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Báo cáo khoa học: Noninvasive imaging of microRNA124a-mediated repression of the chromosome 14 ORF 24 gene during neurogenesis potx

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In our study, the function of miR124a in neurogene-sis was analyzed using biomarker genes of stem cells and neurons, and the expression level of miR124a investigated by qRT-PCR during th

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repression of the chromosome 14 ORF 24 gene during

neurogenesis

Hae Young Ko1,2,3, Dong Soo Lee1,4and Soonhag Kim5

1 Department of Nuclear Medicine, Seoul National University College of Medicine, Korea

2 Interdisciplinary Course of Radiation Applied Life Science, Seoul National University College of Medicine, Korea

3 Institute of Radiation Medicine, Medical Research Center, Seoul, Korea

4 Department of Molecular Medicine and Biopharmaceutical Science, Seoul National University College of Medicine, Korea

5 Laboratory of Molecular Imaging, CHA Stem Cell Institute, CHA University, Seoul, Korea

Introduction

MicroRNAs (miRNAs), a class of small noncoding

RNAs, are  22-nucleotide single-strand RNA

mole-cules that are expressed in both plants and animals

[1,2] In general, miRNAs are incorporated into the RNA-induced silencing complex, and perfectly or imperfectly bind to the 3¢-UTR of its target mRNA to

Keywords

c14orf24; imaging; microRNA124a;

neurogenesis; target gene

Correspondence

S Kim, Laboratory of Molecular Imaging,

CHA Stem Cell Institute, CHA University,

605-21 Yoeksam 1-dong, Gangnam-gu,

Seoul, 135-081, Korea

Fax: +82 2 3468 3373

Tel: +82 2 3468 2830

E-mail: kimsoonhag@empal.com

D S Lee, Department of Nuclear Medicine,

Seoul National University, College of

Medicine, 28 Yongon-dong, Chongno-gu,

Seoul, 110-744, Korea

Fax: +82 2 3668 7090

Tel: +82 2 2072 2501

E-mail: dsl@plaza.snu.ac.kr

(Received 16 April 2009, revised 15 June

2009, accepted 29 June 2009)

doi:10.1111/j.1742-4658.2009.07185.x

The function of microRNAs (miRNAs) is translational repression or mRNA cleavage of target genes by binding to 3¢-UTRs of target mRNA

In this study, we investigated the functions and the target genes of micro-RNA124a (miR124a), and imaged the miR124a-mediated repression of chromosome 14 open reading frame24 (c14orf24, unknown function) during neurogenesis, using noninvasive luciferase systems The expression and functions of miR124a were investigated in neuronal differentiation of P19 cells (P19 is a mouse embryonic carcinoma cell line) by qPCR and RT-PCR The predicted target genes of miR124a were found by searching a bioinformatics database and confirmed by RT-PCR analysis Remarkable repression of c14orf24 by miR124a was detected during neurogenesis, and was imaged using in vitro and in vivo luciferase systems The expression of miR124a was highly upregulated during neuronal differentiation Overex-pression of miR124a in P19 cells resulted in a preneuronal gene exOverex-pression pattern MicroRNA124a-mediated repression of c14orf24 was successfully monitored during neuronal differentiation Also, c14orf24 showed molecu-lar biological characteristics as follows: dominant expression in the cyto-plasm; a high level of expression in proliferating cells; and gradually decreased expression during neurogenesis Our noninvasive luciferease system was used for monitoring the functions of miRNAs, to provide imaging information on related neurogenesis and the miRNA-regulated molecular network in cellular metabolism and diseases

Abbreviations

AA, antibiotic ⁄ antimycotic solution; c14orf24, chromosome 14 ORF 24; CMV, cytomegalovirus; DAPI, 4¢,6-diamidino-2-phenylindole; EdU, 5-ethynyl-2¢-deoxyuridine; Fluc, firefly luciferase; Gluc, Gaussia luciferase; LAMC1, laminin c1; LMNB1, lamin B1; MAP2, microtubule-associated protein 2; miRNA, microRNA; MSC, mesenchymal stem cell; Oct4, octamer4; PTBP1, polypyrimidine tract-binding protein 1; PTPN12, protein tyrosine phosphatase non-receptor type 12; qRT-PCR, quantitative RT-PCR; RA, retinoic acid; RBMS1, RNA-binding motif single-stranded interacting protein 1; ROI, region of interest; SD, standard deviation; USP48, ubiquitin-specific protease 48.

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induce either mRNA degradation or translational

inhi-bition [3–5] Recently, in animals, miRNAs have been

reported to destabilize the mRNA of their targets by

base paring with a continuous six or seven nucleotide

sequence in the 3¢-UTR of the target genes known as a

seed sequence, seed region, or seed match, in spite of

the partial base pairing between miRNAs and targets

[6]

The first reported miRNA, encoded by the

Caenor-habditis elegansgene lin-4, was found to be crucial for

the developmental timing and patterning of

postembry-onic stages [7] Since their identification, miRNAs have

been shown to play important roles in diverse

biolo-gical functions, such as cell differentiation, fat

meta-bolism, cell proliferation, and cell death [8–10] A

recent study found that many miRNAs, such as miR9,

miR9*, miR124a, miR134, miR23a, miR132, and

miR128, are expressed in neurons and regulate

neuro-nal development [11,12] Among them, miR124a is

present at undetectable or very low levels in neural

progenitors, but is expressed at a high level in

differen-tiating and mature neurons [13] A microarray study

of miR124a-treated HeLa cells (human carcinoma

cells) revealed 174 downregulated non-neuronal

tran-scripts [14] The endogenous targets directly bound

and repressed by miR124a include the genes encoding

small C-terminal domain phosphatase 1 [15],

poly-pyrimidine tract-binding protein 1 (PTBP1) (PTB⁄

hnRNP 1) [16], laminin c1, and integrin b1 [17]

Moreover, neurite outgrowth was promoted by

over-expression of miR124a during neuronal differentiation

[18]

It is a great challenge to study the expression and

function of endogenous miRNAs without killing the

animals The current methods, including northern

blot analysis and RT-PCR, used to investigate the

molecular regulation of endogenous miRNAs are

time-consuming, labor-intensive, nonrepeatable, and

not clinically relevant Recently, there have been

significant advances in optical imaging techniques

using multimodal reporter systems; this technology

has been used for noninvasive repeated quantitative

imaging of tumor and stem cells in living animals

[19–22] Previous articles from our laboratory have

reported the expression of miRNA and its target

genes in vitro and in vivo, using these luciferase

systems [21,23,24]

In our study, the function of miR124a in

neurogene-sis was analyzed using biomarker genes of stem cells

and neurons, and the expression level of miR124a

investigated by qRT-PCR during the neuronal

differ-entiation of P19 cells A bioinformatics analysis was

then performed to predict the targets of miR124a, and

showed, by RT-PCR, several genes that were directly regulated by miR124a One of these genes, chromosome

14 ORF 24 (c14orf24), which is of unknown function, was evaluated in our successfully developed luciferase reporter system, both in vitro and in vivo, to determine whether the 3¢-UTR of c14orf24 was directly regulated

by miR124a Also, for the first time, the biological functions of c14orf24 were investigated during cell proliferation

Results

MicroRNA124a is expressed at a high level during neurogenesis

MicroRNA124a is a small RNA composed of 22 nucleotides, and is well conserved from humans to aquatic species To determine and quantify the endoge-nous levels of miR124a during neuronal differentiation

of P19 cells, we performed quantitative RT-PCR (qRT-PCR) (Fig 1) cDNA was synthesized from small RNA of neuronal differentiated P19 cells, at 0,

1, 2, 3, 4, 5 and 6 days after retinoic acid (RA) treat-ment A pair of specific primers for miR124a was used, and the quantities of miR124a for each differentiation day were normalized with U6 small RNA The expres-sion of miR124a gradually increased during neuronal differentiation, and had increased more than three-fold

by the fifth day after RA treatment

4

3

2

1

0 Before 1 day 2 days 3 days

Day after RA treatment

4 days 5 days 6 days

Fig 1 The expression of miR124a during neuronal differentiation

of P19 cells (A) Quantitative RT-PCR analysis of the expression of mature miR124a Endogenous mature miR124a levels were increased during neuronal differentiation Data were normalized to U6 snRNA (DDC T = DC T-before – DC T-day , DC T-before = C T-miRNA⁄ before

– C T-U6RNA ⁄ before , DC T-day = C T-miRNA ⁄ day – C T-U6RNA ⁄ day ) Data are expressed as means ± SD in triplicate samples.

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Preneuronal characteristics of P19 cells were

induced by overexpression of miR124a

To investigate the role of miR124a during

neurogene-sis, overexpression of miR124a was examined in P19

cells We transfected exogenously derived miR124a, at

a concentration of 5 nm, into P19 cells, which are

believed not to be induced into neuronal differentiation

in the absence of RA The programming process of neuronal differentiation was induced by overexpression

of miR124a (Fig 2A) We followed the gradual acqui-sition of neuronal traits over time after transfection with exogenous miR124a Interestingly, 2 days after transfection, none of the P19 cells treated with miR124a showed neuronal morphology, whereas RA-treated P19 cells had the neuronal phenotype

Oct4 NeuroD MAP2

Before

Before

1 day 2 days 3 days 4 days Before 1 day 2 days 3 days 4 days 1 day 2 days 3 days 4 days Day after transfection with

miR124a

Day after anti-miR124a and RA treatment Day after RA treatment

β-actin

Oct4

NeuroD

MAP2

Day after transfection with miR124a

Day after transfection with miR124a

Day after RA treatment

Day after RA treatment

A

C

B

Fig 2 MicroRNA124a-induced preneurogenesis in P19 cells (A) Neuronal differentiation analysis in miR124a-transfected P19 cells Upper panel: P19 cells were changed to preneurons by overexpression of miR124a Lower panel: neuronal differentiation induced by RA treatment,

as a control (B) RT-PCR analysis of P19 cells transfected with miR124a and subjected to RA treatment Oct4, stem cell marker; NeuroD, preneuronal marker; MAP2, neuronal marker b-Actin was used as a control (C) Laser scanning confocal microscopy of immunofluorescence staining using Oct4, NeuroD and MAP2 for P19 cells transfected with miR124a, treated with RA, and treated with both anti-miR124a and

RA for 4 days Red fluorescence represents the cytoplasmic expression of Oct4 (top panel), NeuroD (middle panel), and MAP2 (bottom panel), and the blue fluorescence represents DAPI, which stained the nucleus.

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However, thereafter, miR124a-treated P19 cells

exhib-ited a marked change in cell morphology: there was a

gradual expansion of dendrites from the cells, even

though the rate of dendrite development from

miR124a-treated P19 cells was slower than in the

positive controls By RT-PCR analysis, it was shown

that, in undifferentiated P19 cells, expression of the

stem cell marker octamer 4 (Oct4) was upregulated, but

the differentiation markers NeuroD and

microtubule-associated protein 2 (MAP2) were not detected, as

previously reported [15] (Fig 2B) When P19 cells was

treated only with miR124a, the level of Oct4 transcript

was gradually decreased until 4 days, whereas RA

treatment of P19 cells resulted in the disappearance of

Oct4 expression 2 days after the treatment Both

miR124a-treated and RA-treated P19 cells showed a

significant increase in the expression of the preneuronal

marker NeuroD; however, this was less than in P19

cells with RA treatment Interestingly, the neuron

mar-ker MAP2 was present at high levels only in

RA-trea-ted P19 cells, and was not present at high levels in P19

cells treated with exogenous miR124a Additionally, to

inhibit the function of miR124a, we treated P19 cells

with both RA and miR124a antagomir (synthetic

oligonucleotides that fully complement the miR124a)

Oct4 was continuously expressed until 4 days after

anti-miR124a and RA treatment, but gradually

decreased in level, whereas MAP2 was undetectable

Moreover, NeuroD was detected only 3 days after

treatment with RA and miR124a antagomir These

results showed that miR124a antagomir retarded

RA-induced neuronal differentiation of P19 cells by

blocking miR124a function

To investigate how the protein levels of these

mark-ers were affected by miR124a or RA in P19 cells,

immunofluorescence staining was performed with each

antibody (Fig 2C) The confocal microscope image

showed that the fluorescent signals obtained with Oct4

was found in the cytoplasm of undifferentiated P19

cells, gradually decreased with the treatment with

miR124a, and was undetectable after RA treatment,

owing to the neuronal differentiation of P19 cells

Conversely, the cytoplasmic fluorescent activity of P19

cells with NeuroD gradually increased with treatment

with miR124a or RA by 4 days, with stronger signals

being seen in RA-treated P19 cells, whereas no signal

was found before the treatments The result of

immu-nofluorescence staining using MAP2 showed a gradual

increase of cytoplasmic fluorescence in RA-treated P19

cells for 4 days, but no significant signal either before

or after the miR124a treatment in P19 cells This

sug-gests that the sole function of miR124a could be to

trigger the initial neurogenesis program and that it is

not additionally involved in fully differentiating P19 cells into mature neurons

MicroRNA124a repressed multiple target genes during the neuronal differentiation of P19 cells

To find the genes that are directly regulated by miR124a during miR124a-directed neurogenesis and that contain miR124a seed sequences, microarray data from miR124a-treated HeLa cells [14] and bioinfor-matics data from miR124a-predicted targets from the PicTar database (http://pictar.mdc-berlin.de), an algo-rithm for the identification of miRNA targets using 3¢-UTR alignments, were compared Comparison of

174 microarray-analyzed genes that are significantly regulated by miR124a in HeLa cells and the PicTar database-predicted 787 genes showed 35 genes with overlapping coding sequences that might be directly regulated by miR124a (Fig 3A, Table S1)

For further analysis of miR124a-targeted gene expres-sion by RT-PCR, we randomly selected eight candidates after a review of the literature and determination of the neuronal correlation These included the following: c14orf24, laminin gamma 1 (LAMC1), PTBP1, RNA-binding motif single-stranded interacting protein 1 (RBMS1), hypothetical protein MGC5508 (transmem-brane protein 109), lamin B1 (LMNB1), protein tyrosine phosphatase non-receptor type 12(PTPN12), and ubiqu-itin-specific protease 48 (USP48) Unlike the gradual increase of miR124a expression during the neuronal dif-ferentiation of P19 cells treated with RA, the endogenous gene expression of five of the candidates was gradually decreased over the period of neuronal differentiation (Fig 3B) Unfortunately, three of the eight targets, transmembrane protein 109, Usp48, and RBMS1, could hardly be distinguished, owing to weak expression or technical problems with amplification of their amplicons (data not shown) To determine the direct correlation between the five candidates and miR124a, overexpres-sion analysis of exogenous miR124a was conducted in P19 cells in the absence of RA treatment We could more clearly predict that mRNA transcript levels of the c14orf24, LAMC1, PTBP1, PTPN12 and LMNB1 genes were significantly and directly repressed by miR124a, indicating that mRNA of the five target genes was destabilized by miR124a (Fig 3C)

C14orf24 was directly downregulated by miR124a Most of the miRNA target genes predicted in the Pic-Tar database have one to tens of different miRNA seed sequences at the 3¢-UTR of each mRNA This means that multiple miRNAs can regulate a single

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target in a cell or a tissue at the same time

Fortu-nately, the PicTar database showed that the 3¢-UTR of

c14orf24 has only two predicted miRNA seed

sequences, miR124a and miR128, both of which are

known to be expressed during neurogenesis [12] The

miRNA-related functions of these are unknown; we

sought to determine whether c14orf24 is directly

regu-lated by miR124a or not About 100 bp of the 3¢-UTR

of c14orf24 near the miR124a seed sequence was

cloned into our established luciferase reporter gene

sys-tem, CMV⁄ Gluc ⁄ c14ofr24-3¢UTR, to monitor whether

it is directly downregulated by miR124a (Fig 4A,B)

CMV⁄ Gluc ⁄ c14orf24-3¢UTR was first transfected into

HeLa cells, where miR124a expression is undetectable

The cotransfection with various doses of exogenous

miR124a showed a significant repression of Gaussia

luciferase (Gluc) activity as compared with the control,

CMV⁄ Gluc ⁄ c14orf24-3¢UTRmt construct, which

con-tained a completely mutated miR124 seed sequence of

c14orf24(Fig 4C)

To quantify the in vitro luciferase activity,

represent-ing the miR124a-directed endogenous expression level

of the c14orf24 gene during neurogenesis, CMV⁄

Gluc⁄ c14ofr24-3¢UTR was transfected into RA-treated

P19 cells The Gluc activity of CMV⁄ Gluc ⁄

c14ofr24-3¢UTR slightly decreased to the third day of neuronal

differentiation of P19 cells (Fig 4D) The significant

decrease in Gluc activity from CMV⁄ Gluc ⁄

c14ofr24-3¢UTR was detected 4 days after the neuronal

differen-tiation of P19 cells, as compared with CMV⁄ Gluc ⁄

c14orf24-3¢UTRmt

The in vivo imaging of 2.5· 106 implanted P19 cells

bearing CMV⁄ Gluc ⁄ c14ofr24-3¢UTR or CMV ⁄ Gluc ⁄

c14orf24-3¢UTRmt was monitored for 2 days of

neuro-nal differentiation, and then aneuro-nalyzed by a region of interest (ROI) analysis As compared with the control from the left thigh without RA treatment, the Gluc expression of CMV⁄ Gluc ⁄ c14ofr24-3¢UTR from the RA-treated P19 cells was significantly decreased, and almost disappeared by the second day of neuronal dif-ferentiation, due to the increased expression of miR124a (Fig 4E) Similar to what was found in the

in vitro luciferase assay of CMV⁄ Gluc ⁄ c14orf24-3¢UT-Rmt, the Gluc activity was slightly, but not signifi-cantly, decreased (Fig 4F) The fold ratio from the ROI analysis of CMV⁄ Gluc ⁄ c14ofr24-3¢UTR showed more dramatically decreased expression of c14orf24 in the RA-treated P19 cells than in the undifferentiated P19 cells (Fig 4G)

The c14orf24 gene was expressed in the cytoplasm as a cellular component and functioned biologically in the positive regulation of cell proliferation

Unfortunately, any potentially valuable biological functions of c14orf24 have not yet been studied, and even its antibody was not available Therefore, identifi-cation of the functions of c14orf24 is a considerable challenge To predict how the protein encoded by the c14orf24 mRNA regulated by miR124a functions in the intact cells, we introduced the coding sequence and the full 3¢-UTR containing miR124a seed sequences of c14orf24 into an expression vector, pcDNA3.1⁄ His vector (designated as Xp-c14orf24), that could express c14orf24 at abnormally high levels and represent the miR124a-mediated repression that takes place in cells

in the presence of miR124a First, to investigate the

752 genes

a

139 genes

C14orf24 LAMC1 PTBP1

PTPN12

Before 1 day 2 days 3 days 4 days 5 days 6 days

Untreated +miR124a

Day after RA treatment β-actin

LMNB1

C14orf24 LAMC1 PTBP1

PTPN12

β-actin

LMNB1

35 genes

Fig 3 The predicted target genes of miR124a in P19 cells (A) Prediction of miR124a target genes using bioinformatics By comparison with

787 genes obtained from the PicTar database (a) and 174 genes significantly downregulated in miR124a-treated HeLa cells (b), 35 genes with overlapping coding sequences (c) were discovered (B) RT-PCR analysis showing the expression of five predicted target genes The lev-els of c14orf24, LAMC1, PTBP1, PTPN12 and LMNB1 were repressed by increasing amounts of miR124a during the neuronal differentiation

of P19 cells (C) The target genes downregulated by overexpression of miR124a The five predicted genes (c14orf24, LAMC1, PTBP1, PTPN12, and LMNB1) were downregulated by miR124a transfection into P19 cells.

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A B

E

F

G

c14orf24 mRNA

3 ′UTR

Human Mouse

ORF

2360

* 1.4

1.2

1.0

0.8

0.6

0.4

0.2

0

b

a

b

a

0 h 0.5 1.0

2.0

1.5

0

Time

2 days

10 8

4 6

2 0

400 300 200 100

8

4 6

2

10 n M Concentration of miR124a

*

1.2 1.0 0.8 0.6 0.4 0.2 0

20 n M

Before 1 day 2 days 3 days

Day after RA treatment

4 days 5 days

Seed sequence

ATTGAGC

CMV/Gluc/c14orf24-3 ′ UTR

CMV/Gluc/c14orf24-3 ′ UTRmt

3 ′ UTR

3 ′ UTRmt

Gluc CMV

Gluc CMV

Mutation of seed sequence

Fig 4 C14orf24 regulation by miR124a targeting (A) The genomic locus of the c14orf24 3¢-UTR containing the seed sequence that needs to

be recognized by miR124a The mutation of the seed sequence was designed to interrupt the binding of miR124a into the 3¢-UTR (B) Sche-matic diagram of CMV ⁄ Gluc ⁄ c14orf24-3¢UTR constructs The Gluc activity of CMV ⁄ Gluc ⁄ c14orf24-3¢UTR was downregulated when miR124a bound to the c14orf24 3¢-UTR However, the Gluc activity of the CMV ⁄ Gluc ⁄ c14orf24-3¢UTR mutant was not regulated by miR124a, because miR124a did not bind to the c14orf24 3¢-UTR mutant (C) Luciferase analysis to confirm whether the c14orf24 3¢-UTR was bound by exogenous miR124a in HeLa cells The Gluc activity of CMV ⁄ Gluc ⁄ c14orf24-3¢UTR (black bar) was significantly decreased as the concentration of miR124a increased CMV ⁄ Gluc ⁄ c14orf24-3¢UTRmt (gray bar) was used as a negative control Luciferase activity was normalized to the CMV ⁄ Gluc vector Data are expressed as means ± SD in triplicate samples *P < 0.05; **P < 0.005 (D) Luciferase analysis showing that endogenous miR124a binds to the c14orf24 3¢-UTR in P19 cells after RA treatment The Gluc activity of CMV ⁄ Gluc ⁄ c14orf24-3¢UTR (black bar) was decreased during neuronal differentiation The Gluc activity of CMV ⁄ Gluc ⁄ c14orf24-3¢UTRmt (gray bar), as a negative control, was not regulated Data are expressed as means ± SD in triplicate samples *P < 0.05 (E) Bioluminescence image showing that c14orf24 is the target gene of miR124a P19 cells (2.5 · 10 6 ) transfected with CMV ⁄ Gluc ⁄ c14orf24-3¢UTR were subcutaneously grafted onto the left side (a) and right side (b) of the nude mice On the right side, neuronal differentiation was induced by RA treatment Bioluminescence of grafted P19 cells on the right side was decreased in comparison with those on the left side (three mice) (F) Bioluminescence image showing that CMV ⁄ Gluc ⁄ c14orf24-3¢UTRmt, as a negative control, was not regulated by RT-induced neuronal differentiation We used the same method as that used to obtain the previous biolu-minescence image (three mice) (G) ROI analysis of the biolubiolu-minescence image The ratio differentiated ⁄ undifferentiated ratio reduced as time passed However, in the case of injected P19 cells transfected with CMV ⁄ Gluc ⁄ c14orf24-3¢UTR, the ratio remained unchanged.

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localization of expression of c14orf24 protein,

Xp-c14orf24 was transfected into P19 cells, and double

analysis was performed with the Anti-Xpress antibody

and 4¢,6-diamidino-2-phenylindole (DAPI), which

stains the nucleus As shown in Fig 5A, c14orf24

pro-tein was well expressed in the undifferentiated P19 cells

for 4 days, and showed even localization in the

cyto-plasm However, it was observed that the expression of

c14orf24 protein in the cytoplasm was almost

com-pletely lost 4 days after treatment with either RA or

miR124a of P19 cells These results implied that the

expression of c14orf24 protein is dominantly localized

in the cytoplasm and repressed directly by miR124a

during neuronal differentiation

Next, we investigated how the overexpression of

c14orf24 affects the proliferation and neuronal

differ-entiation of P19 cells Following Xp-c14orf24

transfec-tion, P19 cells displayed more rapidly achieved and

higher cell densities than the cells grown under normal

control conditions (Fig 5B) Additionally, the cellular

morphology demonstrated that treatment with both

RA and Xp-c14orf24 of P19 cells simultaneously

repressed the morphological changes of neuronal

dif-ferentiation, leading to less retraction of cytoplasm

towards the nucleus and a less spherical appearance of

cell bodies, whereas after a single treatment with RA

of P19 cells, the cells increasingly showed the neuronal

traits of a pyramidal and perikaryal appearance To

determine the effect of c14orf24 on P19 cells at the

molecular level, RT-PCR analysis was conducted with

total RNA from P19 cells 4 days after no treatment,

treatment with either RA or Xp-c14orf24, or both

treatments Transfection of Xp-c14orf24 into P19 cells

led to overexpression c14orf24 transcript (Fig 5C)

Similar to the results shown in Fig 4B, RA treatment

of P19 cells resulted in neuronal characteristics of P19

cells, which showed increased gene expression of the

neuron markers MAP2 and NeuroD, and a significant

decrease in that of the stem cell marker Oct4

However, additional treatment of RA-treated P19 cells with Xp-c14orf24 inhibited neuronal differentiation, resulting in lower levels of MAP2 and NeuroD tran-script, and higher levels of Oct4 trantran-script, than in P19 cells treated only with RA

To further verify the effect of c14orf24 on cell prolif-eration and neuronal differentiation of P19 cells, flow cytometry assay was performed with the mitotic marker, 5-ethynyl-2¢-deoxyuridine (EdU) EdU is incor-porated into replicating DNA similarly to 5-bromo-2¢-deoxyuridine (BrdU), and its terminal alkyne group reacts with fluorescent azide [25] The incorporation of the nucleoside analog EdU into cellular DNA of P19 cells was quantified, and demonstrated that the number

of EdU-labeled P19 cells with the overexpression of c14orf24revealed about 2.5-times higher than that with-out treatment (Fig 5D) The number of EdU-labeled P19 cells treated with both Xp-c14orf24 and RA showed 1.7-times higher than that of only RA-treated P19 cells These results indicated a higher rate of DNA synthesis in P19 cells with c14orf24 overexpression

To extend our understanding of the involvement of c14orf24in cellular proliferation and neuronal differen-tiation, the expression of the c14orf24 gene was investi-gated in various cells, including normal, cancer and neuronal precursor cell lines RT-PCR was conducted using total RNA from HT-ori3 cells (normal thyroid cells), L132 cells (lung normal cells), C6 cells (glioma cells), CT-26 cells (colon carcinoma cells), mesenchy-mal stem cells (MSCs), and G2 cells (neural stem cells) In normal cells, HT-ori3 cells, and L132 cells, the c14orf24 gene was expressed at a relatively low level, whereas a higher amount of the c14orf24 ampli-con was expressed in highly proliferating cells, C6 cells, and CT-26 cells (Fig 5E) Also, the c14orf24 gene was highly expressed in both MSCs and G2 Interestingly, when G2 cells were induced to undergo neuronal dif-ferentiation by the deletion of doxycycline from the growth medium, the transcript level of the c14orf24

Fig 5 The biological and cellular functions of the c14orf24 gene in cells (A) Immunocytostaining analysis of c14orf24 by laser scanning con-focal microscopy (red, c14orf24; blue, DAPI) The pcDNA3.1 ⁄ His_c14orf24 containing Xpress epitope was transfected into P19 cells and treated with RA, miR124a or nothing for 4 days The fluorescence image detected by Xpress antibody showed that c14orf24 proteins were predominantly present in the cytoplasm of P19 cells before the treatments, and that they disappeared during the neuronal differentiation of P19 cells induced by RA or after miR124a treatment Magnification: upper panel, · 400; lower panel, · 1000 (B) Cellular morphology charac-teristics of P19 cells affected by the c14orf24 gene Cell morphology was acquired 4 days after no treatment and treatment with either or both of RA or pcDNA3.1 ⁄ His_c14orf24 of P19 cells (C) RT-PCR analysis of P19 cells transfected with the c14orf24 gene Total RNA was extracted from P19 cells 4 days after no treatment and and treatment with either or both of RA or pcDNA3.1 ⁄ His_c14orf24 of P19 cells RT-PCR was conducted for MAP2, NeuroD, Oct4 and c14orf24 using a pair of primers listed in Tables S2 and S4 b-Actin was used as a con-trol (D) EdU-incorporated flow cytometry analysis of P19 cells affected by the c14orf24 gene EdU-labeled cells were measured 4 days after

no treatment and after treatment with either or both of RA or pcDNA3.1 ⁄ His_c14orf24 of P19 cells The y-axis indicates the percentage of EdU-labeled P19 cells Data are expressed as means ± SD in triplicate samples *P < 0.05; **P < 0.005 (E) RT-PCR analysis of c14orf24 expression in various cell lines: HT-ori3 cells (normal thyroid cell line), L132 cells (lung normal cell line), C6 cells (glioma cell line), CT-26 cells (colon carcinoma cell line), MSCs, and G2 cells (neural stem cell line) b-Actin was used as a control.

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gene was dramatically decreased, and the transcript

had almost disappeared on the fourth day of neuronal

differentiation This result showed that the c14orf24

gene must be positive regulator of cellular proliferation

and be downregulated during neurogenesis

Discussion

Hundreds of well-conserved miRNAs have been

reported to be evolutionarily well conserved over

spe-cies and related to cellular metabolism and various

diseases, including cancer, cardiovascular disease, and neurological disease [26] In particular, neuronal-specific miRNAs such as miR124a, miR9, miR128, miR131, miR178 and miR125b have been directly and indirectly shown to have relatively high expression levels during brain development, and are expected to regulate the various genes related to neuronal differen-tiation

Complicated intracellular and extracellular communi-cation could possibly be involved in the differentiation

of stem cells into mature neurons of the eukaryotic

sys-A

B

E

x1000 x1000

x1000 x1000

x1000 x1000

2 days

RA

c14orf24

RA c14orf24

RA

c14orf24

c14orf24

HT-ori3 L132 C6 CT26 MSC G2-un G2-4d G2-6d

+ + +

+ –

– –

MAP2 NeuroD

Oct4

β-actin

c14orf24

β-actin

+ +

+

– –

70 60 50 40 30 20 10 0

Day after transfection with miR124a

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tem In general, a single miRNA is believed to directly

target hundreds of genes, which may indirectly regulate

thousands of coding genes [14] Overexpression of miR1,

which is specifically expressed in cardiac and skeletal

muscle, induced the differentiation of C2C12 myoblasts

into myotubes [27] Transfection of exogenously derived

miR124a into P19 cells in the absence of RA resulted in

guidance of the neuronal program, partly explaining

why miR124a is specifically neuronally expressed

Unlike miR1, the solo miR124a could not sufficiently

induce P19 cells to fully differentiate into neurons, but

could induce only the preneuronal characteristics It is

possible that other, more complex, mechanisms are

required to complete the neuronal differentiation

pro-gram Surprisingly, some experiments in our laboratory

showed that transfection of single, dual or multiple

neu-ronal-specific miRNAs into P19 cells induced a more

neuronally differentiated status than single transfection

with miR124a, and that a cancer-related miRNA

delayed the neuronal differentiation of RA-treated P19

cells (data not shown) These results mean that miRNAs

play an important role in maintaining the stem cells

and inducing neuronal differentiation

As miRNAs have been shown to be correlated with

various diseases, many efforts in both experimental

and in silico studies have been focused on their gene

targets, in order to develop our understanding from

the simple concept of miRNA expression to the more

complicated regulatory interactions between miRNA

and target genes, which can decide cellular fate or

dis-ease progression [28,29] RT-PCR of genes selected by

the analysis from the comparison of microarray data

of miR124a-treated HeLa cells with miR124a-predicted

targets through the PicTar database showed that the

c14orf24, LAMC1, LMNB1, PTBP1 and PTPN12

genes had gradually decreased endogenous gene

expression during the neuronal differentiation of P19

cells when endogenous levels of miR124a were

gradu-ally increased Additiongradu-ally, excessive amounts of

exogenous miR124a in P19 cells resulted in

signifi-cantly direct regulation of these candidates PTPB1,

which is expressed at a high level in non-neuronal cells,

binds to pyrimidine-rich sequences in pre-mRNA and

inhibits the splicing of nearby neuron-specific

alterna-tive exons, but is known to be repressed in the nervous

system by miR124a, to allow the inclusion of

neuron-specific exons in mature mRNA [16] LAMC1, which

is one of the components of a heterodimeric molecule,

laminin-1, comprising laminin a1, laminin b1, and

lam-inin c1 subunits, has also been reported to be an

endogenous target of miR124a [17]

Our experiment using the established luciferase

sys-tem containing the putative 6 bp seed sequence matched

between the 5¢-end of miR124a and the 3¢-UTR of the c14orf24 gene showed a strong evidence of miR124a-mediated repression of c14orf24 during neurogenesis The coding region of c14orf24 (NM_173607), the func-tion of which is not known, possesses 213 amino acids and has a molecular mass of 23 kDa Our findings from PCR and in vitro⁄ in vivo luciferase assays show that c14orf24is expressed before and after neuronal differen-tiation of P19 cells, but its expression is significantly decreased on the day when the endogenous level of miR124a is at its peak Also, the strong suppression of c14orf24 transcript was caused by the exogenous miR124a, through direct binding of miR124a to the miR124a seed sequence of c14orf24 Unfortunately, the

in vivoGluc expression of transiently transfected lucifer-ase systems was almost gone after 3 days of neuronal differentiation For long-term noninvasive imaging, sta-ble cell lines or viral vectors such as lentiviral or adeno-viral vector will be helpful to image the dynamic changes of miR124a-regulated neurogenesis

Additional biological and cellular studies of the c14orf24 gene by immunocytostaining, flow cytometry and RT-PCR analysis showed that c14orf24 was domi-nantly expressed in the cytoplasm and highly expressed

in proliferating cells, but was repressed during neuro-nal differentiation In this study, for the first time, we showed that c14orf24 might function in maintaining cell proliferation, at least in P19 cells, and be involved

in the initial program of neurogenesis via the negative interaction with miR124a

Our noninvasive luciferase imaging systems for moni-toring the repression of the novel targets of miR124a will

be a useful tool for the study of the molecular regulation

of miRNAs related to cellular proliferation, differentia-tion, apoptosis, and various diseases In particular, our ongoing development of a dual luciferase reporter gene system to simultaneously monitor ectopically expressed miRNAs and their targets will provide bidirectional information for cellular therapy and disease diagnosis

Experimental procedures

Quantitative RT-PCR of miR124a With the mirVana miRNA isolation kit (Ambion, Austin,

TX, USA), small RNA was isolated during the neuronal differentiation of P19 cells qRT-PCR was performed using the mirVana qRT-PCR primer Set (Ambion) and mirVana qRT-PCR miRNA detection kits (Ambion), according to the manufacturer’s instructions The PCRs were performed

in triplicate using iCycer (Bio-Rad, Hercules, CA, USA) with SYBR Premix Ex Taq (2·) (Takara, Shiga, Japan) as follows: 95C for 3 min; and 40 cycles of 95 C for 15 s

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and 60C for 30 s) To normalize the experimental samples

for RNA content, the U6 snRNA primer set (Ambion) was

used as a control

Culture of HeLa cells and P19 cells

HeLa cells (cervical carcinoma cell line) were cultured

routinely in RPMI (Jeil Biotechservices Inc., Daegu, Korea)

containing 10% fetal bovine serum (Cellgro, Herndon, VA,

USA) and 1% antibiotics⁄ antimycotic solution (AA)

(Cell-gro) at 37C P19 cells were obtained from the ATCC In

brief, undifferentiated P19 cells were grown at 37C in

a-MEM (Gibco, Grand Island, NY, USA) supplemented

with 2.5% fetal bovine serum (Cellgro), 7.5% bovine calf

serum (Gibco) and 1% AA (Cellgro) in a 5% CO2humidified

chamber For induction of neuronal differentiation using the

monoculture differentiation method [21], P19 cells were

pla-ted on gelatin-coapla-ted culture plates at a density of

5· 103cellsÆcm)2in growth medium prior to growth factor

removal After 24 h, P19 cells were cultured under serum-free

conditions in DMEM⁄ 12 (1 : 1) medium (Gibco)

supple-mented with 1% insulin–transferrin–selenium (Gibco) and

1% antibiotics, and treated with 5· 10)7m all-trans-RA

(Sigma, St Louis, MO, USA) After 2 days, the RA was

removed from the medium, and the cells were cultured

further under serum-free conditions

RT-PCR

Using Trisol reagent (Invitrogen, Grand Island, NY, USA),

total RNA was isolated from P19 cells as during the day after

RA treatment Reverse transcription, for synthesis of the

first-strand cDNA, was carried out using random-hexamer

and SuperScript II reverse transcriptase (Invitrogen),

accord-ing to the manufacturer’s instructions, and this cDNA was

then used as a template for PCR amplification: 94C for

5 min, with 30 amplification cycles [94C for 30 s each Tm

for 30 s (Tables S2 and S4), 72C for 30 s], and 72 C for

4 min The sequences of the primers used for PCR

amplifica-tion are listed in Table S2 and Table S4

Development of vectors monitoring the

miR124a-directed repression of c14orf24

We constructed a Gluc reporter vector bearing a

cytomega-lovirus (CMV) promoter, and 99 nucleotides from the

3¢-UTR of the c14orf24 gene, containing an miR124a seed

sequence, identified through the PicTar database, were used

for imaging of miR124a-regulated repression They were

constructed by incubation with a pair of primers, sense and

antisense primers of the c14orf24 3¢-UTR, in annealing

buf-fer (· 1 TE bufbuf-fer + 50mm NaCl) for 10 min at 60 C,

cloned into CMV⁄ Gluc at the site between XhoI and XbaI,

and cloned into the CMV⁄ Gluc vector labeled

CMV⁄ Gluc ⁄ c14orf24-3¢UTR (Table S3) As a negative control, CMV⁄ Gluc ⁄ c14orf24-3¢UTRmt was constructed by complete mutation of the miR124a seed sequences in the 3¢-UTR of c14orf24, and annealing the oligonucleotides and sense and antisense primers of c14orf24-3¢UTRmt (Fig 4A,B)

Transfection and luciferase assay Transient transfection of various vectors of interest into undifferentiated⁄ differentiated P19 cells was performed by using 0.6 lg of DNA, 3 lL of Plus reagent (Invitrogen), and 1.5 lL of Lipofectamine (Invitrogen) per well After

3 h, the transfection medium was replaced with undifferen-tiated medium (a-MEM; 2.5% fetal bovine serum, 7.5% bovine calf serum, and 1% AA) from undifferentiated P19 cells and differentiated media (DMEM⁄ 12; 1% insulin– transferrin–selenium and 1% AA) from RA-treated P19 cells Gluc expression was analyzed 2 days after transfec-tion All transfections were carried out in triplicate The cells were washed with NaCl⁄ Piand lysed with 200 lL per well of passive lysis buffer (Promega, Madison, WI, USA) Next, 100 lL of cell lysate from each well was used to mea-sure luciferase activity with the Gaussia luciferase assay kit (Targetingsystems, San Diego, CA, USA), according to the manufacturer’s instructions, and measured on a Wallac1420 VICTOR3V (PerkinElmer Life and Analytical Sciences, Waltham, MA, USA) The data are presented as means ± standard deviation (SD) calculated from triplicate wells

Grafting of the cells with reporter gene constructs and in vivo visualization of miR124a and c14orf24 in nude mice All experimental animals were housed under specific patho-gen-free conditions and handled in accordance with the guidelines issued by the Institutional Animal Care and Use Committee of Seoul National University Hospital We performed transient transfection of P19 cells with CMV⁄ Gluc ⁄ c14orf24-3¢UTR and CMV⁄ Gluc ⁄ c14orf24-3¢UTRmt After 48 h of transfection, the cells were counted and resuspended in 100 lL of NaCl⁄ Pi(2.5· 106

cells per

100 lL of NaCl⁄ Pi) These cells were implanted subcutane-ously into male Balb⁄ c nude mice (6 weeks old, weighing 25–27 g) The cells containing CMV⁄ Gluc ⁄ c14orf24-3¢UTR

or CMV⁄ Gluc ⁄ c14orf24-3¢UTRmt were implanted in both thighs; the left thigh, without the RA treatment, was used

as a control, and the right thigh was treated with RA coin-cidently with cell injection Three mice in each group were subsequently anesthetized with 2.5% isofluorane, and trans-ferred into the chamber of an IVIS 100 imager (Xenogen, Alameda, CA, USA) To acquire images of Gluc, the mice were directly injected with 5 lg of coelenterazine The Gluc

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