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The lack of a hydrophobic substrate binding site in the b and b¢ domains of ERp57 and ERp72 indicates that these PDIs either require protein partners that assist in substrate recognition

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A structural overview of the PDI family of proteins

Guennadi Kozlov, Pekka Ma¨a¨tta¨nen, David Y Thomas and Kalle Gehring

Department of Biochemistry, Groupe de Recherche Axe´ sur la Structure des Prote´ines, McGill University, Montre´al, Que´bec, Canada

Introduction

Protein disulfide isomerase (PDI) was the first protein

folding catalyst discovered [1] Since its discovery more

than 40 years ago, studies of this remarkable enzyme

have shown that PDI acts as a dithiol–disulfide

oxido-reductase that is capable of reducing, oxidizing and

isomerizing disulfide bonds Independently of its redox

activity, PDI can also act as a chaperone both in vitro

[2] and in vivo [3] PDI is the founding member of a

family of 20 related mammalian proteins that are

chiefly located and function in the endoplasmic

reticu-lum (ER) (Fig 1) PDI family members are abundant

and play a significant role in protein folding and

qual-ity control in the calcium-rich oxidative environment

of the ER [4] The members vary in length and domain

arrangement, but share the common structural feature

of having at least one domain with a thioredoxin-like

structural fold, babababba Most PDI family members

contain both catalytic and non-catalytic

thioredoxin-like domains that are identified as either a or b based

on the presence or absence of a catalytic motif, with use of the prime symbol to indicate their position in the protein PDI has four such domains, a, b, b¢ and a¢ [5] The a and a¢ domains functionally resemble thiore-doxin, and each contains catalytic Cys-x-x-Cys motifs that react with thiols of newly synthesized proteins to confer disulfide oxidoreductase activity The b and b¢ domains, although structurally similar to thioredoxin,

do not contain catalytically active cysteines Instead, the b and b¢ domains appear to act as spacers, and are often responsible for substrate recruitment [6–9] The non-catalytic domains have lower sequence identity than the catalytic domains across PDI family members, and show more structural variability For instance, the

b domain of ERp44 (ER protein 44 kDa) has an unorthodox arrangement of the secondary structure elements, bbabbba [10] The family members most

Keywords

disulfide; endoplasmic reticulum; ERp44;

ERp57; ERp72; PDI; protein folding; protein

structure; thioredoxin-like; X-ray

crystallography

Correspondence

K Gehring, Department of Biochemistry,

McGill University, 3649 Promenade Sir

William Osler, Montre´al, Que´bec, H3G 0B1,

Canada

Fax: +1 514 398 2983

Tel: +1 514 398 7287

E-mail: kalle.gehring@mcgill.ca

(Received 8 April 2010, revised 11 July

2010, accepted 27 July 2010)

doi:10.1111/j.1742-4658.2010.07793.x

Protein disulfide isomerases (PDIs) are enzymes that mediate oxidative pro-tein folding in the endoplasmic reticulum Understanding of PDIs has historically been hampered by lack of structural information Over the last several years, partial and full-length PDI structures have been solved at an increasing rate Analysis of the structures reveals common features shared

by several of the best known PDI family members, and also unique features related to substrate and partner binding sites These exciting breakthroughs provide a deeper understanding of the mechanisms of oxida-tive protein folding in cells

Abbreviations

CNX, calnexin; CRT, calreticulin; ER, endoplasmic reticulum; PDI, protein disulfide isomerase; SAXS, small-angle X-ray scattering.

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similar to PDI have a short interdomain region between

the b¢ and a¢ domains that is termed the x-linker [11]

Recently determined structures of several PDI family

members have revealed their detailed architecture and

led to mechanistic insights into their function The

most exciting breakthrough came when the full-length

crystal structure was solved Determination of the

structure of yeast Pdi1p (yeast PDI) showed that its

four domains form an overall ‘U’ shape, suggesting

how substrates may be positioned relative to the two

catalytic domains [12] The structure of ERp44 showed

that its three thioredoxin-like domains (abb¢) are

arranged like a cloverleaf A long C-terminal tail folds

back and makes contacts with the a and b¢ domains

[10] This capping function of the C-terminal tail may

have assisted the successful crystallization and

struc-ture determination Recently, the crystal strucstruc-ture of

full-length human ERp57 (ER protein 57 kDa, also

known as protein disulfide-isomerase A3 or 58 kDa

glucose-regulated protein) in complex with tapasin was

also solved [13] The ERp57 structure provides the first

structural insight into protein binding by the catalytic domains Two other crystal structures of PDI-like pro-teins have been solved: human ERp29 (ER protein 29 kDa) [14] and yeast Mpd1p (member of the protein disulfide isomerase family 1) [15] Additionally, the structures of the non-catalytic fragments of human ERp57 [16], human PDI [8,17] and rat ERp72 (ER protein 72 kDa, also known as protein disulfide-iso-merase A4) [18] have been determined

Here we review these structural studies, with special focus on mammalian PDIs and what can be learnt from their similarities and differences Excellent reviews of the process of disulfide bond formation in the ER and the biology of PDIs are also available [19,20]

What constitutes a PDI family member?

The PDI family contains both thiol-reactive and thiol non-reactive members, and this has led to some confusion A thioredoxin-like domain has been loosely

Fig 1 Domain architectures of human

disulfide isomerases (PDIs) Catalytic

thiore-doxin-like domains (a and a¢) are colored

pink, and non-catalytic domains (b and b¢)

are blue The first domain of PDILT, which

does not contain active site cysteines, is

hatched to indicate its strong similarity to

the a domains of other PDIs Yellow boxes

correspond to the linker between the b¢ and

a¢ domains (x-linker) The DnaJ domain of

ERdj5 and the C-terminal helical domain of

ERp29 are shown in green White boxes

indicate transmembrane domains The

sec-ond column lists available structures of

mammalian PDIs Among notable

non-mam-malian structures are two structures of

yeast PDI crystallized at different

tempera-tures (PDB accession numbers 2b5e and

3boa) and yeast Mpd1p (PDB accession

number 3ed3) The structure of full-length

ERp57 (PDB accession number 3f8u) was

determined in complex with tapasin.

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defined as anything with a predicted thioredoxin-like

structure (based on sequence) However, some authors

have used a more specific definition of a

thioredoxin-like domain as one capable of reacting with cysteines

[21] This definition appears to make the most sense,

as members of a disulfide isomerase family should be

capable of reacting with cysteine side chains However,

the founding member of this family, PDI, also exhibits

non-specific polypeptide-binding chaperone activity

[22] Thus, a more inclusive definition of PDI family

members would comprise proteins that contain

non-thiol-reactive thioredoxin-like domains with

chap-erone-like activities for ER folding and secretion of

proteins This includes ERp29 [23] and ERp27 (ER

protein 27 kDa) [24], two PDI family members that do

not contain thiol-reactive active sites The proteins

PDILT (protein disulfide isomerase-like protein of the

testis) and TMX2 (thioredoxin-related transmembrane

protein 2) have catalytic motifs, Ser-x-x-Cys, that lack

the N-terminal cysteine required for full

oxidoreduc-tase activity However, because they contain

C-termi-nal cysteines, and PDILT has been shown to form

mixed disulfides with partners and substrates in vivo

[25], we categorize these as thiol-reactive Their

contri-butions to oxidative protein folding in cells remain

unclear, and because of their inability to act as

oxido-reductases, their chaperone functions are probably

more important [26] Similar considerations apply to

ERp44, AGR2 (anterior gradient protein 2 homolog),

AGR3 (anterior gradient protein 3 homolog) and

TMX5 (thioredoxin-related transmembrane protein 5),

which contain Cys-x-x-Ser motifs that lack the C-terminal

cysteine

PDIs are a diverse family

PDI family members have functions as diverse as their

sequences and domain arrangements In Fig 1, we

dis-tinguish between thiol-reactive and non-reactive PDI

family members Most PDIs contain more than one

active site, and usually contain a combination of active

and inactive thioredoxin-like domains The inactive

domains perform functions such as substrate or

part-ner recruitment Importantly, although in vitro

activi-ties have been demonstrated for most PDIs, their

function in vivo is more difficult to determine, and may

be intrinsically more complex, involving other partners

or specific conditions For example, while PDI

gener-ally promotes protein folding, it can act as an

unfol-dase, favoring ER exit of cholera toxin [27] The

specific function of ERp57 is unclear, but its gene

knockout is embryo-lethal at day 13.5 [28] for reasons

that may relate to its modulation of STAT3 (signal

transducer and activator of transcription 3) signaling [29] A conditional B-cell knockout has adverse effects

on folding of glycosylated influenza virus hemaggluti-nin, but little effect on folding and secretion of the Semliki Forest virus coat spike proteins p62 and E1 ERp57 knockout did not change ER morphology or function drastically, and ER stress levels were not affected, suggesting more functional overlap between PDIs than previously appreciated Remarkably, treat-ment with castanospermine rescued the folding of viral hemagglutinin in ERp57) ⁄ )mouse fibroblasts [30], pre-sumably by preventing its entry into the calnexin cycle, and thereby allowing other disulfide isomerases to act

on it These results highlight the need for in vivo stud-ies to clarify the functions of the various PDIs, and the difficulty in assigning functions to PDIs based on their in vitro activities or structures alone With nota-ble exceptions such as ERp57, ERdj5 [31], and AGR2, which is involved in the production of intestinal mucin [32,33], use of knockouts to address the in vivo functions of mammalian PDIs has not been reported

Catalytic sites

In thioredoxin-like domains, the conserved catalytic Cys-x-x-Cys motif is found at the N-terminus of a long helix, a2 (Fig 2) Within the catalytic motif, the two cysteines play distinct roles The N-terminal cysteine forms a mixed heterodimer with a protein substrate, while the C-terminal cysteine is involved in substrate release [34] Several other residues in the catalytic site contribute to the reaction mechanism A conserved glutamate positioned below the C-terminal cysteine functions in proton transfer during substrate release [35] A neighboring arginine modulates the pKaof this catalytic cysteine by its placement in the active site [36,37] The recent determination of the structure of the ERp72 catalytic domains allowed a glimpse into the effects of local rearrangements of the N-terminal part of helix a2 on positioning of the conserved argi-nine residue [38] In the a0 domain, the arginine side chain is surface-accessible, but in the a domain, the equivalent arginine points towards the catalytic site and forms a salt bridge with the glutamate residue, Glu200, that is implicated in substrate release (Fig 2) This suggests that coordinated arginine–glutamate interactions may serve to modulate the catalytic activ-ity of protein disulfide isomerases

Substrate binding sites

One major area of interest has been how PDI family members recognize and bind substrate molecules In

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pioneering studies, the substrate binding site of

mammalian PDI was identified by cross-linking with

radiolabeled model hydrophobic peptides that mimic

unfolded proteins [6] The major binding site for

unfolded proteins was shown to be the b¢ domain, and

this was confirmed by structural studies that used

NMR chemical shifts to map the binding site of

unfolded RNase A and peptide ligands onto the

struc-ture of the bb¢ fragment of PDI [8,9] The binding site

consists of a large hydrophobic cavity between helices

a1 and a3, comprising Phe223, Ala228, Phe232, Ile284,

Phe287, Phe288, Leu303 and Met307 side chains

(Fig 3A) Determination of the crystal structure of the

b¢x domain of human PDI provided a more detailed

insight into substrate binding, as the x-linker is folded

back and mimics how hydrophobic stretches bind to

the b¢ domain [17] Specifically, the side chains of

Leu343 and Trp347, which are part of the linker

between the b¢ and a¢ domains, are inserted into the

hydrophobic cavity of the b¢ domain The binding sur-face appears to be conserved across species, as the same pocket is fully accessible and well positioned for protein substrate binding in the structure of yeast PDI [12] In human PDI, the b¢a¢ fragment is required for efficient binding of non-native protein substrates [6]

A recent study of Humicola insolens PDI also demon-strated that the a¢ domain assists in substrate binding,

as the b¢a¢ fragment shows extensive contacts with the hydrophobic peptide mastoparan [39]

ERp44 contains three thioredoxin-like domains, a, b and b¢, in addition to a C-terminal regulatory domain [10] There are obvious structural similarities between the b¢ domains of ERp44 and PDI, and most of the substrate binding PDI residues are conserved in ERp44 (Fig 3) The b¢ domain of ERp44 also has a hydrophobic pocket As observed with the PDI b¢x fragment, a hydrophobic stretch C-terminal to the b¢ domain of ERp44 folds back and binds to the a1–a3 cavity as a short helical segment using the side chains

of Phe358 and Leu361 (Fig 3B) The C-terminal tail also partly shields a hydrophobic patch of the a domain, and its removal increases the in vitro activities

of ERp44 as an oxidase, reductase, isomerase and chaperone [10] Strikingly, tail-less ERp44 formed mixed disulfides with endogenous proteins in several cell types [10], suggesting that the C-terminal cap of the substrate binding domains contributes to the speci-ficity of ERp44 How the action of the C-terminal tail

of ERp44 is regulated in cells is an intriguing question Structural studies of two other major PDIs, ERp57 and ERp72, showed that they do not contain hydro-phobic pockets [16,18] Structurally, ERp57 lacks a protein substrate binding site in its b¢ domain Instead, the a1–a3 surface is mostly negatively charged The corresponding surface of ERp72 is likewise polar and

is unable to bind hydrophobic peptides The residues Arg398 and Glu459 of ERp72 form a salt bridge to occlude a potential substrate binding cavity (Fig 3C) This interaction is stabilized by a hydrogen bond between Tyr416 and Glu459 These two positions are characteristic of b¢ domains of protein disulfide isome-rases that do not bind directly to hydrophobic stretches In ERp57, Gln256 forms a salt bridge with the corresponding Glu310 that is stabilized by a hydro-gen bond with Tyr264 (Fig 3D) The charged nature

of the a1–a3 surface explains the inability of ERp57 and ERp72 to bind model peptides in vitro [40,41]

In contrast, several other PDIs are predicted to bind

to substrates via hydrophobic pockets in their b¢ domains PDIp (pancreas-specific protein disulfide iso-merase, also known as PDIA2), PDILT and ERp27 have mostly identical or similar hydrophobic residues

A

B

Fig 2 Role of arginines in the ERp72 catalytic thioredoxin-like

domains in modulating disulfide isomerization activity (A) Phe97

and Pro138 of the a0 domain restrict access of Arg155 to the

Cys-x-x-Cys active site (B) A conformational change in the a

domain a2 helix allows Arg270 to enter the hydrophobic core and

form a salt bridge with the conserved buried residue Glu200.

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as PDI in their hydrophobic pockets (Fig 3E) PDIp

can bind the amphipathic peptide D-somatostatin [42],

as can ERp27 [24] The redox-inactive bb¢ domains of

PDIp exhibit chaperone activity in vitro and in vivo

[43] Binding of liver- and testis-specific PDILT to

D-somatostatin and unfolded bovine pancreatic trypsin

inhibitor has been demonstrated [26], and PDILT has

been shown to form mixed disulfides with substrates in

HeLa cells via its unusual Ser-x-x-Cys motif [25] The

lack of a hydrophobic substrate binding site in the b

and b¢ domains of ERp57 and ERp72 indicates that

these PDIs either require protein partners that assist in

substrate recognition, or that they directly interact

with substrates through the active sites of their

cata-lytic domains

ERp29 utilizes an alternative site for substrate

recog-nition The putative peptide binding site of its single

thioredoxin-like domain is located in the b2–a2 and

a3–b4 loop area of its N-terminal thioredoxin-like

domain [14] This is opposite to the a1–a3 surface used

by PDI ERp29 forms a tight dimer, and its b domain

is sufficient for peptide and substrate binding It binds

peptides with two or more aromatic residues, and favors peptides with basic character [14]

The structure of ERp18 revealed that this protein also adopts a thioredoxin-like fold and has a conserved Pro113 that results in an unusually bent a2 helix when ERp18 is in its oxidized form [44] This conserved pro-line might be important for ERp18 function, although

a specific requirement for ERp18 function has yet to

be determined ERp18 shows specificity for a compo-nent of the complement cascade, the pentraxin-related protein PTX3 [45], and has been implicated in the reduction of gonadotropin-releasing hormone [46] The recently determined ERp57–tapasin structure provided the first structural insights into protein bind-ing by the catalytic domains of a mammalian PDI [13]

In the structure, the a domain of full-length ERp57 is linked to tapasin by a disulfide bond Tapasin is a chaperone associated with editing the peptide cargo of the major histocompatibility complex class I ERp57 specificity for tapasin appears to be determined pri-marily by the catalytic domains [47] It has been sug-gested that the interdomain distance between the a and

C

E

D

Fig 3 A cavity on the a1–a3 surface of the b¢ domain defines the ability of the PDI to bind hydrophobic stretches of protein sub-strates The stretches after the b¢ domain of PDI (A) and ERp44 (B) interact with the hydrophobic surface in the corresponding crystal structures The residues that bind to the hydrophobic groove are labeled The cor-responding surfaces in ERp72 (C) and ERp57 (D) are occluded by polar interactions involving conserved glutamates that also form hydrogen bonds with tyrosine side chains Hydrogen bonds are shown as black dashed lines, and the residues involved are labeled (E) Structure-based sequence align-ment of the b¢ domains from human PDIs and rat ERp72 The glutamates that contrib-ute to the inability of the ERp57 and ERp72 b¢ domains to bind hydrophobic stretches are highlighted in pink, and their polar equiv-alents are shown in turquoise The residues potentially involved in substrate binding are highlighted in gray The alignment includes the N-terminal part of the x-linker that appears to be an integral part of the b¢ domain The consensus secondary structure

is shown above the alignment.

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a¢ domains makes ERp57 particularly suited for

bind-ing to tapasin; however, the mobility of the a and a¢

domains relative to the bb¢ base for each of these

pro-teins (discussed further below) suggests that the a to a¢

interdomain distance is not strictly maintained

(Fig 4A) The suggestion that the interaction is

stabi-lized by cumulative complementary protein–protein

interactions seems more likely, with the a and a¢

domains together providing an avidity effect that

secures binding to tapasin Although most ERp57

resi-dues within 4.5 A˚ of tapasin in the complex are

con-served in ERp57, ERp72 and PDI, three (K366, C92

and R448) are unique to ERp57 Further studies are

required to understand ERp57 specificity for tapasin,

but these results illustrate the importance of

PDI–sub-strate complementarity within catalytic domains

Protein partner binding sites

PDI is highly abundant in the ER, and is a member of

distinct protein complexes with specific functions PDI

is the b-subunit of the prolyl-4-hydroxylase complex

that is important for hydroxylating proline residues of

collagen [48], and also is a subunit of the microsomal

triglyceride transfer protein [49] Binding of PDI to the

prolyl-4-hydroxylase complex minimally requires intact

b¢ and a¢ domains of PDI, but the assembly and

activ-ity of the complex is further enhanced by the addition

of a and b domains [50] Mutagenesis of individual

residues in PDI confirmed the importance of the a and

a¢ domains in the assembly of an active complex but,

surprisingly, mutations in the hydrophobic

substrate-binding site in the b¢ domain had no effect [51], for

assembly of the prolyl-4-hydroxylase complex

Rather than directly binding to substrates, ERp57

requires a protein partner to assist in substrate protein

folding [41] The partner protein, either calnexin (CNX) or calreticulin (CRT), recruits glycoprotein substrates through a lectin domain NMR titrations and mutagenesis studies mapped CNX⁄ CRT binding

to a site centered on the N-terminal half of helix a2 of the b¢ domain of ERp57 [16] This area is abundant in positively charged residues and displays charge com-plementarity to the negatively charged tip of the CNX⁄ CRT P-domain, which is responsible for ERp57 binding [52] In particular, mutations R282A and K214A in ERp57 abrogate or greatly decrease CNX P-domain binding in vitro [16] and inhibit substrate interactions in vivo [31] This region of ERp57 has also been shown to mediate binding to the PDI ERp27 [24] ERp27 has a hydrophobic peptide binding site on its second thioredoxin-like domain and may recruit substrates to ERp57 ERp27 has no catalytic cysteines

of its own

A yeast homolog of CNX, Cne1p, interacts with the oxidoreductase Mpd1p Mpd1p and ERp57 present very different overall architectures Mpd1p contains only two domains: an N-terminal catalytic domain and

a C-terminal non-catalytic domain The recently deter-mined crystal structure of Mpd1p has a positively charged surface at the beginning of the second thiore-doxin-like domain that has been suggested to be a potential Cne1p binding site [15]

ERp72 also does not possess a hydrophobic sub-strate binding site in its b and b¢ domains Compared

to PDI and ERp57, ERp72 contains an additional cat-alytic a0 domain at its N-terminus The recently deter-mined high-resolution crystal structure of the bb¢ fragment of ERp72 reveals strong structural similarity

to ERp57 in terms of both the individual domains and relative domain orientation [18] The ERp72 surface corresponding to the CNX binding site of ERp57 is

a

a

b

b

Fig 4 Involvement of the catalytic domains in protein binding (A) Representation of the ERp57–tapasin structure showing that only the cat-alytic a and a¢ domains of ERp57 (green) interact with tapasin (turquoise) The ERp57 residues that interact with CNX are shown in blue [16] (B) Structural model of ERp72 based on structures of the a 0 a (pink), bb¢ (turquoise) and a¢ (yellow) domain fragments overlaid upon full-length ERp57 The catalytic cysteines and adjacent hydrophobic residues in ERp57 and ERp72 are shown in orange and gray, respectively.

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negatively charged, and consequently does not bind

the CNX P-domain, but shows high sequence

conser-vation among ERp72 proteins from various species,

suggesting functional importance

Given that ERp72 does not interact with CNX, and

lacks a hydrophobic substrate binding site, how might

it interact with substrates? Perhaps ERp72 relies on its

three catalytic domains for specificity toward

sub-strates or partners The relative contributions of each

of the catalytic Cys-x-x-Cys motifs of ERp72 to

reduc-tase activity on insulin in vitro suggest unequal

contri-butions to binding and catalysis [53] Cysteine-to-serine

mutations in the N-terminal a0 domain affect the kcat

for insulin reduction, but the Km is unaffected The

same mutations in the a domain have intermediate

effects on both kcatand Km, while loss of the catalytic

motif of the a¢ domain primarily affects Km[53] These

results were interpreted as indicating that the a0

domain is primarily involved in catalysis, the a domain

has intermediate roles in catalysis and binding, and the

a¢ domain functions primarily to bind substrates

How-ever, the unequal contributions to binding and

cataly-sis of the catalytic domains could also indicate that

other structural features are important for the

activi-ties These kinetic studies on ERp72 must be

inter-preted carefully, because PDI-catalyzed oxidoreductase

reactions do not necessarily follow simple Michaelis–

Menten rules Compared to the other abundant PDIs,

relatively few endogenous substrates were identified for

ERp72 in HT1080 human fibroblasts [45] However,

due to the use of Cys-x-x-Ala substrate-trapping

mutants that form mixed disulfides during reduction or

isomerization reactions, substrates oxidized by ERp72

may have been missed ERp72 may play a more

spe-cialized role in protein oxidation, or act on specific

substrates not readily detected by the methods used

Further work is required to understand

ERp72–sub-strate and ERp72–partner interactions

Recently, the transmembrane PDI TMX4 was

char-acterized and found to interact with ERp57 and

caln-exin [54] Unlike ERdj5, over-expression of TMX4

does not accelerate ER-associated decay of the NHK

(null-Hong Kong) variant of a1-antitrypsin

Interac-tion of TMX4 with ERp57 was dependent on its

cata-lytic active site, suggesting that it may reduce ERp57

in the ER On the other hand, the interaction of

TMX4 with CNX did not require an intact

Cys-x-x-Cys motif Further work is necessary to determine how

TMX4 might interact with substrates, although

sub-strate recruitment by CNX in a fashion analogous to

the ERp57-CNX complex is a possibility While

TMX4 lacks a b¢-like domain that can interact with

the P-domain of CNX, other mechanisms are possible

TMX4 does not isomerize scrambled RNase A in vitro, suggesting that it requires a co-factor⁄ partner for sub-strate recruitment

Interdomain mobility

The question of interdomain flexibility is relevant for PDIs that comprise multiple thioredoxin-like domains Recent crystallographic and small-angle X-ray scatter-ing (SAXS) studies provide support for interdomain mobility This may be the main reason why PDI was resistant to crystallization efforts for a long time Only two-four-domain PDIs (ERp57 and yeast PDI) have been crystallized, and both represent partner- or pseudo-substrate-bound structures ERp57 was crystal-lized as a heterodimer with tapasin, while yeast PDI was crystallized with another yeast PDI molecule mim-icking a bound substrate Crystallization of the three-domain ERp44 structure was potentially favored by reduced mobility due to binding of the protein C-ter-minus to the thioredoxin-like domains Apart from these examples, only the two-domain proteins ERp29 and Mpd1p, and protein fragments of ERp57 and ERp72 (bb¢ for ERp57 and a0

a and bb¢ for ERp72) have been crystallized The best strategy for crystalliz-ing PDIs with domains connected by flexible linkers appears to be immobilization of their domains via sub-strate or partner binding, or focusing on smaller frag-ments instead of the whole protein

The clearest evidence of interdomain flexibility comes from the two crystal structures of yeast PDI (Fig 5A) [12,55] When the structures are superposed, the catalytic domains clearly adopt different positions The ability of human PDI to adopt open and closed conformations was demonstrated by sedimentation equilibrium and SAXS experiments [56,57] Although only one crystal form of ERp57 is available, compari-son of a SAXS reconstruction of the protein free in solution with the ERp57⁄ tapasin crystal structure sug-gests mobility of the catalytic a and a¢ domains [13,16] Among PDIs with x-linkers, mobility between the b¢ and a¢ domains is likely to be much more pronounced than between the a and b domains, which have a short interdomain linker A recent study of domain mobility

in human PDI showed that the sites of greatest prote-ase sensitivity are located between the b¢ and a¢ domains [58]

Another striking example of domain flexibility was provided by recent SAXS, crystallographic and NMR studies of ERp72 [18,38] The crystal structures of the

a0a and bb¢ fragments allow modeling of the full-length protein by overlaying the a and bb¢ domains onto the ERp57 structure (Fig 4B) Interestingly, this generates

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a potential substrate binding site comprising the

cata-lytic a0, a and a¢ domains The ERp72 model presents

one possible orientation of the domains, but does not

illustrate the full range of possible interdomain

confor-mations SAXS measurements of full-length ERp72

revealed multiple relative orientations of the domains,

with the greatest mobility between the a0 and a

domains and the b¢ and a¢ domains [18] Limited

prote-olysis revealed that the a¢ domain is cleaved most

rapidly from ERp72, probably due to flexibility of the

x-linker (P.M., unpublished data) The recently

deter-mined a0

a structure illustrates the difficulty in drawing

conclusions about interdomain mobility based on

crys-tal structures alone [38] Although the a0

a crystal

structure suggests a single conformation, NMR chemi-cal shifts in the a0a fragment and a0 domains suggest that the two domains do not form a rigid pair in solu-tion (G.K., unpublished data)

The bb¢ domains form a rigid spacer between catalytic domains

Although future studies will better define the structural diversity of PDIs, an obvious feature is the limited mobility of the bb¢ fragment in the four- and five-domain PDIs A structural overlay of this region from human PDI, ERp57, ERp72 and yeast PDI showed striking similarity in the domain orientations (Fig 5)

As there are two crystal structures available for yeast PDI (at 4C and room temperature) and for ERp57 (full-length and the bb¢ fragment), more conclusive comparisons can be made concerning the rigidity of these bb¢ pairs The bb¢ domains of the two yeast PDI structures superpose with an rmsd of 1.7 A˚ over 210

Ca atoms Much greater variability is observed in the positions of the catalytic a and a¢ domains (Fig 5A) The bb¢ domain orientation is also similar in human and yeast PDI (Fig 5B) Likewise, overlay of the bb¢ structures from ERp57 and ERp72 results in an rmsd

of 1.7 A˚ for backbone atoms (Fig 5C) The difference results from a small (10) rotation at the interdomain interface A similar comparison between the two full-length ERp57 molecules in the crystal structure with tapasin shows the bb¢ domains overlay with an rmsd

of 1.1 A˚ [13] These examples indicate that the bb¢ tan-dem forms a relatively rigid base, providing a spacer for the attachment of more mobile active site domains that can access substrates from opposite sides simulta-neously The bb¢ domains may also jointly contribute

to functional substrate or protein binding sites As one example, the CNX binding site of ERp57 includes a contribution from Lys214 in the b domain in addition

to the residues in the b¢ domain, which form the majority of the binding site [16] In contrast to the bb¢ base, the catalytic domains in the available structures show a much larger degree of mobility, which may be important for recognition of protein substrates of vari-able sizes as well as adjustment to conformational changes in substrate during folding and disulfide rear-rangement In the proteins most closely related to PDI, the a–b linker is generally very short, while the b¢–a¢ linker (x-linker) is significantly longer It is very likely that other PDI-like proteins such as PDIp and PDILT will display a similar structural arrangement of their domains

The recently determined structure of yeast Mpd1p shows a very different orientation of its two

thioredox-A

Fig 5 The non-catalytic bb¢ fragment provides a relatively rigid

base in PDIs containing four or five thioredoxin-like domains, while

allowing greater mobility of the catalytic domains (A) An overlay of

the yeast PDI structures crystallized at 4 C (pink) and room

tem-perature (yellow) shows high similarity in the orientation of the bb¢

domains and significant differences in orientation of their a and a¢

domains The catalytic cysteines (orange) of the 4 C yeast PDI

structure face each other (B) The b and b¢ domains (gray) of

human PDI are oriented similarly to those from yeast PDI

crystal-lized at room temperature (pink) (C) An overlay of the bb¢

struc-tures of ERp57 (green) and ERp72 (turquoise) shows a very similar

domain orientation (D) An overlay of the bb¢ structures of ERp57

(green) and ERp44 (brown) shows roughly similar domain

orienta-tion (E) Representation of the Mpd1p structure with the N-terminal

a domain shown in similar orientation to the b domains in (A)–(D).

The structure, colored blue to red from the N- to C-terminus,

reveals the C-terminal helix contacts the a domain.

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in-like domains, which are locked in a rigid orientation

by numerous interdomain contacts (Fig 5E) [15] This

led to suggestions that other PDIs cannot be reliably

modeled using yeast PDI and ERp57 structures

Nev-ertheless, there are several reasons why this argument

may not apply to most mammalian PDIs Mpd1p does

not have a close structural homolog in the mammalian

ER Only its N-terminal catalytic domain has

signifi-cant sequence homology to human PDIs P5 (39%

identity) and ERdj5 (34% identity), while the

C-termi-nal non-catalytic domain has no detectable sequence

similarity to human proteins In contrast, mammalian

PDIs have significant (30–40%) sequence identity

(Table 1) that translates into structural similarity

Although the non-catalytic domains have low sequence

similarity, there is strong sequence similarity between

the human PDI bb¢ domains (residues 136–354) and

PDIp (39% identity), ERp27 (32%), PDILT (27%)

and ERp72 (24%) The sequence identity of 28%

between the bb¢ domains of ERp57 and ERp72 results

in a strikingly similar domain orientation, and the bb¢

domains of PDI, ERp57 and ERp72 adopt similar

rel-atively rigid conformations Based on this, the

non-cat-alytic domains of ERp27 are expected to adopt a very

similar structure Although the bb¢ domains of ERp44

show a somewhat different interdomain angle, the

ori-entation is still largely similar to other known

struc-tures of mammalian PDIs (Fig 5D), and could reflect

the effect of the protein C-terminus

In contrast, the a–b interfaces of PDIs are more

dif-ficult to compare to one another Indeed, even within

the same protein, the a–b or b¢–a¢ interfaces assume

different orientations (Fig 5A) As Mpd1p has only

two domains, it may adopt a unique orientation to

provide the catalytic, substrate binding and partner

binding sites afforded by the four domains in ERp57

These observations suggest that Mpd1p is a structural

outlier when compared to mammalian PDIs

Table 1 Sequence identity (%) between human protein disulfide

isomerases.

Domains PDI PDIp PDILT ERp57 ERp72

PDI (residues

26–471)

PDIp (residues

44–492)

PDILT (residues

45–490)

ERp57 (residues

27–478)

ERp72 (residues

179–632)

A

Fig 6 Structural organization of the x-linker (A) Representation of the full-length ERp57 structure with the region recognized as the x-linker colored in red (B) The N-terminal part of the x-linker folds against the b¢ domain in the structures of the ERp57 bb¢ fragment (blue), full-length ERp57 with tapasin (green) and the ERp72 bb¢ fragment (turquoise) The side chain of Leu361 in the ERp57 x-lin-ker inserts into a cavity formed by three aromatic side chains This interaction is also observed in ERp72, involving residues Leu508 of the x-linker and residues Phe484, Phe499 and Phe503 of the b¢ domain Towards the middle of the x-linker, Tyr364 of ERp57 (and Val511 of ERp72) fit into a small hydrophobic pocket below the C-terminus of helix a3 of the b¢ domain For clarity, ERp72 residues are not labeled and side chains of the ERp57–tapasin structure are not shown (C) The structures of the b¢ domains of human PDI (gray) and the two crystal forms of yeast PDI (yellow and pink) show similar interactions Ile334 of the x-linker is inserted into the pocket formed by the three aromatic side chains Phe325, Phe329 and Tyr310 of human PDI (gray) In yeast PDI, the corresponding residues are Ala361 from the x-linker and Tyr325, Leu352 and Phe356 of the b¢ domain (D) An overlay of the a¢ domain from full-length ERp57 (green) and the isolated domain (brown) of ERp57 shows a similar structure for the C-terminal part of the x-linker between residues L365 and P377 The N-terminal part of the x-lin-ker is disordered in the NMR structure of the isolated a¢ domain Only one of many possible conformations is shown (E) An overlay

of the a¢ domains of full-length yeast PDI crystallized in various crystal forms at 4 C (yellow) and room temperature (pink) shows very similar conformations of the C-terminal region of the x-linker between Ser367 and Ser377.

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The x-linker consists of distinct

structural regions

The x-linker is a conserved stretch of approximately 20

residues connecting the b¢ and a¢ domains [5] Recent

progress in the structural characterization of a number

of PDIs has provided new insights into the structural

role of this region The structures of the full-length

proteins ERp57 and yeast PDI reveal that the linker

consists of two distinct regions (Fig 6A) [12,13] The

N-terminal part (approximately seven residues) folds

onto the b¢ domain and runs perpendicular to the

strand b5 The interactions are mostly mediated by the

hydrophobic residues (Leu361 and Tyr364 in ERp57)

that contact the hydrophobic surface at the edge of the

b sheet (Fig 6B) In ERp57, the side chain of Leu361

occupies a hydrophobic pocket formed by the aromatic

rings of Phe336, Phe352 and Tyr356 (Fig 6B) These

interactions are conserved in the structures of ERp72

and PDI, suggesting that the N-terminal region of the

linker is an integral part of the b¢ domain (Fig 6B,C)

In agreement with this conclusion, removal of the

x-linker decreases the midpoint for denaturation of the

PDI b¢ domain from 2.32 m guanidinium chloride to

1.65 m [7]

In contrast, the C-terminal portion of the x-linker

takes on strikingly different conformations in the

structures of the isolated b¢ domain and full-length

proteins In the crystal structure of the b¢x fragment of

human PDI, the C-terminal part of the x-linker turns

back to contact the b¢ hydrophobic surface between

helices a1 and a3 [17] In the full-length proteins

ERp57 (Fig 6D) and yeast PDI (Fig 6E), the

C-termi-nal half of the x-linker is structurally associated with

the a¢ domain, and displays an irregular conformation

without conserved salt bridges or hydrophobic

interac-tions with the a¢ domain Overlay of structures from

full-length ERp57 and the isolated ERp57 a¢ domain

(PDB accession numbers 3f8u and 2dmm) shows that

the linker structure is preserved even in the absence of

the preceding domain (Fig 6D) At least three NMR

solution structures of a¢ domains from human PDI

(PDB accession number 1x5c), human ERp72 (PDB

accession number 2dj3) and H insolens PDI (PDB

accession number 2djj) have been obtained without

residues comprising the x-linker The a¢ domain from

rat ERp72 is also well-folded on its own [38] This

sup-ports the idea that the C-terminal part of the x-linker

loosely interacts with the a¢ domain and does not

con-tribute to its structural integrity As discussed

previ-ously, studies with both PDI and ERp72 have shown

significant interdomain mobility at the b¢a¢ domain

interface

N- and C-terminal extensions

A number of PDIs contain N- or C-terminal tails out-side the thioredoxin-like domains (Fig 1) The C-ter-minal tail of yeast PDI forms a protruding a helix [12] The C-terminus of ERp44 forms short helical turns while folding back and interacting with the b¢ and a domains [10] Likewise, the C-terminus of Mpd1p binds to the N-terminal domain as an a helix [15] Despite these examples, the tails of many mammalian PDIs are unlikely to be structured due to their low sequence complexity and highly charged nature In particular, the C-terminal tail of PDI and N-terminal tail of ERp72 are very acidic, while the C-terminus of ERp57 is positively charged NMR spectra suggest that the above-mentioned acidic stretches are unstruc-tured in solution (unpublished data) They could become more structured during interaction with ligands or protein partners These acidic extensions have been previously implicated in calcium binding, and recent circular dichroism measurements showed that a similar extension of the ER luminal chaperone CRT becomes structured upon binding calcium [59] Although not generally critical for the disulfide isomer-ase activity, the N- and C-termini of PDIs may also be important for mediating protein–protein interactions with other ER chaperones [60]

Concluding remarks

The mammalian PDI family currently consists of 20 proteins with diverse functions in oxidative folding of protein substrates in the ER As the availability

of structures for the PDI family grows, the functions

of its members are becoming clearer Most PDIs con-sist of multiple thioredoxin-like domains with a similar organization: central non-catalytic domains that often form a rigid scaffold for binding substrate or partner chaperones, surrounded by more mobile catalytic domains with active site cysteines The mobility of cat-alytic domains may be beneficial when acting upon incorrectly disulfide-bonded proteins and substrates of various sizes This model of PDI function is consistent with sequence analysis showing that the non-catalytic domains have the greatest sequence diversity while the catalytic domains are more highly conserved, especially

in regions flanking the Cys-x-x-Cys catalytic sites

It is currently unclear why most PDIs have multiple catalytic domains One possibility is that these domains are involved in oxidative folding of complex substrates with many disulfide bonds Another possi-bility is that multiple catalytic domains enhance the avidity for substrates, or provide modules that

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