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The retroviral RNA genome encodes for three enzymes essential for viral replication: HIV-1 protease PR, HIV-1 reverse transcrip-tase RT and HIV-1 integrase IN.. Peptide-based inhibitors

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R E V I E W A R T I C L E

Modulation of the oligomeric structures of HIV-1 retroviral enzymes

by synthetic peptides and small molecules

Nicolas Sluis-Cremer1and Gilda Tachedjian2

1

Department of Medicine, Division of Infectious Diseases, University of Pittsburgh, PA, USA;2AIDS Molecular Biology Unit, Macfarlane Burnet Institute for Medical Research and Public Health, Melbourne, Victoria, Australia

The efficacy of antiretroviral agents approved for the

treat-ment of HIV-1 infection is limited by the virus’s ability to

develop resistance As such there is an urgent need for new

ways of thinking about anti-HIV drug development, and

accordingly novel viral and cellular targets critical to HIV-1

replication need to be explored and exploited The retroviral

RNA genome encodes for three enzymes essential for viral

replication: HIV-1 protease (PR), HIV-1 reverse

transcrip-tase (RT) and HIV-1 integrase (IN) The enzymatic

func-tioning of each of these enzymes is entirely dependent on

their oligomeric structures, suggesting that inhibition of

subunit-subunit assembly or modulation of their quaternary structures provide alternative targets for HIV-1 inhibition This review discusses the recent advances in the design and/or identification of synthetic peptides and small mole-cules that specifically target the subunit–subunit interfaces of these retroviral enzymes, resulting in the inactivation of their enzymatic functioning

Keywords: protease; reverse transcriptase; integrase; oligo-meric structure; inhibiting protein–protein interactions

In 1983 HIV was identified as the etiologic agent of AIDS

[1,2] During the past 18 years a tremendous effort has been

placed in the identification and/or development of

com-pounds that effectively attenuate HIV-1 infection To date,

16 anti-HIV agents have been approved by the United

States FDA for administration to HIV-1 infected

individ-uals These antiviral agents target the active sites of two

retroviral enzymes, protease (PR) and reverse transcriptase

(RT), and can be further divided into three different

therapeutic classes; PR active site inhibitors, nucleoside (and

nucleotide) reverse transcriptase inhibitors (NRTI) and

non-nucleoside reverse transcriptase inhibitors (NNRTI)

However, due to the long-lived nature of the HIV-1

infection as well as the genetic plasticity inherent to the

virus, emergence of viral resistance to these antiretroviral

agents is inevitable Furthermore, as many of the

com-pounds from the same therapeutic class exhibit similar chemical structures and mechanisms of action, the emer-gence of viral resistance to one drug frequently results in cross-resistance to other compounds Thus, the identifica-tion of addiidentifica-tional viral targets and the development of new classes of antiviral compounds are essential in the fight against HIV/AIDS In this regard, many promising com-pounds have been identified that target different steps in the HIV-1 viral life cycle including viral entry and fusion, proviral DNA integration as well as viral assembly (for reviews see [3,4])

Physical interactions between proteins play a critical role in many biological processes including signal trans-duction, cell cycle and gene regulation, and viral assembly and replication [5–7] Furthermore, many protein–protein interactions provide therapeutically worthwhile targets In this regard, inhibitors of protein– protein interactions have been successfully developed that target, amongst others, the interface of the large and small subunits of herpes simplex virus ribonucleotide reductase [8], cytokines (IL-2/IL-2Ra) [9], and growth hormone/receptor binding [10] The three enzymes of HIV (PR, RT and integrase (IN)) are all oligomeric proteins (Fig 1) The enzymatic functioning of each of these enzymes is entirely dependent on their quaternary structure [11–13] Therefore, inhibition of retroviral enzyme protein-protein assembly, or drug-mediated modulation of retroviral enzyme oligomers, provide alternative targets for HIV-1 inhibition

The objective of this review is to describe the unique structural features of the HIV-1 oligomeric enzymes PR, RT and IN and the strategies that have been developed to inhibit enzyme function by modulation of the interfaces between the subunits of the enzymes Each viral enzyme will

be dealt with individually

Correspondence to N Sluis-Cremer, Department of Medicine,

Division of Infectious Diseases, University of Pittsburgh, S808 Scaife

Hall, 3550 Terrace Street, Pittsburgh 15261, PA, USA.,

Fax: + 412 6489653, Tel.: + 412 3838525,

E-mail: CremerN@msx.dept-med.pitt.edu

Abbreviations: IN, integrase; NNRTI, non-nucleoside reverse

transcriptase inhibitor; PR, protease; RNase H, ribonuclease H; RT,

reverse transcriptase; TSAOe 3

T, 1-{spiro[4¢-amino-2¢,2¢-dioxo-1¢,2¢-oxathiole-5¢,3¢-[2¢,5¢-bis-O-(tert-butyldimethylsilyl)-b- D

-ribofurano-syl]]}-3-ethylthymine.

Enzymes: HIV-1 integrase (EC 2.7.7.49); HIV-1 protease

(EC 3.4.23.16); HIV-1 reverse transcriptase (EC 2.7.7.49); HIV-1

ribonuclease H(EC 3.1.26.4, SWISS-PROT entry name:

POL_HV1B1, Pol polyprotein of HIV-1 (BH10 isolate)).

(Received 22 May 2002, revised 28 June 2002,

accepted 29 August 2002)

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H I V - 1 P R O T E A S E

Structure and function of HIV-1 PR

HIV-1 PR catalyzes the hydrolysis of specific peptide bonds

within the HIV-1 Gag and Gag-Pol polyproteins to generate

the various structural and functional proteins essential for

viral replication HIV-1 PR is a symmetrically arranged

homodimeric protein composed of two chemically identical

subunits of 99 amino acids (Fig 1) The PR subunit fold

consists of a compact structure of b strands with a short

a helix near the C-terminus [14] The antiparallel b strands

constituted by residues 44–57 from both subunits, form a

flexible flap region that is thought to fold down over the

active site during catalysis to both bind substrate and

exclude water Protein–protein interactions in the dimer

include interactions between the catalytic triad residues

(D25-G27), I50 and G51 at the tip of the flaps, and the

antiparallel b sheet formed by the four termini in the dimer

(residues 1–5 and 95–99) Additional interactions include a

complex salt bridge between D29 and R87 of one subunit

and R8 of the other subunit Thermodynamic analyses of

the dimeric PR molecule indicates a Gibbs energy of dimer

stabilization of 10 kcal/mol at 25C (pH3.4), consistent

with a dissociation constant of 5· 10)8M [15]

Interest-ingly, the Gibbs free energy of dimerization is not uniformly

distributed along the protein–protein interface [15] Instead,

the interface is characterized by the presence of clusters of

residues (hot spots) that significantly contribute to subunit

association, and other regions that contribute very little In

particular, the four-stranded b sheet formed by the

amino-acid residues at the N- and C-termini of PR contribute close

to 75% of the total Gibbs energy [15] The importance of

this four-stranded b sheet is further emphasized by the fact

that all PR dimerization inhibitors developed by rational

(structure-assisted) design target this region (discussed below)

Peptide-based inhibitors of PR dimerization Short synthetic peptides corresponding to the amino-acid sequences of the N- and C-termini of HIV-1 PR have been shown to inhibit proteolytic activity by binding to the inactive PR subunits and preventing their association into active dimer [16–19] Peptides corresponding to the C-terminal segment of the HIV-1 matrix protein have also been found to elicit the same effect [20] However, the concentration of these peptides (both PR- and matrix-derived) required to effectively inhibit the PR monomer-dimer equilibrium by 50% (IC50) is relatively high (30–100 mM, Table 1) Schramm et al demonstrated that

it was possible to significantly improve their inhibitory properties ( 50–200 fold) through modification of their amino-acid composition and the addition of a hydrophobic moiety, such aminocaproyl or palmitoyl, to the N-terminus

of the peptide [20] The development of these more potent peptide inhibitors firmly established that PR dimerization was a rational target for the development of AIDS therapeutics, and that small-size peptide mimetics exhibiting good bio-availability could be derived for HIV-1 therapy In this regard, N-terminally palmitoyl-blocked peptides con-sisting of only three residues, one of which is a non-natural amino acid, have been developed and shown to exhibit good potency against PR dimerization [21]

Cross-linking of the N- and C-terminal peptides to form a mimic of the HIV)1 PR dimerization interface has provided an alternative strategy for the development of more potent PR dimerization inhibitors The principle of this strategy is illustrated in Fig 2 This approach was initially adopted by Babe et al., who cross-linked the N- and C-terminal PR-derived peptides by a 3.5-A˚ tether composed of three glycine residues, however, the resulting compounds were not potent inhibitors of PR dimerization [18] In the crystal structures of HIV-1 PR, the polypep-tide termini are held at a distance of approximately 10 A˚ (see Fig 2) Accordingly, more potent cross-linked inter-facial peptide compounds have been developed using tethers that bridge this gap [22–24] For example, Zutshi

et al used flexible alkyl-tethers to link the peptide strands [22], while Bouras et al took advantage of a

pyridinediol-or naphthalenediol-based scaffold [23] The supposed advantage of the aromatic conformationally constrained scaffold is that it may allow the two peptide strands to be initially more suitably oriented to permit formation of the antiparallel b sheet with one PR monomer [23] However, very little difference in relative potency is observed between the different tethers (Table 1) Irreversible inhibi-tion of PR dimerizainhibi-tion has also been achieved by designing a cross-linked interfacial peptide molecule that can form a disulfide bond with C95 in HIV-1 PR [25] Other novel strategies involve tethering an active-site peptide inhibitor with the dimerization inhibiting C-terminus peptide, thereby generating a compound that exhibits synergistic inhibition of PR activity [26]

The peptide inhibitors described above were all developed using peptide sequences corresponding to the N- and C-termini of PR, which themselves had been initially tested following the observation of their essential role in linking the

Fig 1 Oligomeric structures of HIV-1 PR (1A3O.pdb), RT

(1HMV.pdb)and IN (1EX4.pdb) The two subunits for each retroviral

enzyme are depicted in magenta and cyan, respectively Residues

contributing to the protein–protein interface are illustrated using a

surface representation.

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two PR subunits through the formation of the four-stranded

b sheet [14] Recently, an elegant strategy for the genetic

selection of dissociative peptide-based inhibitors of HIV-1

PR (and virtually all other designated protein–protein

interactions) has been reported [27] Briefly, this strategy

takes advantage of k-bacteriophage repressor protein (cI)

that binds to its operator (kPR) as a homodimer The

C-terminal dimerization domain of cI can be replaced by

another protein that homodimerizes, in this instance an

inactive variant of PR was used When bacteria are

transformed with a reporter plasmid (that contains the

selection module kPR-lacZ-tet and directs the production of

the cI-PR fusion protein), cI-PR represses the transcription

of the reporter genes and the transformants show a

LacZ(negative)-Tet(sensitive)phenotype [27]

Co-transfor-mation of the reporter plasmid with a peptide plasmid

library allows for the selection of peptides that prevent

NcI-PR dimerization and generate transformants exhibiting

a LacZ(positive)Tet(resistant) phenotype [27] The power

and utility of this technique was demonstrated by the

selection of approximately 300 peptides from 3· 108 cotransformants that exhibited a positive phenotype, rep-resenting a selection frequency of 1 in 106 Further analyses

of the selected peptides identified the peptide IVQVDAEGG

as an inhibitor of PR dimerization, which when tethered in a head-to-head or a tail-to-tail fashion generated a relatively potent inhibitor of PR dimerization (Table 1)

Non-peptide based inhibitors of PR dimerization

To date, two structurally unrelated classes of small-organic molecules have been identified which inhibit PR dimeriza-tion [28,29] The first class of molecules, which exhibit a polycyclic triterpene structure, were identified following a search of the Cambridge Structural Database (www.ccdc.cam.ac.uk) for pharmacophores that could bridge the 10 A˚ gap between the termini of a PR subunit [28] Extensive kinetic analysis of one of these triterpenes, ursolic acid, demonstrated that these compounds inhibited

PR dimerization with relatively high potency (K ¼ 3.4 lM)

Table 1 Peptide and small molecule inhibitors of HIV-1 PR dimerization.

IC 50 (lM) Method of analysis of PR dimerization Ref Peptides Derived from the

N-and C- Termini of HIV-1 PR N-and MA

Modified Peptides Derived from the

C-terminus of HIV-1 PR

Palmitoyl-Tyr-Glu-(p-biphenyl-alanine)-OH0.025 Kinetic analysis [21] Cross-linked Interfacial Peptides

2.0 Gel-filtration; protein cross-linking

PR fluorescence

[22]

Other Peptides

Kinetic analysis; gel-filtration Above peptide cross-linked using

Non-Peptide Based Inhibitors

a

Kinetic analyses were carried out according to the method described by Zhang et al (1991) [16].

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The second class of molecules that inhibited PR

dimeriza-tion include pentaester derivatives of of didemnaketal A

[29] The identification of these classes of small molecules is

significant, as in general many empirical searches for low

molecular mass pharmacological inhibitors (<400) of

protein–protein interactions have routinely failed

H I V - 1 R E V E R S E T R A N S C R I P T A S E

Structure and function of HIV-1 RT

HIV-1 RT is required for conversion of the viral genomic

RNA into a double-stranded proviral DNA precursor This

process is catalyzed by the RNA- and DNA-dependent

polymerase and ribonuclease H(RNase H) activities of the

enzyme in a reverse transcription complex in the cell

cytoplasm [30] HIV-1 RT is an asymmetric heterodimer

composed of a 560-residue 66 kDa subunit (p66)

compri-sing two domains termed DNA polymerase and RNase H,

and a p66-derived 440-residue 51 kDa subunit (p51) The

p51 subunit is produced during viral assembly and

matur-ation via HIV-1 protease-mediated cleavage of the

C-terminal (RNase H) domain of a p66 subunit [31] A

fascinating feature of the HIV-1 RT heterodimer is the

structural asymmetry which exists between the p66 and p51

subunits despite the fact that they are products of the same

gene and exhibit identical amino-acid sequences for the first

440 residues [32–40]

The overall shape of the p66 subunit has been likened to

that of a right-hand [35] The major subdomains of the

polymerase domain of p66 are termed fingers (residues

1–85, 118–155), palm (86–117, 156–237) and thumb (238–

318) The DNA polymerase catalytic aspartate residues

(D110, D185, and D186) reside in the palm subdomain A

fourth subdomain, termed the connection subdomain

(residues 319–426), acts as a tether between the DNA

polymerase and C-terminal RNase H(427–565) domains

The p51 subunit contains the same fingers, thumb, palm and connection subdomains, however, their spatial arrangement differs markedly to those of the p66 subunit [35]

Upon formation of the RT heterodimer from the p66 and p51 monomers, large surface areas of the individual subunits become inaccessible to water [33,41] Approxi-mately 4800 A˚2of protein surface is buried in the RT dimer complex of which  3050A˚2 corresponds to nonpolar atoms Amino-acid residues in the p66 subunit that form part of the dimer interface are derived primarily from the palm, connection and RNase Hdomain, while in the p51 they arise from the fingers, thumb and connection domains Dissection of the contributions of each individual residue to the total buried surface area upon dimerization reveals eight stretches of residues that make the largest contribution to total binding strength These include residues D86-L92, Q373-G384, W406-W410 and P537-G546 in p66 subunit, and P52-P55, I135-P140, C280-T290 and P392-W401 in the p51 subunit [41,42] Three clearly visible clusters are formed between these interfacial residues [33] A single region in the palm domain of p66 (D86-L92) interacts with two regions of the fingers of p51 (P52-P55 and S135-P140) The RNase H residues 537–546 in the p66 subunit interact with the p51 thumb residues 280–290, and the p66 connection residues W406-W410 interact with residues in the p51 connection domain residues (P392-W401) Evident from these clusters, the two subunits are completely asymmetric with respect to one another in that the subunit interface on p51 involves different amino acids than the p66 [33] Contacts between the connection subdomains form the only interactions between equivalent subdomains from each subunit How-ever, even in this case, many equivalent residues make different protein–protein interactions in such a way that the contacts between the two connection subdomains are also intrinsically asymmetric Thermodynamic evaluations of the association between the p66 and p51 subunits of RT have estimated a Gibbs free energy of dimer stabilization of approximately 10–12 kcalÆmol)1, corresponding to a disso-ciation constant of approximately 10 nM[43,44]

Peptide-based inhibitors of RT dimerization

As described above, one of the three clusters of residues formed between the RT p66 and p51 dimer interface are formed through the interactions between the RT p66 connection residues W406-W410 and residues P392-W401

in the p51 connection domain Interestingly, a 19 amino-acid synthetic peptide corresponding to residues 389–407 of the connection domain (N-FKLPIQKETWETWWTEYWQ-C)

of RT was demonstrated to be relatively efficient in retarding the heterodimerization process of HIV-1 RT [45] Further studies indicated that the same peptide was as efficient at inhibiting the heterodimerization process of HIV-2 RT as HIV-1 RT [46] This result is not surprising given that this region in the connection domain is conserved in 1,

HIV-2 and the simian immunodeficiency virus RTs [46] (the corresponding amino-acid sequence in HIV-2 RT is N-FHLPVERDTWEQWWDNYWQ-C) More recently, the length of the peptide was optimized to generate a shorter (10 residue) peptide (corresponding to residues 395–404 of RT) that was synthesized with an acetylated N-terminus and

a cysteamide group at the C-terminus to improve stability and cellular uptake [47] The resulting peptide was a more

Fig 2 Schematic representation of the strategy used to inhibit PR

dimerization by cross-linked interfacial peptides The N-terminus of PR

is indicated (N) The four-stranded b sheet formed by amino-acid

residues at the N- and C-termini of PR is a major binding determinant

in the formation of dimeric PR Cross-linked interfacial peptides

containing a tether region of approximately 10 A˚ inhibit PR

dimeri-zation by permitting the formation of a pseudo antiparallel b sheet

with one of the PR subunits.

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efficient inhibitor of RT dimerization in vitro and was also

shown to inhibit HIV-1 replication in cell culture [47] The

antiviral activity of the peptide was further enhanced by

conjugation to a peptidyl carrier without adverse toxic effects

to cells [47] Remarkably, the concentration of

peptide-carrier complex required to inhibit HIV-1 replication was

significantly less than the peptide concentration required for

the inhibition of RT dimerization in vitro For example,

0.1 nM of peptide-carrier completely suppressed HIV-1

replication for 15 days, whereas a peptide concentration of

240 mMwas required to inhibit RT heterodimerization by

50% in vitro [47] This may suggest that the mechanism of

inhibition of subunit association in vitro is different from the

process in HIV-1 infected cells In HIV-infected cells the RT

polypeptides are translated as part of the Gag–Pol

polypro-tein which is subsequently cleaved by HIV-1 PR to release

the various structural and functional proteins Recent studies

have shown that HIV-1 PR cleaves the Pol region of Gag–

Pol in a sequential manner in which the RT p66 polypeptide

is initially released from the polyprotein precursor Cleavage

of the p66 subunit to generate RT p51 appears to require a

p66/p66 homodimeric intermediate (D Arion, N

Sluis-Cremer & M.A Parniak, unpublished results) The

dissocia-tion constant for p66 homodimerizadissocia-tion is 10)6M, a value

approximately 1000-fold weaker than the interaction

be-tween RT p66 and p51 [43,44] Thus, one could anticipate

that the 10-residue peptide should be a more potent inhibitor

of p66 homodimerization Furthermore, it is interesting to

consider that modulation of RT dimerization may also affect

the interaction between two Gag–Pol molecules that must

dimerize to activate HIV-1 PR [48] Any affects on this

interaction may adversely affect PR activity [49] Hence, the

in vitro study of the effect of peptides on p66 and p51

dimerization does not necessarily reflect the process that is

occurring in HIV-infected cells and may not accurately

predict their impact on HIV-1 replication in cell based assays

Synthetic peptides that inhibit conformational changes

during HIV-1 RT heterodimerization

In vitroformation of active heterodimeric p66/p51 HIV-1

RT from the p66 and p51 monomeric subunits occurs in a

two step process involving an initial bimolecular association

followed by a slow conformational change [50] The

conformational change (or maturation step) appears to be

essential for the complete enzymatic activation of RT [50]

As discussed previously, the RNase Hresidues 537–546 in

the p66 subunit interact with the p51 thumb residues 280–

290 A synthetic peptide derived from a sequence within the

thumb subdomain of HIV-1 RT (residues 284–300) was

found to bind to heterodimeric HIV-1 RT with an apparent

dissociation constant in the nanomolar range and interfere

with the conformational change (or maturation step)

required for activation of heterodimeric RT [51] Based on

this work it was suggested that the activation of RT might

also represent an important target for the design of novel

antiviral compounds

Destabilization of the HIV-1 RT dimer interface

by small nonpeptidic molecules

The complete dissociation of the p66 and p51 subunits of

HIV-1 RT heterodimer may not be entirely necessary for

there to be a negative impact on RT enzymatic function Indeed, recent studies have shown that small molecule binding to the dimer interface of HIV-1 RT may induce conformational changes that impact on the overall stability

of the heterodimeric complex without dissociating the heterodimer complex [44,52] Two structurally unrelated classes of compounds have been found to elicit this effect 2¢,5¢-Bis-O-(tert-butyldimethylsilyl)-b-D -ribofuranosyl]-3¢spiro-5¢¢-(4¢¢-amino-1¢,2¢-oxathiole-2¢,2¢-dioxide)thymine (TSAO-T) is the prototype of an unusual class of non-nucleoside reverse transcriptase inhibitors (NNRTI) which have structures (Fig 3) and mechanism of actions quite distinct from conventional NNRTI [53,54] The N3-ethyl derivative of TSAO-T, TSAO-e3T has been shown to destabilize both the p66/p51 and p66/p66 dimeric forms of HIV-1 RT [44] The Gibbs free energy of RT dimer dissociation is decreased in the presence of increasing concentrations of TSAOe3T, resulting in loss of dimer stability of 4.0 and 3.2 kcalÆmol)1for p66/p51 and p66/p66 forms of HIV-1 RT, respectively [44] This loss of energy is not sufficient to induce subunit dissociation in the absence

of denaturant [44] High-level drug resistance to TSAO is mediated by the E138K mutation in the p51 subunit of HIV-1 RT [55] The introduction of this mutation into RT significantly diminishes the ability of TSAO to bind to and inhibit the enzyme [55] and accordingly TSAO-e3T is unable

to destabilize the subunit interactions of the E138K mutant enzyme [44] Modeling experiments have suggested that TSAO may bind to a site in RT that is overlapping with, but

Fig 3 Chemical structures of NNRTI that modulate the RT dimeri-zation process The NNRTI nevirapine, efavirenz, and UC781 act as chemical enhancers of HIV-1 RT dimerization [57] Unlike other NNRTI, delavirdine has no effect on RT dimerization [57] TSAOe3T and BBNHbinding to HIV-1 RT destabilizes the quaternary structure

of the enzyme [43,51].

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distinct from, the NNRTI binding site where it appears to

make significant interactions with the p51 subunit of the

enzyme [41,44] On the basis of this model, the

TSAO-induced changes in RT dimer stability likely arise from

conformational perturbations that affect the p66/p51 RT

interface [41,44]

N-(4-tert-butylbenzoyl)-2-hydroxy-1-naphthaldehyde

hydrazone (BBNH) is a multitarget inhibitor of HIV-1 RT

that binds to both the DNA polymerase and RNase H

domains of the enzyme, and inhibits both enzymatic

activities [56,57] BBNHbinding to HIV-1 RT also impacts

on the dimeric stability of the heterodimeric enzyme [52] in

that BBNHbinding to p66/p51 RT decreases the value of

the Gibbs free energy of RT dimer dissociation by

3.8 kcalÆmol)1 To evaluate whether this loss of Gibbs free

energy was mediated by BBNHbinding to one or more sites

in RT, a variety of BBNHanalogs were synthesized and

evaluated for their ability to destabilize (or weaken) the

protein–protein interactions of the heterodimer [52] In this

regard, it was found that N-acyl hydrazone binding in the

DNA polymerase domain alone was sufficient to elicit the

observed decrease in Gibbs free energy In this regard, it has

been speculated that BBNHbinds to HIV-1 RT in a manner

analogous to TSAOe3T [52]

Small molecules that enhance RT dimerization

It is clear that either dissociation or destabilization of the

RT subunits is detrimental to enzyme function Conversely,

enhancement of the HIV)1 RT subunit interactions may

also represent a novel approach to modulating RT activity

In this regard, it has recently been reported that several

NNRTI exhibit an unexpected capacity to dramatically

increase the association of the p66 and p51 RT subunits [58]

Using a yeast two hybrid RT dimerization assay that

specifically detects the interaction between the p66 and p51

RT subunits [59] it was shown that several NNRTI,

including efavirenz, nevirapine, UC781, 8-Cl-TIBO, HBY

097 and a-APA, can significantly increase the

b-galactosi-dase readout in a yeast reporter strain [58] This increase in

b-galactosidase activity suggested an enhancement of RT

heterodimer subunit interaction, an effect that was

con-firmed by in vitro binding assays using recombinant p66 and

p51 [58] Enhanced homodimerization of the RT p66

subunits by efavirenz has also been observed in both the

Y2Hassay and in in vitro binding assays (G Tachedjian,

unpublished observations) Furthermore, this

NNRTI-induced enhancement effect on RT dimerization requires

drug binding to the NNRTI binding site in the p66 subunit

as introduction of the drug resistance mutation, Y181C, in

the NNRTI-binding pocket negates the enhancement effect

mediated by nevirapine [58] The mechanism by which these

small molecules enhance RT dimerization remains unclear

However, the mode of NNRTI binding to RT appears to be

important Delavirdine, also an NNRTI, does not enhance

RT dimerization [58] This drug, in contrast to other

NNRTI, is longer and does not sit exclusively in the

NNRTI binding pocket but protrudes outside this site [60]

The unique characteristics of the interaction of delaviridine

with the HIV-1 RT suggests that it binds to p66 in a way

that does not favor the enhancement of RT dimerization

[58] Elucidation of the differences in RT binding between

delavirdine and other NNRTI may provide important

information for the design of potent enhancers of RT dimerization and consequently potent inhibitors of DNA polymerization [58]

H I V - 1 I N T E G R A S E

Structure and function of HIV-1 IN HIV-1 IN is a polynucleotidyltransferase that catalyzes the integration of the DNA copy of the viral genome into the genome of the host cell In order to accomplish this goal, IN has evolved to catalyze two separate reactions, each proceeding by direct transesterification reactions catalyzed

at a single active site in the enzyme’s core [61] In the first reaction, 3¢ processing, IN removes two nucleotides from the from the 3¢-end of each strand of the nascent viral DNA, leaving a recessed 3¢CA dinucleotide After migra-tion into the nucleus of the infected cell as part of the nucleoprotein complex, IN covalently attaches each 3¢ processed viral end to the host cell DNA, a reaction termed strand transfer

HIV-1 IN comprises three independently folding domains; an N-terminal domain, a catalytic core domain, and a C-terminal domain (for a review see [62]) The N-terminal domain, residues 1–51, contains a conserved HH-CC motif that binds zinc in a 1 : 1 stoichiometry [63] The central catalytic core domain, residues 52–210, contains the catalytic site characterized by three invariant essential acidic residues, D64, D116 and E152 The C-terminal domain, residues 220–288, appears to significantly contrib-ute to DNA binding [64] and is linked to the catalytic core

by residues 195–220, an extension of the final helix of the core domain Efforts to crystallize the full length HIV-1 IN have been hampered by poor solubility However, the three-dimensional structure of each domain has been solved [65–67] as have structures of two domain INs containing either the catalytic core and C-terminal domain [68], or the N-terminal domain and the catalytic core [69] In all structures reported to date, the quaternary structure of IN is dimeric, however, the full enzyme is likely to function as at least a tetramer [13] The dimer interface in the catalytic core–C-terminal two domain fragment involves the strong helix-to-helix contacts a1 (residues 99–108):a5¢(residues 168–185) and a5:a1¢, where both hydrophobic and electro-static interactions contribute to dimer stabilization In the N-terminal –catalytic core two domain structure, additional subunit interface interactions are provided from the N-terminal domain, in particular residues 29–35

Peptide inhibitors of HIV-1 in oligomerization

As described above, protein–protein interactions between the two catalytic core domains involve interactions from the a1 and a5 helices of both subunits Synthetic peptides corresponding to the respective sequences (93–107 and 167– 187) were found to strongly inhibit the 3¢-processing and strand-transfer activities of IN [70] Furthermore, both peptides were found to perturb the association-dissociation equilibrium of both the full-length IN enzyme, as well as the individually isolated catalytic cores [70] Interestingly, peptide binding to IN also appeared to alter the overall conformation of the protein subunits, suggesting that enzyme deactivation, subunit dissociation and protein

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unfolding are events which parallel one another

Fluores-cence studies suggested that the peptide corresponding to

residues 167–187 physically interacts with helix a1 in the

dimer interface of the catalytic core domains thus providing

a rational for the observed dissociation of IN oligomers [70]

C O N C L U S I O N S

The enzymatic activities of HIV-1 PR, RT and IN are all

coupled to their quaternary (or oligomeric) structures

Accordingly, modulation of the protein–protein

inter-actions of these enzymes has been proposed as a rational

target for the development of anti-HIV compounds In this

regard, our review highlights the many peptidic and small

molecule compounds that have been identified to exhibit

such a mode of action However, in most cases, the

structural and kinetic characterization of their mechanisms

of action has primarily been carried out in an in vitro

environment, using recombinantly purified enzyme

Although some of the molecules described above have

been shown to exhibit antiviral activity in cell culture

[17,47,53,56], no studies have rigorously evaluated their

effect on either Gag-Pol processing or enzyme oligomer

formation in the virus Thus to date, there is essentially no

evidence to confirm that their mechanisms of action in vivo

are similar to those proposed in vitro In these authors’

opinions, such studies obviously represent the next logical

step in the unfolding story of the modulation of the

oligomeric structures of HIV-1 viral enzymes by synthetic

peptides and small molecules

A C K N O W L E D G E M E N T S

The authors would like to acknowledge Dominique Arion for critical

reading of the manuscript The research of N.S.-C has been funded, in

part, by a University of Pittsburgh Medical Center (UPMC)

Compet-itive Medical Research Fund (CMRF) G.T was supported in part by a

C.J Martin Fellowship 977373 awarded by the National Health and

Medical Research Council of Australia.

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