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coli cells, deletion of the C-terminal half of pyrrhocoricin signi-ficantly reduced the peptide’s ability to enter bacterial or mammalian cells.These findings highlighted pyrrhocoricin’s s

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Identification of crucial residues for the antibacterial activity

of the proline-rich peptide, pyrrhocoricin

Goran Kragol1, Ralf Hoffmann2, Michael A Chattergoon1, Sandor Lovas3, Mare Cudic1, Philippe Bulet4, Barry A Condie1, K Johan Rosengren5, Luis J Montaner1and Laszlo Otvos Jr1

1

The Wistar Institute, Philadelphia, PA, USA;2Biologisch-Medizinisches Forschungszentrum, Heinrich-Heine-Universita¨t,

Du¨sseldorf, Germany;3Department of Biomedical Sciences, School of Medicine, Creighton University, Omaha, NB, USA;

4

Institut de Biologie Moleculaire et Cellulaire, Strasbourg, France;5Institute for Molecular Bioscience, University of Queensland, Brisbane, Australia

Members of the proline-rich antibacterial peptide family,

pyrrhocoricin, apidaecin and drosocin appear to kill

responsive bacterial species by binding to the multihelical lid

region of the bacterial DnaK protein.Pyrrhocoricin, the

most potent among these peptides, is nontoxic to healthy

mice, and can protect these animals from bacterial challenge

A structure–antibacterial activity study of pyrrhocoricin

against Escherichia coli and Agrobacterium tumefaciens

identified the N-terminal half, residues 2–10, the region

responsible for inhibition of the ATPase activity, as the

fragment that contains the active segment.While

fluo-rescein-labeled versions of the native peptides entered E coli

cells, deletion of the C-terminal half of pyrrhocoricin

signi-ficantly reduced the peptide’s ability to enter bacterial or

mammalian cells.These findings highlighted pyrrhocoricin’s

suitability for combating intracellular pathogens and raised

the possibility that the proline-rich antibacterial peptides can deliver drug leads into mammalian cells.By observing strong relationships between the binding to a synthetic fragment of the target protein and antibacterial activities of pyrrhocori-cin analogs modified at strategic positions, we further veri-fied that DnaK was the bacterial target macromolecule

In addition, the antimicrobial activity spectrum of native pyrrhocoricin against 11 bacterial and fungal strains and the binding of labeled pyrrhocoricin to synthetic DnaK D-E helix fragments of the appropriate species could be correla-ted.Mutational analysis on a synthetic E coli DnaK frag-ment identified a possible binding surface for pyrrhocoricin Keywords: antimicrobial peptides; cell penetration; heat shock proteins; mutational analysis; pharmacophore

In the desperate fight against antibacterial-resistant bacteria,

the proline-rich peptide family can come to the rescue [1]

The most active member of these peptides, pyrrhocoricin, is

nontoxic to healthy mice and eukaryotic cells, and at low

doses can protect mice from bacterial challenge [2].Because

at high doses pyrrhocoricin is toxic to compromised

animals, pyrrhocoricin analogs were developed that lack

in vivotoxicity, even in infected mice, and show improved

stability in mammalian sera [2,3].These designed peptides

efficiently kill various resistant strains and clinical isolates

[4].The key feature to the ability of these peptides to kill

resistant bacteria is their novel mechanism of action

Pyrrhocoricin, and other family members such as drosocin

and apidaecin appear to exhibit their antimicrobial activity

by binding to the bacterial heat shock protein DnaK [5],

preventing chaperone assisted protein folding and inhibiting

the strongly related ATPase activity of DnaK [6]

Similar to other antimicrobial peptides [7], and generally

peptides that penetrate mammalian cells [8], the proline-rich

peptides are also rich in cationic residues, arginine, lysine

and histidine.Cationic peptides interact electrostatically

with the negatively charged bacterial phospholipids and then insert into the model membranes of planar bilayers or liposomes [9,10].Although these processes often involve the formation of channels or pores, alternative mechanisms were also suggested that either cooperatively destroy the membrane barrier without channel formation [11] or just create brief disruptions in permeability [12].It is clear that permeabilization of the cytoplasmic membrane to destroy the membrane potential is not lethal per se [13].This is especially true for peptides that are rich not only in positively charged residues but prolines as well [14].For example, it was argued that apidaecin or its mammalian analog PR-39, enter bacterial cells without any disruption of the membrane structure, and consecutively meet their intracellular target biopolymer [15,16].The special role of proline residues has been best documented for buforin II,

an antimicrobial peptide acting on bacterial DNA and RNA [17].Confocal fluorescence microscopy studies show that buforin II analogs with an intact proline hinge penetrate Escherichia coli membranes without permeabili-zation and accumulate in the cytoplasm [18].When the key proline is replaced with leucine, the preferred amino acid residue in antibacterial peptides with amphipathic a-helical structure [19], the ability of the peptide to accumulate intracellularly is decreased, and therefore the antibacterial activity is reduced

The above results suggest that the DnaK-binding domain

of pyrrhocoricin may not span the entire molecule, and a shorter active site can be identified that might not share the

Correspondence to L.Otvos, Jr., The Wistar Institute, 3601 Spruce

Street, Philadelphia, PA 19104, USA.

Fax: + 1 215 898 5821, Tel.: + 1 215 898 3772,

E-mail: Otvos@wistar.upenn.edu

(Received 24 May 2002, revised 2 July 2002,

accepted 11 July 2002)

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sequence with the fragment responsible for bacterial or

eukaryotic cell entry.Therefore, we studied the antibacterial

activity of a series of pyrrhocoricin analogs, in which either

each residue was replaced with alanine (Ala-scan) or

arginine-containing modules were inserted into strategically

important positions in the peptide.We also wanted to know

whether a pyrrhocoricin-based delivery mechanism could

promote entry into the less permeable mammalian cells [20]

When we ascertained that the Asp2–Pro10 fragment is the

most crucial domain for killing of E coli and

Agrobacte-rium tumefaciens, we further investigated the interaction

between fluorescein-labeled pyrrhocoricin or its active or

inactive analogs and E coli DnaK.In another line of

investigation, four pyrrhocoricin-responsive and seven

unresponsive bacterial and fungal strains were selected and

pyrrhocoricin’s binding to the corresponding DnaK D-E

helix was studied by fluorescence polarization.The

anti-bacterial activity correlated strongly with the binding to the

multihelical lid region of DnaK.Finally an Ala-scan on the

synthetic E coli DnaK D-E helix fragment identified a

series of residues that could not be replaced without losing binding to pyrrhocoricin.In the crystal structure of the DnaK protein, these residues comprise a continuous binding surface

M A T E R I A L S A N D M E T H O D S Rationale for the design of the modified pyrrhocoricin analogs

Modified pyrrhocoricin analogs were designed for studying important residues and regions in the hypothetical active site and delivery module (Table 1).First we concentrated on the pharmacophore.In peptide Pyrr-mod1, a characteristic turn-forming Pro8-Arg9 dipeptide motif in the active site was replaced with helix-forming residues Glu and Ala.To maintain the overall number of positive charges, the apparently freely replaceable Ser5 was substituted with Arg.The same design principles were applied to peptide Pyrr-mod2 with an additional positive charge at the

Table 1 Synthetic peptides used in this study Where appropriate, gaps are inserted to help the comparison of the different peptide sequences Unlabeled pyrrhocoricin analogs

Fluorescein-labeled full-sized pyrrhocoricin derivativesa

Pyrrhocoricin fragments (unlabeled and labeled) b

DnaK and Hsp70 D-E helix fragments:

a The fluorescein-label was directly coupled to the amino-termini of the peptides b The fluorescein-label was directly coupled to the amino-termini of the peptides, except for the Pyrr-10-20-K(Fl) derivative in which the fluorescein was coupled to the side chain of the extra C-terminal lysine residue.The Fl-K-pyrrhocoricin 1-9 and Fl-K-pyrrhocoricin 1-10 peptides behaved very similarly in every experiment, and therefore data are provided only for the 1-9 analog that had been scrutinized for its ATPase inhibitory activity earlier [6].The fluorescence signal of the two C-terminal analogs was quenched very rapidly.They are part of our ongoing efforts to find a suitable carboxy-terminal substrate.

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C-terminus to further promote cell entry.In addition, the

nonessential and freely replaceable Ile-Tyr dipeptide was

substituted with the Glu-Arg dipeptide to maintain good

solubility at any pH.Later we made changes only in the

C-terminal, nonessential region.In peptide Pyrr-mod3,

while the active site was kept intact, the characteristic

Pro12-Pro13 dipeptide fragment of the C-terminal domain was

disrupted, by inserting two hydroxy-amino acids that are

not foreign to pyrrhocoricin, but are normally located

outside the Pro-Arg-Pro repeats.To compensate for the

reduction of the percentage of Pro, an additional proline

was inserted after the last Pro, before the final IYNRN

segment.Finally, in peptide Pyrr-mod4 the same design

principles were used, except the inserted two hydroxy-amino

acids were separated, and the positive charge of the putative

cell-penetrating region was increased by inserting an

addi-tional Arg residue

N-Terminally fluorescein-labeled peptides included

full-sized native pyrrhocoricin, apidaecin and drosocin [6], and

the N-terminal 1–9 fragment of pyrrhocoricin.In these

derivatives, 5(6)-carboxy-fluorescein was coupled to the

a-amino group of an extra N-terminal lysine residue.The

addition of this Lys was needed, because the antibacterial

activity of the pyrrhocoricins is lost upon directly blocking

the N-terminus without an extra positive charge ([2] and

Table 2).This positive charge apparently plays a role in the

interaction with bacterial membranes [21].The Pyrr-mod1–

4 peptides were also synthesized with an N-terminal

fluorescein label, but because these peptides were designed

for fluorescence polarization studies, they did not contain

the extra lysine, except peptide Fl-Pyrr-mod4, which was

made both with and without the N-terminal Lys.In support

of the earlier findings, the peptide without the lysine lost its

antibacterial activity (Table 2).Table 2 also demonstrates

that the addition of the N-terminal lysine did not turn an

otherwise inactive peptide into an active one against E coli

JC7623

A series of peptides corresponding to the D-E helix regions

of different bacterial and eukaryotic DnaK/Hsp70 sequences

were synthesized.The different Hsp70 sequences were

dissimilar towards to C-termini of the proteins.The decision

as to which residues to select as C-termini depended upon where a gap between the extended D-E helix and the short extreme C-terminal domain was found.We observed the earliest gaps for the H pylori and the mouse sequences.To have bacterial DnaK fragments of similar size, the H pylori peptide was extended by two residues toward the D-helix at the N-terminus.The Salmonella typhimurium DnaK 584–

616 fragment has not been prepared because it is essentially identical to the E coli sequence except for a conservative TAGfi AGS change very close to the C-terminus For the Ala-scan of the DnaK D-E helix region, all native residues were replaced with alanine.The three native alanines, Ala591, Ala601 and Ala606 were replaced with phenylalanine.The sequence was not continued after Thr610 because the C-terminal hydrophobic residues signi-ficantly contribute to the low aqueous solubility of the full 583–610 fragment

Peptide synthesis and purification N-Terminally Fmoc-protected amino acids [22] were used for the synthesis of the peptides.In the Ala-scans each residue in pyrrhocoricin or each residue in the 583–610

E coliDnaK fragment was replaced with alanine.The Ala-scan peptides were assembled on a SYRO multiple peptide synthesizer, and after cleavage were purified by RP-HPLC All other peptides were made individually, on a MilliGen

9050 continuous-flow automated synthesizer and were purified by RP-HPLC until MALDI-MS revealed only single species with the expected molecular ions.The mass spectra were taken at the Wistar Institute Protein Micro-chemistry Facility

Fluorescence polarization Binding of the synthetic DnaK fragments or the mutated derivatives to fluorescein-labeled pyrrhocoricin or its ana-logs was assessed by fluorescence polarization [23].For these experiments, the unlabeled peptides were serially diluted in Tris buffer (pH 7.4) in 50 lL final volume in

6· 50 mm disposable glass borosilicate tubes.The fluores-ceinated pyrrhocoricin peptides were added to each tube in a 50-lL aliquot to a final concentration of 1 nMand tubes were incubated at 37C for 5 min The extent of cence anisotropy was measured on a Beacon 2000 fluores-cence polarization instrument (PanVera, Madison, WI, USA) and calculated as millipolarization values.The filters used were 485 nm excitation and 535 nm emission with

3 nm band width.During fluorescence polarization, it is assumed that one molecule of the labeled compound interacts with one molecule of the unlabeled partner, and while the concentration of the unlabeled larger molecule determines the association constant, the concentration of the labeled partner determines the fluorescence intensity The 1 nM tracer concentration, andother parameters used herein, were successfully used in our laboratory earlier to study peptide–peptide [6,24], peptide–protein [5] and pep-tide–nucleic acid interactions [25]

In vitro antibacterial assays Antibacterial assays were performed in sterile 96-well plates (Nunc F96 microtiter plates) with a final volume of 100 lL

Table 2 In vitro antibacterial activity of some pyrrhocoricin analogs

that are discussed in the text In addition to the fluorescein-labeled

Pyrr-mod1 – Pyrr-mod4 pyrrhocoricin derivatives, the same peptide analogs

were labeled with biotin at their N-termini.Similar to the

fluorescein-derivatives, none of the biotin-labeled peptides exhibited activity up to

40 l M

Peptide

IC 50 against

E coli JC7623 (l M )

L -pyrrhocoricin-1-10- D -pyrrhocoricin-11-20 > 40

Fl-K-pyrrhocoricin 1-20 20–40

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as described previously [26].Briefly, 90 lL of a suspension

of a mid-logarithmic phase bacterial culture at an initial

600 nm UV absorbance of 0.001 in media required for good

growth of the various bacterial strains was added to 10 lL

of serially diluted peptides in sterilized water.The final

peptide concentrations ranged between 0.06 lM(lower) and

20–80 lM (upper).Plates were incubated at 37C for

16–24 h with gentle shaking, and growth inhibition was

measured by recording the increase of the UV absorbance at

600 nm on a microplate reader

Cell penetration assay

To study the ability of the antibacterial peptides and their

fragments to enter cells, fluorescein-labeled peptides were

added to E coli JC7623 cells or mouse macrophages at final

concentrations of 5 lgÆmL)1and 100 ngÆmL)1, respectively

The cells were allowed to acquire the substrate for 60 min at

37C, the excess substrate was removed and the cells were

washed extensively with NaCl/Pi, pH 6.8 The cells were

fixed with NaCl/Pibuffer containing 1% paraformaldehyde

and visualized using a Leica TCS SPII laser scanning

confocal microscope (E coli) or Leica DMIL fluorescence

microscope (macrophages)

Circular dichroism measurements

CD spectra were performed on a Jasco J720 instrument at

room temperature in a 0.2 mm pathlength cell Doubly

distilled water was used as solvent.The peptide

concentra-tions were 0.5 mgÆmL)1, determined each time by

quan-titative RP-HPLC [27].Curves were smoothed by the

algorithm provided by Jasco.Mean residue ellipticity

([h]MR) is expressed in degreesÆcm2Ædmol)1by using mean

residue masses of 110

R E S U L T S

Identification of crucial residues for the antibacterial

activity of pyrrhocoricin

An Ala-scan was performed to identify key residues that

could not be replaced in pyrrhocoricin without a major loss

in antibacterial activity.For the bacterial strains, we selected

E coliand A tumefaciens because in our earlier studies we

had observed some differences in the potency of truncated

pyrrhocoricin peptides in killing these strains

[28].Accord-ing to the Ala-scan, pyrrhocoricin had an identical bioactive

fragment against both strains (Fig.1) No activity was

detected (up to 20 lM peptide) against either bacterium

when Asp2, Lys3, Tyr6, Leu7, Pro8, Arg9 or Pro10 were

replaced with alanine, indicating that the most crucial

residues for antibacterial activity were in the Asp2–Pro10

peptide fragment.Further investigations are required to

reveal whether the Asp2–Lys3 dipeptide is a functional part

of the pharmacophore.All other amino acids could be

replaced without a major loss in the antimicrobial activity of

the peptide, except Val1, Arg14 and Arg19, residues, which

are needed for the full antibacterial activity, at least against

A tumefaciens.Some key N-terminal residues were also

replaced with tyrosine (Tyr-scan), resulting in identical

activity data

Antibacterial activity of pyrrhocoricin analogs The Ala- and Tyr-scans identified the N-terminus as the fragment carrying essential residues for the antibacterial activity and the C-terminus as a necessary domain, but suitable for substitutions.To provide further support for this notion, the antibacterial activity of the Pyrr-mod1– Pyrr-mod4 analogs was studied in detail against clinical strains of E coli, A tumefaciens, S typhimurium, H influ-enzae, Klebsiella pneumoniae, P aeruginosa and S aureus Table 2 shows the half inhibitory concentration (IC50) values against these bacteria.As we showed earlier, the pyrrhocoricin analogs are inactive against S aureus [5], and

no activity could be obtained with the new peptides, even if the number of the positive charges was increased.When modifications were made in the Tyr6–Pro10 active segment (peptides Pyrr-mod1 and Pyrr-mod2) the activity was completely lost against all five originally responsive strains Most, but not all, activity could be recovered by peptide Pyrr-mod3, which had no modification in the Tyr6–Pro10 region.However in peptide Pyrr-mod4, a repeat of the characteristic Thr11–Pro12 linker between the active seg-ment and the C-terminal domain, coupled with the increase

of the positive charges needed for cell entry, resulted in a peptide variant with increased antibacterial activity against two of the five strains.When we synthesized pyrrhocoricin fromL-amino acid residues at the N-terminus (Val1-Pro10) and D-amino acid residues at the C-terminus (Thr11-Asn20), no activity against E coli JC7623 was observed (Table 2).Antibacterial peptides with mainly membrane-disrupting mode of action are insensitive to the stereochem-ical configuration of the constituent residues [29]

Peptide entry intoE coli cells The experiments above indicated that the proline-rich peptide family had multiple functions and functional domains, and perhaps carried separate modules for cell entry and other tasks leading to bacterial killing.Therefore,

we studied the ability of fluorescein-labeled pyrrhocoricin, its fragments and modified analogs to enter E coli JC7623 cells.Because these peptides are currently considered

promi-Fig 1 In vitro antibacterial activity of alanine-substituted pyrrhocoricin peptides (Ala-scan) against Escherichia coli D22 and Agrobacterium tumefaciens The data represent the IC 50 values, which were read as half ultraviolet absorbance between full bacterial growth (no peptide added) and medium.The assay was performed in poor broth medium.

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sing therapeutic drug candidates [1,3,4], and many emerging

resistant bacterial species are intracellular pathogens, we also

wanted to know whether the proline-rich antibacterial

peptide family was able to penetrate macrophages where

facultative or obligate bacteria often propagate

N-Terminally labeled pyrrhocoricin, containing a lysine

residue between the label and the antibacterial peptide

(Fl-K-pyrrhocoricin 1–20), entered the bacterial cells very

efficiently (Fig.2, top left image).The staining appeared to

be homogenous throughout the entire cell body.Although

the isolated 1–9 fragment of pyrrhocoricin is inactive in the

in vitroantibacterial efficacy test [2], this fragment binds to

the D-E helix region of bacterial DnaK and inhibits the

ATPase activity of the heat shock protein [6].We wanted to

see whether the lack of antibacterial activity could be

correlated with less efficient cell entry.Indeed, much weaker

labeling of the E coli cells was observed for the N-terminal

1–9 fragment (Fl-K-pyrrhocoricin 1–9), carrying the

fluorescein chromophore and the extra lysine residue in a

position identical to that of full-sized pyrrhocoricin (Fig.2,

top right image).Not only fewer cells were labeled with the

shortened pyrrhocoricin fragment, but perhaps more

importantly, the fluorescence intensity seemed to

concen-trate to the cell surface as the staining pattern of single cells

indicated (Fig.2, middle images).Widespread distribution

of the drug inside bacterial cells is needed for efficient inhibition of the putative intracellular target protein.When

a similarly labeled construct of Pyrr-mod4 was studied (Fl-K-Pyrr-mod4), the bacterial cell penetration profile was not significantly different from that of full-sized pyrrhoco-ricin, indicating that the function of the C-terminal module was not compromised with the amino acid changes and insertions in this domain.The removal of the Lys residue that connected the fluorescein moiety and Val1 of the antibacterial peptide analog resulted in a major decrease in the number of E coli cells labeled by Fl-Pyrr-mod4, consistent with the significant reduction in the antibacterial activity (Table 2).Nevertheless, when Fl-Pyrr-mod4 with-out the extra lysine did enter bacterial cells, the peptide analog was homogenously distributed all over the cell interior (Fig.2, bottom right image).Fl-Pyrr-mod1 without the extra lysine entered similar number of cells and underwent intracellular distribution in an identical fashion (Fig.2., bottom left image), indicating that there were no major differences in the cell penetration and distribution pattern of naked Pyrr-mod1 or Pyrr-mod4.Rather, the considerably stronger antibacterial activity of naked Pyrr-mod4 (Table 3) was due to its actions on the intracellular target molecule.In addition to bacterial cells, fluorescein-labeled analogs of the three native peptides with the Lys

Fig 2 Confocal fluorescence microscopic images of E coli JC7623 cells upon incubation with fluorescein-labeled pyrrhocoricin analogs The peptide substrates are as follows: Fl-K-pyrrhocoricin 1–20, top and middle left; the analogous N-terminal half, Fl-K-pyrrho-coricin 1–9, top and middle right; the Fl-Pyrr-mod1 peptide without a Lys between the fluorescein label and the peptide analog, bottom left; and a similarly built Fl-Pyrr-mod4 construct, bottom right.

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inserted at the N-terminus between the label and the

peptides were added to murine macrophage cultures

Pyrrhocoricin, apidaecin and drosocin penetrated into

mammalian cells without having preference to any

partic-ular cell compartment, including the cell membrane (not

shown).Similar to the results observed on bacterial cells, the

identically labeled pyrrhocoricin 1–9 fragment penetrated

into the mouse macrophages much less effectively.This also

confirmed that while the N-terminus served as the

pharma-cophore and hence the interactive domain with the bacterial

target protein, the C-terminal half aided the delivery of the

pharmacophore into the interior of cells

These studies require a detailed analysis of the

permis-sibility of fluorescein substitutions at various positions in the

peptide.Addition of fluorescein significantly reduces the

antibacterial activity of pyrrhocoricin, much more than any

other substitution does ([2] and Table 2), even if the Lys

residue is incorporated between the label and the peptide

Therefore, the current cell penetration studies were

restric-ted to peptides that could be labeled with fluorescein at the

original N-termini in an identical manner.The fluorescence

intensity of C-terminally labeled pyrrhocoricin fragments

quenched very rapidly (compare with Table 1).In addition,

the extra lysine residue alone did not generate antibacterial

activity above that present without this addition.The

K-Pyrr-1–10 peptide fragment was just as inactive against

E coli JC7623 as any other pyrrhocoricin fragments

truncated to half size

Connection between antibacterial activity

and pyrrhocoricin binding to the D-E helix region

of DnaK

Previously, we documented that pyrrhocoricin binds to a

synthetic version of the D-E helix region of E coli, a

responsive species, but fails to bind to the homologous

fragment of the unresponsive species S aureus [6].Here, we

extended these studies to four responsive and seven

unresponsive bacterial and fungal species.Table 3 shows

that native pyrrhocoricin killed E coli, A tumefaciens,

S typhimurium, and H influenzae, among others, and

failed to kill S aureus.Very weak activity, often read as

inactive [3], was found against P aeruginosa.Some other

pyrrhocoricin-unresponsive bacterial and fungal strains

include S pyogenes, C albicans [4], S.pneumoniae, H

duc-reyiand H pylori.In addition, the peptide is not toxic to

healthy mice or eukaryotic cells [2].Fragments of the

DnaK/Hsp70 sequences of the above-listed bacterial and

fungal strains and of mouse and human were synthesized

and their binding to N-terminally fluorescein-labeled pyr-rhocoricin (with the extra lysine inserted) was studied by fluorescence polarization.The synthetic DnaK peptides encompassed the D and E helices and a short fragment of the unordered region C-terminal to the multihelical lid assembly (Table 1).Because the corresponding sequences of

E coliand S typhimurium DnaK are almost identical, and from other studies we know that the TAGADA C-terminal hexapeptide fragment of the E coli sequence (where these minor mutations are found) is not needed for the binding, the S typhimurium peptide was not synthesized.The Kd between E coli D-E DnaK and fluorescein-labeled pyrrho-coricin is in the order of 50 lM[6], is expected to be lower for the full-sized protein, and correlates well with the 5–10 lM antibacterial activity of the same fluorescein-labeled pyrrhocoricin against E coli [2].One set of the binding studies was carried out at a receptor fragment concentration of 15 lM, below the peptide–peptide associ-ation constant, serving the data as baseline values.The highest concentration of the synthetic DnaK fragment was

250 lM, where all responsive sequences were expected to bind.In summary, strong pyrrhocoricin binding to the D-E DnaK helix region of the three (four) responsive strains was observed, and no or very low binding was found to the DnaK fragments of the seven nonresponsive, or very weakly responsive strains (Fig.3) Significantly, no binding was detected to the mouse or human Hsp70 fragment, confirm-ing the lack of specific bindconfirm-ing to the human equivalent of DnaK [5]

While modifications in the active segment of pyrrhoco-ricin resulted in the complete disappearance of the antibac-terial activity, changes made in the C-terminal delivery unit retained at least some activity (Table 3).To further correlate the ability to kill certain bacteria and DnaK binding, we studied the interaction between the E coli D-E helix DnaK fragment and fluorescein labeled Pyrr-mod1–4 peptides.In essence, the fluorescence anisotropy was not increased compared to the baseline when the inactive Pyrr-mod1 or Pyrr-mod2 peptides were added to the synthetic DnaK fragment.However, when the active mod3 and Pyrr-mod4 peptides were added, significant increases in the fluorescence anisotropy were detected, once again showing full correlation between DnaK binding and antimicrobial activity (Fig.4).These experiments further confirmed the location of the pharmacophore (i.e DnaK binding site) between Asp2 and Pro10 in pyrrhocoricin.It needs to be mentioned that in these labeled Pyrr-mod1–4 peptides, no cationic amino acid, needed for the antibacterial activity of N-terminally labeled pyrrhocoricin analogs [2], was inserted

Table 3 In vitro antibacterial activity of modified pyrrhocoricin peptides One quarter strength of Muller-Hinton broth was used as the medium for the growth of E coli, A tumefaciens, K pneumoniae and P aeruginosa; 1/4 strength of brain-heart infusion for S typhimurium and H influenzae (for the latter one the medium was supplemented with NAD) and poor broth medium was used for S aureus.

Peptide

IC 50 (l M ) against

E coli A tumefaciens S typhimurium K pneumoniae H influenzae P aeruginosa S aureus

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between the label and Val1.A combination of these data

and those represented by Fig.2 demonstrated that while an

increased number of positive charges at the amino terminus

was required for the gross antibacterial activity, the DnaK

binding and cell distribution studies could proceed with

analogs lacking the lysine insert

Conformation of the DnaK fragments

Although we assumed that the lack of pyrrhocoricin

binding to some of the DnaK D-E helix sequences directly

reflected the lack of pyrrhocoricin activity against these bacteria, it was possible that alterations in the physical or biochemical properties of the synthetic protein fragments played roles in our inability to detect positive interaction between the peptides.One of the potential sources of altered behavior can be a change in the trademark helical conformation of the DnaK fragments.To exclude this scenario, we compared the secondary structure of the D-E helix peptides by CD spectroscopy.CD spectra were taken in water, and water/trifluoroethanol mixtures [30] All DnaK fragments exhibited very similar spectra regardless of whether they bound to pyrrhocoricin or not.Here we present the data on E coli and S aureus (Fig.5) In water, the CD spectra of both DnaK fragments could be assigned as a type C spectrum [31], and reflected the dominance of type I (III) b-turns, or a mixture of type I and type II turns [32].Addition of 5% trifluoroethanol (v/v) resulted in a redshift of both pp* bands to 190 and 204 nm, respectively, accompanied by

an increase of the intensity of the positive band which is a clear indication of the appearance of helical structures The two DnaK fragments behaved identically, at least in spectral terms.While the spectral features of well-devel-oped a-helices could already be seen at a trifluoroethanol concentration as low as 10%, the intensity was increased with increasing trifluoroethanol content.The spectra of the E coli and S aureus peptides remained very similar in all water/trifluoroethanol compositions studied.If any difference could be detected it was a minor intensity increase throughout and some redshift (around 1 nm) at low trifluoroethanol concentrations for the S aureus sequence compared to the E coli peptide.This could be explained by the increased number of potential salt bridges along the helix barrel.The S aureus peptide contained 3 and 5 potential Glu–Lys salt bridges in i,i+3 and i,i+4 positions, respectively.These figures for the

E coli fragment were 2 and 0.The isolated peptide fragments thus exhibited all helical features of the complete DnaK multihelical lid; the fragments were very similar but not identical.Having said this, pyrrhocoricin binding was not directly related to the helical content as determined by fluorescence polarization (data not shown) The millipolarization values between E coli DnaK 583–

615 and Fl-K-pyrrhocoricin decreased as the trifluoroeth-anol content of the solvent increased in the 0–10% trifluoroethanol range.No fluorescence anisotropy could

be detected when the solvent contained more the 10% trifluoroethanol

The CD studies further verified the binding of pyrrho-coricin to the E coli DnaK fragment and the lack of a sequence analogous to that of S aureus (Fig.6).To study the interaction of the antibacterial peptide and the synthetic DnaK D-E helix fragments, we looked at the conformation

of the E coli DnaK 583–615 or the analogous S aureus DnaK 554–585 peptide–pyrrhocoricin mixtures as shown

by CD.If the sum of the individual CD curves of the two interacting partners is different from the CD of the ligand– receptor mixture, it is a clear indication of not only binding, but also of a conformational change upon interaction Indeed, such a spectral alteration was detected for the binding fragment of the responsive strain E coli, but not for the nonbinding fragment of the unresponsive strain

S aureus(Fig.6).If any consequence for the nature of the

Fig 4 Interaction of 1 n M fluorescein-labeled pyrrhocoricin analogs

with the E coli DnaK 583–615 fragment The concentration of the

unlabeled protein fragment was 125 l M The fluorescence anisotropy

data are expressed as the difference in the millipolarization values

between samples containing both the labeled antibacterial peptides and

the E coli receptor fragment, or the appropriate labeled pyrrhocoricin

analogs alone.

Fig 3 Binding of fluorescein-labeled pyrrhocoricin to various synthetic

DnaK D-E helix fragments as studied by fluorescence polarization In

these experiments, the N-terminally fluorescein-labeled pyrrhocoricin

derivative at 1 n M concentration was added to synthetic D-E helix

DnaK fragments that were applied at either 250 l M or 15 l M The

data are expressed as the difference in the millipolarization values

between samples containing both the labeled antibacterial peptide and

the given receptor fragments, or labeled pyrrhocoricin alone.The

fig-ure shows the average of 10 readings taken after a 5 min incubation

period.These experiments were repeated two times with basically

identical outcomes.

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conformational change was to be drawn, the decrease of the

intensity of the 200 nm band representing the unordered

structure suggests the generation of a more ordered

conformation

Identification of the pyrrhocoricin binding surface

onE coli DnaK

Encouraged by the success of the Ala-scan in identifying the

essential residues of pyrrhororicin for antibacterial activity,

we used a similar strategy to identify essential residues in

E coliDnaK for pyrrhocoricin binding.For this purpose

each residue in the 583–610 D-E helix fragment was

replaced with alanine.Phenylalanine was substituted for

the three native alanines in the sequence.Binding to

N-terminally fluorescein-labeled and Lys extended

pyrrho-coricin was studied by fluorescence polarization.The

fluorescence anisotropy showed a ladder-type interaction

pattern that probably reflected the presence of vital residues

for the binding and flanking residues likely needed to

maintain the structural integrity at the interaction sites

(Fig.7).The major drop in the fluorescence polarization

signal was observed when Glu7 (Glu589 in the full protein),

Gln13 (Gln595) or Met16 (Met598) were replaced with Ala

Additional important residues were Gln6 (Gln588), Gln10

(Gln592), Leu15 (Leu597), Ala19 (Ala601), Gln20 (Gln602)

and Gln22 (Gln604).Figure 7 shows an average of three

independent measurements and is cut at 12 millipolarization

units, which represents pyrrhocoricin binding to the native

Fig 6 Circular dichroism spectra of DnaK

D-E helix–pyrrhocoricin mixtures in water.

First, the individual peptide spectra were

col-lected, followed by the spectra of the

anti-bacterial peptide–protein fragment mixtures.

While the measured spectrum of the E coli

fragment–pyrrhocoricin mixture is different

from the mathematical sum of the individual

spectra, and indicates a conformational

change upon interaction, no similar alteration

could be detected for the unresponsive strain

S aureus.

Fig 5 Circular dichroism spectra of E coli

and S aureus DnaK D-E helix fragments in

trifluoroethanol-water mixtures The spectra

were taken at room temperature and the

peptide concentration was approximately

0.5 mgÆmL)1.Both peptides underwent a

characteristic turn–helix transition as the

tri-fluoroethanol concentration increased.

Fig 7 Binding of fluorescein-labeled pyrrhocoricin to alanine-substi-tuted E coli DnaK D-E helix analogs as studied by fluorescence polar-ization In these experiments, the N-terminally fluorescein-labeled pyrrhocoricin derivative at 1 n M concentration was added to the Ala-scan of synthetic D-E helix DnaK fragments that were applied at

150 l M The data are expressed as the difference in the millipolariza-tion values between samples containing both the labeled antibacterial peptide and the D-E helix analogs, or labeled pyrrhocoricin alone.The figure shows the average of three independent experiments conducted from freshly purified samples; in each, 10 readings were taken after a

5 min incubation period.

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E coliDnaK 583–610 sequence.In reality, substitution of

some residues increased rather than decreased the

fluores-cence anisotropy.The reason for this finding is still unclear;

it may indicate stronger binding to the mutated peptides, an

increased fluorescence signal due to a somewhat shifted

binding site or simply solubility differences

D I S C U S S I O N

Proline-rich delivery modules

To reach the cell interior, antibacterial peptides have to cross

the bacterial membrane.To fight pathogens living inside

host cells, peptides generally have to enter eukaryotic cells as

well.In the current study, we looked at the entry of

pyrrhocoricin, drosocin and apidaecin into E coli cells and

macrophages, host cells of intracellular bacteria such as

Mycobacterium tuberculosisor Mycobacterium leprae.The

full-sized peptides entered the cells and became distributed in

all cellular compartments, suggesting an efficient transport

mechanism for these molecules.In our previous report, we

suggested that in addition to the target protein, DnaK, the

60 kDa chaperonin protein family, GroEL in bacteria and

Hsp60 in mammalian cells, can serve such a purpose [5]

Remarkably, when full-sized peptides were studied, no

selective surface staining of the cells could be detected by

confocal fluorescence microscopy.Such surface staining

is characteristic for alternating arginine-leucine

contain-ing biooligomers and other membrane-destabilizing

antimicrobial peptides, including magainin 2 [18].In

con-trast, the PRP or similar repeat sequences in the proline-rich

antibacterial peptide family assist the entry into the host and

subsequently into bacterial cells without any potential to

destabilize the cells, and therefore become toxic to

eukar-yotes.The antibacterial activity of the native products is

provided by the independently functioning active site,

capable of binding to the D-E helix region of bacterial

DnaK and preventing chaperone-assisted protein folding [6]

Proline-rich cell penetration modules may be general for

antibacterial peptides in nature.Bovine myeloid cathelicidins

carry precursors of broad spectrum antimicrobial peptides

characterized by N-terminal amphipathic a-helices and

C-terminal hydrophobic tails [33].Antibacterial peptides

with amphipathic helices or sheets are considered to act on

the negatively charged bacterial membranes by creating a

strong dipole moment and destroying the energy gradient

across the membrane [34].Indeed, the C-terminal tails of the

cathelicidins are not needed for the antibacterial activity with

the truncated peptides containing only the amphipathic

helices being equally active against a selection of both

Gram-negative and Gram-positive bacteria [33].However, a 5- to

10-fold higher concentration of the truncated analogs is

required to achieve a kinetics of permeabilization similar to

that of the respective parent peptides, suggesting a somewhat

less effective initial interaction with the target bacterial

membranes [33].An examination of the cathelicidin

hydro-phobic tail sequences (PVIPLLHR and PIIVPIIRI) reveal

strong similarities to C-terminal tails of pyrrhocoricin

(PPRPIYNRN), drosocin (SHPRPIRV) or apidaecin

(PHPRI).Apparently, regardless of the mechanism for

peptide-mediated killing of bacteria, the highly similar

cell-penetrating modules may serve as first interaction points

with the membrane surface and help the internalization of

the peptides.In turn, this may suggest that some individual antibacterial peptides or peptide families for which we originally assumed a membrane-only mode of action [1] may have intracellular targets too, just the sequences responsible for nonmembrane activity are disguised by the ubiquitous presence of membrane-targeting delivery modules

Interaction between pyrrhocoricin and bacterial biopolymers from the viewpoint of the antibacterial peptide

The essential region of pyrrhocoricin for the antibacterial activity was located between Asp2 and Pro10.The identi-fication of the Asp2–Pro10 active segment is in full agreement with our earlier model of pyrrhocoricin–DnaK interactions.In our previous report [6], we showed a model

of how pyrrhocoricin remains close to the E-helix of E coli DnaK after applying a flexible docking algorithm.When we look at this model from the angle of the E helix barrel, it is evident that the pyrrhocoricin fragment that most intimately touches DnaK is exactly the experimentally identified Tyr6– Pro10 hexapeptide segment [35].The pyrrhocoricin 1–9 fragment alone (or pyrrhocoricin 1–10 for that matter) penetrated into cells very inefficiently but was an absolute requirement for antibacterial activity.Pyrrhocoricin is shown to bind to DnaK both inside and outside the peptide-binding pocket [6].The N-terminal region might play roles in the association with molecular chaperones (DnaK or GroEL) that serve as transport vehicles for the peptide.Consistent with this, an application of the predi-cative algorithm for nonspecific interaction of peptides with the conventional peptide binding pocket of E coli DnaK [36] reveals a high probability of the N-terminal trideca-peptide fragment of pyrrhocoricin associating with the heat shock protein (energy score )5.0), but not when the 13-residue window starts from Gly4 (energy score +4.7) Significantly, the side-chain of Thr11, located at the border

of the pharmacophore and the delivery unit, appeared not

to drastically influence the antimicrobial activity.This observation is compatible with our earlier findings showing that the native sugar addition to Thr11 has no influence on the gross antibacterial activity [28].The presence of the carbohydrate side-chain, however, may modify either the binding to the target protein or the ability of the peptide to penetrate cells via through-space effects.The importance of Arg19 was documented previously, when we reported that the deletion of the C-terminal Arg19–Asn20 dipeptide results in a reduction of the activity against A tumefaciens [28].The partial loss of activity after replacing the two C-terminal arginines suggests that this region is responsible for the initial interaction of pyrrhocoricin with the negat-ively charged bacterial surface and the ensuing entry into bacterial cells

The modified C-terminal delivery module featuring an increased number of the Arg–Pro segments had an advant-age against two strains that have more resistant membrane structures, S typhimurium and H influenzae.Even if the C-terminal fragment was not directly involved in binding to the target protein, it still interacted with a biopolymer in a stereospecific manner.The weak activity of native pyrrho-coricin against P aeruginosa, a gram negative strain known

to have an outer membrane that is difficult to penetrate [37], may reflect either only a low ability of the peptide to

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disintegrate this membrane structure or very weak binding

to the target protein.A Lys1-containing analog of

pyrrho-coricin shows improved activity against both E coli and

P aeruginosa, potentially shifting the dominant mode of

action from DnaK inhibition to membrane disintegration

[21].N-Terminally blocked cecropin analogs, known to kill

bacteria by disassembling the membrane structure, lack

antibacterial activity [29].All these suggest that the

N-terminal charges play an equally important role in the

interaction with bacterial membranes, but this might be

decoupled from the distribution of the peptides in the cells

The N-terminally labeled Pyrr-mod4 analog without the

extra lysine bound to DnaK, was well dispersed inside

E colicells, but labeled a smaller number of bacterial or

mammalian cells, and therefore was unable to kill E coli

(Table 2).It is tempting to speculate that antimicrobial

peptides with intracellular targets form a loop and interact

with the negatively charged bacterial membranes with both

termini and not with only one side, as was impressively

demonstrated previously [15].Alternatively, the two

charged domains could interact with spatially and

tempor-ally separated bacterial components

Concerning the specific interaction between the

antimi-crobial peptides and bacterial DnaK, the antibacterial

activity and selective binding to the D-E helix region of the

70 kDa heat shock protein was strongly

correlated.Pyr-rhocoricin bound to the representative synthetic fragments

of the highly responsive strains E coli, S typhimurium,

A tumefaciensand H influenzae, but failed to bind to the

homologous fragments from the nonresponsive strains

S aureus, S pyogenes, H pylori, H ducreyi, S pneumoniae,

C albicansor the weakly responsive strain P aeruginosa

Further support for the connection between binding to the

D-E helix of DnaK and antibacterial activity came from the

Pyrr-mod1–4 analogs.The antibacterial activity of

pyrrho-coricin analogs that were modified in either the active site or

in the C-terminal unit and binding to the protein fragment

was strongly correlated.In addition, the native peptide

lacked binding to the homologous region of mouse or

human Hsp70 confirming the lack of specific peptide

binding to full-sized human Hsp70 [5] or toxicity to

mammalian cells and healthy mice [2]

Interaction between pyrrhocoricin and DnaK from the viewpoint of the heat shock protein

To identify a potential pyrrhocoricin-binding surface on

E coliDnaK, here we analyzed the important residues for the interaction based on the published X-ray structure of the protein.In Fig 8, we displayed the surface of the C-terminal domain (residues 533–606) in semitransparent gray [38], then marked the synthetic 583–606 fragment in ball and stick inside the surface.Due to the lack of ordered structure C-terminal to Ala606, the rest of the synthetic DnaK peptide is excluded from this graphical representation.The crucial residues for pyrrhocoricin binding based on Fig.7 are colored in Fig.8 blue (most important) and green (important).This exercise identifies an L-shaped binding surface, including the primary binder linear surface com-prising residues Gln13 (Gln595 in the full protein), Met16 (Met598), Ala19 (Ala601) and Gln20 (Gln602), and the secondary surface attached in an 80 degrees angle featuring Gln6 (Gln588) and Gln10 (Gln592).Additional essential residues, Lys14 (Lys596), Leu15 (Leu597) and Ile18 (Ile600) are involved in the stabilization of the correct fold of DnaK for pyrrhocoricin binding.Of these, Leu597 and Ile600 have

no or very little surface exposure (calculated by [39]), but make hydrophobic contacts with neighboring essential residues.While Leu597 strongly influences the proper side-chain orientation of the highly important residue Met598, Ile600 touches the aliphatic portion of the side chain of Lys596.The apparently very significant residues Glu7 (Glu589) and Gln22 (Gln604) have surface exposure, but are facing the opposite direction.These hydrophilic residues probably maintain the aqueous solubility required for the successful fluorescence polarization assay of the otherwise not very soluble synthetic DnaK D-E helix peptide fragment

This model would well explain the lack of activity against all the negative bacterial strains and higher organisms

H pylori, P aeruginosa, S aureus, S pyogenes, S pneumo-niae, C albicans, M musculusand H sapiens all lack both Met16 and Gln20 (compare with Table 1).These residues are present in the responsive strain H influenzae and the homologous unresponsive strain H ducreyi.The two

Fig 8 Possible pyrrhocoricin-interacting

resi-dues in E coli DnaK The published structure

of the C-terminal domain of E coli DnaK [38]

is displayed in semitransparent.The synthetic

583–606 fragment, used for the Ala-scan is

marked with ball and stick representation

inside the surface.The crucial residues for

pyrrhocoricin binding based on the Ala-scan

are labeled blue (most important) and green

(important).The likely primary and secondary

binding surfaces are highlighted by blue and

green patches, respectively.

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