Frontiers in Molecular Neuroscience,
Journal Year:
2019,
Volume and Issue:
12
Published: Dec. 5, 2019
Neuromuscular
and
neurodegenerative
diseases
are
mostly
modelled
using
genetically
modified
animals
such
as
mice.
However,
animal
models
do
not
recapitulate
all
the
phenotypes
that
specific
to
human
disease.
This
is
mainly
due
genetic,
anatomical
physiological
difference
in
neuromuscular
systems
of
human.
The
emergence
direct
indirect
somatic
cell
reprogramming
technologies
may
overcome
this
limitation
because
they
enable
use
disease
patient-specific
cellular
enhanced
platforms
for
drug
discovery
autologous
cell-based
therapy.
Induced
pluripotent
stem
cells
(iPSCs)
urine-derived
(USCs)
increasingly
employed
pathophysiology
various
diseases.
Recent
modelling
approaches
utilizes
highly
complex
differentiation
faithfully
mimic
tissue-
organ-level
dysfunctions.
In
review,
we
discuss
promising
models,
USC-
iPSC-based
approaches,
currently
being
used
model
Cell,
Journal Year:
2024,
Volume and Issue:
187(5), P. 1076 - 1100
Published: Feb. 1, 2024
Genome
editing
has
been
a
transformative
force
in
the
life
sciences
and
human
medicine,
offering
unprecedented
opportunities
to
dissect
complex
biological
processes
treat
underlying
causes
of
many
genetic
diseases.
CRISPR-based
technologies,
with
their
remarkable
efficiency
easy
programmability,
stand
at
forefront
this
revolution.
In
Review,
we
discuss
current
state
CRISPR
gene
technologies
both
research
therapy,
highlighting
limitations
that
constrain
them
technological
innovations
have
developed
recent
years
address
them.
Additionally,
examine
summarize
landscape
applications
context
health
therapeutics.
Finally,
outline
potential
future
developments
could
shape
coming
years.
Acta Pharmaceutica Sinica B,
Journal Year:
2023,
Volume and Issue:
13(6), P. 2483 - 2509
Published: Feb. 15, 2023
New
drug
discovery
is
under
growing
pressure
to
satisfy
the
demand
from
a
wide
range
of
domains,
especially
pharmaceutical
industry
and
healthcare
services.
Assessment
efficacy
safety
prior
human
clinical
trials
crucial
part
development,
which
deserves
greater
emphasis
reduce
cost
time
in
discovery.
Recent
advances
microfabrication
tissue
engineering
have
given
rise
organ-on-a-chip,
an
International Journal of Molecular Sciences,
Journal Year:
2018,
Volume and Issue:
19(4), P. 936 - 936
Published: March 21, 2018
The
rising
interest
in
human
induced
pluripotent
stem
cell
(hiPSC)-derived
organoid
culture
has
stemmed
from
the
manipulation
of
various
combinations
directed
multi-lineage
differentiation
and
morphogenetic
processes
that
mimic
organogenesis.
Organoids
are
three-dimensional
(3D)
structures
comprised
multiple
types,
self-organized
to
recapitulate
embryonic
tissue
development
vitro.
This
model
been
shown
be
superior
conventional
two-dimensional
(2D)
methods
mirroring
functionality,
architecture,
geometric
features
tissues
seen
vivo.
review
serves
highlight
recent
advances
3D
technology
for
use
modeling
complex
hereditary
diseases,
cancer,
host-microbe
interactions,
possible
translational
personalized
medicine
where
cultures
were
used
uncover
diagnostic
biomarkers
early
disease
detection
via
high
throughput
pharmaceutical
screening.
In
addition,
this
also
aims
discuss
advantages
shortcomings
utilizing
organoids
modeling.
summary,
studying
diseases
using
hiPSC-derived
may
better
illustrate
involved
due
similarities
architecture
microenvironment
present
an
organoid,
which
allows
drug
responses
properly
recapitulated
Circulation Research,
Journal Year:
2018,
Volume and Issue:
123(2), P. 224 - 243
Published: July 5, 2018
Life
threatening
ventricular
arrhythmias
leading
to
sudden
cardiac
death
are
a
major
cause
of
morbidity
and
mortality.
In
the
absence
structural
heart
disease,
these
arrhythmias,
especially
in
younger
population,
often
an
outcome
genetic
defects
specialized
membrane
proteins
called
ion
channels.
heart,
exceptionally
well-orchestrated
activity
diversity
channels
mediates
action
potential.
Alterations
either
function
or
expression
can
disrupt
configuration
potential,
abnormal
electrical
that
sometimes
initiate
arrhythmia.
Understanding
pathophysiology
inherited
be
challenging
because
complexity
disorder
lack
appropriate
cellular
vivo
models.
Recent
advances
human
induced
pluripotent
stem
cell
technology
have
provided
remarkable
progress
comprehending
underlying
mechanisms
channel
disorders
channelopathies
by
modeling
complex
arrhythmia
syndromes
vitro
dish.
To
fully
realize
potential
cells
elucidating
mechanistic
basis
channelopathies,
it
is
crucial
basic
knowledge
myocyte
electrophysiology.
this
review,
we
will
discuss
role
various
electrophysiology
molecular
highlighting
promise
cell-cardiomyocytes
as
model
for
investigating
testing
antiarrhythmic
strategies.
Overall,
review
aims
provide
understanding
related
clinicians
research
scientists
cardiovascular
field
with
limited
background.
Human Genomics,
Journal Year:
2020,
Volume and Issue:
14(1)
Published: June 26, 2020
Abstract
Human-induced
pluripotent
stem
cells
(hiPSCs)
and
CRISPR/Cas9
gene
editing
system
represent
two
instruments
of
basic
translational
research,
which
both
allow
to
acquire
deep
insight
about
the
molecular
bases
many
diseases
but
also
develop
pharmacological
research.
This
review
is
focused
draw
up
latest
technique
applied
on
hiPSCs,
exploiting
some
genetic
manipulation
directed
discovery
innovative
therapeutic
strategies.
There
are
expediencies
provided
by
use
can
a
disease
model
clinically
relevant
predictive,
with
great
potential
if
associated
technology,
tool
powered
ease
precision
never
seen
before.
Here,
we
describe
possible
applications
hiPSCs:
from
drug
development
screening
therapy
induction
immunological
response
specific
virus
infection,
such
as
HIV
SARS-Cov-2.
Frontiers in Cell and Developmental Biology,
Journal Year:
2022,
Volume and Issue:
10
Published: Nov. 2, 2022
Over
the
past
2
decades,
mesenchymal
stem
cells
(MSCs)
have
attracted
a
lot
of
interest
as
unique
therapeutic
approach
for
variety
diseases.
MSCs
are
capable
self-renewal
and
multilineage
differentiation
capacity,
immunomodulatory,
anti-inflammatory
properties
allowing
it
to
play
role
in
regenerative
medicine.
Furthermore,
low
tumorigenicity
immune
privileged,
which
permits
use
allogeneic
therapies
that
eliminate
need
collect
directly
from
patients.
Induced
pluripotent
(iPSCs)
can
be
generated
adult
through
gene
reprogramming
with
ectopic
expression
specific
pluripotency
factors.
Advancement
iPS
technology
avoids
destruction
embryos
make
cells,
making
free
ethical
concerns.
iPSCs
self-renew
develop
into
plethora
specialized
useful
resource
medicine
they
may
created
any
human
source.
also
been
used
treat
individuals
infected
SARS-CoV-2
virus.
undergone
more
clinical
trials
than
due
high
tumorigenicity,
trigger
oncogenic
transformation.
In
this
review,
we
discussed
overview
induced
cells.
We
briefly
present
approaches
COVID-19-related
diseases
using
iPSCs.
Cytotherapy,
Journal Year:
2024,
Volume and Issue:
unknown
Published: June 1, 2024
Since
their
first
production
in
2007,
human
induced
pluripotent
stem
cells
(iPSCs)
have
provided
a
novel
platform
for
the
development
of
various
cell
therapies
targeting
spectrum
diseases,
ranging
from
rare
genetic
eye
disorders
to
cancer
treatment.
However,
several
challenges
must
be
tackled
iPSC-based
therapy
enter
market
and
achieve
broader
global
adoption.
This
white
paper,
authored
by
Japanese
Society
Regenerative
Medicine
(JSRM)
-
International
Cell
Therapy
(ISCT)
iPSC
Committee
delves
into
hurdles
encountered
pursuit
safe
economically
viable
therapies,
particularly
standpoint
industry.
It
discusses
differences
guidelines
regulatory
frameworks,
outlines
series
quality
control
tests
required
ensure
safety
therapy,
provides
details
important
considerations
around
cost
goods
(COGs),
including
impact
automated
advanced
manufacturing.
