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A catalytically inactive b1,4- N -acetylglucosaminyltransferase IIIGnT-III behaves as a dominant negative GnT-III inhibitor Hideyuki Ihara, Yoshitaka Ikeda, Souichi Koyota, Takeshi Endo,

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A catalytically inactive b1,4- N -acetylglucosaminyltransferase III

(GnT-III) behaves as a dominant negative GnT-III inhibitor

Hideyuki Ihara, Yoshitaka Ikeda, Souichi Koyota, Takeshi Endo, Koichi Honke and Naoyuki Taniguchi Department of Biochemistry, Osaka University Medical School, Suita, Osaka, Japan

b1,4-N-Acetylglucosaminyltransferase III (GnT-III) plays a

regulatory role in the biosynthesis of N-glycans, and it has

been suggested that its product, a bisecting GlcNAc, is

involved in a variety of biological events as well as in

regu-lating the biosynthesis of the oligosaccharides In this study,

it was found, on the basis of sequence homology, that

GnT-III contains a small region that is signi®cantly homologous

to both snail b1,4GlcNAc transferase and b1,4Gal

trans-ferase-1 Subsequent mutational analysis demonstrated an

absolute requirement for two conserved Asp residues

(Asp321 and Asp323), which are located in the most

homologous region of rat GnT-III, for enzymatic activity

The overexpression of Asp323-substituted, catalytically

inactive GnT-III in Huh6 cells led to the suppression of the activity of endogenous GnT-III, but no signi®cant decrease

in its expression, and led to a speci®c inhibition of the for-mation of bisected sugar chains, as shown by structural analysis of the total N-glycans from the cells These ®ndings indicate that the mutant serves a dominant negative e€ect on

a speci®c step in N-glycan biosynthesis This type of Ôdomi-nant negative glycosyltransferaseÕ, identi®ed has potential value as a powerful tool for de®ning the precise biological roles of the bisecting GlcNAc structure

Keywords: GnT-III; glycosyltransferase; bisecting GlcNAc; N-glycan synthesis; dominant negative e€ect

b1,4-N-Acetylglucosaminyltransferase III (GnT-III)

cata-lyzes the transfer of GlcNAc from UDP-GlcNAc, a glycosyl

donor, to a core b-mannose residue in N-linked

oligosac-charides via a b1 ® 4 linkage, resulting in the formation of

a bisected sugar chain [1] The resulting GlcNAc residue is

referred to as a bisecting GlcNAc, and is known to play a

role in regulating the biosynthesis of N-glycans, as the

addition of this unique structure inhibits the action of other

N-acetylglucosaminyltransferases, such as GnT-IV and

GnT-V, both of which are involved in the formation of

multiantennary sugar chains [2] Thus, GnT-III can be

regarded as a key glycosyltransferase in N-glycan

biosyn-thetic pathways

It has been suggested that GnT-III and/or the bisecting

GlcNAc residue are involved in a variety of biological

processes, such as the intracellular sorting of

glycopro-teins [3], secretion of apo B100 from the liver [4], cell

adhesion [5] and cancer metastasis [6,7], as evidenced by

gene transfection experiments using GnT-III cDNA

Although studies using GnT-III-de®cient mice have

revealed that a defect in GnT-III suppresses

diethylni-trosamine-induced hepatocarcinogenesis [8,9], they do not

provide any explanations for the changes caused by an overexpression of GnT-III, as mentioned above The mechanisms that underlie such a variety of biological events, which are caused by ectopic expression, overex-pression and defects in GnT-III, remain obscure In addition, the issue of whether phenotypes associated with altered expression of GnT-III are the actual consequences

of N-glycan modi®cation by GnT-III remains unresolved

To address these issues, a catalytically inactive GnT-III and/or dominant negative mutant GnT-III would be valuable because it may potentially de®ne whether bisected N-glycan structures are actually important Alternatively, the issue of whether GnT-III protein is directly involved regardless of formation of bisected sugar chains could also be examined

The identi®cation of catalytic residues, the absence of which leads to the complete loss of activity, is required to produce a catalytically inactive GnT-III The enzymatic properties of this enzyme have been extensively studied in terms of substrate speci®city towards acceptors and donors, and these collective data provide an enzymatic basis for the role of the bisecting GlcNAc in the regulation

of N-glycan biosynthesis [1,10,11] Nevertheless, because the catalytic mechanism of GnT-III has not yet been analyzed in suf®cient detail, the chemical basis of the enzymatic reaction is not known The reaction catalyzed

by GnT-III is accompanied by inversion at the anomeric center of the transferred monosaccharide and the forma-tion of a b1 ® 4 linkage, while chemically similar reactions are also catalyzed by other glycosyltransferases such as b1,4GlcNAc transferase (b1,4GlcNAcT) from snail, Lymnaea stagnalis [12], and mammalian b1,4Gal transferases, (b1,4GalTs), both of which are mutually homologous [13] It seems more likely that the common properties of these three enzymes are associated with the catalytic mechanism, rather than substrate binding, as the

Correspondence to N Taniguchi, Department of Biochemistry, Osaka

University Medical School, 2-2 Yamadaoka, Suita, Osaka 565-0871,

Japan Fax: + 81 6 6879 3429, Tel.: + 81 6 6879 3420,

E-mail: proftani@biochem.med.osaka-u.ac.jp

Abbreviations: GnT-III, b1,4-N-acetylglucosaminyltransferase III;

PNGase, peptide-N-glycosidase; HRP, horseradish peroxidase;

DMEM, Dulbecco's modi®ed Eagle's medium.

Enzyme: b1,4-N-acetylglucosaminyltransferase III (EC 2.4.1.144).

Note: a web site is available at http://www.med.osaka-u.ac.jp/pub/

biochem/index.html

(Received 17 April 2001, revised 15 October 2001, accepted 29 October

2001)

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donor and acceptor substrates are divergent Therefore, as

the residues that are conserved among these enzymes

would be expected to be involved in the presumed

common catalytic mechanism, such residues must be

essential for enzyme activity

In this study, amino-acid residues of GnT-III, which are

conserved in mammalian b1,4GalT-1 and snail

b1,4GlcN-AcT were identi®ed by a comparison of sequences using a

dot matrix analysis, and were then examined for their

requirement with respect to GnT-III activity In addition,

the effect of the inactive mutant GnT-III, in which a residue

identi®ed as being essential was replaced, was examined in

the biosynthesis of bisected sugar chains in cells These

experiments were performed, in order to determine if the

mutant serves as a Ôdominant negative glycosyltransferaseÕ

toward a speci®c step in the oligosaccharide biosynthesis

E X P E R I M E N T A L P R O C E D U R E S

Materials

Restriction endonucleases and DNA-modifying enzymes

were purchased from Takara (Kyoto, Japan), Toyobo

(Shiga, Japan) and New England Biolabs (UK)

UDP-GlcNAc and UDP-GlcNAc were obtained from Sigma (MO,

USA) Oligonucleotide primers were synthesized by Greiner

Japan (Tokyo, Japan) Antibodies were obtained from

following sources: monoclonal anti-(GnT-III) Ig from

Fujirebio Inc (Tokyo, Japan); horseradish peroxidase

(HRP)-conjugated anti-(mouse IgG) Ig from Promega

(WI, USA) Peptide-N-glycosidase F (PNGase F) was

obtained from Roche Diagnostics (IN, USA) Standards of

pyridylaminated sugar chains were purchased from Takara

and Seikagaku Corp (Tokyo, Japan) Other common

chemicals were obtained from Wako pure chemicals

(Osaka, Japan), Nacalai Tesque (Kyoto, Japan) and Sigma

Construction of expression plasmids

For transient expression in COS-1 cells, a cDNA encoding

rat GnT-III [14] was subcloned into the EcoRI sites of an

SV40-based expression vector, pSVK3 (Amersham

Phar-macia Biotech, Buckinghamshire, UK) When the enzyme

was stably expressed in Huh6 cells, the cDNA was

subcloned into the EcoRI sites of another expression vector,

pCXNII, which contains a neorgene [15] In this vector, the

GnT-III was expressed under the control of the b-actin

promoter and the CMV enhancer

Site-directed mutagenesis

Site-directed mutagenesis experiments were carried out

according to Kunkel [16], as described previously [17] A

0.6-kb fragment obtained by digestion of rat GnT-III cDNA

with EagI and HindIII was subcloned into pBluescript

KS+, and the resulting plasmid was used for transformation

of CJ236 (dut±, ung±) The uracil-substituted ssDNA was

prepared by infection of the transformed CJ236 with a

helper phage M13K07 This template was then used with

oligonucleotide primers to replace the conserved aspartic

acid residues with alanine The primers used in this study

were

5¢-TTTATCATCGACGCCGCGGACGAGATCC-3¢ for replacement of Asp321 (designated D321A),

5¢-ATCATCGACGACGCCGCGGAGATCCCTGCGT-3¢ for Asp323 (D323A), and 5¢-ATCCCTGCGCGTGCC GGCGTGCTGTTCCTGAAG-3¢ for Asp329 (D329A) The resulting mutations were veri®ed by dideoxy sequencing using a DNA sequencer (model 373 A, Applied Biosystems,

CA, USA), and the entire sequences that had been subjected

to mutagenesis were also veri®ed The corresponding region

of the wild-type cDNA was replaced by each mutant sequence The plasmids for the expression of these mutants were constructed, as were those for the wild-type enzyme, and used for transfection

Cell culture Huh6 cells, a human hepatoblastoma cell line, and COS-1 cells were maintained in Dulbecco's modi®ed Eagle's medium (DMEM) containing 10% fetal bovine serum,

100 UámL)1 penicillin, 100 lgámL)1 streptomycin and

5 gáL)1glucose under a humidi®ed atmosphere of 95% air and 5% CO2

Protein determination Protein concentration was determined with BCA Kit (Pierce, IL, USA) using BSA as a standard

Electrophoresis and immunoblot analysis SDS/PAGE was carried out on 8% gels, according to Laemmli [18] The separated proteins were transferred onto

a nitrocellulose membrane (PROTORAN, Schleicher & Schuell Inc., NH, USA) The resulting membrane was blocked with 5% skimmed milk and 0.5% BSA in NaCl/Pi

containing 0.05% Tween-20, and was then incubated with

an anti-(GnT-III) Ig After washing with NaCl/Pi that contained 0.05% Tween-20, the membrane was reacted with

a HRP-conjugated goat anti-(mouse IgG) Ig The immuno-reactive protein bands were visualized by chemiluminescence using an ECL system (Amersham Pharmacia) Ponceau staining of the transferred membrane was performed before blocking with skimmed milk and BSA to verify equal amounts of proteins loaded For digestion by PNGase F, the samples were denatured by boiling for 3 min in 20 mM

phosphate buffer (pH 7.0) containing 0.2% SDS, 1% 2-mercaptoethanol and 0.5% Triton X-100 Deglycosyla-tion by PNGase F was performed according to the manu-facturer's instructions

DNA transfection Expression plasmids were transfected into cells by electro-poration [19] using a Gene Pulser (Bio-Rad, CA, USA), as described previously [14] In a typical experiment, the cells were washed with Hepes-buffered saline and resuspended in the same solution Plasmids (30 lg), puri®ed by CsCl gradient ultracentrifugation, were added to the cell suspen-sion, followed by electri®cation For transient expression in COS-1 cells, the transfected cells were harvested after an appropriate growth period When stable transfectants of Huh6 cells were established, the transfected cells were subjected to selection by geneticin resistance The expression

of GnT-III was veri®ed by immunoblot analysis and enzyme activity assay for GnT-III

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Enzyme activity assays for glycosyltransferases

GnT-III and GnT-V activities were assayed using a

pyridylaminated biantennary sugar chain as an acceptor

substrate, as described previously [20,21] A large-scale

preparation of pyridylaminated biantennary sugar chain

was performed as reported previously [22±24] Standard

assays were performed in a ®nal volume of 15 ll of 125 mM

Mes/NaOH buffer (pH 6.25) containing 0.5% Triton

X-100, 200 mMGlcNAc The assay mixture also contained

10 mM MnCl2 for GnT-III assay or 10 mM EDTA for

GnT-V The concentration of pyridylaminated biantennary

sugar chain was 10 lM for both enzymes The

concentra-tions of the donor, UDP-GlcNAc, for GnT-III and GnT-V

were 20 and 40 mM, respectively After incubation for 2±4 h,

the reactions were terminated by rapidly heating to 100 °C

The reaction mixtures were then centrifuged at 10 000 g, for

10 min and the resulting supernatants were applied to an

HPLC equipped with a TSK-gel, ODS-80TM column

(4.6 ´ 150 mm) (Tosoh, Tokyo, Japan) in order to separate

and quantitate the products Elution was performed

isocratically at 55 °C using a 20-mM acetate buffer

(pH 4.0) containing 0.3% and 0.15% butanol for GnT-III

and GnT-V assays, respectively The column eluate was

monitored for ¯uorescence using a detector (model

RF-10AXL, Shimadzu, Kyoto, Japan) operating at

excita-tion and emission wavelengths of 320 and 400 nm,

respec-tively The amounts of products were estimated from the

¯uorescence intensity b1,4GalT activity was also assayed

using a pyridylaminated biantennary sugar chain as an

acceptor substrate [25] The assay mixture for b1,4GalT

consisted of 50 mMMops buffer (pH 7.4) containing 20 mM

MnCl2, 0.5% Triton X-100, 5 mM UDP-Gal and 10 lM

pyridylaminated biantennary sugar chain After incubation

for 2 h, the reaction was stopped, and the reaction mixture

was analyzed by normal phase HPLC In this assay, a

TSK-gel Amide-80 column (4.6 ´ 250 mm) (Tosoh) was used at

40 °C The elution buffer was 1.14% acetic

acid/triethyl-amine (pH 7.3) that also contained 62% acetonitrile

Structural analysis of sugar chains

Huh6 cells and the transfectant cells, which were harvested

from 10 10-cm dishes of con¯uent cultures were sonicated

and lyophilized Each preparation of the whole cells was

then hydrazinolyzed to liberate Asn-linked oligosaccharides,

as described previously [23,24,26] The free oligosaccharides

were then re-N-acetylated in 10 mL saturated ammonium

bicarbonate with 554 ll acetic anhydride, followed by

desalting on a column of AG 50 W-X12 resin (Bio-Rad)

Fluorescence labeling of the sugar chains was carried out by

reductive amination involving the use of a ¯uorescent

reagent, 2-aminopyridine and sodium cyanoborohydride

[24] After pyridylamination, the excess reagents were

removed by gel ®ltration with HW-40F (Toyopearl, Tosoh)

The pyridylaminated sugar chains were then desialylated,

degalactosylated and defucosylated by sialidase

(Arthro-bacter ureafaciens, Nacarai tesque), b-galactosidase (jack

bean, Seikagaku Corp.) and a-fucosidase (bovine kidney,

Sigma), respectively The digested samples were then

analyzed by HPLC using a TSK-gel ODS-80TM column

(4.6 ´ 150 mm), and elution was performed at 55 °C by a

linear gradient of butanol from 0.1% to 0.25% in 20 mM

ammonium/acetate buffer (pH 4.0) The eluted sugar chain peaks were identi®ed by comparison with standard pyr-idylaminated sugar chains (Takara and Seikagaku Corp.) RT-PCR

Total RNA from parental Huh6 cells and various transfec-tants was prepared using TRIZOL (Gibco-BRL, MD, USA), and the cDNAs were synthesized by reverse transcriptase with an oligo dT-adaptor primer from RNA

LA PCR Kit (Takara) In order to speci®cally detect the expression of endogenous human GnT-III, PCR was performed with the selective primers for human GnT-III

in a PCR Thermal Cycler 480 (Takara) The primers used in this study were designed to detect mRNA for human GnT-III but not rat GnT-GnT-III: 5¢-AAGACCCTGTCCTAT-3¢ (nucleotide position 85±99 in the ORF) for sense, and 5¢-GTTGGCCCCCTCAGG-3¢ (position 415±429) for antisense This differential detection was con®rmed by PCR using plasmid DNA containing human and rat GnT-III cDNAs as templates The primers to detect b-actin mRNA

as a control were 5¢-CAAGAGATGGCCACGGCTGCT-3¢ (nucleotide position 673±693 in the ORF of human b-actin)and5¢-TCCTTCTGCATCCTGTCGGCA-3¢(posi-tion 927±947), for sense and antisense, respectively The sizes of the products that were yielded by the PCR using these primers were expected to be 345 bp and 275 bp for human GnT-III and b-actin, respectively The absence of ampli®cation of the genomic DNA was veri®ed by subject-ing total RNA directly to the PCR without reverse transcription, because the open reading frame of GnT-III

is encoded by a single exon

R E S U L T S A N D D I S C U S S I O N

Comparison of the amino-acid sequence of GnT-III with snail b1,4GlcNAcT and mammalian b1,4GalT-1

In order to identify the essential residues that are important for GnT-III activity, the candidate residues for examination were selected on the basis of amino-acid sequence homology amongratGnT-III,mammalianb1,4-galactosyltransferase-1 (b1,4GalT-1) and snail b1,4-N-acetylglucosaminyltrans-ferase (b1,4GlcNAcT) Dot matrix analyses comparing the GnT-III sequence with that of either of the other enzymes showed signi®cant similarities in the small region, in which three aspartic acid residues and several other residues are perfectly conserved (Fig 1A,B) Only this homologous region was detected in all three enzymes These Asp residues correspond to Asp321, Asp323 and Asp329 in the rat GnT-III sequence, as shown by sequence alignment (Fig 1C) The sequence of Asp321-Val322-Asp323 appears to corre-spond to the D-X-D motif, as have been suggested for the catalytic importance in many other glycosyltransferases [27±41], and, thus, the sequence comparison suggests that these Asp residues represent likely candidates for investiga-tion as having a role in GnT-III activity

Site-directed mutagenesis of the conserved aspartic acid residues in GnT-III

To explore the requirement of the candidate amino acids, Asp321, Asp323 and Asp329, for enzyme activity, mutant

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GnT-IIIs in which these Asp residues were replaced by Ala

were prepared using site-directed mutagenesis, and the

respective mutants were designated as D321A, D323A and

D329A When these mutants were transiently expressed in

COS-1 cells, their protein expression levels were found to be

similar to that of wild-type, as indicated by immunoblot

analysis (Fig 2A,B) However, in the standard activity

assay, the D321A and D323A mutants had no detectable

catalytic activity, whereas the D329A mutant was fully

active, as shown by an activity similar to that of the

wild-type enzyme (Table 1) No activity was detected in the

D321A and D323A mutants even under the conditions of

longer incubation time and UDP-GlcNAc concentration as

high as 100 mM, indicating that the replacement of Asp321

and Asp323 lead to a complete loss of activity These data

suggest that Asp321 and Asp323 play an essential role in the

activity of enzyme, while Asp329 does not, even though this

Asp residue is conserved Our previous study showed that

three N-linked glycosylation sites in rat GnT-III are fully

glycosylated when expressed in COS cells [42], and all

mutants used in this study were also found to be fully

glycosylated, as indicated by the digestion with PNGase F

(Fig 2C) Immuno¯uorescence microscopic analysis

showed that the intracellular localization of the mutants are identical to that of the wild-type enzyme, indicating that the replacements of Asp321 and Asp323 have no effect on intracellular localization (data not shown) Thus, it seems unlikely that these mutations lead to gross conformational alterations or misfolding of the protein, but it is more likely that the loss of the activity is the result of the deletion of the active site residues

The possible role of Asp321 and Asp323

in the catalysis of GnT-III The absolute requirement for Asp321 and Asp323 and their conservation in b1,4GalT-1 and snail b1,4GlcNAcT suggest that the short sequence of Asp321-Val322-Asp323 in GnT-III plays a role that is analogous to the function of the D-X-D motif, found in b1,4GalT-1 [13] Although an essential role for the D-X-D motif has been demonstrated in many glycosyltransferases [27±35], the function of this motif seemed divergent While a large clostridial glucosyltransfer-ase requires the D-X-D motif for UDP or UDP-sugar binding [27], this motif appears not to be critical for nucleotide binding in GM2 synthase [34] and Fringe [31] in

Fig 1 Dotplot analyses for rat GnT-III vs snail b1,4GlcNAcT or bovine b1,4GalT-1 Dotplots for (A) rat GnT-III vs L stagnalis b1,4GlcNAcT and (B) rat GnT-III vs bovine b1,4GalT-1 are shown The amino-acid sequences were compared under the conditions of a window size of 10 residues and 50% of identity using the computer software program, ALIGN The numbers beside the axis indicate the residue number of each enzyme Diagonal plots show the homologous regions, which satisfy the above conditions The sequences of the most homologous region are given outside the matrices (C) Multiple alignment of the homologous regions of GnT-III, b1,4GalT-1 and L stagnalis b1,4GlcNAcT were carried out by

CLUSTALV The amino-acid residues which are conserved in all enzymes are highlighted by shaded boxes, and two homologous residues are also indicated by grey-shaded boxes The conserved aspartic acid residues that were examined by mutational analysis are indicated by arrowheads GenBank accession numbers for the glycosyltransferases are: GnT-III (human), D13789; GnT-III (rat), NM_019239; GnT-III (mouse), NM_010795; b1,4GlcNAcT (L stagnalis), X80228; b1,4 GalT-1 (human), X14085; b1,4 GalT-1 (bovine), X14558; b1,4 GalT-1 (mouse), J03880; b1,4 GalT-2 (mouse), AB019541.

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spite of its requirement for their enzyme activities On the other hand, crystallographic analyses of b1,4GalT-1 [36] as well as other glycosyltransferases [37±41] have suggested that this motif serves the coordination of a divalent cation such as Mn2+along with a phosphoryl group of the donor The motif would thereby allow the enzyme to interact with a nucleotide portion of the donor nucleotide sugar and also may facilitate the reaction via electrostatic catalysis involv-ing the divalent cation Although the function of the D-X-D motif is not known for GnT-III, it is possible that the D-X-D motif in GnT-III plays a similar role to that in b1,4GalT-1 because of the signi®cant sequence homology in the region containing this motif In a large clostridial glucosyltransferase mutant similar to the GnT-III D321A and D323A mutants, the activity could be recovered in the presence of extremely high concentrations of Mn2+, supporting the suggestion that the equivalent aspartic acid residues in the motif are involved in the coordination with the divalent cation [27] Nevertheless, in the case of GnT-III, activity was not detected even at concentrations of Mn2+as high as 100 mM, suggesting an absolute requirement of Asp321 and Asp323 for the coordination of Mn2+during the reaction of GnT-III (data not shown)

Expression of a catalytically inactive GnT-III

in Huh6 cells, a hepatoblastoma cell line

In order to determine whether a catalytically inactive GnT-III mutant serves a dominant negative function by prevent-ing the action of the wild-type endogenous enzyme, Huh6 cells, a human hepatoblastoma cell line, were transfected with the rat GnT-III mutants because a structural pro®le of the N-glycans in these cells had been previously character-ized through a structural analysis of N-linked sugar chains

of a-fetoprotein produced by the cells [43] Huh6 cells express relatively high levels of GnT-III and produce bisected sugar chains, the products of this glycosyltransfer-ase [43] Following the selection of the transfected cells by geneticin resistance, clones were grown separately, and the expression of the rat mutant enzyme was veri®ed by immunoblot analysis As a result, we obtained three clones, which overexpress the D323A GnT-III mutant (Fig 2D) In Huh6 cells, as was in COS-1 cells, the D323A mutant was expressed at a similar level to the wild-type, and appeared to

be fully glycosylated (Fig 2D,F) In the case of the other mutant, D321A, however, we were not successful in establishing such clones

The speci®c suppression of endogenous GnT-III activity by expression of the catalytically inactive D323A mutant in Huh6 cells

When assays for GnT-III activity were performed in the transfected cells, it was found that the activity in the transfected cells were as low as less than 5% of the activity in the parental or mock-transfected Huh6 cells, which were used as controls On the other hand, the activities of GnT-V, another GlcNAc transferase which is involved in the formation of b1,6-branches, and b1,4GalT were not essen-tially affected in the transfected cells, indicating that the overexpression of the D323A mutant has no effect on these glycosyltransferase activities (Fig 3) Therefore, it appears that the overexpression of the mutant does not impair

Fig 2 Expression of the wild-type and mutant GnT-III proteins The

wild-type and mutant enzymes were transiently expressed in COS-1

cells (A±C), and stably expressed in Huh6 cells (D±F) (A,D) The cell

homogenates were separated on 8% SDS-gels and were analyzed by

immunoblot using anti-(GnT-III) Ig (B,E) The amounts of protein

loaded were veri®ed by Ponceau staining, prior to the immunoblot

analysis (C,F) The samples were treated with PNGase F, followed by

SDS/PAGE and immunoblotting COS-1 and pSVK3 indicate

non-transfected and vector-non-transfected (mock) COS-1 cells, respectively.

Huh6, Huh6/D323A and Huh6/WT represent parental

nontrans-fected, D323A-transfected and wild-type-transfected Huh6 cells,

respectively Details of the conditions are described in Experimental

procedures.

Table 1 Activities of the wild-type and mutant GnT-IIIs transiently

expressed in COS-1 cells GnT-III activity was determined as described

in Experimental procedures The mock plasmid was transfected with a

vector, pSVK3 ND, not detectable.

Plasmid GnT-III activity(nmoláh )1 ámg protein )1 )

Not transfected ND

Wild-type 0.98

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glycosyltransferase activities in a nonspeci®c manner, for example, via the downregulation of their expression or damage to the Golgi apparatus Quite similar results were obtained in all three of the obtained clones These results suggest that the overexpression of the D323A mutant speci®cally suppresses the activity of endogenous GnT-III

To examine whether the mutant GnT-III inhibits the intrinsic GnT-III in vitro, the extracts from the parental cells were mixed with the extract from the D323A-transfected cells, and were then analyzed by the activity assay As shown in Fig 4, no inhibitory effect of the mutant was observed, and, thus, it seemed unlikely that the in vivo inhibition by the mutant is due to direct competition for substrate Furthermore, RT-PCR using a primer set that is speci®c to the human GnT-III sequence showed that the mRNAs for endogenous human GnT-III were not signif-icantly decreased (Fig 5), suggesting that the decreased GnT-III activity is not due to the downregulation of expression of the intrinsic human GnT-III gene Therefore,

it is more likely that the activity of the intrinsic human enzyme is decreased as the result of control at the translational or post-translational level including protein± protein interactions

Blockage of a speci®c step, namely the formation

of a bisecting GlcNAc residue, in N-glycan biosynthesis via the expression of the D323A mutant

In order to further examine the issue of whether the biosynthesis of bisected sugar chains are inhibited by overexpression of the D323A mutant, total N-linked sugar chains were prepared from the cells and labeled with 2-aminopyridine, a ¯uorescent reagent The resulting free oligosaccharides were digested with sialidase, b-galactosi-dase and a-fucosib-galactosi-dase, and then subjected to reversed phase HPLC, in order to analyze the core structures, i.e the addition of a bisecting GlcNAc and the extent of branching

As shown in Fig 6, when parental cells, which express various sugar chains with bi-, tri- and tetra-antennae were examined, substantial fractions (20±30%) of these sugar chains were found to contain the bisecting GlcNAc Almost

Fig 4 E€ect of the D323A mutant on the endogenous GnT-III activity

in vitro After indicated amounts of the extracts from parental Huh6 cells and the D323A-transfected cells were mixed, GnT-III activity was assessed The data are expressed as the relative value to the activity determined in the absence of the mutant extract.

Fig 3 GnT-III, b1,4GalT and GnT-V activities in the

D323A-trans-fectant Activities of GnT-III (A), b1,4GalT (B) and GnT-V (C) were

assayed using a pyridylaminated oligosaccharide acceptor, as described

in Experimental procedures Data for the transfectants are expressed as

mean values from three di€erent clones for D323A and 2 clones for the

wild-type GnT-IIIs and mock-transfected cells In the

D323A-trans-fectants, standard deviations are also shown.

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the same pro®le was obtained in the case of the

mock-transfected cells (data not shown) On the other hand, the

D323A-transfected cells were found to express negligible

levels of bisected sugar chains These results indicate that the

overexpression of the D323A mutant blocks the

biosynthe-sis of bisected sugar chains as the result of a decrease in the activity of endogenous GnT-III The results demonstrate that the catalytically inactive mutant enzyme acts as a dominant negative glycosyltransferase toward the forma-tion of a bisecting GlcNAc in vivo

Fig 5 Detection of mRNA for endogenous human III in the D323A-transfectant by RT-PCR mRNA expression of endogenous human GnT-III in parental Huh6 cells and the transfectants for the D323A and wild-type GnT-GnT-IIIs were investigated by RT-PCR (upper panel) Reverse transcription was omitted to verify the absence of ampli®cation of genomic DNA in the lanes indicated by RT (±) Human b-actin mRNA expression was also examined as a control (lower panel) Speci®city to the endogenous human enzyme was con®rmed by PCR using plasmid DNAs Lanes: human GnT-III and rat GnT-III indicate PCR-ampli®cation of plasmids containing cDNAs for human and rat enzyme, respectively Details regarding the PCR procedures are described in Experimental procedures.

Fig 6 Structural analysis of Asn-linked sugar

chains from parental and transfected Huh6

cells Elution pro®les of pyridylaminated sugar

chains in reversed phase HPLC are shown for

parental Huh6 cells (a), wild-type

GnT-III-transfectant (b) and D323A-GnT-III-transfectant (c).

Numbers at the top indicate peaks for bisected

sugar chains: 1, asialo-agalacto-bisected

biantennary sugar chain; 2,

agalacto-bisected tetraantennary sugar chain; 3,

asialo-agalacto-bisected triantennary sugar chain

containing a b1,4-GlcNAc residue on the

Mana1,3 arm Arrowheads indicate

nonbi-sected sugar chains: left arrowhead,

overlap-ping peaks of asialo-agalacto biantennary and

asialo-agalacto tetraantennary sugar chains;

right, asialo, agalacto triantennary sugar chain

containing a b1,4-GlcNAc residue on the

Mana1,3 arm These structures are shown

above the pro®le.

Trang 8

C O N C L U S I O N S

Our present study identi®ed Asp321 and Asp323 as being

essential residues in the enzyme activity of GnT-III, and

provides further support for the view that the D-X-D motif

in glycosyltransferases is important and signi®cant, even

though the exact roles of Asp321 and Asp323 in GnT-III

remains to be elucidated On the other hand, the utility of

the catalytically inactive GnT-III as a dominant negative

glycosyltransferase toward bisecting GlcNAc formation is

clearly demonstrated The ®ndings contribute to the

estab-lishment of a distinct strategy for in vivo oligosaccharide

manipulation, which permits the speci®c inhibition of a

particular glycosylation step in the biosynthetic pathway in

a manner that is independent of gene targeting Although a

similar attempt was made for a1,3GalT [44], the mechanism

of the inhibition is not known It is generally thought that

dominant negatively acting molecules involve competition

with the corresponding endogenous wild-type molecules

Such competition could also occur in the case of the

dominant negative GnT-III Possible steps of the

competi-tion could involve: (a) competicompeti-tion for localizacompeti-tion in the

Golgi, as observed in competition for cell surface b1,4GalT-1

[45]; (b) homophilic interaction involved in the formation of

the active enzyme; (c) association with a presently unknown

molecule that activates the enzyme; and (d) activation of the

enzyme by post-translational modi®cation Although the

mechanism of action of the dominant negative mutant is not

yet known, an understanding of this mechanism could lead

to the discovery of a novel regulatory mechanism for

glycosyltransferase activity

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

We thank Dr Milton S Feather for correcting this manuscript This

research was supported, in part, by a Grant-in-Aid for Scienti®c

Research on Priority Area no 10178104 from the Ministry of

Education, Culture, Sports, Science and Technology of Japan, and

by the Sumitomo Foundation.

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