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Tiêu đề The effect of mutations surrounding and within the active site on the catalytic activity of ricin A chain
Tác giả Catherine J. Marsden, Vilmos Fulop, Philip J. Day, J. Michael Lord
Trường học University of Warwick
Chuyên ngành Biochemistry
Thể loại Journal article
Năm xuất bản 2004
Thành phố Coventry
Định dạng
Số trang 10
Dung lượng 707,54 KB

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Michael Lord Department of Biological Sciences, University of Warwick, Coventry, UK Models for the binding of the sarcin–ricin loop SRL of 28S ribosomal RNA to ricin A chain RTA suggest

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The effect of mutations surrounding and within the active site

on the catalytic activity of ricin A chain

Catherine J Marsden, Vilmos Fu¨lo¨p, Philip J Day* and J Michael Lord

Department of Biological Sciences, University of Warwick, Coventry, UK

Models for the binding of the sarcin–ricin loop (SRL) of 28S

ribosomal RNA to ricin A chain (RTA) suggest that several

surface exposed arginine residues surrounding the active site

cleft make important interactions with the RNA substrate

The data presented in this study suggest differing roles for

these arginyl residues Substitution of Arg48 or Arg213 with

Ala lowered the activity of RTA 10-fold Furthermore,

substitution of Arg213 with Asp lowered the activity of RTA

100-fold The crystal structure of this RTA variant showed it

to have an unaltered tertiary structure, suggesting that the

positively charged state of Arg213 is crucial for activity

Substitution of Arg258 with Ala had no effect on activity,

although substitution with Asp lowered activity 10-fold

Substitution of Arg134 prevented expression of folded

pro-tein, suggesting a structural role for this residue Several

models have been proposed for the binding of the SRL to the

active site of RTA in which the principal difference lies in the conformation of the second G in the target GAGA motif

in the 28S rRNA substrate In one model, the sidechain

of Asn122 is proposed to make interactions with this G, whereas another model proposes interactions with Asp75 and Asn78 Site-directed mutagenesis of these residues of RTA favours the first of these models, as substitution of Asn78 with Ser yielded an RTA variant whose activity was essentially wild-type, whereas substitution of Asn122 reduced activity 37.5-fold Substitution of Asp75 failed to yield significant folded protein, suggesting a structural role for this residue

Keywords: ricin; ribosome; site-directed mutagenesis; heterologous expression

Ricin is a potent toxin found in the seeds of the Ricinus

communis plant It is a member of a large family of

ribosome-inactivating proteins (RIPs) that exist in various

tissues of many plants, fungi and bacteria [1] Ricin

consists of a catalytic A chain (RTA) joined by a single

disulphide bond to a lectin B chain (RTB) that facilitates

both cell surface binding and entry of the catalytically

active A chain into the cytoplasm [2] RTA inactivates

eukaryotic ribosomes by catalysing the depurination of a

specific adenine residue of 28S rRNA, rendering the

ribosome unable to bind elongation factors and hence

abolishing protein synthesis [3] The site of depurination

by RTA lies within a highly conserved purine-rich region

of 28S rRNA termed the sarcin–ricin loop Both the

NMR structure [4,5] and X-ray structure [6] of an

oligoribonucleotide that mimics this region show it to be

a compact structure with a stem and four base loop, GAGA, at its centre Each of the four nucleotides in this GAGA tetraloop are necessary for the action of RTA, thus, implying that the N-glycosidase activity of RTA not only requires specific interactions with the target adenine but also direct recognition of other bases in the tetra-nucleotide [7] Unlike this tetratetra-nucleotide, the sequence of the stem does not appear to be important as long as it is

at least three base-pairs in length [8] Interactions between RTA and the rRNA backbone in this area might be required to maintain the tetraloop in the optimum conformation for catalysis Both the catalytic role of RTA and its recognition and binding of the substrate RNA have been investigated by chemical modification [9], X-ray crystallography [10–12] and site-directed mutagen-esis [13–17] Chemical modification showed RTA to be inactivated by treatment with the arginyl-specific reagent, phenylglyoxal [9], although it is likely that the inactivation observed in this study was primarily due to modification

of the crucial catalytic residue, Arg180 [18] Crystal structures of small molecules bound in the active site of RTA have been solved [10,12,19], but to date there are no structures of complexes of RTA with larger substrate analogues

The structure of the ribosome and of small RNA oligonucleotides show that the target adenine is not in a conformation that is compatible with binding to RTA [6] Monzingo and Robertus [10] generated three models of complexes of a hexanucleotide (C1G2A3G4A5G6, where A3

is the target for depurination by RTA) and RTA The

Correspondence to J M Lord, Department of Biological Sciences,

University of Warwick, Coventry CV4 7AL, UK.

Fax: + 44 2476523701, Tel.: + 442476523598,

E-mail: mlord@bio.warwick.ac.uk

Abbreviations: ApG, adenyl (3¢ fi 5¢) guanosine; DMEM, Dulbecco’s

modified Eagles medium; FMP, formycin monophosphate; RIP,

ribosome-inactivating protein; RTA, ricin A chain; RTB,

ricin B chain; SRL, sarcin-ricin loop.

*Present address: AstexTechnology, 436 Cambridge Science Park,

Milton Road, Cambridge, CB4 0QA, UK.

(Received 25 September 2003, revised 28 October 2003,

accepted 10 November 2003)

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models were based on the tetraloop structure of such a

hexanucleotide, where the conformation of the target

adenine had been altered, using the known structures of

complexes of RTA with FMP (formycin monophosphate)

and ApG (adenyl 3¢ fi 5¢-guanosine), to facilitate docking

into the active site of RTA

Here, two of the models proposed [10] have been

examined (Fig 1) The third model in which the

hexanucle-otide is bound to the active site of RTA in an open

conformation is not included in this study as, although

several favourable interactions between the hexanucelotide

and RTA are maintained, many additional interactions are

proposed that involve poorly conserved residues Each of

the two models tested proposed that several arginyl residues

were involved in electrostatic interactions with the

phos-phdiester backbone of the hexanucleotide The role of four

such arginyl residues (Arg48, Arg134, Arg213 and Arg258) has been examined here The most significant difference between the two models is the identity of the residues involved in interactions made between RTA and base G4of the hexanucleotide (Fig 1) In this study those amino acid residues (Asp75, Asn78 and Asn122) around the active site cleft of RTA that have a putative role in the binding of G4in each of the two models have been examined

Experimental procedures

Materials (35S)Methionine was from Amersham, RTB was from Vector Laboratories (Peterborough, UK) and microbridges were from Crystal Microsystems (Oxford, UK)

R258

R213

R180

N78 D75

E177

R134

R258

R213

R180

N78 E177

D75

R134

R258

R213

D75

E177 Y123

Y80

N122 R134

R258

R213

E177

D75

Y123

Y80

R134

N122

A

B

Fig 1 Models of hexanucleotide binding in the active site of RTA (based upon [10]) The structures are shown as stereo images with the

C 1 G 2 A 3 G 4 A 5 G 6 (where A 3 is the target for depurination by RTA) in red C 1 of the hexanucleotide is at the bottom left and the target adenine

is at the top of each model The sidechains of RTA are shown in blue (A) Model 1 has the tetraloop bound in the active site of RTA with G 4 stacked upon the G 2 -A 5 pair and able to make interactions with Asn122 (B) Model 2 is a variation of model 1 with the tetraloop bound in a conformation where G 4 stacks with Tyr80 and makes interactions with Asp75 and Asn78 Drawn with MOLSCRIPT [34,35].

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Creation of ricin A chain variants

Ricin A chain variants containing single amino acid

sub-stitutions were generated by PCR mutagenesis and standard

recombinant DNA techniques Using the RTA expression

plasmid pUTA [14] as a PCR template, appropriate base

changes were introduced to encode for the single amino acid

substitutions R48A, R134A, R134Q, R213A, R213D,

R258A, R258D, N78S, D75A, D75S, D75N, N122A All

substitutions were confirmed by DNA sequencing

Expression and purification of ricin A chain variants

A single colony of Escherichia coli JM101 transformed with

the pUTA vector [14] containing the appropriate

RTA-variant sequence was used to inoculate 50 mL of 2YT and

grown overnight at 37C This starter culture was used to

inoculate 500 mL of 2YT, and the culture was grown for 2 h

at 30C Expression was induced by adding isopropyl

thio-b-D-galactoside to a final concentration of 0.1 mMfor 4 h at

30 C Cells were harvested by centrifugation at 2740 g,

resuspended in 15 mL of 5 mMsodium phosphate buffer

(pH 6.5), and lysed by sonication on ice Cell debris was

pelleted by centrifugation at 31 400 g at 4C for 30 min

and the supernatant loaded onto a 50 mL CM-Sepharose

CL-6B column (Amersham Biosciences) The column was

washed with 1 L of 5 mM sodium phosphate, pH 6.5

followed by 100 mL of 100 mM NaCl in 5 mM sodium

phosphate, pH 6.5 and RTA was eluted with a linear

gradient of 100–300 mMNaCl in the same buffer Fractions

containing RTA were pooled and stored at 4 C at a concentration of no more than 1 mgÆmL)1 Typical yields

of purified wild-type RTA and RTA variants were between

10 and 12 mgÆL)1unless otherwise stated in the text

Crystallization, X-ray data collection and refinement

of ricin A chain variants Crystals were grown in the tetragonal space group P41212

by the sitting-drop method using microbridges (Crystal Microsystems, UK) and the conditions described for wild-type RTA crystallization [11] Data were collected at 100 K and processed using the HKL suite of programs [20] Refinement of the structures was carried out by alternate cycles of REFMAC[21] and manual refitting using O [22], based on the 1.8 A˚ resolution model of wild-type RTA [11] (Protein Data Bank code 1ift) Water molecules were added

to the atomic model automatically using ARP [23] at the positions of large positive peaks in the difference electron density, only at places where the resulting water molecule fell into an appropriate hydrogen bonding environment Restrained isotropic temperature factor refinements were carried out for each individual atom Data collection and refinement statistics are given in Table 1

Assay of theN-glycosidase activity of ricin A chain variants

The activity of each of the RTA variants was determined

by assessing their ability to depurinate 26S rRNA of

Table 1 Data collection and refinement statistics Numbers in parentheses refer to values in the highest resolution shell.

Data collection

Unit cell dimensions (A˚) a ¼ b ¼ 67.3, c ¼ 140.7 a ¼ b ¼ 67.5, c ¼ 140.6

R sym

a

Refinement

Non-hydrogen atoms 2492 (including 2 sulphate

406 water molecules)

2596 (including 2 sulphate, 1 acetate and 510 water molecules)

R cryst

b

Rmsds from ideal values

a R sym ¼ S j S h |I h,j – <I h >|/S j S h <I h > where I h,j is the jth observation of reflection h, and <I h > is the mean intensity of that reflection b

R cryst ¼ S||F obs | – |F calc ||/S|F obs | where F obs and F calc are the observed and calculated structure factor amplitudes, respectively.cR free is equivalent to R for a 4 % subset of reflections not used in the refinement [24].

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purified yeast (Saccharomyces cerevisiae) ribosomes For

each reaction, 20 lg of yeast ribosomes were incubated at

30 C for 1 h with the relevant RTA variant in 25 mM

Tris/HCl (pH 7.6), 25 mM KCl, 5 mM MgCl2, in a total

volume of 20 lL Reactions were stopped by the addition

of 100 lL of 2· Kirby buffer [25] and 80 lL of H2O, and

rRNA was obtained by precipitation after two phenol–

chloroform extractions Four micrograms of rRNA were

treated with 20 lL of acetic-aniline for 2 min at 60 C,

and rRNA was precipitated and resuspending in 15 lL of

60% de-ionized formamide/0.1· TPE (3.6 mMTris, 3 mM

NaH2PO4, 0.2 mM EDTA) and heated at 65 C for

5 min Ribosomal RNA fragments were separated on a

1.2% agarose, 0.1· TPE, 50% (v/v) formamide gel

rRNAs were quantified from digital images of ethidium

bromide-stained gels, using IMAGEQUANT software, and

depurination was calculated by relating the amounts of

the small aniline-fragment and 5.8S rRNA and expressing

values as a percentage

Reassociation and quantification of ricin A-chain variants

Purified RTA (100 lg) was mixed with 100 lg of RTB

(Vector Laboratories) and made up to a final volume of

2 mL with NaCl/Picontaining 0.1Mlactose and 2% (v/v)

2-mercaptoethanol This was dialysed for 24 h against 1 L

of NaCl/Picontaining 0.1Mlactose, followed by a further

36 h against 5 L of NaCl/Pi Reassociated holotoxin was

separated from free RTA on a 0.5 mL immobilized

a-lactose column The dialysate was loaded onto the

column three times and the column was then washed with

10 mL of NaCl/Pi before eluting bound holotoxin (and

free RTB) in 5 mL of NaCl/Pi containing 75 mM

galactose Eluted protein was dialysed for 16 h against

1 L of NaCl/Pi to remove the galactose, before

quanti-fying against know quantities of RTA on a silver-stained

SDS/polyacrylamide gel using Molecular Dynamics

IMAGEQUANTversion 3.3

Cytotoxicity assay

Cells were plated out in a volume of 100 lL in 96-well plates

at a density of 1.5· 105cellsÆmL)1and incubated for 18 h at

37C Toxin dilutions (100 lL) in DMEM were added in

quadruplicate and the plates were incubated for 4 h at 37C

Protein synthesis was measured by incubating the plates for

90 min at 37C in the presence of 1 lCi of (35S)methionine

in 50 lL of NaCl/Pi per well Proteins were precipitated

by washing three times with ice-cold 5% (v/v)

trichloroace-tic acid and, after the addition of 200 lL of scintillant

(OptiPhase Supermix) to each well, plates were counted

in a Wallac 1450 MicroBeta Triluxliquid scintillation

counter

Results

N-glycosidase activity of RTA variants containing

arginine substitutions

RTA variants in which arginyl residues 213 or 258 were

substituted with Ala, or Asp or in which Arg48 had been

substituted with Ala, expressed to levels equivalent to

wild-type RTA and were readily purified to homogeneity Each

of these RTA variants had the same stability to digestion

by trypsin as wild-type RTA (data not shown) Substitu-tion of Arg134 with either Ala or Gln resulted in barely detectable expression levels and, as such, these mutants could not be purified To assess the effect of substitutions made at each of the arginyl residues on catalytic activity of RTA, the ability of each of the purified RTA variants to depurinate yeast ribosomes was compared to that of wild-type RTA Conversion of Arg213 to either alanine or aspartate (R213A and R213D, respectively) reduced the

D50 (concentration giving half maximal depurination) by 10-fold and over 100-fold, respectively (Fig 2A) Substitu-tion of Arg258 with Ala (R258A) had no effect on activity whereas substitution of Arg258 with Asp (R258D) lowered the activity niinefold (Fig 2B) Finally, substitution of Arg48 with Ala (R48A) lowered the activity by 10-fold (Fig 2C)

Cytotoxicity of RTA variants containing arginine substitutions

Each of the RTA variants described above were reassoci-ated with RTB and the cytotoxicity of each of the resultant ricin variants was compared to wild-type ricin Ricin R213A and ricin R213D were 12-fold and more than 2000-fold less cytotoxic than ricin, respectively (Fig 3A) Ricin R258A was equally as cytotoxic as ricin (Fig 3B) whereas ricin R258D (Fig 3B) and ricin R48A (Fig 3C) were 10-fold less cytotoxic The reductions in cytotoxcity compared to native ricin were comparable to the decrease

in catalytic activity against ribosomes in vitro for each of the substitutions except for ricin R213D, whose cytotoxi-city was reduced by 20-fold more than the catalytic activity

of RTA R213D

The X-ray structure of RTA R213D

In the absence of structural data for each of the RTA variants discussed, it is possible that the reduction in activity might be attributed to structural changes of the enzyme The most substantial decrease in catalytic activity was seen when

a substitution was made at Arg213 (R213D) The crystal structure was solved to determine whether the reduction in activity of this RTA variant could be attributed solely to the change in charge and size of the sidechain of this single residue The structure of RTA R213D is essentially identical

to that of recombinant wild-type RTA with an root mean square deviation (RMSD) from the Ca atoms of the wild-type crystal structure [11] of 0.33 A˚ The electron density in the area local to the substitution is shown in Fig 4 The positions of catalytic residues and all other residues local to the substitution site do not differ significantly from the wild-type crystal structure

Binding of the GAGA tetraloop to RTA

In order to better understand the specific interactions that RTA makes with the GAGA tetraloop, the models of RTA–substrate interactions proposed by Monzingo and Robertus [10] have been examined The first model examined here has the sequence CGAGAG (where A

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is the target for depurination by RTA) modelled into the

active site with the first base C1 of the hexanucleotide

forming a Watson–Crick pair with the last base G6 This

both closes the tetraloop and allows G2 and A5 to be

stacked upon this base pair forming a non-Watson–Crick

pairing (Fig 1A) The target adenine residue A3 projects

out of the tetraloop and is positioned in the same location as

indicated by the crystal structures of both the FMP and ApG complexes [10], forming stacking interactions with the aromatic groups of Tyr80 and Tyr123 The first model has

G4 stacked on the G2-A5 pair of the hexanucelotide allowing two hydrogen bonds to be formed between the base G4and the sidechain of Asn122 In the second model (Fig 1B) the structure of the hexanucelotide is, on the whole, unchanged and the majority of the interactions that were seen in the first model are maintained However, the interaction between G4and Asn122 can no longer be made

as G4is in an altered position making a continuous curved stack of aromatic groups, Tyr123, A, Tyr80, G In this

Fig 3 Dose dependent cytotoxicity assay of ricin variants, ricin R213A and ricin R213D towards Vero cells Vero cells were challenged with increasing concentrations of toxin at 37 C for 4 h Remaining protein synthesis after this time was measured by the incorporation of ( 35 S)methionine Symbols indicate the mean of four replicate samples, error bars represent the SD (A) ricin, d, ricin R213A, j; and ricin R213D, m (B) ricin, d; ricin R258A, j, and ricin R258D, m; (C) ricin, d; ricin R48A, j.

Fig 2 Assessment of the N-glycosidase activity of RTA variants

con-taining arginine substitutions Isolated yeast ribosomes (20 lg) were

incubated with RTA or RTA variants for 60 min at 30 C rRNA was

isolated and 4 lg was aniline-treated and electrophoresed on an

ag-arose/formamide gel rRNAs were quantified from digital images using

IMAGEQUANT software The depurination was calculated by relating

the amounts of small diagnostic aniline-fragment [3] and 5.8S rRNA

and expressing values as a percentage Symbols indicate the

experi-mental data, error bars represent the SD and solid lines represent

best-fitted curves (A) RTA, d; RTA R213A, j, and RTA R213D, m (B)

RTA, d; RTA R258A j, and RTA R258D, m; (C) RTA, d; RTA

R48A, j.

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model alone G4is proposed to make two hydrogen bonds

with Asn78 and a further hydrogen bond with Asp75 In

order to test these models, substitutions were made at each

of the residues proposed to be involved – Asp75, Asn78 and

Asn122

N-glycosidase activity of RTA variants with substitutions

at Asp75 and Asn78

Substitution of either Asp75 or Asn78 with Ala resulted in

very low expression levels and purification of these variants

was not achieved However, although a very low expression

level was again seen on substitution of Asp75 to Ser,

substitution of Asn78 to Ser (RTA N78S) produced a protein

that expressed as well as wild-type RTA and had equal

stability to digestion by trypsin as wild-type RTA (data not

shown) Further substitutions were made at Asp75 to Asp,

Arg and Gln However, all were expressed at very low levels,

and purification to homogeneity, to allow quantitative

activity assays of these RTA variants, was not achieved To

assess whether substitution at Asn78 with Ser changed the

catalytic activity of RTA, the N-glycosidase activity of RTA

N78S against yeast ribosomes was determined and compared

to that of wild-type RTA (Fig 5A): there was less than a

twofold reduction in activity between them

Cytotoxicity of RTA N78S

RTA N78S was reassociated with RTB and its cytotoxicity

was compared to ricin (Fig 5B) Ricin containing RTA

N78S was approximately twofold less cytotoxic than

wild-type ricin This difference in cytotoxicity was comparable

to the small reduction in N-glycosidase activity seen for

RTA-N78

N-glycosidase activity of RTA variants with

substitutions at Asn122

Conversion of Asn122 to Ala (RTA N122A) produced an

RTA variant that expressed to high levels (equivalent to

wild-type RTA) RTA N122A had equal stability, based on

sensitivity to trypsin digestion, as the wild-type protein (data not shown) and was readily purified to homogeneity To assess whether the substitution at Asn122 for Ala had caused any change in the catalytic activity of RTA, the N-glycosidase activity of RTA N122A against yeast ribo-somes was determined and compared to that of wild-type RTA (Fig 6A) The reduction in activity of this RTA N122A was 37.5-fold

Cytotoxicity of RTA N122A RTA N122A was reassociated with RTB and its cytotox i-city was compared to ricin (Fig 6B) Substituting Ala for Asp at residue 122 in RTA reduced the cytotoxicity of RTA

by 30-fold (correlating well with the in vitro N-glycosidase activity)

The X-ray structure of RTA N122A

In order to confirm that the reduction in catalytic activity of RTA N122A could be attributed solely to the change in charge and size of the sidechain of this single residue, the crystal structure of RTA N122A was solved The RMSD from the Caatoms of the wild-type RTA crystal structure [11] was 0.33 A˚ indicating that the structure of RTA N122A

is essentially identical to recombinant wild-type RTA The electron density in the area of the substitution is shown in Fig 7 The positions of catalytic residues and all other residues local to the substitution site do not differ from the wild-type crystal structure

Discussion

When the structure of ricin A-chain was solved to 2.5 A˚ [26], it was suggested that a number of arginyl residues were likely to be responsible for the binding of the rRNA substrate Several of these arginyl residues have subse-quently been proposed to form an arginine-rich binding motif [27] Furthermore, when RTA is treated with the arginyl specific reagent, phenylglyoxal, it is readily inacti-vated leading to the proposal that this inactivation

Fig 4 Electron density of RTA R213D in the vicinity of residue 213 The backbone and sidechains of the R213D substitution are shown as stereo images in thick ball and stick and the position of the Arg213 side-chain of the wild-type enzyme is overlayed and shown in thin ball and stick The SIGMAA [33] weighted 2mF o -DF c electron density using phases from the final model is contoured at 1 r level, where r represents the rms electron density for the unit cell Contours more than 1.4 A˚ from any of the displayed atoms have been removed for clarity Drawn with MOLSCRIPT [34,35].

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involved the modification of arginyl residues 196, 213, 234

and 235 [9] Of theses residues, only Arg213 is in the

vicinity of the active site of RTA In order to establish the

role of arginyl residues around the active site of RTA in

the binding of rRNA, a number of RTA variants have

been constructed Four such arginyl residues were selected

as targets for site-directed mutagenesis We appreciate that

the mutagenic approach we have taken does not

distin-guish between a role for particular residues in substrate

binding or the catalytic reaction itself Both roles are

required for RTA catalysis and hence for the cytoxicity of ricin However, it does not seem unreasonable to assume,

at least as a broad generalization, that residues lying within the active site cleft are involved in the reaction catalysed, while those positively charged residues surrounding but outside the active site are probably involved in binding the negatively charged RNA substrate

Arg48 is a variable residue that lies in a loop on the edge

of the active site cleft of RTA that has been implicated in substrate binding by modelling studies [28] Substitution of this RTA residue with alanine reduced catalytic activity

Fig 5 Assessment of the N-glycosidase activity and cytotoxicity of

RTA N78S and ricin N78S (A) N-glycosidase activity of RTA N78S.

Isolated yeast ribosomes (20 lg) were incubated with RTA (d) or

RTA N78S (j) for 60 min at 30 C rRNA was isolated and 4 lg was

aniline-treated and electrophoresed on an agarose/formamide gel.

rRNAs were quantified from digital images using IMAGEQUANT

soft-ware The depurination was calculated by relating the amounts of

small aniline-fragment and 5.8S rRNA and expressing values as a

percentage Symbols indicate the experimental data, error bars

repre-sent the SD and solid lines reprerepre-sent best-fitted curves (B) Dose

dependent cytotoxicity assay of ricin N78S Vero cells were challenged

with increasing concentrations of ricin (d), or ricin N78S (j) at 37 C

for 4 h Remaining protein synthesis after this time was measured by

the incorporation of (35S)methionine Symbols indicate the mean of

four replicate samples, error bars represent the SD.

Fig 6 Assessment of the N-glycosidase activity and cytotoxicity of RTA N122A and ricin N122A (A) N-glycosidase activity of RTA N122A Isolated yeast ribosomes (20 lg) were incubated with RTA (d) or RTA N122A (j) for 60 min at 30 C rRNA was isolated and

4 lg was aniline-treated and electrophoresed on an agarose/forma-mide gel rRNAs were quantified from digital images using IMAGE-QUANT software The depurination was calculated by relating the amounts of small aniline-fragment and 5.8S rRNA and expressing values as a percentage Symbols indicate the experimental data, error bars represent the SD and solid lines represent best-fitted curves (B) Dose dependent cytotoxicity assay of ricin N122A Vero cells were challenged with increasing concentrations of ricin (d) or ricin N122A (j) at 37 C for 4 h Remaining protein synthesis after this time was measured by the incorporation of ( 35 S)methionine Symbols indicate the mean of four replicate samples, error bars represent the SD.

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10-fold, consistent with the role predicted for this residue by

modelling, but in contrast to an earlier study [29] The

apparent discrepancy with this earlier study is probably due

to the type of the assays used The earlier study [29] used

single point assays which may not have been sufficiently

sensitive to observe a 10-fold decrease in activity that is

readily observed in the dose–response assay used here

Arg258 is another variable residue that Monzingo and

Robertus [10] suggested might form an ion-pair with the

phosphodiester backbone of rRNA, specifically with that of

the second guanine in the GAGA motif Olson and Cuff [28]

also proposed that this residue was involved in substrate

binding, interacting with the loop-closing guanine base (the

first G after the GAGA) and, due to it’s variable nature,

could perhaps be responsible for the differing binding

affinities of RIPs for their substrates Substitution of Arg258

with Ala had no effect on activity, implying that this residue

is not necessary for the activity of RTA This is supported

by the earlier observation showing that deletion of residues

258–262 did not inactivate RTA [30] However, although

this residue is not essential for activity, reversing the charge

of this sidechain resulted in a 10-fold reduction in activity,

consistent with this residue being close to the

phosphodi-ester backbone of the substrate but not making an essential

interaction with it

Arg134 is highly conserved in RIPs, and it has been

proposed that it could make a forked interaction with both

of the phosphodiester backbones of the target adenine and

of the preceding guanine residue in the GAGA motif [10]

Substitution of Arg134 with Ala or Gln abolished

expres-sion of folded protein, and consequently it was concluded

that Arg134 played a critical structural role Although

Arg134 is predicted to be able form an ionic interaction

with the phosphodiester backbone of the substrate, the

structure of RTA also shows that this residue is at the

centre of a hydrogen bonding network, forming H-bonds

with Glu127, Asn209 and Glu208 Arg134 also forms a p-stacking interaction with Tyr123, a residue directly involved in substrate binding Substitution of residues Asn209, Glu208 and Tyr123 permitted RTA folding and reduced activity between threefold and 10-fold, suggesting that they form only modest interactions with the substrate [14,18] Thus, while it remains possible that Arg134 makes

a significant contribution to substrate binding, this can not

be readily tested by site-directed mutagenesis due to its structural role

Arg213 is a weakly conserved residue, the amino acyl residue at this position being positively charged in nearly 60% of ribosome-inactivating proteins [28] The Monzingo and Robertus models [10], and the 29mer oligonucleotide-binding model of Olson and Cuff [28], show this residue forming an ion-pair with the phosphodiester backbone of the first cytosine residue in the CGAGAG tetraloop motif Furthermore, an RTA mutant in which Arg213 had been deleted was found to be inactive [31] Substitution of Arg213 with Ala reduced activity 10-fold against both purified ribosomes and whole cells, while reversal of the charge at this site reduced activity a further 10-fold This additive effect of changing the charge of residue

213 strongly suggests that it is the charged nature of Arg213 that is responsible for effects on enzyme activity rather than any structural changes induce by the substitutions This is confirmed by the finding that the structure of RTA R213D is identical to that of the wild-type enzyme except for the sidechain of residue 213 Thus, Arg213 forms a significant electrostatic interaction with the substrate ribosome, prob-ably via interaction with the phosphodiester backbone Individual substitution of arginyl residues around the active site cleft of RTA resulted in different effects on the activity of RTA Whereas, one had no effect on the activity

of the enzyme (R258A), others reduced activity by one (R213A, R258D, R48A) or by two (R213D) orders of

N122A

Y80

N122A Y80

Fig 7 Electron density of RTA N122A in the vicinity of residue 122 The backbone and sidechains of the N122A substitution are shown as stereo images in thick ball and stick and the position of the Asn122 side-chain of the wild-type enzyme is overlayed and shown in thin ball and stick The SIGMAA [33] weighted 2mF o -DF c electron density using phases from the final model is contoured at 1 r level, where r represents the rms electron density for the unit cell Contours more than 1.4 A˚ from any of the displayed atoms have been removed for clarity Drawn with MOLSCRIPT [34,35].

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magnitude It then appears that, in addition to catalytic site

residues, a number of the Arg residues located around the

active site cleft play a significant role in the activity of RTA

As none of these arginyl residues have been implicated in

catalysis, the reduction in activity of each of these RTA

variants is probably due to a loss of electrostatic interactions

that are critical for the stable binding of the substrate RNA

molecule in the active site pocket Although the contribution

of each individual residue is relatively small, the cumulative

effect of these residues would make a substantial

contribu-tion to substrate binding This contribucontribu-tion is probably

relatively nonspecific in nature such that these residues

probably make a major contribution to binding, but do not

greatly contribute to specificity

Further interactions of the substrate RNA with RTA

were studied by modelling a hexanucleotide into the active

site [10] whose structure was based upon the crystal

structure of a GNRA loop which had been solved [19] A

series of RTA variants were made based on two of the

models proposed by Monzingo and Robertus [10]

Site-directed mutagenesis was used to make RTA variants with

substitutions at either Asp75 or Asn78, both of which are

highly conserved in the RIP family and were proposed to

make interactions with the second G4 in the GAGA

tetraloop in the second of the models (Fig 1B) Whereas

none of the substitutions to Ala, Ser, Asp, or Arg were

tolerated at Asp75 implying that this residue might play an

important structural role, RTA N78S had the same catalytic

activity as wild-type RTA suggesting that this residue may

not play a significant role in substrate binding, although it

remains possible that a substituted serine could still make

the hydrogen bond normally made by Asn78 In the first

model (Fig 1A) G4 is proposed to interact with Asn122

and, in agreement with this, substitution of Ala for Asp at

this site was shown to lower the N-glycosidase activity

37.5-fold, and cytotoxicity of the reassociated holotoxin by

30-fold, without affecting the overall structure or the

position of either active site residues or residues in the

vicinity of the substitution That the RTA N78S variant had

no effect on activity, but the RTA N122A variant reduced

catalytic activity over 30-fold compared to wild-type RTA,

is consistent with the first of the models proposed by

Monzingo and Robertus [10]

In order to understand more fully the precise role of

Asn122 in the catalytic activity of RTA, it would be of

value to solve the structure of RTA N122A in the

presence of small nucleotides such as ApG that can be

diffused into RTA crystals RTA cleaves a single

N-glycosidic bond from among over 4000 in 28S rRNA

[32] Ultimately, complete understanding of the specificity

of RTA for its ribosomal RNA substrate, and the role

that recognition and binding of the RNA stem and loop

might play in this, may only be fully achieved when

crystal structures of complexes of RTA with much larger

RNA fragments become available

Acknowledgements

This work was supported by the UK Biotechnology and Biological

Sciences Research Council grant 88/B16355 and Wellcome Trust

Programme Grant 063058/Z/00/Z VF is a Royal Society University

Fellow.

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