LETTER
doi:10.1038/nature12885
C/EBPa poises B cells for rapid reprogramming into
induced pluripotent stem cells
Bruno Di Stefano
1,2
, Jose Luis Sardina
1,2
*, Chris van Oevelen
1,2
*, Samuel Collombet
3,4,5
, Eric M. Kallin
1,2
{, Guillermo P. Vicent
1,2
,
Jun Lu
6
, Denis Thieffry
3,4,5
, Miguel Beato
1,2
& Thomas Graf
1,2,7
CCAAT/enhancer binding protein-a (C/EBPa) induces transdif-
ferentiation of B cells into macrophages at high efficiencies and
enhances reprogramming into induced pluripotent stem (iPS) cells
when co-expressed with the transcription factors Oct4 (Pou5f1),
Sox2, Klf4 and Myc (hereafter called OSKM)
1,2
. However, how C/EBPa
accomplishes these effects is unclear. Here we find that in mouse
primary B cells transient C/EBPa expression followed by OSKM
activation induces a 100-fold increase in iPS cell reprogramming
efficiency, involving 95% of the population. During this conversion,
pluripotency and epithelial–mesenchymal transition genes become
markedly upregulated, and 60% of the cells express Oct4 within 2 days.
C/EBPa acts as a ‘path-breaker’ as it transiently makes the chromatin
of pluripotency genes more accessible to DNase I. C/EBPa also induces
the expression of the dioxygenase Tet2 and promotes its transloca-
tion to the nucleus where it binds to regulatory regions of pluripo-
tency genes that become demethylated after OSKM induction. In
line with these findings, overexpression of Tet2 enhances OSKM-
induced B-cell reprogramming. Because the enzyme is also required
for efficient C/EBPa-induced immune cell conversion
3
, our data indi-
cate that Tet2 provides a mechanistic link between iPS cell reprogram-
ming and B-cell transdifferentiation. The rapid iPS reprogramming
approach described here should help to fully elucidate the process
and has potential clinical applications.
Reprogramming of somatic cells into induced pluripotent stem (iPS)
cells by the OSKM transcription factors (also called Yamanaka factors)
can be divided into a stochastic and a deterministic phase
4,5
. During
this process cells undergo a mesenchymal–epithelial transition (MET)
and activate endogenous pluripotency genes, paralleled by changes in
histone marks, nucleosome positioning and chromatin accessibility
6
.
In addition, their promoters become de-methylated, preceded by oxida-
tion of methylated CpGs through the dioxygenases Tet2 and Tet1, both
of which have been implicated in the establishment of pluripotency
7,8
.
Earlier work showed that co-expression of C/EBPa with OSKM increases
the reprogramming efficiency of B cells ,15-fold, reaching ,3% of the
population
1
. Here we describe that a pulse of C/EBPa followed by
OSKM overexpression permits the rapid reprogramming of B cells into
iPS cells by activating Tet2 and facilitating accessibility of pluripotency
gene promoters to Oct4 binding. Highly efficient reprogramming of
somatic cells has recently also been reported with a loss-of-function
approach
9
.
Committed B-cell precursors (hereafter referred to as B cells) can be
induced to transdifferentiate into macrophages at 100% efficiency by
forced C/EBPa expression, deregulating ,7,500 genes
10
. Reasoning
that chromatin of cells in transition might be more ‘open’ than that of
end stages, we tested the effect of transiently exposing B cells to C/EBPa,
followed by OSKM expression. B cells were isolated from the bone
marrow of reprogrammable mice (containing a tetracycline-controlled
transactivator (rtTA) and a doxycycline-responsive OSKM cassette
11
),
infected them with C/EBPa-ER-hCD4 retrovirus, sorted human CD4
1
cells 4 days later and incubated them for different times with b-estradiol
(E2) followed by a wash-out (Fig. 1a). Subsequently, OSKM was induced
by doxycycline treatment and Nanog
1
colonies scored 12 days post
induction (d.p.i.). B cells continuously co-expressing C/EBPa with OSKM
showed an 11-fold enhancement in reprogramming efficiency com-
pared to cells induced with OSKM alone (B1OSKM cells), confirming
earlier reports
1,12
. In contrast, cells treated for 18h with E2 and then
treated with doxycycline (Ba91OSKM cells) exhibited a 103-fold colony
increase, with cells pulsed for 6h already showing a 74-fold increase
(Fig. 1b–d). Clonal assays showed that 92–94% of viable colonies were
Nanog
1
after 12 days (Extended Data Fig. 1a–c). OSKM induction of B
cells pre-treated with E2 did not increase Nanog
1
colony numbers nor
Oct4 expression levels (Extended Data Fig. 2a). A mutant of C/EBPa
(BRM-2 (ref. 13)) defective for DNA binding failed to enhance iPS
cell generation (Extended Data Fig. 2b). Induced Ba91OSKM cells
remained .50% viable (Extended Data Fig. 2c). C/EBPa pulses after
OSKM induction had no effect on reprogramming efficiency (Extended
Data Fig. 2d), indicating that C/EBPa acts as a path-breaker for OSKM-
induced reprogramming. Stable iPS cell lines derived from Ba91OSKM
cells (aiPS cells) displayed similar gene expression profiles as embry-
onic stem (ES) cells, differentiated into all three germ layers in vitro and
in vivo, and efficiently contributed to coat colour chimaerism (Fig. 1e
and Extended Data Fig. 3a–e).
Ba91OSKM-derived iPS cell colonies could be identified as early as
4 d.p.i. and their numbers increased modestly after 8days (Fig. 1f, g),
whereas B1OSKM colonies continued to increase 8–10 d.p.i. (Fig. 1f, g).
Retroviral hCD4 expression was found to be silenced within 2–4 days in
Ba91OSKM cells compared to ,8 days in B1OSKM cells (Fig. 1h). To
test the effect of C/EBPa on OSKM-induced transgene independence,
Ba9 and B cells were doxycycline-treated for different times (Fig. 1i).
The first transgene-independent iPS cell colonies were observed after
4 days for Ba91OSKM cells, compared to 9–10 days for B1OSKM
controls (Fig. 1j). Together these results show that the C/EBPa pulse
accelerates iPS cell reprogramming by 4–6 days.
Gene expression analyses of Ba91OSKM cells 8 d.p.i. showed the
upregulation of 764 out of 1,668 genes expressed more highly in ES
cells than in B cells, including all well described pluripotency genes
(Fig. 2a). Unsupervised hierarchical clustering analysis revealed that
8 d.p.i. Ba1OSKM cells clustered with ES/iPS cells (Extended Data
Fig. 4a). A large part of known pluripotency genes were activated
2–6 d.p.i. (Fig. 2b, c and Extended Data Fig. 4b), reaching levels com-
parable to aiPS cells and ES cells within ,1 week (Fig. 2c and Extended
Data Fig. 4c). In contrast, they remained essentially silent in B1OSKM
cells (Fig. 2b, c). In addition, five genes described to be activated very late
(21 d.p.i.) during fibroblast reprogramming
14
became upregulated within
2–4 days in Ba91OSKM cells (Extended Data Fig. 4d). Using Oct4–
GFP reporter mice
12
crossed with reprogrammable mice
11
,Ba91OSKM
*These authors contributed equally to this work.
1
Gene Regulation, Stem Cells and Cancer Programme, Centre for Genomic Regulation (CRG), Dr Aiguader 88, 08003 Barcelona, Spain.
2
Universitat Pompeu Fabra (UPF), Dr Aiguader 88, 08003 Barcelona,
Spain.
3
Ecole Normale Supe ´ rieure, Institut de Biologie de l’ENS, 45 Rue d’Ulm, Paris F-75005, France.
4
Inserm, U1024, Paris F-75005, France.
5
CNRS, UMR 8197, Paris F-75005, France.
6
Yale Cancer
Center, Yale University School of Medicine, 333 Cedar Street, New Haven, Connecticut 06510, USA.
7
Institucio ´ Catalana de Recerca i Estudis Avançats (ICREA), Pg Lluis Companys 23, 08010 Barcelona,
Spain. {Present address: Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York 10065, USA.
13 FEBRUARY 2014 | VOL 506 | NATURE | 235
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