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Tiêu đề Deamidation of labile asparagine residues in the autoregulatory sequence of human phenylalanine hydroxylase
Tác giả Therese Solstad, Raquel N. Carvalho, Ole A. Andersen, Dietmar Waidelich, Torgeir Flatmark
Trường học University of Bergen
Chuyên ngành Biochemistry and Molecular Biology
Thể loại báo cáo khoa học
Năm xuất bản 2003
Thành phố Bergen
Định dạng
Số trang 10
Dung lượng 445,92 KB

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Deamidation of labile asparagine residues in the autoregulatorysequence of human phenylalanine hydroxylase Structural and functional implications Therese Solstad1, Raquel N.. Andersen2,

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Deamidation of labile asparagine residues in the autoregulatory

sequence of human phenylalanine hydroxylase

Structural and functional implications

Therese Solstad1, Raquel N Carvalho1, Ole A Andersen2, Dietmar Waidelich3and Torgeir Flatmark1

1 Department of Biochemistry and Molecular Biology and the Proteomic Unit, University of Bergen, Norway; 2 Department of Chemistry, University of Tromsø, Norway;3Applied Biosystems, Applera Deutschland GmbH, Langen, Germany

Two dimensional electrophoresis has revealed a

micro-heterogeneity in the recombinant human phenylalanine

hydroxylase (hPAH) protomer,that is the result of

sponta-neous nonenzymatic deamidations of labile asparagine

(Asn) residues [Solstad,T and Flatmark,T (2000) Eur J

Biochem 267,6302–6310] Using of a computer algorithm,

the relative deamidation rates of all Asn residues in hPAH

have been predicted,and we here verify that Asn32,followed

by a glycine residue,as well as Asn28 and Asn30 in a loop

region of the N-terminal autoregulatory sequence (residues

19–33) of wt-hPAH,are among the susceptible residues

First,on MALDI-TOF mass spectrometry of the 24 h

expressed enzyme,the E coli 28-residue peptide,L15–K42

(containing three Asn residues),was recovered with four

monoisotopic mass numbers (i.e., m/z

3108.474 and 3109.476,of decreasing intensity) that differed

by 1 Da Secondly,by reverse-phase chromatography,

isoaspartyl (isoAsp) was demonstrated in this 28-residue

peptide by its methylation by protein-L-isoaspartic acid

O-methyltransferase (PIMT; EC 2.1.1.77) Thirdly,on

incubation at pH 7.0 and 37C of the phosphorylated form

(at Ser16) of this 28-residue peptide,a time-dependent

mobility shift from tR 34 min to  31 min (i.e.,to a more

hydrophilic position) was observed on reverse-phase chro-matography,and the recovery of the tR 34 min species decreased with a biphasic time-course with t0.5-values of 1.9 and 6.2 days The fastest rate is compatible with the rate determined for the sequence-controlled deamidation of Asn32 (in a pentapeptide without 3D structural interfer-ence),i.e.,a deamidation half-time of 1.5 days in 150 mM Tris/HCl,pH 7.0 at 37C Asn32 is located in a cluster of three Asn residues (Asn28,Asn30 and Asn32) of a loop structure stabilized by a hydrogen-bond network Deami-dation of Asn32 introduces a negative charge and a partial b-isomerization (isoAsp),which is predicted to result in a change in the backbone conformation of the loop structure and a repositioning of the autoregulatory sequence and thus affect its regulatory properties The functional implications

of this deamidation was further studied by site-directed mutagenesis,and the mutant form (Asn32fiAsp) revealed a 1.7-fold increase in the catalytic efficiency,an increased affinity and positive cooperativity of L-Phe binding as well as substrate inhibition

Keywords: phenylalanine hydroxylase; microheterogeneity; deamidation; asparagine; structure and function

The irreversible,spontaneous,nonenzymatic deamidation

of asparagine (Asn) residues is a common

post-trans-lational modification known to occur in a large number of

mammalian proteins [1],and it represents an important

source of protein instability at biologically relevant

conditions [2,3] The deamidation of Asn at neutral pH has been reported to proceed primarily by a succinimide mechanism involving the formation of a succinimide intermediate via nucleophilic attack on the amide carbonyl

of Asn by the nitrogen of the peptide group linking the Asn to the following residue [4–6]

intermediate may occur on either side of the imide nitrogen,the Asp residue produced by the deamidation reaction will be linked to the subsequent residue by a normal

3 a-aspartyl (Asp) or by a b-aspartyl (or isoaspartic acid – isoAsp) bond In the latter case,the b-carbon is part of the polypeptide backbone,and the a-carboxyl group is present as an atypical one carbon carboxylic acid side-chain available for methylation by isoaspartyl O-methyltransferase [7] In general,Asn residues deami-date faster and more frequently than do glutamine residues,due to a more energetically favourable formation

of a cyclic intermediate (reviewed in [8,9]) The rate of Asn deamidation in proteins has been shown to depend primarily on their nearest (to the Asn [10]) neighbour amino acid C-terminal,their localization in the 3D

Correspondence to: T Flatmark,Department of Biochemistry and

Molecular Biology,University of Bergen,A˚rstadveien 19,

N-5009 Bergen,Norway Fax: +47 5558600,Tel.: +47 55586428,

E-mail: torgeir.flatmark@ibmb.uib.no

Abbreviations: hPAH,human phenylalanine hydroxylase; rPAH,rat

phenylalanine hydroxylase; H 4 ,biopterin (6R)- L

-erythro-5,6,7,8-tetrahydrobiopterin; PIMT,protein- L -isoaspartate

O-methyltrans-ferase; MALDI-TOF,matrix-assisted desorption/ionization time of

flight; IPTG,isopropyl-thio-a- D -galactoside; L -Phe, L -phenylalanine;

MBP,maltose binding protein; wt,wild-type.

Enzyme: Phenylalanine 4-monooxygenase or phenylalanine

hydroxylase (EC 1.14.16.1).

(Received 9 October 2002,revised 23 December 2002,

accepted 8 January 2003)

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structure [9,10] as well as on environmental factors such as

pH,temperature and ionic strength [8,11] and including

some specific ion effects [12] Two dimensional

electro-phoresis of the 50-kDa subunit of purified monkey,

human and recombinant human PAH (hPAH) has

revealed a marked microheterogeneity in which the

individual molecular forms of the protomer have the

same apparent molecular mass ( 50 kDa),but differ in

their pI by about 0.1 pH unit [13]

was proven to be the result of progressive,spontaneous,

nonenzymatic deamidations of labile amide containing

amino acid residues [13] Based on the specific

deamida-tion rates in a cellular system (expression in E coli) and

the experimental conditions in vitro required for the

deamidation reactions to occur,the labile amide groups

were concluded to represent Asn residues [13]

Interest-ingly,a comparison of the catalytic properties of

non-deamidated and highly non-deamidated enzyme revealed that

the catalytic efficiency (kcat/[S]0.5) was almost threefold

higher for the tetramer (as isolated by size-exclusion

chromatography) with multiple deamidated protomers

(generated by 24 h expression in E coli at 28C) than for

the essentially nondeamidated form (generated by 2 h

expression) [13] Therefore,the unambiguous identification

of the Asn residues susceptible to deamidation at

biologi-cally relevant conditions represents a major challenge for the

characterization and understanding of the

catalytic,regula-tory and stability properties of this homotetrameric enzyme

Using a recently developed computer algorithm [9,10], that

accurately ( 95%) predicts the relative deamidation rates

of Asn residues within a single protein,when its 3D

structure is known,several candidate Asn residues have

been predicted in hPAH and rPAH [14] Interestingly,based

on this predictive algorithm and the 2D electrophoretic

patterns of wt-hPAH and the truncated form DN(1–102)/

DC(429–452)-hPAH,two of the labile Asn residues have

been located in the catalytic domain structure In addition,

three residues in the designated N-terminal autoregulatory

sequence (residues 19–33),extending over the active site

pocket as a lid [15],were also predicted to be susceptible to

deamidation This sequence contains a cluster of three Asn

residues (Asn28,Asn30 and Asn32) as well as the

phosphorylation site for PKA (Ser16) in close proximity,

and we here present experimental data verifying the

computational prediction of Asn32 as the most susceptible

residue to undergo deamidation to Asp/isoAsp

Materials and methods

Materials

The restriction protease,enterokinase,was delivered by

Invitrogen (the Netherlands) The IsoQuant protein

isoaspartic acid detection kit was purchased from Promega

The Sigma Chemical Co delivered TPCK-treated trypsin

and soybean trypsin inhibitor The catalytic subunit of

cAMP-dependent protein kinase (PKA) was purified to

homogeneity from bovine heart and was a generous gift

from S O Døskeland,Department of Anatomy and Cell

Biology,University of Bergen A synthetic 28-residue

peptide representing the N-terminal tryptic peptide,LSD

FGQETSYIEDNCNQNGAISLIFSLK (L15–K42),was

synthesized by Research Genetics,AL,

and S-adenosyl-L-[methyl-3H]methionine were obtained from Amersham Pharmacia Biotech,U.K Other chemicals were of the highest purity available,and some specific chemicals are referred to in the text

Site-specific mutagenesis The Asn32fiAsp mutation was introduced into the pMAL-hPAH expression system containing the entero-kinase cleavage site (D4K) (New England Biolabs) using the QuikChangeTM site-directed mutagenesis kit (Stratagene) The following primers (provided by MWG-Biotech AG) were used for mutagenesis: (forward) 5¢-AAGACAACT GCAATCAAGATGGTGCCATATCACTGATC-3¢ and (reverse) 5¢-GATCAGTGATATGGCACCATCTTGATT GCAGTTGTCTT-3¢ (the mismatched nucleotides are shown in boldtype) The authenticity of the mutagenesis was verified by DNA sequencing in an ABI PrismTM377 DNA Sequencer (Perkin Elmer) using the oligonucleotides malE,13B [16] and A674[17] and the Big DyeTMTerminator Ready Reaction Mix (Perkin Elmer Applied Biosystems) The analysis of the electropherograms was carried out with the programsCHROMAS1.6 (Technelysium Pty,Ltd) andCLUSTAL X(1.8) The DNA was introduced into E coli TB1 cells by electroporation using a Gene Pulser II (Bio-Rad)

Expression and purification of recombinant hPAH The pMAL expression system was used for the production

of the wild-type fusion protein MBP-(D4K)ek-hPAH [18] with maltose binding protein as the fusion partner Cells were grown at 37C,and expression was induced at 15 C

or 28C by the addition of 1 mM isopropyl

thio-b-D-galactoside (IPTG); the cells were harvested after 2 h or

24 h of induction The fusion protein was cleaved by enterokinase for 5 h at 4C using 4 U protease per mg fusion protein,and the tetrameric forms were isolated to homogeneity by size-exclusion chromatography [18] Protein measurements

Purified enzyme was measured by the absorbance at

280 nm,using the absorption coefficient A280 (1 mgÆmL)1

cm)1)¼ 1.63 for the fusion protein MBP-(D4K)ek-hPAH and 1.0 for the isolated hPAH protein [18]

Phosphorylation of hPAH The enzyme was phosphorylated by PKA as described previously [19] The standard reaction mixture contained

15 mM Na/Hepes (pH 7.0),0.1 mM ethylene glycol bis-(a-amino ether)-N,N,N¢,N¢-tetraacetic acid,0.03 mM EDTA,1 mM dithiothreitol,10 mM MgAc2, [c-32P]ATP,

60 lM ATP,100 nMof the catalytic subunit of PKA and

20 lMof hPAH; 30C for 30 min

Trypsination of hPAH Tryptic proteolysis of hPAH was performed in 20 mM Na/Hepes buffer,pH 7.0 at 30C for 2 h at a trypsin to

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substrate ratio of 1 : 10 (by mass) Soybean trypsin inhibitor

was added at the end with a protease to inhibitor ratio of

1 : 1.5 (by mass) for the analyses of peptides by

reverse-phase chromatography

Methylation of tryptic peptides

The repair enzyme protein-L-isoaspartate

O-methyltrans-ferase (PIMT; EC 2.1.1.77) catalyses the methylation of the

a-carboxyl in isoAsp residues with S-adenosyl-L-methionine

as the methyl donor The products of this reaction are the

formation of peptide/proteinL-isoaspartartyl methyl ester

and S-adenosyl-L-homocysteine The reaction was

per-formed as described in the manual for the IsoQuant

protein isoaspartic acid detection kit with

S-adenosyl-L-[methyl-3H]methionine as the methyl donor

Reverse-phase chromatography

Reverse-phase chromatography of the tryptic peptides was

performed using a ConstaMetric Gradient System

(Labor-atory Data Control) and a 4.6 mm· 10 cm Hypersil

ODS C18 column (Hewlett Packard,USA) fitted with a

2-cm guard column of the same material Solvent A was

50 mMammonium acetate (pH 8.0) and solvent B,50 mM

ammonium acetate in 70% (v/v) acetonitrile (pH 8.0),and a

linear gradient of 10–50% solvent B was used at a flow rate

of 1 mLÆmin)1 for 60 min Samples were collected every

15 s and the elution pattern of32P-labelled and3H-labelled

peptides was analysed and resolved into individual

compo-nents,assuming a Gaussian distribution of each peptide,

using thePEAKFITsoftware program (SPSS Inc.,IL,USA);

the AutoFit-peak II-Residuals was used with the

confid-ence level set at‡ 95%

2D electrophoresis

Isoelectric focusing (IEF) was performed as described [13]

SDS/PAGE was performed at 200 V for 3–4 h on 10%

(w/v) polyacrylamide gels [20] The gels were stained by

0.5% (w/v) Coomassie Brilliant Blue R250 (Bio-Rad) in

30% (v/v) ethanol and 10% (v/v) acetic acid The apparatus

used for IEF and electrophoresis (EPS 3500XL power

supply,Protean Xi 2D Cell and Tube cell) were from

Bio-Rad Finally,the 2D gels were dried on a slab gel dryer

(Bio-Rad model 443) and scanned on a Hewlett Packard

Scan Jet 4C/T

Mass spectrometry

Matrix-assisted desorption/ionization time of flight

(MALDI-TOF) spectra and tandem (MS/MS) mass

spec-trometry of target peptides were acquired on a 4700

Proteomic Analyser (Applied Biosystems) in the reflectron

positive-ion mode,and the spectra were mass calibrated

externally The tryptic peptides were diluted (1 : 100 or

1 : 50) in 25% (v/v) acetonitrile and 0.1% trifluoroacetic

acid with a-cyano-4-hydroxycinnamic acid (Aldrich) as the

matrix Samples were spotted on the sample plate and

allowed to crystallize at room temperature The instrument

was supplied with a software tool that uses a scanning

algorithm to isotopically deconvolute the mass spectra; for

theoretical consideration see [21] The deconvolution method is particularly useful to detect labile Asn residues

in peptides as deamidation of Asn to Asp/isoAsp increases the monoisotopic mass ([M + H]+) of the peptide by only

1 Da

Assay of hPAH activity The hPAH activity was assayed as described [18],the catalase concentration was 0.1 lgÆlL)1and the enzyme was activated by prior incubation (5 min) with L-Phe The enzyme source was the isolated tetrameric forms and the reaction time was 1 min 0.5% (w/v) BSA was included in the reaction mixture to stabilize the diluted,purified enzyme The steady-state kinetic data were analysed by nonlinear regression analysis using SIGMA PLOT (Jandel Scientific Software) and the modified Hill equation of LiCata and Allewell [22] for cooperative substrate binding

as well as substrate inhibition [13,23]

Results Expression, purification and 2D electrophoresis

of recombinant hPAH The expression of wild-type hPAH and its Asn32fiAsp mutant form in the E coli pMAL-system resulted in the expected high yields of the recombinant fusion protein MBP-(D4K)ek-hPAH [18] using an IPTG induction period of 2–24 h at 28C or 24 h at 15 C After cleavage of the affinity purified fusion proteins with the restriction protease enterokinase,the tetrameric forms were isolated by size-exclusion chromatography Whereas the wild-type protomer gave a single band on 1D SDS/PAGE,this was not the case when subjected to 2D electrophoresis As described previ-ously [13,24], recombinant wt-hPAH expressed as MBP-(D4K)ek-hPAH fusion protein for 24 h at 28C revealed multiple ( 5) molecular forms of the protomer (Fig 1) that differed in their isoelectric point by about 0.1 pH unit,but shared the same apparent molecular mass ( 50 kDa) Labile asparagine residues in the regulatory domain Based on the microheterogeneity of the protomer in a double truncated form of hPAH

(DN(1–102)/DC(428–452)-Fig 1 2D-electrophoresis pattern of full-length wt-hPAH obtainedas a fusion protein after 24 h of induction in E coli at 28 °C Approximately

30 lg of enterokinase cleaved fusion protein MBP-(D 4 K) ek -hPAH was subjected to 2D electrophoresis and stained with Commassie Brilliant Blue The multiple molecular forms of the protomer (denoted hPAH I-IV [13]) differed in pI by  0.1 pH unit,but shared the same apparent molecular mass of  50 kDa.

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hPAH),expressed in E coli,it has been concluded that at

least two of the labile Asn residues are located in the

catalytic domain of the protomer [13] Furthermore,on

the basis of a nearest neighbour amino acid analysis of all

the Asn residues in wt-hPAH and taking into account the

contribution of the 3D structure (PDB accession numbers,

1PAH and 1PHZ) to the instability of Asn residues [10],

three residues in the N-terminal regulatory domain are

predicted to deamidate nonenzymatically at biologically

relevant conditions ([14] and Table 1) Notably,Asn32 in

hPAH and rPAH (and in addition,Asn8 in rPAH) is thus

predicted to be a very labile residue with a theoretical

deamidation coefficient (CD) of 0.5 and a theoretical

first-order half-time of 1.5 days in 150 mMTris/HCl,pH 7.5

at 37C ([14] and Table 1) Asn32 is located in a small cluster of Asn residues,including Asn28 and Asn30,that have predicted higher deamidation coefficients (7.9 and 5.0) and longer deamidation half-times (54 and 60 days) From Fig 2 it is seen that this cluster of Asn residues is in a loop structure and that Asn30 Od1 is stabilized by a hydrogen bond to Gln134 Ne2 The conformation of the loop structure is further stabilized by hydrogen bonds as shown

in Fig 2 As can be seen from Table 1,none of the other Asn residues in the N-terminal regulatory domain are predicted to contribute to the microheterogeneity of the wild-type protomer Asn58,which is located at the end of an a-helix (Ra1) [15],is particularly unlikely to undergo nonenzymatic deamidation [25]

Table 1 Asn residues in the N-terminal domain, their nearest neighbour amino acids, secondary structure position and their theoretical half-times for nonenzymatic deamidation/deamidation coefficients N.D.,not determined.

First-order deamidation half-times (t 0.5 )a(days)

Deamidation coefficient (C D )b

a The values were obtained from the estimated half-times of penta-peptides containing the sequence GlyXxxAsnYyyGly [1] b The values were predicted by the computer algorithm developed by Robinson and Robinson [10] based on the crystal structure data obtained for the truncated dimeric rat PAH (PDF id code,1PHZ) containing the regulatory and catalytic domains [15].

Fig 2 Stereo picture of the three Asn residues in the N-terminal autoregulatory sequence of rPAH Figure based on the crystal structure of the ligand-free,phosphorylated dimeric DC(428–452)-rPAH (PDB id code,1PHZ),which contains both the regulatory and the catalytic domains (residues 1–427) The N-terminal autoregulatory sequence is highly homologous in rPAH and hPAH,including the conserved Asn residues at positions 28,

30 and 32 [14] The Asn residues and the surrounding residues are shown by ball-and-stick representation The figure was produced using MOLSCRIPT [38].

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Mass spectrometry

To identify the labile Asn residues in the N-terminal

autoregulatory sequence,tryptic peptides of wt-hPAH

(Fig 3) were analysed by MALDI-TOF using a scanning

algorithm to isotopically deconvolute the mass spectra The

theoretical isotopic distribution was calculated on the basis

of the elemental composition (C135 H209 N34 O48 S1) for

the peptide L15–K42 (Fig 4A,D) This acidic and

hydro-phobic 28-residue tryptic peptide (containing Asn28,Asn30

and the predicted most labile residue,Asn32) revealed an

isotopic mass spectrum identical to the theoretical spectrum,

and on deconvolution,a single monoisotopic mass peak

([M + H]+) of m/z 3106.514 Da when obtained from

wt-hPAH isolated after 2 h at 28C of induction with IPTG (Fig 4B,C) Tandem (MS/MS) mass spectrometry of this peptide revealed a fragmentation pattern which iden-tified Asn residues in positions 28,30 and 32 By contrast, the same tryptic peptide obtained from wt-hPAH isolated after 24 h induction at 28C,revealed three additional monoisotopic mass peaks (m/z 3107.470,3108.474 and 3109.476) of decreasing intensity on deconvolution of the mass spectrum (Fig 4E,F)

of the additional peaks corresponds to the increase in mass

as expected from deamidation of 1,2 or 3 Asn residues, respectively The apparent intensity ratio of 3106.455 to

S (3107.470,3108.474 and 3109.476) was  4 : 3 and may give an estimate of the residual amount of nondeamidated peptide (m/z 3106.455); note,however,the lower S/N ratio

of the spectra in Fig 4E than in 4B that has an effect on the accuracy of the deconvoluted spectrum

Lability of the Asn residues in the autoregulatory sequence

The 28-residue tryptic peptide,L15–K42 with the cluster of Asn residues also contains the phosphorylation site (Ser16) which is a preferred substrate for PKA [26] 32P-labelled tetrameric hPAH (obtained after 24 h of induction at 15C

Fig 3 Tryptic peptides of wt-hPAH containing Asn28, Asn30 and

Asn32 The alternative cleavage sites for trypsin are indicated by

arrows,which may contribute to the heterogeneity of phosphopeptides

after reverse-phase chromatography (Fig 5).

Fig 4 MALDI-TOF mass spectra of the N-terminal 28-residue tryptic peptide L15–K42 obtained from wt-hPAH isolated after 2 and 24 h of induction

at 28 °C in E coli Panels A and D represent the theoretical isotopic mass spectrum of the 28-residue peptide L15–K42 (elemental composition C135 H209 N3 O48 S1) Panels B and E represent the mass spectra of this peptide obtained from the 2 h and 24 h expressed wt-hPAH at 28 C, respectively,and panels C and F represent the corresponding deconvoluted monoisotopic mass spectra ([M + H]+) Note that the S/N ratio is slightly better in the isotopic mass spectrum of B than in E.

17

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in E coli),that had been fully phosphorylated by PKA,was

digested with trypsin and subjected to reverse-phase

chro-matography that resolved one major (tR  34 min) and

some minor phosphopeptides (Fig 5,inset) The minor

components represent peptides generated by alternative

tryptic cleavage (see Fig 3) and/or the presence of a mixture

of peptides with either

(and eventually at positions 28 and/or 30) In order to study

further the stability of the three Asn residues,the

phospho-peptides were incubated at 37C and pH 7 (15 mM Na/

Hepes containing 1 mM dithiothreitol),and at timed

intervals,aliquots were subjected to reverse-phase

chroma-tography The main phosphopeptide (with tR 34 min)

revealed a time-dependent mobility shift to a more

hydro-philic position with tR 31 min By making corrections for

the decay of32P-radioactivity and any loss of peptide,the

amount of phosphopeptide with tR 34 min was found to

decrease with a biphasic time-course and with calculated

half-times of 1.9 days (r¼ 0.98) and 6.2 days (r ¼ 0.92),

assuming pseudo first-order kinetics for the deamidation [1,13,27]

Demonstration of isoaspartate in tryptic peptides

of wt-hPAH The enzyme protein-L-isoaspartate O-methyltransferase (PIMT) specifically methylates isoAsp residues in peptides and a broad range of proteins [7] at substoichiometric levels [28, 29, 30] Thus, PIMT can be used to identify isoAsp formed due to nonenzymatic deamidation or spontaneous isomerization of Asp [30] In order to detect the presence of isoAsp residues in hPAH,the wt-hPAH (obtained after 24 h induction with IPTG at 28C) was digested with trypsin, and the peptides methylated by PIMT with

S-adenosyl-L-[methyl-3H]methionine as the methyl donor and analysed

by reverse-phase chromatography The3H-labelled peptides were resolved into several components with retention times

in the range of 20–60 min (Fig 6A),with major peaks at

tR 28 min, tR 34 min, tR 36 min and tR 51 min

A major3H-labelled peptide was eluted at the end of the gradient,this was also the case for the synthetic 28-residue peptide L15–K42 (Fig 6B) In the latter case,multiple, closely spaced,but nonresolved peaks of3H-radioactivity were observed and indeed expected from the MALDI-TOF mass spectrum of the peptide as isolated (Fig 6B,inset), including the full-length form with a monoisotopic m/z of 3106.396 (theoretical monoisotopic m/z of 3106.467); such

a heterogeneity was expected on the synthesis of this 28-residue peptide and possibly also as a result of partial deamidation to Asp/isoAsp during the synthesis procedure However,the pattern of nonresolved methylated peptides eluted between 50 and 60 min (Fig 6B) demonstrated the presence of isoAsp residues that increased markedly upon its incubation in 150 mMTris/HCl buffer,pH 7.5 at 37C; i.e.,

at the standard incubation conditions previously selected for deamidation of model peptides [1]

In order to demonstrate that the phosphopeptides of wt-hPAH (Fig 5) also contained isoAsp,the 32P-labelled tryptic peptides of wt-hPAH were incubated for 1 week at

37C,pH 7.5 and then subjected to methylation by PIMT and reverse-phase chromatography The elution pattern of the tryptic peptides revealed distinctly different profiles for

32P (phosphopeptides) and3H (methylated peptides),but with some major overlapping peaks at tR  29 min,

 36 min and  39 min In addition,several minor over-lapping peaks were also observed,thus proving that the presence of one or more isoAsp residues in the N-terminal tryptic peptide of wt-hPAH are generated because of nonenzymatic dedamidation in vitro

Steady-state kinetic analysis of tetrameric wt-hPAH and its Asn32fiAsp mutant form

The progressive deamidation events observed on 24 h expression of hPAH in E coli have been shown to alter the catalytic properties of the tetrameric form when isolated

by size-exclusion chromatography [13] Partially deamida-ted enzyme (24 h induction at 28C in E coli) revealed a 3-fold higher catalytic efficiency (kcat/[S]0.5),resulting from a higher Vmax and an increased affinity for the substrate -Phe,a lower affinity for its pterin cofactor and a

Fig 5 Time-course for the spontaneous deamidation of the major

tryptic phosphopeptide of wt-hPAH containing the residues Asn28,

Asn30 andAsn32 Full-length wt-hPAH expressed for 24 h at 28 C

was phosphorylated by PKA and subjected to digestion with trypsin.

(Inset) The elution pattern of the phosphopeptides separated by

reverse-phase chromatography on a Hypersil C18 column; the column

was equilibrated with 50 m M ammonium acetate (pH 8.0) and the

peptides eluted with a linear gradient of 10–50% (v/v) of 50 m M

ammonium acetate in 70% (v/v) acetonitrile (pH 8.0) at a flow-rate of

1 mLÆmin)1 Detection of the 32 P-radioactivity revealed a

heteroge-neity of the phosphopeptides that may be related to alternative cleavage

sites for trypsin (see Fig 4) and partial deamidation(s) of AsnfiAsp

and AsnfiisoAsp; main peak at t R  34 min (Main figure) The

phosphopeptides were further incubated in phosphorylation medium

at pH 7.0 and 37 C,and at timed intervals,aliquots ( 150 lg

pep-tide) were subjected to reverse-phase chromatography Fractions

(250 lL) were collected every 15 s followed by scintillation counting

and analysis of the data by the PEAKFIT software program Each data

point represents the average value obtained in three separate

experi-ments,and the two lines were calculated by linear regression analysis

with the correlation coefficients r 1 ¼ 0.98 and r 2 ¼ 0.92 The

time-course for the remaining32P-radioactivity in the t R  34 min peak (log

d.p.m t R  34 min) was thus resolved into two deamidation half-times

of 1.9 and 6.2 days,assuming pseudo first-order kinetics.

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pronounced substrate (L-Phe) inhibition when compared to

the nondeamidated tetramer (2 h induction at 28C)

Steady-state kinetic analysis of the Asn32fiAsp mutant

form (2 h induction at 28C),demonstrated kinetic

properties that where comparable qualitatively to those

observed for highly deamidated wt-hPAH with a 1.7-fold

increase in its catalytic efficiency,a 34% decrease in the

[S]0.5-value for L-Phe,increased cooperativity on L-Phe

binding and substrate inhibition (Fig 7,Table 2)

Discussion The observed microheterogeneity of recombinant wt-hPAH

on isoelectric focusing and 2D electrophoresis (Fig 1) has been demonstrated to be the result of spontaneous non-enzymatic deamidation of labile amide containing amino acid residues and has implications both for the catalytic efficiency and stability properties of the enzyme [13] Based

on the rate of the multiple deamidation reactions,their pH and temperature dependence,the labile amide residues were concluded to be Asn,and at least two of them were found to

be present in the double truncated form DN(1–102)/ DC(428–452)-hPAH including the catalytic core domain

of the enzyme [13]

Deamidation of Asn residues can occur by several alternative mechanisms,but in proteins and peptides the most common is a nonenzymatic deamidation via the b-aspartyl shift mechanism (see Introduction),that proceeds with a high frequency in proteins at neutral to basic pH [31] Asn,followed by Gly,Ser,Thr or Lys residues are most commonly subjected to deamidation [9,31], particularly when the Asn residue is located in a flexible segment of the protein [32] Under physiological conditions (with respect to temperature,pH and ion composition) the ratio of Asp to isoAsp is reported to be 1 : 2 for Asn–Gly or Asn–Ser sequences [33],and ratios of 1 : 3 have been reported in model pentapeptides [6,34],however,conformational con-straints in the protein may have an effect on this ratio

Labile Asn residue(s) in the N-terminal regulatory domain of wt-hPAH

On the basis of nearest neighbour analyses of the Asn residue in wt-hPAH and the recently solved 3D crystal structures of rPAH and hPAH,a computer algorithm has predicted that the most labile Asn residue in rPAH/hPAH is Asn32,together with Asn8 in rPAH ([14] and Table 1) Its nearest neighbour amino acid (Gly33),in a loop structure, favours the deamidation of Asn32 with the formation of Asp and isoAsp at a ratio of  1 : 2 [33] MALDI-TOF mass spectrometry analyses have confirmed this prediction Thus,the 28-residue tryptic peptide {residues 15–42 with a theoretical monoisotopic ([M + H]+) m/z of 3106.467,see Fig 4A or D} obtained from wt-hPAH isolated after 24 h induction at 28C revealed on deconvolution of the mass spectrum four monoisotopic peaks (Fig 4F) at m/z values

of decreasing intensity (3106.455,3107.474,3108.474 and 3109.476; Fig 4F)

9 These m/z values correspond to that

expected for nondeamidated (m/z 3106.455),mono-deami-dated (+ 1 Da),double-deami3106.455),mono-deami-dated (+ 2 Da) and triple-deamidated (+ 3 Da) forms of the peptide This conclusion

is further supported by the existence of only one monoiso-topic peak (i.e.,at m/z 3106.514) for the peptide obtained from the 2 h expressed enzyme (Fig 4C) That this peptide represents the nondeamidated form,with Asn residues at positions 28,30 and 32,was confirmed by the fragmentation pattern obtained by tandem (MS/MS) mass spectrometry Thus,the MALDI-TOF spectra in Figs 4E,F are compa-tible with a progressive deamidation of the Asn residues at positions 28,30 and 32 However,attempts to further confirm this conclusion by the MS/MS fragmentation pattern of the selected precursor ion were not successful As

Fig 6 Reverse-phase chromatography of 3 H-labelledmethylatedtryptic

peptides of wt-hPAH and the synthetic N-terminal peptide, L15–K42.

(A) The pattern of 3 H-labelled methylated tryptic peptides obtained

from wt-hPAH isolated after induction for 24 h at 28 C Peptides

from  2.5 mg of enzyme were subjected to methylation and

reverse-phase chromatography as described in the legend to Fig 5 Fractions

(250 lL) were collected every 15 s and the 3 H-radioactivity was

counted (B) Approximately 100 lg of the 28-residue synthetic peptide

(L15–K42 of hPAH) was subjected to methylation by protein

iso-aspartyl methyltransferase and then to reverse-phase chromatography.

The bottom trace (thin line) represents the radioactivity profile of the

peptide as isolated and the upper trace (thick line) the profile obtained

after its incubation for 7 days at 37 C in 150 m M Tris/HCl,pH 7.5.

(Inset) The MALDI-TOF mass spectrum of the synthetic peptide

isolated with a main component of m/z 3106.396 (theoretical m/z-value

for the peptide is 3106.467) and several minor components,including

peptides with nonreleased blocking groups (in the

high-molecular-mass region).

Trang 8

a further approach to study the multiple deamidations in

this 28-residue tryptic peptide,the wt-hPAH was labelled

with 32P-phosphate (at Ser16) and the phosphopeptides

separated by reverse-phase chromatography (Fig 6,inset)

When the phosphopeptides were further incubated at 37C

and pH 7.0 (15 mM Na/Hepes),a half-time of 1.9 days

(Fig 6) was estimated for the nonenzymatic deamidation of

the major phosphopeptide (as estimated by its

time-dependent change in retention time from tR 34 min to a

more hydrophilic position (with tR 31 min) This is close

to the half-time estimated for an Asn in a pentapeptide

containing the identical nearest neighbour amino acids

xQNGx as in hPAH,i.e., 1.5 days when incubated at

37C and 150 mMTris buffer,pH 7.5 [1]

other Asn–Gly sequence in the human enzyme,our data

(Fig 6) support the conclusion that Asn32 is the most labile

Asn residue in hPAH The longer t0.5–value (6.2 days)

calculated from the second slope in Fig 6 most likely

reflects the deamidation of Asn28/Asn30 which are both

predicted to be more stable than Asn32 (Table 1),notably

Asn30 which is stabilized by a hydrogen bond to Gln134

(Fig 2) The deamidations of these Asn residues were

further substantiated by the chromatography

containing 32P-labelled S16 and 3H-labelled (methylated) isoAsp of wt-hPAH on reverse-phase chromatography and the demonstration of isoAsp in a synthetic peptide contain-ing both the full-length species,L15–K42 – of the expected monoisotopic mass 3106.467 – and a number of lower molecular mass peptides,recovered as a mixture of peptides

on reverse-phase chromatography

Deamidation of Asn32 during isoelectric focusing and 2D electrophoresis

The rapid rate of deamidation of Asn32 in wt-hPAH observed in vitro in the present study (Fig 6) explains why

we have not been able to obtain a single component on isoelectric focusing and 2D electrophoresis of the 2 h (28C) expressed enzyme [13] as expected from its MALDI-TOF mass spectrum Thus,no microheterogeneity was observed for the Asn containing peptides on MALDI-TOF and tandem (MS/MS) mass spectrometry of tryptic peptide obtained from wt-hPAH (2 h induction at 28C),

in particular not for the sequence L15–K42 (containing the

Fig 7 Effect of L-Phe (A andC) andH 4

biopterin (B andD) concentration on the catalytic activity of recombinant wt-hPAH andhPAH(Asn32fiAsp) obtainedafter 2 h

of induction in E coli at 28 °C Assay conditions and nonlinear regression ana-lysis are described in the Methods section.

In each graph,both the experimental (closed circle) and fitted (open circle) data are shown The kinetic constants obtained are presented in Table 2.

Table 2 Steady-state kinetic parameters for recombinant wt-hPAH and hPAH(Asn32fiAsp) Wt-hPAH was obtained after 2 h and 24 h of induction with IPTG in E coli at 28 C The Asn32fiAsp mutant form was obtained after 2 h at 28 C The PAH activity was assayed and the apparent kinetic constants were calculated by nonlinear regression analysis as described in the Materials and methods section The substrate concentrations were 1 m M L -Phe (H 4 biopterin variable) and 75 l M H 4 biopterin ( L -Phe variable) Values are shown as means ± SEM, n ¼ 3.

18

Enzyme

tetramer

V maxa

(nmolÆmin)1Æmg)1)

½S0:5a (l M )

k cat /[S] 0.5

(l M Æmin)1) h

Substrate inhibition

V max

(nmolÆmin)1Æmg)1)

K m b

(l M )

a The kinetic parameters were calculated from a modified Hill equation as described in the Materials and methods section b The kinetic parameters were calculated from the Michaelis–Menton equation.

Trang 9

predicted most labile residue Asn32; Fig 4B,C) hPAH

requires 20 h to reach the equilibrium position in the pH

gradient at 20 C,i.e.,conditions that favour

deamida-tion of at least Asn32 and,therefore,represents a major

contributor to the observed microheterogeneity of hPAH

isolated after 2 h of induction with IPTG in E coli [13]

Thus,the double spot seen,) e.g.,for the most basic

component of the protomers on 2D electrophoresis

(denoted hPAH I [13]) for the 24 h enzyme – (Fig 1) is

most likely explained by a minor partial deamidation of

Asn32 during isoelectric focusing for 20 h (8M urea,

 20 C) and represents deamidated protomers where

Asp/isoAsp have not yet reached the equilibrium position

in the pH gradient

12

Structural and functional implications of Asn32

deamidation

In the reported crystal structure of DC(429–452)-rPAH

(PDB id code,1PHZ) [15],Asn32 is located in the loop

structure including residues D27–G33 (Fig 2),preceding

the first b-sheet (Rb1) of the regulatory domain [15],and is

stabilized by several hydrogen bonds,including a hydrogen

bond between Asn30 Od1 and Gln134 Ne2 (see Fig 2)

Consequently,the deamidation of Asn32 is likely to affect

the higher order structure of the NH2terminus in several

ways First,on deamidation of Asn32fiAsp/isoAsp,the

resulting charge shift will lead to a repulsive electrostatic

interaction between the negatively charged carboxyl groups

of Asp/isoAsp32 and Asp84 Moreover,the presence of

isoAsp at position 32 in one of the generated isoforms will

also change the backbone conformation by adding an

extra carbon (methylene group) to the polypeptide chain

[35] Interestingly,in the reported crystal structure of

rPAH,the sequence around Asn32 revealed high

displace-ment factors of up to 105 A˚2[15] reflecting poor electron

density High displacement factors are a consequence of

dynamic disorder (vibrations of the atoms) in the crystal as

well as static disorder caused by discrepancies between the

different unit cells in the crystal Combining the crystal

structure information with the results of this study,we

conclude that the recombinant enzyme used in the

crystal-lographic study of rPAH (PDB id code,1PHZ) most likely

contained a mixture of isomeric dimer forms with either Asn,

Asp and isoAsp at position 32 in the protomers Thus,the

conserved residue Asn32 is expected to be deamidated to the

same extent in hPAH as in rPAH,as its nearest neighbour

amino acids and the loop structure are the same in the two

proteins

The perturbation of the overall conformation of the loop

structure as a result of deamidation of Asn32 to Asp/isoAsp

have immediate implications for the function of the

homotetrameric enzyme as they occur in a conformationally

sensitive part of the protein Thus,the loop structure

including Asn32 is part of the designated autoregulatory

sequence (residues 19–33) [15] extending like a lid over the

active site pocket and thus may control the access of

substrate and pterin cofactor to this site [15,36]

Phosphory-lation of hPAH at Ser16 by PKA results in a 1.4-fold

increase in the basal activity and a 1.7-fold increase in

catalytic efficiency [37] Molecular modelling based on the

crystal structure of the recombinant rat enzyme has revealed

that phosphorylation induces a local conformational change that is in agreement with the observed increased accessibility

of the substrate to the active site [37] The functional effect

of deamidation of Asn32fiAsp/isoAsp is rather similar and can be explained by a related mechanism Thus,the substitution of Asn32fiAsp by site-directed mutagenesis results in a 1.7-fold increase in its catalytic efficiency,a 34% decrease in the [S]0.5-value for L-Phe,an increased Hill coefficient of substrate binding as well as substrate inhibi-tion (Fig 7,Table 2)

13 These changes in kinetic properties are all characteristics observed because of multiple deami-dations of wt-hPAH on 24 h expression of the recombinant enzyme in E coli [13] It should be noted,however,that the main-chain loop conformation may be slightly differ-ent in the Asn32fiAsp/isoAsp deamidated form and the Asn32fiAsp mutant form,which may account for the quantitative differences observed between the kinetic pro-perties of the two events That Asn32 has a regulatory function in hPAH is further supported by experiments demonstrating that both deamidation of Asn32 and muta-tion of Asn32fiAsp are accompanied by a  20% increase

in the initial rate of phosphorylation at Ser16 by PKA [39] Thus,it appears that phosphorylation of Ser16 and deamidation of Asn32 act synergistically in the regulation

of hPAH

Acknowledgements

This work was supported by grants from the Research Council (NFR), the Norwegian Council on Cardiovascular Diseases,Rebergs legat,

L Meltzers Høyskolefond,the Novo Nordisk Foundation,the European Commission and the Fundac¸a˜o para a Cieˆncia e Tecnologia, Portugal (SFRH/BD/1100/2000) We thank Ali Sepulveda Mun˜oz for expert technical assistance in preparing the recombinant hPAH fusion proteins.

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