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In addition, we report that the replacement of of Arg80 by Ser significantly decreases the cytotoxic activity of BS-RNase and the stability of the NCD form with respect to the parent prot

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ribonuclease: new insights from heat and chemical

denaturation studies on variants

Concetta Giancola1, Carmine Ercole1, Iolanda Fotticchia1, Roberta Spadaccini2, Elio Pizzo3,

Giuseppe D’Alessio3and Delia Picone1

1 Department of Chemistry ‘Paolo Corradini’, University of Naples ‘Federico II’, Italy

2 Department of Biological and Environmental Sciences, Universita’ degli Studi del Sannio, Benevento, Italy

3 Department of Structural and Functional Biology, University of Naples ‘Federico II’, Italy

Keywords

calorimetric analysis; chemical denaturation;

cytotoxic ribonucleases; domain-swapping;

structure–activity relationships

Correspondence

D Picone or C Giancola, Dipartimento di

Chimica, Universita` di Napoli ‘Federico II’,

Complesso Universitario di Monte

Sant’Angelo, Via Cintia, 80126 Napoli, Italy

Fax: +39 081674409; +39 081674499

Tel: +39 081674406; +39 081674266

E-mail: delia.picone@unina.it;

concetta.giancola@unina.it

(Received 29 June 2010, revised 17

September 2010, accepted 25 October

2010)

doi:10.1111/j.1742-4658.2010.07937.x

Bovine seminal ribonuclease (BS-RNase), a homodimeric protein displaying selective cytotoxicity towards tumor cells, is isolated as a mixture of two isoforms, a dimeric form in which the chains swap their N-termini, and an unswapped dimer In the cytosolic reducing environment, the dimeric form

in which the chains swap their N-termini is converted into a noncovalent dimer (termed NCD), in which the monomers remain intertwined through their N-terminal ends The quaternary structure renders the reduced pro-tein resistant to the ribonuclease inhibitor, a propro-tein that binds most ribo-nucleases with very high affinity On the other hand, upon selective reduction, the unswapped dimer is converted in two monomers, which are readily bound and inactivated by the ribonuclease inhibitor On the basis

of these considerations, it has been proposed that the cytotoxic activity of BS-RNase relies on the 3D structure and stability of its NCD derivative Here, we report a comparison of the thermodynamic and chemical stability

of the NCD form of BS-RNase with that of the monomeric derivative, together with an investigation of the thermal dissociation mechanism revealing the presence of a dimeric intermediate In addition, we report that the replacement of of Arg80 by Ser significantly decreases the cytotoxic activity of BS-RNase and the stability of the NCD form with respect to the parent protein, but does not affect the ribonucleolytic activity or the dissociation mechanism The data show the importance of Arg80 for the cytotoxicity of BS-RNase, and also support the hypothesis that the reduced derivative of BS-RNase is responsible for its cytotoxic activity

Abbreviations

BS-RNase, bovine seminal ribonuclease; DSC, differential scanning calorimetry; GSH, glutathione; hA-BS-RNase, G16S ⁄ N17T ⁄ P19A ⁄ S20A variant of bovine seminal ribonuclease; hA-mBS, G16S ⁄ N17T ⁄ P19A ⁄ S20A variant of the monomeric N67D variant of bovine seminal ribonuclease with Cys31 and Cys32 linked to glutathione moieties; mBS, monomeric N67D variant of bovine seminal ribonuclease with Cys31 and Cys32 linked to glutathione moieties; MxM, dimeric form of bovine seminal ribonuclease in which the chains swap their N-termini; M=M, unswapped dimer of bovine seminal ribonuclease; NCD, noncovalent dimer; PDB, Protein Data Bank; RI, ribonuclease inhibitor; RNase A, bovine pancreatic ribonuclease; S80-BS-RNase, R80S variant of bovine seminal ribonuclease; S80-hA-BS-RNase,

R80S ⁄ G16S ⁄ N17T ⁄ P19A ⁄ S20A variant of bovine seminal ribonuclease; S 80 -hA-mBS, R80S ⁄ G16S ⁄ N17T ⁄ P19A ⁄ S20A variant of the

monomeric N67D variant of bovine seminal ribonuclease with Cys31 and Cys32 linked to glutathione moieties; S 80 -mBS, R80S variant of the monomeric N67D variant of bovine seminal ribonuclease with Cys31 and Cys32 linked to glutathione moieties.

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The outstanding feature of bovine seminal

ribonucle-ase (BS-RNribonucle-ase), proposed initially by Piccoli et al.,

(1992) [1] on the basis of biochemical data and

struc-turally proven a few years later [2,3], is the formation

of a dimeric form in which the chains swap their

N-termini (MxM) This phenomenon was later found

in many proteins, and is now well known under the

name of ‘3D domain swapping’ In most cases, the

swapping is associated with new biological functions,

and it has also been proposed as a possible

mecha-nism for protein aggregation in misfolding-associated

pathologies [4] To date, more than 150 structures of

swapped proteins are present in the Protein Data

Bank (PDB) Among these, BS-RNase still represents

a special case, because the native protein is isolated as

a mixture of two dimeric isoforms, MxM and an

M=M [1], with or without the exchange of N-termini

respectively, in a molar ratio of about 2 : 1 Therefore,

only for this protein, the swapping is a physiological,

equilibrium process consisting of a dimer-to-dimer

interconversion; that is, it is not associated with

varia-tion in the quaternary structure The swapping is

considered to be a prerequisite for most additional

biological properties accompanying the basal

enzy-matic activity, including a selective cytotoxic activity

towards malignant tumor cells [5] However, the X-ray

structures of the two isoforms have revealed only

minor differences [2,3], located essentially at the loop

connecting the dislocated arms to the main body of

the protein On the other hand, a so-called ‘buried

diversity’ [6] becomes evident when the protein is

con-sidered under different environments, such as cytosolic

reducing conditions

In vitro, under mild reducing conditions, the two

interchain disulfides bridging the subunits of BS-RNase

undergo selective cleavage, so that M=M is converted

into two monomers, whereas MxM maintains a

dimeric structure, stabilized by noncovalent

interac-tions of the N-termini [1] The monomeric form of

BS-RNase is readily neutralized by the ribonuclease

inhibitor (RI) [7], a protein that is abundant in

mam-malian cells, whereas the quaternary structure of the

reduced dimer, henceforth called the noncovalent dimer (NCD), allows this protein to evade RI binding [8] It has been proposed that RI prevents endogenous RNA degradation by binding monomeric ribonucleases with very high affinity [9] A schematic representation

of the different forms that BS-RNase can adopt in different environments is given in Fig 1, to emphasize that, in the reducing conditions of the cytosol, BS-RNase exists both as a monomer and as swapped NCD stabilized by noncovalent interactions NCD, which is a transient species because, in solution, it dis-sociates into two monomers, is considered to be the form responsible for the cytotoxic activity of the enzyme, given its resistance to RI Furthermore, it has been reported that the structural determinants that favor the proper quaternary structure [6,10] and the stability in solution of the swapped form play a significant role in the additional biological properties

of the enzyme

In this study, we have investigated the relationship between the cytotoxic activity and the stability of the NCD form of BS-RNase in comparison with variants obtained by replacing the 16–20 hinge loop region and⁄ or Arg80 with the corresponding residues of

Structured digital abstract

l MINT-8050499 : BS-RNase (uniprotkb: P00669 ) and BS-RNase (uniprotkb: P00669 ) bind ( MI:0407 ) by biophysical ( MI:0013 )

l MINT-8050482 : BS-RNase (uniprotkb: P00669 ) and BS-RNase (uniprotkb: P00669 ) bind ( MI:0407 ) by classical fluorescence spectroscopy ( MI:0017 )

l MINT-8050435 : BS-RNase (uniprotkb: P00669 ) and BS-RNase (uniprotkb: P00669 ) bind ( MI:0407 ) by circular dichroism ( MI:0016 )

Fig 1 Crystal structures of the multiple forms of BS-RNase: mBS, PDB code 1N1X; M=M, PDB code 1R3M; MxM, PDB code 1BSR; NCD, PDB code 1TQ9.

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bovine pancreatic ribonuclease (RNase A) It is

worth noting that none of these residues is actually

implicated in the catalytic activity We have already

reported that neither substitution, i.e the changes in

the 16–20 region or the change at Arg80, significantly

affects the swapping propensity of BS-RNase [11,12]

However, when the R80S mutation is inserted into the

construct containing the 16–20 hinge loop region of

RNase A [R80S⁄ G16S ⁄ N17T ⁄ P19A ⁄ S20A variant of

BS-RNase (S80-hA-BS-RNase)], the MxM⁄ M=M

molar ratio in the equilibrium mixture is changed from

2 : 1 to 1 : 2 [12] On the basis of the hypothesis that

ascribes the special functions of BS-RNase to the

swapped form, in this study we investigated the

biolog-ical activity of this mutant, and found that it loses

almost all of the cytotoxic activity Interestingly, the

single R80S substitution is sufficient to reduce the

anti-tumor activity almost to the same extent, leading us to

assign to this residue a pre-eminent role in the

cyto-toxic activity of BS-RNase, independently of the hinge

sequence Furthermore, evaluation of the chemical and

thermal stability of the NCD forms of the variant

pro-teins in comparison with those of the parent one

sup-ports the hypothesis that the reduced swapped dimer

represents the bioactive form of BS-RNase

Results

BS-RNase and its R80S variant (S80-BS-RNase), its

G16S⁄ N17T ⁄ P19A ⁄ S20A variant (hA-BS-RNase) and

R80S/G16S/N17T/P19A/S20A variant (S80-hA-BS-RNase)

were expressed in monomeric form, with Cys31 and

Cys32 linked covalently and reversibly to two

glutathi-one (GSH) moieties, as already reported [12,13] The

correctness of the fold of each monomeric protein was

confirmed by comparing their CD spectra and Kunitz

enzymatic activity on yeast RNA [14] with those of

parent monomeric BS-RNase (data not shown)

Fur-thermore, we compared the 2D NMR spectra of all

the monomeric variant proteins with that of the parent

monomeric N67D variant with Cys31 and Cys32

linked to GSH moieties (mBS), and found that the

res-onances of most amide signals are almost coincident,

the differences being essentially restricted to the

back-bone and side chain of the mutated residue(s) and of

those closest (in space) This is illustrated in detail for

the R80S variant of mBS (S80-mBS) in Fig 2: panel

(A) shows the overlay of the 1H-15N-HSQC spectrum

with that of mBS, and panel (B) gives a detail of the

3D structure of monomeric BS-RNase (PDB

code 1N1X), showing the local environment of Arg80

It is very evident that the shifted residues belong to

the region encompassing Arg80 (78–84), to the hinge

(15 and 16) and to regions 45–49 and 101–103, which are less than 4 A˚ from the Arg80 side chain The overlay of the 1H-15N-HSQC spectrum of mBS with those of its G16S⁄ N17T ⁄ P19A ⁄ S20A mBS and R80S⁄ G16S ⁄ N17T ⁄ P19A ⁄ S20A variants, indicated as hA-mBS and S80-hA-mBS respectively, is shown

in Fig S1

Biological activity The monomeric proteins were converted into dimers

by removal of the protecting GSH moieties and

15 N (p

1 H (p.p.m.)

A

B

Fig 2 (A) Overlay of the 1 H– 15 N-HSQC spectra of the monomeric derivatives of BS-RNase (black) and S 80 -BS-RNase (red) at 300 K Residues whose resonances are shifted are labeled (B) Details of the 3D structure of monomeric BS-RNase (PDB code 1N1X), show-ing the local environment of Arg80 Residues that are less than 4 A˚ from the Arg80 side chain are indicated.

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reoxidation of the intersubunit disulfides, followed by

incubation at 37C to allow the interconversion of

M=M and MxM to reach equilibrium The cytotoxic

activity of the proteins towards tumor cells was

mea-sured by adding increasing concentrations (ranging

from 12.5 to 100 lgÆmL)1) of each variant to

malig-nant SVT2 cells, using BS-RNase as a positive control

For a negative control, the proteins were also assayed

on nontumor 3T3 cell cultures at a final concentration

of 100 lgÆmL)1, and found to be nontoxic (Fig S2)

The percentage of SVT2 cells surviving, illustrated in

Fig 3, show that the replacement of Arg80 by Ser

induced a significant drop in cytotoxic activity,

inde-pendently of the hinge sequence In contrast, we found

that changes in the 16–20 hinge region had only small

effects, as the cytotoxic activity of BS-RNase was only

slightly higher than that of hA-BS-RNase, and that of

S80-BS-RNase was very close to that of S80

-hA-BS-RNase

Stability of NCD versus monomeric forms

In the search for the molecular basis for the induction

of the loss of cytotoxic activity of BS-RNase variants

reported in Fig 3, we followed by CD the thermal

denaturation process of NCD derivatives, measuring

the molar ellipticity at 222 nm as a function of

temper-ature (Fig 4A) As a comparison, the melting curves

of the corresponding monomers are reported in

Fig 4B The melting temperatures (Tm values) of

NCD derivatives, which represent the midpoint of

the denaturation curve (Fig 4A), were 59.4C for

BS-RNase and 59.0C for hA-BS-RNase, i.e very

close to each other In turn, significantly lower, and

comparable, Tm values of 54.3C and 53.6 C were

found for S80-hA-BS-RNase and S80-BS-RNase,

respec-tively A similar trend was observed for the CD melting

temperatures of monomeric derivatives (Fig 4B) and

[12], which can be separated into two groups, corre-sponding to Tmvalues around 58.0C and 54.0 C for the proteins with Arg80 or Ser80, respectively

The CD melting curves of the monomers were used

to calculate the denaturation enthalpy changes by using the van’t Hoff equation (Eqn 3 in Experimental procedures), which describes two-state NMD transi-tions The data obtained, reported in Table 1, indicate that DH0

v:H: values of the monomers follow the same trend of Tm values, with those of mBS and hA-mBS close to each other and higher than the DH0

v:H: values

of S80-mBS and S80-hA-mBS, which, in turn, are close

to each other

As a further step, we performed a calorimetric analysis of all the proteins by standard differential

0

25

50

75

100

Fig 3 SVT2 cell survival after 48 h of incubation with different

amount of BS-RNase ( ), S80-BS-RNase (h), hA-BS-RNase ( ) and

S80-hA-BS-RNase ( ).

0

1 A

B

Temperature (°C)

Temperature (°C)

0

1

Fig 4 Thermal unfolding curves obtained following the change of

CD signal at 222 nm of NCDs (A) and monomeric derivatives (B) of BS-RNase ( ), hA-BS-RNase (x), S80-BS-RNase (•), and S 80 -hA-BS-RNase (h) The unfolded fraction of protein was calculated as (Q –

Q min ) ⁄ (Q max – Q min ); Q is the ellipticity at 222 nm at a given temper-ature, and Q max and Q min are the maximum and minimum values of ellipticity corresponding to the denaturated state and native state of proteins, respectively.

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scanning calorimetry (DSC) measurements The

calori-metric profiles of all proteins are reported in Figs S3

and S4 for monomers and NCDs respectively The

denaturation enthalpies of the monomeric derivatives

obtained from the DSC curves, reported as DH0

cal in Table 1, were in good agreement with the van’t Hoff

enthalpies derived from DSC and CD curves, thus

confirming that the thermal denaturation process for

these proteins is a two-state transition process An

inspection of the whole set of thermodynamic

parame-ters of monomeric forms, collected in Table 1, shows

that hA-mBS and mBS have comparable stabilities,

indicating that the substitution of four residues in the

hinge region does not significantly perturb the global

stability of the monomeric form of BS-RNase On the

other hand, the single mutation R80S leads to

decreases of about 4C in the melting temperature

and of about 100 kJÆmol)1 in the value of DH0

cal This destabilization is well reflected in DG0 values, showing

that Arg80 is crucial for the stability of monomeric

form of BS-RNase

For the NCD forms, thermal denaturation was

found to be an irreversible process, because there was

no refolding upon cooling of the protein solutions

The irreversibility of the denaturation process does not

allow Gibbs energy calculations, but only a

compari-son of the melting temperatures and unfolding

enthalpy changes DH0

caland DH0

v:H: values, both calcu-lated from calorimetric profiles, are very similar and

the DH0

cal=DH0

v:H: ratio is in the range 0.98–1.07,

sug-gesting that the unfolding of the dimers is close to

being a one-step process (Table 1) The DH0

v:H: from

CD and DSC curves, relative to the unfolding of the secondary and tertiary structure respectively, are for each dimer very close, indicating simultaneous collapse

of both structures As also indicated in Table 1, the enthalpy changes for the NCD forms are very similar

to each other For a comparison of the DH0

cal values of the dimers with those of the corresponding monomers, the enthalpy changes of NCD forms were calculated

at the melting temperatures of the corresponding monomers, using the Kirchhoff equation Values of

607 kJÆmol)1, 642 kJÆmol)1, 581 kJÆmol)1 and 605 kJÆmol)1 were obtained for NCD forms of BS-RNase, hA-BS-RNase, S80-BS-RNase and S80-hA-BS-RNase, respectively All values are less than twice those of the respective monomers This indicates a loss of interac-tions of the monomers in the dimeric structures In conclusion, all of the reported data indicate that the R80S mutation is crucial for the loss in the enthalpic content of NCDs of BS-RNase, engendering a lower melting temperature of the R80S variants

Urea denaturation of NCD forms The conformational stability of the NCD forms against the denaturing action of urea in comparison with the corresponding monomers was investigated by means of steady-state fluorescence and CD measure-ments at pH 7.0

Monomeric proteins showed sigmoidal transition curves when the change in fluorescence intensity was

Table 1 Thermodynamic melting parameters of the unfolding process of monomers and NCDs of BS-RNase mutants T m , denaturation temperature; DH0(T m ), calorimetric enthalpy change; DH 0

v:H: , van’t Hoff enthalpy change; DC 0 ðT m Þ, excess heat capacity change; DS 0

(T m ), entropy change; DG 0

298 , denaturation Gibbs energy change at 298 K.

Tm(C)

DH 0 (Tm) (kJÆmol)1)

DH 0 v:H:

(kJÆmol)1)

DC 0 ðT m Þ (kJÆmol)1ÆK)1)

DS 0 (Tm) (kJÆmol)1ÆK)1)

DG 0 298 (kJÆmol)1)

408 ± 16 a

420 ± 17a

328 ± 13 a

316 ± 13 a

528 ± 21a

588 ± 17 a

553 ± 22 a

547 ± 21 a

a DH 0

v:H: from CD measurements.

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recorded at the wavelength maximum, Imax, as a

func-tion of urea concentrafunc-tion (insets in Fig 5), whereas

the curves of NCDs displayed two transitions (Fig 5)

The values of urea concentration at half-completion of

transition, C½, are shown in Table 2, which also

reports the values found by monitoring the molar

ellip-ticity at 222 nm with CD measurements; these reflect

conformational changes of the secondary structures for

monomers (insets in Fig 6) and NCDs (Fig 6) Also

in this case, two distinct C½ values, the first value in

the 2–3 m range and the second close to the C½ value

of the corresponding monomeric form (Table 2), were

observed for the dimers To investigate in more detail

the mechanism of urea denaturation, we followed this

process at different protein concentrations, but

focus-ing on the parent BS-RNase and on the sfocus-ingle-point

R80S variant The results are reported inFig 7, where

the folded fraction is reported as a function of the urea

concentration at four different protein concentrations,

in the range 0.1–25 lm According to Rumfeldt et al

[15], the variation in the curve shape from sigmoidal to

biphasic observed when the protein concentration

increases confirms the presence of a dimeric

intermedi-ate in the dissociation process of both NCD variants

The biphasic curves for the NCD forms of BS-RNase

and S80-BS-RNase at the highest concentration, where

the intermediate is present in significant amounts, were

analyzed according to the three-state equilibrium

model (N2MI2M2U) [16] The following values for the

Gibbs energy changes and m-values were obtained:

DG1= 14 kJÆmol)1, m1= 5 kJÆmol)1Æm)1, DG2 = 80

kJÆmol)1, m2= 19 kJÆmol)1Æm)1 for NCD BS-RNase;

and DG1= 12 kJÆmol)1, m1= 6 kJÆmol)1Æm)1, DG2=

43 kJÆmol)1, m2= 15 kJÆmol)1Æm)1 for S80-BS-RNase The Gibbs energy values indicate that the perturbative action of the urea is greater for the second transition,

I2M2U, than for the first transition, N2MI2, for both dimers The m-values also indicate that the surface area exposed to solvent in the first transition is smaller than that in the second transition A comparison between the two NCD forms shows that the R80S mutation decreases the stability mainly in the step

I2M2U, and, if we assume that the final state is the same for both NCD forms, this suggests that the R80S mutation decreases the stability of the intermediate

Structural models of the NCD forms

In the search for the possible origin of the reduced activity of S80 variants in both aggregation states, i.e

in the monomeric and noncovalent swapped dimeric forms, we examined the corresponding 3D structures All attempts to obtain crystals suitable for X-ray anal-ysis of any form of the two S80-BS-RNase variants had hitherto been unsuccessful Supported by the similarity of NMR spectra (Figs 2 and S1) of the monomers, suggesting that the global architecture of all the variant proteins is very similar to that of the parent BS-RNase, and by the close similarity among the X-ray structures of swapped isoforms of hA-BS-RNase and BS-RNase [11], models of the 3D structures of all proteins were obtained starting from the X-ray structure of the corresponding form of the parent BS-RNase, i.e the monomeric derivative (PDB

0.0 0.2 0.4 0.6 0.8 1.0

[Urea] M

0.0

0.2

0.4

0.6

0.8

1.0

[Urea] M

0.0

0.2

0.4

0.6

0.8

1.0

[Urea] M

0.0 0.2 0.4 0.6 0.8 1.0

[Urea] M

Fig 5 Urea-induced transition curves for NCDs of BS-RNase variants and for the corresponding monomers (inset), followed

by fluorescence spectroscopy.

(A) BS-RNase (B) hA-BS-RNase.

(C) S80-BS-RNase (D) S80-hA-BS-RNase The unfolded fraction represents the fraction

of denaturated protein, calculated as (I ) I min ) ⁄ (I max ) I min ); I is the fluorescence intensity at a given temperature, and I max and Iminare the maximum and minimum values of fluorescence intensity corresponding to the denaturated state and native state of proteins, respectively.

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code 1N1X) and the noncovalent swapped dimer (PDB

code 1TQ9) A representation of the structural models

built for S80-hA-BS-RNase, which, among the variants

examined in this study, is the one hosting the highest

number of substitutions, is reported inFig 8

A careful inspection of these structures reveals that

all of the variant proteins examined, and notably both

of the S80 variants, are characterized by the presence

of a decreased number of hydrogen bonds with respect

to the parent protein This trend is even more evident

in the NCD derivatives: in these forms, all of the

mutants have fewer intersubunit hydrogen bonds

than the native protein In particular, focusing on

residue 80, a contact between the side chains of Arg80 and Ser18 is detectable only in parent BS-RNase Fur-thermore, the same protein and hA-BS-RNase are sta-bilized by a contact between a core residue of one subunit (Gln101) and the hinge residues of the other subunit (Ser20 in the case of BS-RNase and Ser18 in the case of hA-BS-RNase)

Discussion

The antitumor activity of dimeric ribonucleases relies

on their quaternary structure, which enables the pro-teins to avoid inhibition by RI and provides good sta-bility in solution We have already shown that Pro19, Leu28 and, possibly, Gly16 play a relevant role in the cytotoxicity, because they ensure the correct quater-nary assembly of the NCD derivative of BS-RNase [17] However, the hinge residues and Leu28 have a synergistic effect, because to observe a drastic reduc-tion of the cytotoxic activity they have to be replaced simultaneously [6,18]

In contrast, the data reported in this article show that the substitution of the whole hinge region induces only a small reduction in the basal cytotoxic activity

of BS-RNase, as in the case of the single mutants P19A and L28Q [6] Surprisingly, the replacement of Arg80 by Ser significantly reduces the cytotoxic activ-ity, as both S80variants are less active than the parent BS-RNase On the one hand, this shows the impor-tance of Arg80, although it is irrelevant for the cata-lytic activity or the swapping extent of BS-RNase [12];

on the other hand, it indirectly confirms that the

Table 2 Urea-induced denaturation parameters of monomers and

dimers of BS-RNase mutants, monitored by fluorescence and CD

spectroscopy [Urea]1⁄ 2values were the denaturant concentrations

at half-completion of the transition.

[Urea]1⁄ 2 (M) CD

[Urea]1⁄ 2 (M) Fluorescence

5.60 ± 0.04

3.00 ± 0.06 5.70 ± 0.03

NCD S 80 -hA-BS-RNase 1.60 ± 0.05

4.50 ± 0.15

1.60 ± 0.20 4.60 ± 0.20

5.60 ± 0.01

2.62 ± 0.03 5.64 ± 0.01

NCD S80-BS-RNase 2.07 ± 0.02

5.12 ± 0.02

2.17 ± 0.03 5.09 ± 0.02

0.0 0.2 0.4 0.6 0.8 1.0

[Urea] M

0.0 0.2 0.4 0.6 0.8 1.0

[Urea] M

0.0 0.2 0.4 0.6 0.8 1.0

[Urea] M

0.0 0.2 0.4 0.6 0.8 1.0

[Urea] M

Fig 6 Urea-induced transition curves for

NCDs of BS-RNase variants and for the

corresponding monomers (inset), followed

by CD spectroscopy (A) BS-RNase (B)

hA-BS-RNase (C) S80-BS-RNase (D)

S 80 -hA-BS-RNase The unfolded fraction of

protein was calculated as (Q ) Q min ) ⁄

(Q max ) Q min ); Q is the ellipticity at 222 nm

at a given temperature, and Q max and Q min

are the maximum and minimum values of

ellipticity corresponding to the denaturated

state and native state of proteins,

respectively.

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exchange of the N-terminal arms in BS-RNase-like

proteins is not sufficient to elicit the antitumor activity

[10] We are aware that, in principle, the substitution

of a basic residue on the protein surface might reduce

the cytotoxic activity, by affecting the electrostatic

interaction with the cell membrane and perhaps the

internalization process [19], but, as shown by

Notomi-sta [20], the side of BS-RNase with the strongest

posi-tive potential is the one hosting the N-termini, which

is located opposite to Arg80 (Fig 8)

The observation of models built with the X-ray

structure of the reduced dimer of BS-RNase (PDB

code 1TQ9) as template indicate that all of the variants

maintain a quaternary structure very close to that of

the parent protein, but are characterized by weaker

interactions between subunits This result is also in

agreement with thermodynamic data and thermal and

chemical dissociation, which indicate a lower stability

of the variant proteins with respect to BS-RNase,

prin-cipally for both Ser80 variants

We also investigated the dissociation mechanism, and found that it is not affected by the mutations investigated here The experimental data related to thermal denaturation processes of both Ser80 variant proteins can be interpreted by surmising a simple two-step process, from native dimer to denatured monomers, whereas the presence of biphasic chemical denaturation profiles, exhibited by both fluorescence and CD curves, suggests the existence of a thermody-namically stable intermediate induced by urea This discrepancy is not unexpected, because the two dena-turation processes are induced by different perturbing agents, proceed through different mechanisms (in the case of the thermal denaturation process, the interme-diate state is not present in significant amounts) and end with completely distinct denatured states [21] It is possible that the chaotropic effect of urea initially causes a small perturbation of the secondary and ter-tiary structures of the proteins (DG1 for the N2MI2 transition is smaller than DG2 of the I2M2U transi-tion), and urea then stabilizes the intermediate through its hydrogen-bonding ability Furthermore, the com-parison of the values obtained for the first step indi-cates that, in all variants examined, the C½ value of the first step of the denaturation process is decreased with respect to that of the parent protein, indicating

0.0

0.2

0.4

0.6

0.8

1.0

[Urea] M A

0.0

0.2

0.4

0.6

0.8

1.0

[Urea] M B

Fig 7 Urea-induced transition curves of NCDs of BS-RNase (A)

and S80-BS-RNase (B), followed by fluorescence spectroscopy at

different protein concentrations: (•), 0.1 lM; ( ), 1 lM; (h), 7 lM;

( ), 25 lM.

A

B

Fig 8 (A) Crystal structure of the NCD form of BS-RNase (PDB code 1TQ9) (B) Homology model of S 80 -hA-BS-RNase The Arg80– Ser18 and Ser20–Gln101 hydrogen bonds, which are detectable only in BS-RNase, are indicated.

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that the interactions between the hinge region and

resi-due 80 are involved in the early stages of the chemical

denaturation process As a consequence, S80

-hA-BS-RNase is the most prone to unfolding

Our data suggest a correlation between the cytotoxic

activity of BS-RNase and its derivatives and the

stability of the corresponding reduced swapped forms

This is also in agreement with the finding that

cyto-toxic RNases are, in general, very stable enzymes, and

with the relationship between resistance to unfolding

and cytotoxic activity observed for different variants

of RNase A [22,23] In conclusion, the enhancement of

the conformational stability of the NCD derivative

represents a good approach to increase the toxicity of

BS-RNase towards cancer cells In addition, the

find-ing that the structure and stability of the dimeric

inter-mediate, shown by urea denaturation studies, play a

key role in the dissociation process suggests further

investigations of the dissociation mechanism that may

help in the design of new cancer chemotherapeutic

agents based on BS-RNase

Experimental procedures

Protein samples

All of the experimental procedures for obtaining significant

amounts of BS-RNase and its variants starting from the

cor-responding pET-22b(+) plasmid cDNA have already been

described in detail elsewhere [12,13] As in the previous

stud-ies, all of the constructs were coding for an Asp residue at

position 67, instead of an Asn as in the wild-type protein, to

avoid side effects caused by the spontaneous deamidation of

Asn67 [24,25] All of the proteins were expressed in

Escheri-chia colicells and purified in monomeric form, with Cys31

and Cys32 linked to two GSH molecules Monomers with

Cys31 and Cys32 in the reduced form, prepared as described

previously [12], were either carboxyamidomethylated with

iodoacetamide [26], to obtain the monomeric proteins used

directly for analysis, or dialyzed against 0.1 m Tris⁄ acetate

(pH 8.4) for 20 h at 4C, to obtain dimers In both cases,

the last step of the purification procedure was gel filtration

chromatography on a G-75 column (75· 3 cm)

Freshly prepared dimeric proteins were essentially made

least 72 h, to allow the mixture to reach equilibrium

The protein concentration was measured by UV

spectro-photometry, assuming e = 0.5 (0.1%, 278 nm, 1 cm) for

monomers and e = 0.465 for dimers

NCDs

The NCDs were prepared according to the protocol

previ-ously described by Piccoli et al [1], from the exchanged

form of the corresponding dimer The reduction of the

under nonreducing conditions The NCD forms were kept

at 4C until used for kinetic or thermodynamic analyses

Cytotoxicity studies

Cytotoxicity was evaluated by performing the 3-(4,5-dim-ethylthiazol-2-yl)-2,5-diphenyltetrazolium bromide reduc-tion inhibireduc-tion assay as described previously [27] Simian virus-40-transformed mouse fibroblasts (SVT2 cells) and the parental nontransformed Balb⁄ C 3T3 line (3T3 cells) were obtained from the ATCC (Manassas, VA, USA) The cells were plated on 96-well plates at a density of 2.5· 103

cells per well in 100 lL of medium containing BS-RNase or one

of its variants (12.5, 25, 50 or 100 lgÆmL)1), and incubated for 24 and 48 h at 37C Cell survival is expressed as the absorbance of blue formazan measured at 570 nm [27] with

an automatic plate reader (Victor3 Multilabel Counter; Per-kin Elmer, Shelton, CT, USA) Each curve reports the aver-age of three independent assays Standard deviations in all assays were in the range of 5–10%

DSC

DSC measurements were performed on a third-generation

were analyzed with a previously described program [28] The excess heat capacity of the protein in solution in the sample cell was measured against a reference cell filled with the buf-fer solution in the temperature range of 4–80C The excess heat functionhDC0

Pi was obtained after baseline subtraction, assuming that the baseline is given by the linear temperature dependence of the native state heat capacity The denatur-ation enthalpies, DH0(Tm), were obtained by integrating the area under the heat capacity versus temperature curves Tm

is the melting temperature, and corresponds to the maximum

of each DSC peak The entropy changes corresponding to the thermal denaturation of the monomers, DS0(Tm), were determined by integrating the curve obtained by dividing the heat capacity curve by the absolute temperature

Thermodynamic analysis

The van’t Hoff enthalpies were obtained by DSC profiles, utilizing the equation [29]:

DH0 v:H:¼ aRT2

mDC0

PðTmÞ=DH0ðTmÞ ð1Þ where a originates from the stoichiometry of the reaction (a = 4 or a = 6 for monomer or dimer denaturation,

DC0

PðTmÞ is the value of the excess heat capacity function at

Tm, and DH0(Tm) is the calorimetric enthalpy calculated by direct integration of the area under the DSC peak For the

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monomers, the entropy changes, DS0(Tm), were determined

by integrating the curve obtained by dividing the heat

capacity curve by the absolute temperature, and the

dena-turation Gibbs energies at 298 K were calculated by

com-bining the classical Kirchoff equations:

DG0ð298Þ ¼ DH0ðTmÞ Tm 298

Tm

 DC0

pðTmÞ

ðTm 298Þ þ 298 DCp0ðTmÞ lnTm

298

ð2Þ

NMR

Two-dimensional NMR spectra were acquired at 300 K on

on Bruker DRX600 spectrometer by using standard pulse

sequence libraries For the natural abundance 1H–15

N-HSQC spectra, the protein concentration was 2.5 mm in

95% H2O⁄ 5% D2O (pH 5.65) 1H chemical shifts are

rela-tive to the water signal at 4.70 p.p.m at 300 K, and 15N

chemical shifts were indirectly referenced to the 1H

chemi-cal shifts according to gyromagnetic ratios [30]

CD spectroscopy

CD measurements were performed on a JASCO 715 CD

spectrophotometer equipped with a thermoelectrically

con-trolled cell holder (JASCO PTC-348) that allows

measure-ments at a controlled temperature Quartz cuvettes with

0.1 cm optical pathlength were used Unless otherwise

reported, the protein concentration was 0.2 mgÆmL)1 The

CD spectra were recorded from 250 to 200 nm at 4C, and

normalized by subtraction of the buffer spectrum Molar

ellipticity per mean residue [h] in deg cm2Ædmol)1was

calcu-lated from the equation [h] = 100[h]obs⁄ lC, where [h]obs is

the ellipticity measured in degrees, l is the pathlength of the

cell (cm) and C is the protein molar concentration CD

spectra were recorded with a response of 4 s, a 1.0 nm

denatur-ation curves were recorded in temperature mode at 222 nm,

and a scan rate of 1.0CÆmin)1 The enthalpy changes were

calculated using origin 7.5 to fit CD melting curves by the

van’t Hoff equation:

@ln K

@ð1=TÞ

P

0

where K is the equilibrium constant and R is the gas

con-stant

The urea-induced transition curves at 4C were obtained

by recording the CD signal at 222 nm for each independent

sample All of the measurements were performed after

over-night incubation of the samples at 4C The values of urea

concentration at half-completion of transition, [urea]1 ⁄ 2,

were calculated with the Boltzmann equation of origin 7.0

Fluorescence analyses

Intrinsic protein fluorescence was recorded with a Perkin Elmer LS50B spectrofluorimeter equipped with a circulating water bath Unless otherwise stated, the protein

monomers and 7 lm for the dimers The excitation wave-length was set at 274 nm, and the emission was measured between 250 and 400 nm The spectra were recorded at 4C with a 1 cm cell and a 10 nm emission slit width, and cor-rected for background signal The urea-induced transition curves were obtained by recording the change in fluorescence intensity at maximum wavelength as a function of denatur-ant concentration The maximum emission wavelength of proteins was recorded at 303 nm Measurements were per-formed after overnight incubation of samples at 4C The values of urea concentration at half-completion of transi-tion, [urea]1 ⁄ 2, were calculated as for the CD measurements The biphasic curves at 25 lm were analyzed according to the three-state equilibrium model with a dimeric intermedi-ate, N2MI2M2U, where N2 is the native dimeric stintermedi-ate, I2 the dimeric intermediate and U the unfolded monomer, respectively [16] Fitting of the data was performed with the

changes, DG1and DG2, and the values of m1 and m2 for the N2MI2 and I2M2U transitions, respectively The m-value is a measure of the dependence of DG on denatur-ant concentration [31], and is proportional to the amount

of protein surface area exposed upon unfolding [32]

Molecular modeling

The structures of monomeric and dimeric variants of BS-RNase were calculated from the NMR and X-ray tures deposited at the PDB In particular, the NMR

whereas for the dimeric NCD form three crystallographic structures were used, corresponding respectively to the NCD BS-RNase derivative (PDB code 1TQ9), to the N-dimer of RNase A (PDB code 1A2W) and to the PM8 human pancreatic RNase variant (PDB code 1H8X) The atomic coordinates of the above-mentioned protein struc-tures were used as a template to predict the 3D structure of the variants, using modeller 8v5 [33] The quality of the structural models was evaluated with modeller, using the score of variable target function method [34] Model analy-ses were performed with molmol [35] and pymol [36]

Acknowledgements

C Ercole is a fellow of the Department of Chemistry

‘Paolo Corradini’ of University of Naples ‘Federico II’, supported by a training grant from the ‘Compag-nia di San Paolo di Torino’ We thank T Tancredi for help with NMR spectra acquisition, L Petraccone for

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