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Three regions of hTFAM [HMG-like domain 1 HMG1 and HMG-like domain 2 HMG2, as well as the tail region] can effect nuclear accumulation of enhanced green fluo-rescent protein EGFP fusions.

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multiple subcellular targeting signals

Viktoriya Pastukh1, Inna Shokolenko1, Bin Wang2, Glenn Wilson1and Mikhail Alexeyev1,3

1 Department of Cell Biology and Neuroscience, University of South Alabama, Mobile, AL, USA

2 Department of Mathematics and Statistics, University of South Alabama, Mobile, AL, USA

3 Institute of Molecular Biology and Genetics, Kyiv, Ukraine

Mitochondrial transcription factor A (TFAM, mtTFA)

is a member of a high-mobility group (HMG) of

pro-teins named on the basis of their electrophoretic

mobil-ity in polyacrylamide gels This group is composed of

nonhistone chromatin proteins and transcription factors

that can bind DNA either nonspecifically or in a

sequence-dependent manner [1] TFAM is encoded

in the nucleus and is synthesized on cytoplasmic

ribosomes as a precursor, which is converted, upon mitochondrial importation, into a 24.4 kDa, 204 amino acid mature form The N-terminal sequence of the pre-cursor has not been determined, and therefore it is possi-ble that translation can start on either of two N-terminal methionines, resulting in either 246 amino acid (29 kDa) or 240 amino acid (28.4 kDa) precursors [2] The mature form contains two HMG boxes,

Keywords

chemotherapy; cisplatin; etoposide;

mitochondrial transcription factor A; nuclear

localization sequence

Correspondence

M Alexeyev, Department of Cell Biology

and Neuroscience, University of South

Alabama, 307 University Blvd., MSB1201,

Mobile, AL 36688, USA

Fax: +1 251 460 6771

Tel: +1 251 460 6789

E-mail: malexeye@jaguar1.usouthal.edu

(Received 29 July 2007, revised 12 October

2007, accepted 25 October 2007)

doi:10.1111/j.1742-4658.2007.06167.x

The mitochondrial transcription factor A (TFAM) is a member of a high-mobility group (HMG) family represented mostly by nuclear proteins Although nuclear localization of TFAM has been demonstrated in some tumors and after treatment of tumor cells with anticancer drugs, the signifi-cance of these observations has not been fully elucidated Here we report that both TFAM overexpression and impairment of its mitochondrial tar-geting can result in nuclear accumulation of the protein Both M1 and M7 methionines of human TFAM (hTFAM) can be used for translation initia-tion with almost equal efficiency resulting in two polypeptides The shorter polypeptide, however, is not located in the nucleus, despite truncation in the mitochondrial targeting sequence, and both isoforms are targeted to mitochondria with similar efficiency We further demonstrate that nuclear TFAM confers significant cytoprotection against the chemotherapeutic drugs etoposide, camptothecin, and cisplatin Three regions of hTFAM [HMG-like domain 1 (HMG1) and HMG-like domain 2 (HMG2), as well

as the tail region] can effect nuclear accumulation of enhanced green fluo-rescent protein (EGFP) fusions The HMG1 domain contains a bipartite nuclear localization sequence whose identity is supported by site-directed mutagenesis However, this bipartite nuclear localization sequence is weak, and both N-terminal and C-terminal flanking sequences enhance the nuclear targeting of EGFP Finally, several mutations in the HMG1 domain increased the mitochondrial targeting of the EGFP fusions, sug-gesting that the mitochondrial targeting sequence of hTFAM may extend beyond the cleavable presequence

Abbreviations

EGFP, enhanced green fluorescent protein; HMG, high-mobility group; HMG1 and HMG2, HMG-like domains of human mitochondrial transcription factor A; hTFAM, human mitochondrial transcription factor A; MTS, mitochondrial targeting sequence; NLS, nuclear localization sequence; N ⁄ C, nucleus-to-cytoplasm; SOD2, manganese superoxide dismutase; Tc, tetracycline; TFAM, mitochondrial transcription factor A.

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HMG-like domain 1 (HMG1) and HMG-like domain 2

(HMG2) (Fig 1A), joined by a basic 36 amino acid

lin-ker and followed by a basic 27 amino acid tail The gene

for TFAM spans about 10 kb and consists of seven

ex-onsandsixintrons[3,4].Inhumanandrat,exon 5cansplice

alternatively, resulting in two TFAM isoforms [4,5]

TFAM is required for mtDNA transcription and

maintenance Inactivation of both TFAM alleles

results in embryonic lethality accompanied by severe

depletion of mtDNA [6] Tissue-specific inactivation of

TFAM in cardiomyocytes, skeletal muscle cells,

pan-creatic b-cells and pyramidal neurons is associated

with mtDNA depletion, reduced levels of

mitochon-drial transcripts, and severe respiratory chain

defi-ciency [7–11] TFAM levels generally correlate well

with mtDNA content, and upon transient depletion of

mtDNA with ethidium bromide, cellular TFAM

con-tent diminishes as well [12] Conversely, both mtDNA

and TFAM levels are restored upon ethidium bromide

withdrawal, although TFAM appears to lag behind

mtDNA [12]

Like many other members of the HMG family,

TFAM can bind DNA in a nonsequence-specific

man-ner, although it appears to show a higher affinity for

mitochondrial heavy strand promoter and light strand

promoter [13] TFAM binding to DNA induces

unwinding and bending [14], and the mitochondrial

TFAM content (approximately one TFAM molecule per 10 bp) has been suggested to be high enough for TFAM to cover mtDNA completely [15,16] This, together with TFAM’s high affinity for DNA contain-ing cisplatin adducts and 8-oxo-7,8-dihydroguanine raises the possibility of its involvement in recognition and⁄ or repair of mtDNA damage [17]

TFAM effects could be modulated by its interaction with p53 [18] and acetylation [19] Another interesting possibility is the regulation of the effects of TFAM by its subcellular targeting In the mouse and chicken, but not in the human, expression of a special nuclear iso-form of TFAM was demonstrated This isoiso-form is generated by alternative splicing of the duplicated first exons, resulting in a protein that lacks a mitochondrial

A

B

C

D

Fig 1 Nuclear localization of hTFAM and hTFAM–EGFP fusion

pro-teins upon overexpression (A) Structure of TFAM The figure is

drawn to scale The domain boundaries are in accordance with the

Entrez Protein Database entry Q00059 Numbers indicate the

amino acid position (B) Structures of constructs 1760 and 2463

encoding constitutively expressed and Tc-inducible hTFAM–EGFP

fusion proteins, respectively Numbers on the right indicate plasmid

designations Crossed ATG, deleted or mutated translation initiation

site (C) Top row: Flp-in T-Rex cells were transiently transfected

with constitutively expressed hTFAM–EGFP fusion construct

(con-struct 1760) Bottom two rows: inducible expression of hTFAM–

EGFP construct in stably transfected (single copy) Flp-in T-Rex

cells Left images: green, EGFP fusion proteins Middle images:

red, MitoTracker Red (mitochondrial stain) Right images: overlay;

yellow, regions of colocalization – Tc and + Tc, cells were either left

uninduced, or induced with 2 lgÆmL)1Tc for 48 h (D)

Accumula-tion of hTFAM in the nuclei of transfected cells upon

overexpres-sion The Flp-in T-Rex293 cells were stably transfected with

construct 2462, which encodes full-length hTFAM Nuclear

frac-tions (12 lg of total protein) from the parental cell line (T-Rex),

un-induced (2462 unind) and un-induced (2462 ind) 2462 cell line as well

as the mitochondrial fraction (Mito) from the induced 2462 cell line

were subjected to western blot analysis, using antibodies against

nuclear lamin A ⁄ B (loading control) and SOD2 to verify the purity

of the fractions (top panel), as well as with antibodies against

lamin A ⁄ B (loading control) and antibody to hTFAM to determine

levels of hTFAM in the nuclei (the bottom panel).

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targeting sequence (MTS) However, the order of

‘nuclear’ and ‘mitochondrial’ exons in genomic DNA

of these species is opposite [20–22] Nuclear

localiza-tion of TFAM was observed in rat hepatoma, where it

correlates with 10-fold overexpression of this protein

[23] Also, TFAM was isolated recently from rat liver

nuclei, where it was found to be bound to chromatin

[24] Presently, both the mechanism(s) and the

physio-logical consequences of the nuclear localization of

TFAM remain unclear Here we identify the nuclear

localization sequences (NLSs) of human TFAM

(hTFAM), and demonstrate that nuclearly localized

hTFAM can exert significant sensitizing and

cytopro-tective effects in response to chemotherapeutic drugs

Results and Discussion

HTFAM overexpression results in nuclear

localization of hTFAM–EGFP fusion proteins

As nuclear localization of TFAM correlates with

ele-vated levels of this protein in rat hepatoma cells [23],

we were interested in whether TFAM overexpression,

by itself, is sufficient for the relocalization of a fraction

of this protein to the nucleus To this end, the con-struct encoding TFAM–enhanced green fluorescent protein (EGFP) fusion protein under the control of the CMV promoter (construct 1760) was assembled and introduced into HeLa and Flp-in T-Rex293 cell lines by transient transfection In both cases, a fraction

of the fusion protein accumulated in both the cyto-plasm and the nucleus (Figs 1 and 2B) To confirm that nuclear localization of the fusion protein was indeed due to overexpression, we stably integrated an identical fusion construct under the control of a CMV-tet promoter (construct 2463) into the genome of the Flp-in T-Rex293 cell line The Flp-integrase-mediated insertion occurs in a single defined site in the Flp-in T-Rex293 genome Therefore, our stable integrants, unlike cells that received a similar construct by tran-sient transfection, contained a single copy of the fusion construct, and expressed lower levels of the fusion pro-tein In agreement with our hypothesis, the lower levels

of expression attained in the Flp-in T-Rex293-2463 cell

Fig 2 Schematic diagrams (A) and

subcellu-lar localization (B) of hTFAM deletion

con-structs The constructs were generated by

PCR and transfected using Polyfect reagent

as described in Experimental procedures.

Left images: green, EGFP fusion proteins.

Middle images: red, MitoTracker Red

(mito-chondrial stain) Right images: overlay;

yellow, the regions of colocalization.

EF1-alpha, a construct expressing unfused

EGFP.

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line in response to induction did not lead to nuclear

accumulation of the hTFAM–EGFP fusion protein, as

detectable by confocal microscopy, and instead

com-plete colocalization of the fusion protein and

mito-chondria was observed (Fig 1, yellow color in the

overlay) To rule out the possibility that EGFP

non-specifically interferes with mitochondrial targeting, we

established a similar inducible stable cell line that

expressed unfused hTFAM (construct 2462), and used

subcellular fractionation techniques in combination

with more sensitive detection by western blotting This

experiment also revealed accumulation of the hTFAM

in the nucleus in response to increased expression

(induction; Fig 1D, lower panel) This accumulation

was not due to contamination of the nuclear fraction

with mitochondria, as shown by blotting for a mito-chondrial marker, manganese superoxide dismutase (SOD2; Fig 1D, upper panel)

Translation of hTFAM can be initiated on both N-terminal methionines with similar efficiency The above observations suggest either that the two specialized isoforms of hTFAM, nuclear and mito-chondrial, are produced from a single cDNA, or that

a single hTFAM polypeptide possesses an intrinsic nuclear localization signal and is unevenly partitioned between the mitochondria and the nucleus Indeed, DNA ligase III has been shown to produce both nuclear and mitochondrial isoforms by using

alterna-B

EF1alpha

1760

1788

1789

1790

1804

1805

2078 1819 1818 1817 1807 1806

Fig 2 (Continued).

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tive translation initiation signals In this case, a

shorter, nuclear isoform lacks the first 87 amino acids

encoding the MTS [25] Similarly, hTFAM has two

methionines in its N-terminal region, M1 and M7, and

either one can potentially be used for translation

initia-tion To verify whether this is indeed the case, the

5¢-region of hTFAM cDNA, including the 5¢-UTR,

was fused in frame with the luciferase gene, and either

M1 or M7, or both, were mutated to isoleucine (Fig 3

and Experimental procedures) The luciferase assays

demonstrated that although translation initiation on

M7 occurs with somewhat lower efficiency as

com-pared to M1, these differences do not reach the level

of statistical significance (n¼ 3, two-tailed t-test;

Fig 3) To evaluate the subcellular distribution of the

shorter hTFAM variant with a truncated MTS, amino

acids 7–246 were fused to EGFP and the resulting

con-struct was transiently transfected into HeLa cells No

substantial differences in the subcellular distribution were detected between the full-length and the truncated hTFAM variants (supplementary Fig S1, 7–246) The S12T polymorphism in the MTS of hTFAM has been identified as a risk factor for Alzheimer’s disease [26]

We attempted to link this risk with altered nuclear tar-geting of the shorter hTFAM variant containing the S12T mutation However, the patterns of subcellular targeting of hTFAM(7–246) and hTFAM(7–246)S12T were essentially identical (supplementary Fig S1) As both full-length and truncated [hTFAM(7–246)] products are efficiently targeted to mitochondria, the existence of a shorter hTFAM variant cannot account for the nuclear accumulation of the EGFP fusion pro-teins Therefore, it is more likely that intrinsic NLS(s) mediate the nuclear accumulation of hTFAM

HTFAM possesses multiple NLSs

To determine whether hTFAM possesses intrinsic NLS(s), a series of 5¢- and 3¢-deletions were introduced into the hTFAM gene (Fig 2) All three 3¢-deletions (constructs 1788, 1789, and 1790) retained both the MTS and HMG1 domain and demonstrated promi-nent mitochondrial localization of the fusion proteins with some nuclear fluorescence In contrast, all 5¢-dele-tions lacked the MTS and exhibited predominantly nuclear and⁄ or cytoplasmic fluorescence We further fused individual hTFAM segments (HMG1, linker, HMG2, tail) to EGFP to locate putative NLS(s) Sur-prisingly, three of the four constructs tested (HMG1, HMG2, and tail fusions) accumulated in the nucleus, suggesting the presence of NLSs The strength of these signals can be ranked on the basis of nuclear⁄ cyto-plasmic partitioning of the fusion proteins as HMG1 > tail > HMG2 (Fig 2B) Another unex-pected result was that in approximately 2% of the cells expressing the HMG1–EGFP fusion protein, a fraction

of the fusion protein was localized to mitochondria, suggesting that mitochondrial targeting determinants

of hTFAM may extend beyond the cleavable MTS

Lys96 and Lys97 are critical for the nuclear targeting of HMG1

Human SRY protein, a nuclear transcription factor expressed early in embryonic development, is arguably the best studied member of the HMG family [27] SRY contains two distinct NLSs, at either end of a single HMG box Both NLSs are highly conserved in SRY among mammals and are believed to be required for complete nuclear localization [28] The N-terminal NLS is bipartite and consists of two clusters of

Fig 3 Translation initiation efficiency at M1 versus M7 of hTFAM.

(A) The structure of the reporter constructs The bent arrow

indi-cates the initiating methionine (B) Levels of luciferase activity

when initiated at either M1 or M7 Activities were normalized for

transfection efficiency using cotransfection with Renilla luciferase

and a dual luciferase assay system.

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positively charged residues separated by 12 amino

acids (Fig 4B) Whereas the first of these clusters is

conserved in both HMG1 and HMG2 as well as in all

TFAMs aligned in Fig 4B, the second cluster is absent

in mammalian TFAMs As both HMG1 and HMG2

of hTFAM appear to possess NLSs, amino acid

resi-dues conserved between either HMG1 and HMG2

(Fig 4A), or between HMG1 domains of TFAMs

from different species (Fig 4B), were interrogated by

site-directed mutagenesis to identify residues that may

constitute the HMG1 NLS (Table 1; Fig 4;

supple-mentary Fig S2) Mutations in only four (K51, E63,

P73, and E106) of the 12 residues that are invariant

between HMG1 domains in all TFAMs aligned in

Fig 4B had no effect on subcellular redistribution of

EGFP fusion proteins (Table 1; supplementary

Fig S2) Of the eight remaining residues, mutations in

five (P50, P53, Y57, R104, and Y110) resulted in a

sig-nificant impairment of nuclear accumulation of fusion

proteins, implying involvement of these residues in

nuclear targeting Interestingly, mutations in three

invariant residues (P66, W88, and K96) resulted in a

significant increase in the proportion of cells that

displayed mitochondrial partitioning of EGFP fusion proteins (Table 1; Fig 5A) On closer examination, a putative bipartite NLS that consists of the R82-R83 duet and the K95-K96-K97 triplet separated by a spacer of 11 amino acids (Fig 4A) was found in the HMG1 domain Consistent with this observation, a double mutation K96A + K97A completely elimi-nated nuclear localization of the HMG1 domain (Table 1) The nucleus-to-cytoplasm (N⁄ C) index in cells transfected with this mutant was not statistically different from that of the cells transfected with a con-struct encoding unfused EGFP (results not shown) However, this putative NLS, by itself, was unable to effect nuclear accumulation of EGFP fusion proteins (Fig 4A, construct 2163), and both N-terminal and C-terminal flanking sequences enhanced nuclear target-ing of EGFP by this NLS (Fig 4A, constructs 2208 and 2209, respectively) Unlike mutations W88R and Y99A in HMG1, which resulted in increased mito-chondrial localization of the fusion proteins, the corre-sponding mutations W189R and Y200A in HMG2 did not result in any detectable mitochondrial localization, and led instead to increased nuclear accumulation

Fig 4 Alignments of HMG domains from various sources (A) Alignment of HMG1 and HMG2 domains of hTFAM The invariant amino acid residues are in bold and italic The solid black lines below alignment indicate boundaries of deletion constructs, whose designations appear

to the left The P-values, which appear in the brackets next to construct designations, are from one-way ANOVA with Dunnett’s post hoc test comparisons with a construct expressing cytoplasmic EGFP The statistically significant difference indicates nuclear accumulation of corre-sponding EGFP fusion constructs The HMG1 and HMG2 amino acid residues interrogated by site-directed mutagenesis are indicated by arrows above and below the alignment, respectively The brackets above the alignment designate components of the putative HMG1 NLS (B) Alignment of human SRY versus TFAMs from various species The invariant amino acid residues are in bold and italic Amino acid resi-dues interrogated by site-directed mutagenesis are indicated by arrows below the alignment The components of SRY bipartite NLS are indi-cated by the brackets above the alignment, and corresponding amino acid residues in TFAMs from different species are indiindi-cated by brackets below the alignment.

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(Table 1; Fig 5; supplementary Fig S3) In general,

unlike HMG1 mutations, none of the mutations in the

HMG2 domain led to detectable mitochondrial

parti-tioning of EGFP fusion proteins (Table 1) Mutations D207A and E214A in HMG2, which affect residues cor-responding to E106 and E113, respectively, behaved like their HMG1 counterparts and did significantly affect nuclear localization (Table 1; supplementary Fig S3)

Mitochondrial targeting determinants of hTFAM may extend beyond the cleavable MTS

Perhaps the most unexpected finding of the site-direc-ted mutagenesis experiments was that several HMG1 mutations resulted in a significant increase in mito-chondrial targeting of the HMG1–EGFP fusion proteins As mentioned above, in 2% of cells, HMG1– EGFP fusion proteins are partially localized to mito-chondria Mutations P66R, P66A, W88R, W88A, W88A + D93A, K96I and Y99A (Table 1; Fig 5A) significantly increased the fraction of cells with mito-chondrial partitioning of the HMG1 fusion proteins The effect of the P66R and W88R mutations was addi-tive, and the HMG1 P66R + W88R double mutant localized to mitochondria in 60% of transfected cells,

as judged by subcellular distribution of its EGFP fusion protein (Fig 5A) Mitochondrial relocalization

of mutatnt HMG1–EGFP fusion proteins did not pre-vent nuclear targeting of the same constructs, and dual mitochondrial and nuclear localization was typically observed (Fig 5A) Interestingly, the P66E mutation, unlike P66R and P66A, did not cause increased mitochondrial localization of fusion proteins (Fig 5A) This is consistent with the notion that N-terminal posi-tively charged amphiphilic a-helices, which are poor in aspartic and glutamic acid residues, serve as mitochon-drial targeting signals [29] Further supporting the notion that mutant HMG1 domains are targeted to mitochondria using determinants similar to those found in the N-terminal presequences, the placement

of HMG1 P66R + W88R at the C-terminus of EGFP completely abolished the mitochondrial targeting effect

of the mutations, while having no effect on nuclear targeting of this fusion protein (Table 1; Fig 5A) Finally, subcellular fractionation of cells transfected with either wild-type HMG1–EGFP fusion construct

or with the double P66R + W88R mutant HMG1– EGFP fusion construct has revealed increased mito-chondrial accumulation of EGFP in cells transfected with mutant construct This accumulation was accom-panied by the presence of putative processing products

in both whole cell lysates and in purified mitochondria (Fig 5B) Such products are characteristic of precursor proteins cleaved by mitochondrial processing pepti-dase, which removes presequences to produce mature mitochondrial proteins Collectively, these results

Table 1 Effects of mutations in HMG domains on their subcellular

distribution ND, the N ⁄ C ratio was not determined for mutants

playing mitochondrial retargeting, due to the existence of two

dis-crete populations of transfected cells; flN, decreased nuclear

accumulation; ›N, increased nuclear accumulation; ›M,

mitochon-drial redistribution of fusion proteins; ››M, strong mitochondrial

redistribution of fusion proteins; NS, not significantly different from

the wild type (WT).

Mutation(s)

N ⁄ C index (mean ± SEM) P-value (n)

Trend in the subcellular redistribution

of mutants EGFP (EF1-alpha) 1.01 ± 0.04

HMG1–EGFP fusion proteins

P50G + Y110C 2.71 ± 0.32 P < 0.05 (8) flN

P50G + E112A 8.17 ± 2.2 P > 0.05 (6) NS

K96A + K97A 1.12 ± 0.08 P < 0.01 (8) flN

E106A, E113A 2.24 ± 0.24 P < 0.01 (8) flN

HMG2–EGFP fusion proteins

D207A, E214A 1.63 ± 0.06 P > 0.05 (8) NS

EGFP–HMG1 fusion proteins

P66R + W88R 4.36 ± 0.68 P > 0.05 (8) NS

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B

Fig 5 Increased mitochondrial partitioning

of some HMG1 mutants (A) Partitioning as

observed by fluorescence microscopy Cells

with mitochondrial localization of mutants

are indicated by white arrows Note that

P66A and P66R mutations, but not P66E

mutations, increase mitochondrial targeting

of HMG1–EGFP fusion proteins Left

images: green, EGFP fusions Middle

images: red, MitoTracker Red (mitochondrial

stain) Right images: overlay; yellow,

regions of colocalization (B) Partitioning as

observed by subcellular

fraction-ation ⁄ western blotting HEK293FT cells

were transfected with either construct 1817

(wild-type HMG1–EGFP fusion construct,

Fig 3) or construct 1925 [P66R + W88R

HMG1–EGFP fusion construct (A)], and 48 h

after transfection, cells were lysed to

pro-duce whole cell (wc) lysates, or

mitochon-dria were isolated using a Pierce

mitochondrial isolation kit Twenty

micro-grams of wc lysates and 10 lg of

mitochon-drial fraction (mito) were separated by

SDS ⁄ PAGE and subjected to western

blot-ting with antibody to mitochondrial HSP60

(a-HSP60, loading control) or antibody to

GFP (a-GFP) Asterisk: putative processing

products cleaved by mitochondrial

process-ing peptidase, which removes MTS from

mitochondrial precursor proteins.

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indicate that a cryptic mitochondrial targeting

determi-nant may be present in the HMG1 domain This

deter-minant is likely to play an accessory role in the

context of the full-length protein Taken out of that

context, this signal, by itself, is insufficient to effect

mitochondrial localization of EGFP fusion proteins

However, as a consequence of mutations in the HMG1

domain, this determinant can be strengthened,

result-ing in the retargetresult-ing of the EGFP fusion proteins to

mitochondria The role of this cryptic determinant in

the mitochondrial import of hTFAM remains to be

determined It is likely that it acts cooperatively with

the MTS to effect the mitochondrial localization of the

mature polypeptide Importantly, we found no

evi-dence for the presence of a similar cryptic determinant

in the HMG2 domain

Nuclearly targeted hTFAM exerts cytoprotective

effects

TFAM has been found to preferentially bind to DNA

damaged by the genotoxic drugs cisplatin and

N-acet-oxyacetylaminofluorene [17,24,30] This, in

combina-tion with observacombina-tions of TFAM accumulacombina-tion in

transformed cells [23], and its presence in nuclear

extracts of normal liver cells [24], raises the possibility

of the involvement of hTFAM in cellular responses to

chemotherapy [31,32] Indeed, HMG proteins have

been reported to both impede [33,34] and enhance [35–

37] repair of damaged DNA Therefore, we established

a cell line with tetracycline (Tc)-inducible expression

of nuclear, MTS-less, hTFAM (construct 2476) Upon

the induction of nuclear hTFAM synthesis, the

suscep-tibility of this cell line to treatment with three different

chemotherapeutic drugs, etoposide, camptothecin, and

cisplatin, was tested As compared to the similarly

treated parental cell line, the susceptibility to treatment

with etoposide, camptothecin and cisplatin was

decreased by 6.8%, 3.9% and 9.6%, respectively, in

the 2476 line (Table 2) Therefore, although nuclear

hTFAM may affect a tumor’s susceptibility to chemo-therapy, and may represent a defensive mechanism, the amplitude of this response with the drugs tested is too low to be of practical significance

The mitochondrial localization of hTFAM may rep-resent an example of ‘eclipsed distribution’, the phe-nomenon of uneven protein distribution between two

or more cellular compartments, where accumulation of protein in one compartment impedes its detection in another [38] Nsf1 protein, which is involved in the maturation of FeS proteins in mitochondria, represents

a prototypical example of such distribution Similar to that of hTFAM, the nuclear localization of Nsf1 pro-tein is undetectable by physical means However, it has been demonstrated that Nsf1 possesses an internal NLS, and that impairment of either nuclear or mito-chondrial targeting of Nsf1 is lethal [39,40] The embryonic lethality of the TFAM knockout [6] appears

to extend the similarity between these two proteins However, more studies are needed to identify the exact physiological role of nuclear TFAM

Experimental procedures Plasmids

pEF1a is a pcDNA3-derived plasmid in which the elonga-tion factor 1a promoter drives expression of the EGFP gene The plasmid encoding full-length cDNA of hTFAM was purchased from Open Biosystems (Huntsville, AL) hTFAM fusion, deletion and mutant constructs were assembled under the control of the CMV promoter Con-structs for generation of Tc-inducible cell lines were gener-ated in a modified pcDNA5⁄ FRT ⁄ TO vector

Site-directed mutagenesis and gene fusion Site-directed mutagenesis was performed by an overlap extension method [41] using Taq and Vent DNA poly-merases All mutations were verified by sequencing For all C-terminal fusions with HMG domains, an EGFP gene lacking the initiating ATG codon was used The ATG-less EGFP gene was generated by PCR, cloned, and sequenced This was done to exclude expression of unfused EGFP by means of leaky ribosomal scanning

Cell culture and transfection HeLa and Flp-in T-Rex293 cells were grown in DMEM supplemented with 10% fetal bovine serum, 100 unitsÆmL)1 penicillin, and 100 lgÆmL)1streptomycin Cells were seeded into 35 mm tissue culture dishes at a density of 3· 105cells per dish, and transfections were performed using Polyfect

Table 2 Effect of nuclear hTFAM expression on susceptibility to

treatment with genotoxic drugs.

Drug

2476 Mean a

(%)

SEM (%)

P-value (n ¼ 3)

a

Viability as compared to the parental cell line.

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transfection reagent (Qiagen, Valencia, CA) according to

the manufacturer’s recommendations Cells were observed

by confocal microscopy 40 h after transfection

Generation of inducible cell lines

Cell lines with Tc-inducible expression of hTFAM or its

derivatives were generated with the help of a Flp-in T-Rex

system according to the manufacturer’s recommendations

(Invitrogen, Carlsbad, CA) Protein expression was induced

with 2 lgÆmL)1Tc for 48 h

Subcellular fractionation

Cells were collected by trypsinization, washed with NaCl⁄ Pi,

and resuspended in buffer A (10 mm Hepes, pH 7.9, 10 mm

KCl, 5 mm MgCl2), to which NP40 was added to a final

con-centration of 0.4% Cells were vortexed for 1 min, and 1 m

sucrose in buffer A was added to a final concentration of

200 mm to make the solution isotonic Nuclei were collected

by centrifugation at 850 g for 3 min at 4C and washed in

the same buffer with sucrose The supernatant was

centri-fuged at 15 000 g for 10 min at 4C to pellet mitochondria

Nuclei and mitochondria were lysed in 10 mm Tris (pH 8.0),

1 mm EDTA, and 0.5% SDS, and sonicated, and the protein

concentration was determined by the Bradford method

Cell viability studies

The effect of expression of the hTFAM derivatives on cell

viability in response to various drug treatments was

evalu-ated using Alamar Blue fluorescence

Microscopy

Confocal microscopy was performed on live cells using a

Leica DM RXE microscope and a TCS SP2 confocal system

(Leica Microsystems Inc., Bannockburn, IL) in combination

with a 63· water immersion objective Prior to microscopy,

mitochondria were stained with 200 nm MitoTracker Red

(Invitrogen) for 15 min at 37C in an atmosphere of 5%

CO2 The nuclear accumulation of EGFP fusion proteins

was quantitated using the N⁄ C distribution index To

calcu-late this index, average fluorescence intensities (pixel

densi-ties) in nuclear and cytoplasmic regions were determined

with the image j program (National Institutes of Health),

and nuclear fluorescence was divided by cytoplasmic

fluores-cence Statistical analyses of N⁄ C indices were performed

using one-way anova with Dunnett’s post hoc test

Luciferase assays

The pGL3 basic reporter plasmid was modified by

intro-ducing the CMV promoter and by removing the N-terminal

methionine of luciferase The latter modification makes luciferase expression dependent upon the upstream methio-nine, which can be provided by a fusion partner Then,

143 bp of hTFAM cDNA encompassing the 5¢-UTR and the first 21 bp of the hTFAM gene was cloned upstream

of, and in frame with, the luciferase gene Finally, three constructs were generated by replacing either M1, M7 or both with isoleucine (constructs 1969, 1970 and 1971, respectively) Luciferase assays were performed using a dual-luciferase reporter assay system (Promega, Madison, WI) This system allows for the internal normalization of results using cotransfection with a second plasmid encoding Renilla luciferase The light output was measured using a TD-20 luminometer (Turner BioSystems, Inc., Sunnyvale, CA)

Susceptibility to anticancer drugs Flp-in T-Rex293 cells were stably transformed with con-struct 2476, which encodes an MTS-less mature form of hTFAM The resulting cell line, 2476, accumulates hTFAM

in the nucleus in response to Tc induction It was plated at

100 000 cells per well and pretreated with Tc for 24 h, where necessary Subsequently, cells were subjected to one

of four treatments: (a) carrier (dimethylsulfoxide) alone; (b)

Tc (2 lgÆmL)1) alone; (c) drug (etoposide, 20 lgÆmL)1; camptothecin, 20 lgÆmL)1; or cisplatin 75 lgÆmL)1) alone; and (d) drug plus Tc for 24 h Viability was determined using Alamar Blue fluorescence The fluorescence readings from each cell line that received the four different treat-ments (triplicate wells) were normalized as follows: dividing the reading obtained under treatments 2, 3 and 4 in each experiment by the average of the triplicate readings obtained under treatment 1 This normalized the readings

in different experiments on different days and made the changes in readings comparable A four-way anova was used to evaluate the effects of nuclear hTFAM expression The mean changes, together with the SEMs, were also com-puted and listed on the basis of the normalized data obtained under treatment 4 between Flp-in T-Rex293 and

2476 The analyses were performed with the sas 9.1 soft-ware package (SAS Institute Inc., Cary, NC)

Acknowledgements The work in G L Wilson’s laboratory was supported

by National Institutes of Health Grants ES03456 and AG19602

References

1 Bustin M (1999) Regulation of DNA-dependent activi-ties by the functional motifs of the high-mobility-group chromosomal proteins Mol Cell Biol 19, 5237–5246

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