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Chapter II Materials and methods

2.5 Western blot

Whole cell lysates are extracted by lysis buffer at 4 for 15 minutes. Lysates℃

were cleared of debris by centrifugation at 14,000 rpm (Eppendorf) for 15 minutes.

Equal amounts of samples were resolved in 7 % or 10 % sodium dodecyl

sulfate-polyacrylamide gels (SDS-PAGE), followed by transfering to polyvinylidene

difluoride membranes (PVDF) and immunoblotting with the indicated antibodies.

15 2.6 Cytosolic and nuclear extracts

Cells were washed twice with 1XPBS, and then scraped off of the dish and the

cell pellets were obtained by centrifugation (300 xg, 5 minutes, 4℃). Cells was

resuspended in RSB-G40 buffer and nuclei were centrifugated at 10,000 xg to obtain

the cytosolic supernatant. Nuclear extraction was obtained from resuspend nuclei by

using nuclear extraction buffer.

2.7 Co-immunopreciptation (CoIP)

Equal amount of whole cell lysetes were immunopreciptated using anti-HA

antibodies in cell lysis buffer, which was conjugated with protein G beads (Millipore).

Western blotting was performed as described in 2.5 using the indicated antibodies.

2.8 Chromatin immunoprecipitation (ChIP)

MEFs were stimulated with IFN-α4 for 30 minutes, and then fixed in 1.4 %

formaldehyde for 15 minutes at room temperature. Cells were lysed with

immunoprecipitation buffer prior to sonication (Nelson et al., 2006). Chromatins were

sheared by sonication with a Vibra-Cell VCX 130 sonicator (Sonics & Materials).

Cells were sonicated using 1 sec on/ 1 sec off pulses for 5 min at 70% power output to

shear the DNA to ~200 to 500 base pairs. Protein G-Sepharose beads (Millipore) were

16

added to cell lysates preincubated with corresponding antibody overnight at 4°C.

After extensive washes, bead-bound DNA was reverse-crosslinked by incubation

overnight at 67°C. Protein were removed by incubating with 20 μg proteinase K in

proteinase K buffer at 55°C for 4 hours. Recovered DNA from ChIP was analyzed by

QPCR using primers specific for corresponding ISRE elements in the promoters of

the indicated genes. Primer sequences are as follows:

(1) ISRE of IFIT1 promoter

Forward: 5'- GTGGAGAATGCAGTAGGGCAAAC -3'

Reverse: 5'- GTCACACCAACTGGAAGCTCAGG -3'

(2) ISRE of MDA5 promoter

Forward: 5'-ACCAAAGTCCTCACCTAAC-3'

Reverse: 5'-TATTGCCTTCCACCCAC-3'

2.9 In vitro antiviral state assay

MEF cells were pretreated with or without 2-fold serial dilution of IFN-α4 from

240 IU/ml for 24 hours. Cells were infected with EMCV at am MOI of 0.1. After

infection for 18 hours, the medium was removed and cells were fixed with 10 %

formaldehyde for 20 minutes at RT. After fixation, cells were visualized with crystal

violet. The excessive dye was then removed by immersing the plate in water.

17

2.10 Single primer based site-directed mutagenesis

pLPC-FH2-mSTAT3 was subjected to site-directed mutagenesis using one

primer in a single cloning step (Makarova et al., 2000). After PCR, the construct was

digested by DpnI twice to remove the parental unmutated strand DNA and then

transformed into Ecoli.. DNA sequencing was performed to confirm the mutation

sites.

The primers used for mutagenesis are as follows

(1) K49Q: GGCATATGCAGCCAGCCAAGAGTCACATGCCAC Tm= 60 ℃

(2) K87Q: CAACCTTCGAAGAATCCAGCAGTTTCTGCAGAG Tm= 60 ℃

(3) K49R: GCATATGCAGCCAGCAGAGAGTCACATGCCACG Tm= 60 ℃

(4) K87R: AACCTTCGAAGAATCAGGCAGTTTCTGCAGAGC Tm= 58 ℃

(5) K685R: GAGGAGGCATTTGGAAGGTACTGTAGGCCCGAG Tm= 56 ℃

PCR conditions are as follows

95 , 5 min. 98 ℃ ℃, 20 sec. Tm ℃, 20 sec. 72 ℃ 4 min. 72 ℃, 10 min.

2.11 Statistics

A student’s T test (two-tailed) was performed for statistical analysis.

18

Chapter III Results

3.1 STAT3 negatively regulates type I IFN-mediated response

Using STAT3KO MEFs, we have previously reported that STAT3 could suppress

type I IFN-mediated antiviral response (Wang et al., 2011). Since WT MEFs and

STAT3KO MEFs were generated from different mice, it is likely that the epigenetic

modifications are different, which may have additional effects independent of STAT3.

In this study a different approach was taken, STAT1 and STAT3 double knockout

(DKO) MEFs were restored with STAT1, STAT3, or both molecules. In principle, the

genetic makeup of different STAT-restored DKO MEFs should be similar. DKO

MEFs restored with empty vector (EV), Flag- and HA-tagged STAT1 or STAT3, or

both molecular (Fig. 1) with retroviral transduction were stimulated with IFN-α4 for

30 minutes (Fig. 2). STAT1 or STAT3 was expressed in WT, STAT1-restored,

STAT3-restored, and STAT1/3-restored DKO MEFs, and all of their STAT1 or STAT3

were phosphorylated in response to IFN-α4. As expected, STAT1 and STAT3 can also

be detected by anti-Flag and anti-HA antibodies in the restored cells. More

importantly, the phosphorylation of STAT1 and STAT3 in the restored cells was

comparable to that in WT MEFs. Interestingly, phosphorylation of STAT2 was

increased in cells restored with STAT1. These results suggested that DKO MEFs had

19

been successfully restored with Flag- and HA-tagged STAT1 and/or STAT3. We next

examined the functions of the restored STAT1 and STAT3 in these cells. Expression

of STAT1- or STAT3-downstream genes was measured by RT-QPCR. After IFN-α4

stimulation for 1 hour and 2 hours, Socs3 and JunB, two STAT3-dependent genes,

were upregulated, respectively, in STAT3- and STAT1/3-restored DKO MEFs (Fig. 3).

ISGs, including PKR, IP-10, IRF7, IFIT1, and IFIT2, were induced in STAT1 and

STAT1/3-restored DKO MEFs. Consistent with previous studies, the expressions of

ISGs were decreased in the STAT1/3-restored DKO MEFs as compared to

STAT1-restored DKO MEFs (Fig. 4). Besides, we also perform microarray to do

whole gene profiling, and found that there were 137 type I IFN-induced genes, such

as STAT2, IRF1, OAS2, and MX1, displaying the same phenotype (Fig. 5). The

scatterplot, which showed relationship of gene expression, showed that it a good

approach to do the experiment (Fig.6 and Fig. 7A). It revealed that STAT3 could exert

suppressive effect in STAT1/3-induced DKO MEFs as compared with STAT1-restored

DKO MEFs (Fig. 7B). Furthermore, we used antiviral state assay to confirm negative

effect of STAT3 on type I IFN-mediated antiviral response. As shown in Fig. 8 EV

and STAT3-restored DKO MEFs were susceptible to EMCV infection, which was due

to the absence of STAT1. STAT1- and STAT1/3-restored DKO MEFs were resistant to

EMCV infection. Nevertheless, single restoration of STAT1 showed more resistant to

20

EMCV infection than did STAT1/3 double restoration in DKO MEFs. These results

indicate that the phenotype of STAT1- and STAT3-restored DKO MEFs indeed

mimicked the phenotypes seen in STAT3KO and WT MEFs, respectively.

3.2 Suppression of type I IFN response by STAT3 is independent of phosphorylation and nuclear translocation of STAT1 or STAT2

Negative regulation of type I IFN by STAT3 may due to competition for receptor

occupancy to decrease the phosphorylation of STAT1 or STAT2. To examine this

possibility, STAT-restored DKO MEFs were treated with IFN-α4 for different times

and activation of STAT1 and STAT2 was assessed. As shown in Fig. 9,

phosphorylation of STAT1 was transient and decreased in the time-dependent manner,

and the activation of STAT2 was prolonged and remained, at least, for 18 hours.

However, STAT1/3-restored DKO MEFs failed to alter IFN-α-induced activation of

STAT1 and STAT2 as compared to STAT1-restored DKO MEFs. These results

suggested that STAT3 did not affect kinetics of phosphorylation of STAT1 or STAT2,

and it also implied that STAT3 does not compete receptor occupancy with STAT1 or

STAT2. We next investigate whether STAT3 influences nuclear translocation of

STAT1 or STAT2. After IFN-α4 treatment, the level of nuclear STAT1 or STAT2 was

comparable in STAT1- and STAT1/3-restored DKO MEFs, suggesting that STAT3 did

21

not alter nuclear translocation of activated STAT1 and STAT2 following stimulation

(Fig. 10).

3.3 STAT3 suppresses type I IFN-mediated response through blocking the recruitment of ISGF3 complex to ISRE in the ISGs promoters

Since STAT3 did alter phosphorylation or nuclear translocation of STAT1 and

STAT2, we next examined whether STAT3 influenced the binding of ISGF3 complex

binding to ISRE containing promoter using ChIP assay. After IFN-α4 stimulation,

STAT1-containing ISGF3 complex was recruited to the ISRE of IFIT1 and MDA5 in

STAT1-restored DKO MEFs. However, the binding of STAT1 on ISRE site of IFIT1

and MDA5 was reduced in STAT1/3-restored DKO MEF as compared to

STAT1-restored DKO MEFs (Fig. 11), suggesting that STAT3 attenuated ISGF3

complex binding and/or recruitment to the promoter of ISRE of ISGs.

3.4 STAT3 negatively regulates type I IFN-induced gene expression through acetylation-dependent mechanism by HDAC inhibitor

It has been reported that the interaction of STAT1 with p300 can increase the

binding ISGF3 complex to ISRE (Zhang et al., 2005b). However, whether STAT1 and

histone of ISRE promoter require acetylation is still controversial. We next

investigated if acetylation was involved in the suppressive effect of STAT3 by using a

22

HDAC inhibitor, SAHA. The results showed that ISRE gene expression decreased in

the high dose of SAHA treatment, but in 0.1 μM SAHA stimulation, type I

IFN-mediated gene induction decreased only in STAT1- but not STAT1/3-restored

DKO MEFs (Fig. 12). These results suggested that STAT3 may regulate type I IFN

signaling response through acetylation mechanism.

3.5 Lysine 49 and lysine 87 of STAT3 are important for inhibition of type I IFN-mediated gene induction

We have previously shown that NTD alone is sufficient to suppress

STAT1-dependent gene expression (Wang et al., 2011). NTD of STAT3 has two lysine

residues at positions 49 and 87. Acetylation of Lys 49 and 87 is shown to be critical

for STAT3 activation (Ray et al., 2005). K to R mutations in these two positions

decreases the interaction of STAT3 with p300 and HDAC1 (Hou et al., 2008; Ray et

al., 2008). Therefore, we next examined whether acetylation at NTD of STAT3 is

crucial for the suppressive effect of STAT3. Site-directed mutagenesis was performed

to generate STAT3 acetylation-deficient K49R or/and K87R (STAT349R, STAT387R,

and STAT3RR) mutants, and STAT3 acetylation mimics K49Q or/and K87Q

(STAT349Q, STAT387Q, and STAT3QQ) mutants. EV, WT STAT3, or mutant STAT3s

was transfected into STAT3KO MEFs, and the protein expression level was shown to

23

be comparable (Fig. 13). We further detected acetylation of mutant STAT3, and found

RR mutant STAT3 can not be acetylated (Fig. 14). WT STAT3, STAT349Q, STAT387Q,

and STAT3QQ could induce Scos3 and JunB expression in response to IFN-α4

stimulation, while the expression was diminished in STAT349R, STAT387R, and

STAT3RR mutant-restored cells (Fig. 15). However, both STAT3 acetylation-deficient

and STAT3 acetylation mimics mutant failed to negatively regulate type I

IFN-triggered gene expression except for STAT387R mutant (Fig. 16). These results

suggested that suppression effect of STAT3 is dependent on posttranslational

modification of Lys49 and Lys87.

3.6 Acetylation of STAT3 at Lys 685 also plays a critical role for suppression of type I IFN-induced gene production

K685 of STAT3 blocks acetylation, decreases STAT3 downstream gene induction,

and inhibits the interaction with DNMT1 (Lee et al., 2012; Yuan et al., 2005). Hence,

we further investigated whether K685 of STAT3 is involved in suppression of type I

IFN-mediated gene expression. K685R mutant STAT3 was transfected to STAT3KO

MEFs, and revealed that STAT3685R also failed to suppress type I IFN-mediated gene

expression as compared to WT STAT3 (Fig. 17), suggesting that K49, K87, and K685

of STAT3 are important to exert suppressive function.

24

Chapter IV Discussion

Using gain-of-fuction approach by restoring STAT1 and/or STAT3 into DKO

MEFs, we have confirmed the regulatory role of STAT3 in type I IFN response.

STAT1-restored DKO MEFs induced higher level of ISGs expressions and were more

resistant to EMCV infection than STAT1/3-restored DKO MEFs (Fig. 4, Fig. 5, and

Fig. 8). The negative effect of STAT3 is not working through activation or nuclear

translocation of STAT1 and STAT2 (Fig. 9 and Fig. 10), instead, it affects ISGF3

complex binding to ISRE in the promoter of ISGs (Fig. 11). In addition, acetylation of

STAT3 K49 and K87 at NTD and K685 plays a critical role in suppressive effect.

Nevertheless, the detailed mechanism remains to be determined.

4.1 Restored DKO MEFs could reduce the epigenetic difference between different cell lines

We generated STAT1- and/or STAT3-restored DKO MEFs to reduced variation

of epigenetics in WT and STAT3KO MEFs. Although retroviral transduction may

cause some gene overexpression or blockage, and affect cells phenotype, the

microarray results suggest the basal gene expression before IFN treatment were

comparable between different restored cells, confirming our hypothesis. In addition,

25

IFN-α-induced gene expression in STAT1- and STAT1/STAT3-restored DKO MEFs

were at similar magnetitude, despite a negative effect of STAT3 (Fig. 6). Moreover,

most of genes induced by IFN-α4 were known ISGs and the induction was eliminated

in EV-restored DKO MEFs (Fig. 7A), suggesting that gain-of-function is better than

loss-of-function approach in determing function of genes of interests.

4.3 Acetylated site of STAT3 is critical for the negative regulation

It has been shown that STAT3 could inhibit the expression of tumor suppressor

genes through the interaction with DNMT1 (Zhang et al., 2005a). In malignant T

lymphocytes, STAT3 was bound to the promoter of SHP-1 with DNMT1 and HDAC1,

resulting in DNA methylation and gene silencing. Besides, acetylation of STAT3 at

K685 was important for the interaction with DNMT1 (Lee et al., 2012). Acetylated

STAT3 was also increased in melanoma tissue as compared to normal skin cells,

which enhanced methylation of tumor suppressor genes through DNMT1. Taken

together, these data indicated that acetylated STAT3 could interact with DNA

modifying-enzymes, leading to suppression of gene expression. In this study, we

found that K685 of STAT3 also blocked its suppressive effect. In addition to K685,

K49 and K87 at STAT3 NTD are also involved in suppressing type I IFN-mediated

responses. For the moment, it is still unclear how K49 and K87 may contribute to the

26

effect. We propose that acetylation of STAT3 in K49, K87, and K685 is required for

interacting with HDAC1 or p300, which facilitates the binding to DNMT1, resulting

in methylation of ISRE in the promoters of ISGs and blocking the recruitment of

ISGF3.

4.4 STAT3 directly suppressed type I IFN-induced gene expression

Yu’s group showed that STAT3 and DNMT1 could bind to promoter of STAT1,

which is also an ISG, in cancer cells upon tumor conditioned medium treatment (Lee

et al., 2012). Interaction of acetylated STAT3 with DNMT1 resulted in DNA

methylaton, and gene silencing. Furthermore, in our microarray data, we found that

IFIT2 was decreased in STAT3-restored DKO MEFs after IFN-α4 treatment (Fig.

18A). It implies that STAT3 could directly inhibit type I IFN-mediated gene

expression independent of STAT1. In addition to IFIT2, endonuclease domain

containing 1 (ENDOD1), which is upregulated upon type I IFN treatment in

peripheral blood mononuclear cell (PBMC) (Baechler et al., 2003), was also suppressed by STAT3 in STAT3-restored DKO MEFs (Fig. 18B). These results indicated STAT3 not only affect ISGFs binding to promoter of ISRE and indirectly

regulates some ISGs, but it also directly silences gene expression.

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