 Protein was analyzed in 50 m M formate and adjusted to the

pH indicated by the addition of concentrated HCl or NaOH For

clarity reasons, traces have been moved arbitrarily on the y-axis (B)

Plot of T m (ÆÆÆÆ) and DH vH /DH cal (––) as a function of pH Values have

been taken from the data presented in (A).

Fig 4 Study of gp41(24–157), at different concentrations, by DSC Superimposition of DSC scans of gp41(24–157) at 36 l M (ÆÆÆÆ) and at

1 l M (––) Derived thermodynamic parameters are listed in Table 1 Proteins were analyzed in 50 m M formate, pH 2.8 Proteins were incubated for 5 h prior to analysis.

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(Fig 5, Table 1) Most interestingly, the thermodynamic

parameters of glycosylated gp41 (Fig 5A, Table 1), which

shares 99% sequence identity with the corresponding

sequence of gp160, were very similar to the corresponding parameters of the third transition seen for gp160

The Tmvalues of the major unfolding events of glycosyl-ated gp41 (112.6C) and nonglycosylated gp41 (110.4 C) were very similar (Table 1, Figs 2 and 5) Differences in DH values of the major events can rather be attributed to the pre-sence of two minor transitions in the scan of the glycosylated gp41 (which might be attributed to misfolded protein) than to differences between the proteins The unfolding of glycosylated gp41 was less reversible than that of the nonglycosylated form (Table 1, Figs 2C and 5B) However,

in interpreting these data it has to be taken into account that, although the sequences of both proteins are almost identical, the molecular boundaries of glycosylated gp41(34–170) and nonglycosylated gp41(24–157) differ slightly

Analysis of gp41(24–157) with a single point mutation Our vaccine approach is based on the generation of gp41 mutants that resemble a fusion intermediate of the protein

It is generally accepted that this fusion intermediate state is trimeric and that helices N and C do not interact Ways have been explored to destabilize the interaction between helices

N and C by site-directed mutagenesis Alternatively, muta-tions were carried out to stabilize the interaction between the three helices N in order to favour the trimeric state of gp41

An initial series of mutations involved amino acids in positions a and d, which are critical for helix interaction Replacement of T58 with I resulted in an increase in Tm

of 2.2C (Fig 6A, Table 2) This mutation results in the generation of an additional hydrophobic cluster between helices N I124 is located at position a in helix C and interacts, at the same time, with two hydrophobic amino acids located at two neighbouring helices N Replacement

of I124 with S and D significantly destabilizes the protein (Fig 6, Table 2) Interestingly, replacement of I124 with D rendered the unfolding irreversible

Analysis of gp41(24–157) with several point mutations Amino acids Q64 and Q66 are on positions c and e, respectively Their substitution with A resulted in a decrease

in Tmof 5 C (Fig 6B, Table 2), and no changes in the

CD spectrum were noted

There is nowevidence that the loop region of gp41 interacts with gp120 [39] This loop contains a highly conserved di-cysteine motif [40], which forms an intrasub-unit disulfide bond It has recently been suggested that the loop region of the mutant lacking the disulfide bond may be less stable and more dynamic than that of the wild type [40] Here, we show that the replacement of both cysteines with serine has a modest impact on the thermal stability (Fig 6B, Table 2) of the protein, showing a downshift of 2.3C in Tm compared to the wild type These data are consistent with the DSC analysis of native gp41 after reduction with TCEP, which resulted in a decrease in Tmof 1.3C (Table 1) The exposed surface area of the C-terminal half of helix C

is highly charged, whereas helix N is mainly neutral Several

of these charged residues of helix C have been mutated to alanine Both double mutants E143A/K144A and K144A/ E148A showed a decrease in Tm of  4 C (Fig 6B, Table 2)

Fig 5 DSC analysis of gp160 and gp41(34–170) See the Materials and

methods for further information (A) Both proteins are glycosylated.

Proteins were analyzed at pH 2.8, 3.4 and 4.0 in 50 m M formate and

adjusted to the pH indicated by the addition of concentrated NaOH.

Protein, at pH 5.5, was analysed in 50 m M Mes DSC scans of gp160

and gp41(34–170) were performed at different pH values gp41(34–

170) shares 99% sequence identity with the corresponding sequence

in gp160 (B) Shown are segments of two consecutive up-scans of

gp41(34–170) from 5 to 129 C, at pH 2.8 Scans have been moved

arbitrarily on the y-axis Derived data are given in Table 1.

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The DSC scan of a triple mutant, in which three glutamine residues (at positions 40, 41 and 51, all located

on the N-terminal half of the N-terminal helix) were replaced with alanine, showed a major unfolding event

at 119.1C (Fig 7A, Table 2), which is significantly higher than that found for the native protein (110.4C) This unfolding transition was preceded by a broad transition centred at 97 C

Another triple mutant was aimed at combining mutations which stabilize the interaction between the three N-terminal helices (Q51I/T58I) with a mutation that destabilizes the interaction between helices N and C (I124D) The stabilizing effect of the T58I exchange has been described above, and the Q51 is located in a similar position in the heptad repeat

as T58, indicating that its replacement with Ile has a similar effect as the T58 replacement I124 is located on the interface between helices N and C, and its replacement with glutamate dramatically altered the thermodynamics of protein unfolding (see above) In contrast to I124D, mutant Q51I/T58I/I124D unfolds in a single transition, character-ized by a moderate downshift in Tmby 4 C, as compared

to the native protein (Fig 7A, Table 2)

All four amino acids replaced in the quadruple mutant W6OA/I124D/I131D/Q142N are located in the interface between helices N and C W60 is located on helix N and is part of the tryptophan cluster comprising W117 and W120

of helix C The other three amino acids mutated (I124, I131 and Q142) form part of helix C These four mutations have dramatically changed the thermodynamics of protein unfolding Protein unfolding starts at  60 C (Fig 7B) and the peak can be deconvoluted into three transitions centred on 72, 76 and 81C At  95 C, the protein starts

to aggregate The CD spectrum of this quadruple mutant was significantly different from that of the native protein (Fig 1) The percentage of a-helix calculated for that mutant was 56% [33], significantly lower than for the native protein (75%)

In another mutant, three solvent-accessible hydrophobic amino acids (L91, I92, W103) in the loop region were replaced with charged residues (K, K, D, respectively) in order to render this protein soluble at neutral pH This mutant was soluble to 80 lgÆmL)1 in 10 mM Na2HPO4/ NaH2PO4, 0.05% Tween-20, pH 7.5, a considerable improvement on the native protein which is retained qualitatively on 0.22 lm filters after dialysis into this buffer The DSC scan of this triple mutant, at pH 2.8, shows a single event at 108C, indicative of only small alterations to protein stability as a result of these three, nonconservative replacements (Fig 7A, Table 2) When the DSC analysis

is repeated at pH 7.5, a single unfolding event, with a

Tmof 110.8C, is seen (Fig 7A)

Analysis of gp41 constructs of directly fused helices

We created mutants in which helices N and C (or vice versa) were joined directly in order to generate con-straints that might prevent the adoption of this fusogenic state and favour an alternative arrangement This alternative arrangement might be characterized by the exposure of epitopes which are hidden in the helix bundle arrangement Details on these mutants are found

in Table 3

Fig 6 DSC analysis of gp41(24–157) mutants containing single (A) or

double (B) point mutations Thermodynamic parameters derived are

listed in Table 2 Proteins were analysed in 50 m M formate, pH 2.8,

and traces were moved arbitrarily on the y-axis.

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The CD spectra of these five mutants showthe

characteristics of a highly a-helical protein (data not

shown) All five constructs are very thermostable and their

unfolding is at least partially reversible (Fig 7C, Table 3)

The size of the cooperatively unfolding unit was between

1.7 and 2.8, indicating some sort of monomer association

Interestingly, the two NC mutants (fusion of helix N to

helix C), which are closer to the native form, are less thermostable than the CN constructs (fusion of helix C to helix N) CN3 is characterized by an upshift in Tm of

 2 C, accompanied by an increase in the enthalpy change

as compared to the wild-type protein (Table 3) Mutants CN1, CN2 and CN3 differ only in tw o or, respectively, three additional amino acids on the C-terminal extension

Table 2 Thermodynamic parameters for the major unfolding event for the DSC analysis of gp41(24–157) mutants containing one to four point mutations (Figs 6 and 7) The second column shows the location of point mutations in the structure in the six-helix bundle of gp41; letters N, C, and

L correspond to helix N, helix C and loop, respectively; lower case letters indicate the position of the mutation in the heptad repeat ND, not determined.

Sample

Position of the mutation

in the structure

T m

(C)

DH cal

(kcalÆmol)1)

DH vH

(kcalÆmol)1) DH vH /DH cal

Reversibility (%)

96.0 36 ± 3.8 159 ± 15 4.4 0 105.3 33 ± 1.6 127 ± 7 3.8 0 Q64E/Q66E N-c/N-e 105.5 69 ± 2.0 172 ± 4 2.5 66 C87S/C93S L/L 108.1 90 ± 0.5 300 ± 2 3.3 33 E143A/K144A C-f/C-g 106.6 56 ± 1.2 263 ± 8 4.7 ND K144A/E148A C-g/C-d 105.6 61 ± 1.3 216 ± 5 3.5 ND Q40A/Q41A/Q51A N-g/N-a/N-d 119.1 55 ± 0.4 164 ± 2 3.0 55 Q51I/T58I/I124D N-d/N-d/C-a 106.2 77 ± 0.8 243 ± 3 3.1 52 L91K/I92K/W103D L/L/L 108.0 91 ± 0.4 293 ± 2 3.2 59 W6OA/I124D/I131D/Q142N N-f/C-a/C-a/C-e 71.7 13 ± 2.5 125 ± 8 9.8 ND

a Parameters of the three transitions.

Fig 7 Analysis of gp41(24–157) mutants by DSC (A) DSC scans of gp41(24–157) mutants containing three point mutations The upper three scans were carried out with protein in in 50 m M formate, pH 2.8, the lower trace with protein in 10 m M Na 2 HPO 4 /NaH 2 PO 4 , 0.05% Tw een-20, pH 7.5 (B) DSC scan of a mutant containing four point mutations; note the difference in scale of the y-axis Derived thermodynamic parameters are shown

in Table 2 (C) DSC scans of recombinant constructs of gp41(24–157) in which helices N and C have been joined directly A definition of these molecules and the thermodynamic parameters derived are given in Table 3.

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of helix C, whereas the fragment corresponding to helix N

is unchanged

Discussion

Proteins are the result of evolution and their features reflect

an optimal adaptation to a multitude of environmental,

mechanistic and other factors Some organisms have

evolved in a way which enables them to live in a

hyperthermophilic environment characterized by

tempera-tures of > 80C Elevated growth temperature has thus

been a driving force for the evolution of their proteins

towards hyperthermostability Early studies aimed at

understanding thermostability were based on sequence

comparisons, but the increasing availability of 3D structures

has made it possible to identify structural determinants of

protein hyperthermostability by comparing 3D structures of

hyperthermophilic organisms with their mesophilic

coun-terparts [41,42] At least 15 physical and chemical factors

giving rise to thermostability have been identified [43]

However, a major role in achieving thermostability has been

attributed to an increase in salt bridges and H-bonds, better

hydrophobic internal packing, enhanced secondary

struc-ture propensity, helix dipole stabilization and burying of

a hydrophobic accessible surface area [41]

gp41 is a special case in terms of thermal stability The

protein has evolved to be hyperthermostable, but, in

contrast to the studies described above, elevated growth

temperatures can be excluded as a factor driving protein

evolution This hence raises the question of whether the

characteristics of thermostable proteins, which have evolved

as an adaptation to high temperature, are different from a

protein that has evolved to be hyperthermostable as a

consequence of the adaptation to a factor other than

elevated temperature

The availability of an increasing number of complete

genome sequences has allowed comparison of the amino

acid composition of entire genomes of mesophiles and

hyperthermophiles [44,45] It emerged that proteins of

hyperthermophilic organisms have a higher percentage of

charged residues (K,R,D,E) and a lower number of polar

noncharged residues (Q,N,S,T) than mesophiles The

difference between charged and polar noncharged residues

(termed CvP bias) was found to be the only criterion that

distinguishes mesophiles (CvP: )1 to 5%) from

hyper-thermophiles (CvP: 10–15%) on a global basis [45] The

authors conclude that the evolutionary adaptation of

proteins to elevated temperatures (in hyperthermophile

species) is dominated by the replacement of polar

noncharged residues with charged ones This implies that the formation of ion bonds is the dominating mechanism leading to hyperthermostability as a result of adaptation to elevated temperatures This statistical approach is suppor-ted by experimental data Lowering the pH results in the perturbation of electrostatic interaction, including ion pairs, which is complete at pH 2.0 [46] For a large number of proteins from hyperthermophiles, monitoring thermostabil-ity as function of pH shows a dramatic decrease as the pH approaches 2.0 [46–49], consistent with protein destabiliza-tion by perturbadestabiliza-tion of ionic interacdestabiliza-tion

This was not the case for gp41 The native protein was shown to be hyperthermostable over the pH range of 1–4 (Fig 3) and a similar thermostability has been observed at

pH 7.5 for the soluble triple mutant L91K/I92K/W103D (Fig 7), indicating that protonation has little effect on the thermal stability of the protein Interestingly, the CvP bias for gp41 was, at )13%, the opposite from proteins in hyperthermophile organisms (CvP¼ 10–15%) A dense internal hydrophobic packing is thus more likely to determine thermostability, which is supported by the fact that gp41 contains 29% aliphatic amino acids, above the average found in mesophilic and thermophilic species [45]

It can be concluded that the evolution of gp41 towards hyperthermostability was not dominated by an increase in ionic or electrostatic interaction, shown to be the major feature of proteins from thermophilic species It can thus be hypothesized that structural features giving rise to thermo-stability are different for proteins which have evolved in adaptation to elevated temperatures and hyperthermostable proteins found in mesophile organisms

It appears that the formation of trimeric coiled-coil structures is a common principle of viral membrane fusion [34] It has been demonstrated that the influenza virus fusion protein, HA2 [50,51], and the paramyxovirus fusion protein [52], are equally thermostable It can further be hypothes-ized that this trimeric coiled-coil fold might be associated with thermostability

DSC analysis of the double cysteine to alanine mutant of SIV gp41(27–149) (in 50 mMformate, pH 3.0) has recently been reported [38] The protein was equally hyperthermo-stable with a Tm of 110.7C, and the cooperatively unfolding unit was shown to contain a trimer (DHvH/

DHcal¼ 2.91) Here we report very similar data (Tm¼ 110.7 C, DHvH/DHcal¼ 3.53) for the analysis of HIV gp41(24–157) using the same buffer conditions Based

on the observations of Wingfield et al [20], that SIV gp41 can be expressed and refolded with substantially higher yields than HIV gp41, the authors [37,38] have used the SIV

Table 3 Parameters derived from the DSC analysis of the helix–fusion constructs of gp41(24–157) (see Fig 7C) Molecules are defined in the second column of the table All molecules contain the C-terminal tag, GGGGSHHHHHH.

Sample

Position of the mutation

in the structure

T m

(C)

DH cal

(kcalÆmol)1)

DH vH

(kcalÆmol)1) DH vH /DH cal

Reversibility (%) NC1 A30-D78_N113-K154 91.0 103 ± 3.1 176 ± 4 1.7 65

NC2 A30-K77_W117-K154 86.5 84 ± 1.0 234 ± 3 2.8 71

CN1 W117-K154_A30-K77 101.9 59 ± 1.3 140 ± 5 2.4 37

CN2 W117-A156_A30-K77 106.8 81 ± 0.5 206 ± 2 2.5 39

CN3 W117-W159_A30-K77 112.5 140 ± 0.6 241 ± 1 1.7 18

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