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A random Abstract
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Adult human liver mesenchymal progenitor cells express phenylalanine hydroxylase.
[phenylketonuria]
Phenylketonuria
(
PKU
)
is
one
of
the
most
prevalent
inherited
metabolic
diseases
and
is
accountable
for
a
severe
encephalopathy
by
progressive
intoxication
of
the
brain
by
phenylalanine
.
This
results
from
an
ineffective
L-
phenylalanine
hydroxylase
enzyme
(
PAH
)
due
to
a
mutated
phenylalanine
hydroxylase
(
PAH
)
gene
.
Neonatal
screening
programs
allow
an
early
dietetic
treatment
with
restrictive
phenylalanine
intake
.
This
diet
prevents
most
of
the
neuropsychological
disabilities
but
remains
challenging
for
lifelong
compliance
.
Adult-derived
human
liver
progenitor
cells
(
ADHLPC
)
are
a
pool
of
precursors
that
can
differentiate
into
hepatocytes
.
We
aim
to
study
PAH
expression
and
PAH
activity
in
a
differenciated
ADHLPC
.
ADHLPC
were
isolated
from
human
hepatocyte
primary
culture
of
two
different
donors
and
differenciated
under
specific
culture
conditions
.
We
demonstrated
the
high
expression
of
PAH
and
a
large
increase
of
PAH
activity
in
differenciated
LPC
.
The
age
of
the
donor
,
the
cellular
viability
after
liver
digestion
and
cryopreservation
affects
PAH
activity
.
ADHLPC
might
therefore
be
considered
as
a
suitable
source
for
cell
therapy
in
PKU
.
