Frontiers in Cell and Developmental Biology,
Journal Year:
2024,
Volume and Issue:
12
Published: Nov. 15, 2024
Retinal
pathologies
are
major
causes
of
vision
impairment
and
blindness
in
humans,
inherited
retinal
diseases
(IRDs),
such
as
retinitis
pigmentosa,
Leber
congenital
amaurosis,
Stargardt
disease,
greatly
contribute
to
this
problem.
In
vitro
disease
modeling
can
be
used
for
understanding
the
development
pathology
screening
therapeutic
pharmaceutical
compounds.
preclinical
research
phase,
models
complement
vivo
by
reducing
animal
studies,
decreasing
costs,
shortening
timelines.
Additionally,
may
not
always
accurately
replicate
human
phenotype.
This
review
examines
types
cells
that
create
IRD
models,
including
retina-specific
cell
lines,
primary
cells,
induced
pluripotent
stem
(iPSCs),
more.
Special
attention
is
given
mesenchymal
(MSCs),
which
characterized
various
isolation
sources,
relative
ease
isolation,
straightforward
differentiation.
MSCs
derived
from
bone
marrow
(BM),
adipose
tissue
(AT),
dental
(DT),
umbilical
cord
(UC),
other
sources
differentiate
into
photoreceptor
pigment
epithelial
(RPE)
dysfunction
most
commonly
associated
with
IRDs.
Subsequent
differentiation
carried
out
via
methods:
culturing
induction
media
supplemented
certain
growth
factors,
co-culturing
or
their
conditioned
media,
regulating
gene
expression
viral
vector-delivered
transcription
factors
(TFs)
microRNAs
(miRNAs).
Compared
popular
iPSCs,
example,
MSC-based
significantly
cheaper
faster
obtain,
making
them
more
feasible
large-scale
drug
screening.
Nevertheless,
existing
methods
need
further
optimization
promising
platform
receive
success
it
deserves.
Heliyon,
Journal Year:
2024,
Volume and Issue:
10(16), P. e35836 - e35836
Published: Aug. 1, 2024
Stem
cell
transplantation
has
emerged
as
a
promising
avenue
in
regenerative
medicine,
potentially
facilitating
tissue
repair
degenerative
diseases
and
injuries.
This
review
comprehensively
examines
recent
developments
challenges
stem
transplantation.
It
explores
the
identification
isolation
of
various
types,
including
embryonic,
induced
pluripotent,
adult
cells
derived
from
multiple
sources.
Additionally,
highlights
tissue-specific
applications
these
cells,
focusing
on
bone
cartilage
regeneration,
treatment
neurological
disorders,
management
hematological
conditions.
Future
advancements
effective
resolution
current
will
be
crucial
fully
realizing
potential
medicine.
With
responsible
ethical
practices,
field
can
transform
disease
injury
treatment,
ultimately
improving
quality
life
for
countless
individuals.
Frontiers in Oncology,
Journal Year:
2025,
Volume and Issue:
15
Published: Feb. 7, 2025
This
review
summarizes
the
applications
and
research
progress
of
organoid
models
in
colorectal
cancer
research.
First,
high
incidence
mortality
rates
are
introduced,
emphasizing
importance
organoids
as
a
model.
Second,
this
provides
detailed
introduction
to
concept,
biological
properties,
organoids,
including
their
strengths
mimicking
structural
functional
aspects
organs.
article
further
analyzes
adult
stem
cell-derived
pluripotent
discusses
advancements
for
basic
research,
drug
development,
personalized
treatment
evaluation
prediction,
regenerative
medicine.
Finally,
prospects
applying
technology
its
significant
value
improving
patient
survival
rates.
In
conclusion,
systematically
explains
highlighting
tremendous
potential
promising
Regenerative Therapy,
Journal Year:
2024,
Volume and Issue:
26, P. 382 - 386
Published: June 1, 2024
Retinal
organoids
are
three-dimensional
(3D)
microscopic
tissues
that
induced
and
differentiated
from
stem
cells
or
progenitor
in
vitro
have
a
highly
similar
structure
to
the
retina.
With
optimization
development
of
3D
retinal
culture
system
improvement
differentiation
technology,
broad
application
prospects
development,
regenerative
medicine,
biomaterial
evaluation,
disease
mechanism
investigation,
drug
screening.
In
this
review
we
summarize
recent
their
applications
ophthalmic
medicine.
particular,
highlight
promise
challenges
use
modeling
discovery.
Investigative Ophthalmology & Visual Science,
Journal Year:
2024,
Volume and Issue:
65(13), P. 23 - 23
Published: Nov. 13, 2024
Purpose:
CLN3
Batten
disease
(also
known
as
juvenile
neuronal
ceroid
lipofuscinosis)
is
a
lysosomal
storage
disorder
that
typically
initiates
with
retinal
degeneration
but
followed
by
seizure
onset,
motor
decline
and
premature
death.
Patient-derived
induced
pluripotent
stem
cell–RPE
cells
show
defective
phagocytosis
of
photoreceptor
outer
segment
(POS).
Because
modifier
genes
are
implicated
in
disease,
our
goal
here
was
to
investigate
direct
link
between
mutation
POS
defect.
Methods:
Isogenic
control
mutant
cell
lines
were
generated
CRISPR-Cas9-mediated
biallelic
deletion
exons
7
8.
A
transgenic
CLN3Δ7–8/Δ7–8
(CLN3)
Yucatan
miniswine
also
used
study
the
impact
on
phagocytosis.
cultured
RPE
analyzed
Western
blotting
immunohistochemistry.
Electroretinogram,
optical
coherence
tomography
histological
analysis
wild-type
eyes
carried
out
at
6,
36,
or
48
months
age.
Results:
(CLN3
RPE)
displayed
decreased
binding
consequently
uptake
compared
isogenic
cells.
Furthermore,
phagocytosed
less
efficiently
than
POS.
Consistent
phagocytosis,
lipofuscin/autofluorescence
36
age
almost
complete
loss
photoreceptors
Conclusions:
CLN3Δ7
–
8/
Δ7
8
(which
affects
≤85%
patients)
both
leads
disease.
primary
dysfunction
independently
contribute
impaired
Translational Vision Science & Technology,
Journal Year:
2024,
Volume and Issue:
13(12), P. 28 - 28
Published: Dec. 17, 2024
Inherited
retinal
degeneration
(IRD)
disease
and
age-related
macular
(AMD)
are
leading
causes
of
irreversible
vision
loss
blindness.
Although
significant
progress
has
advanced
the
field
in
past
5
years,
challenges
remain.
The
current
article
reviews
accomplishments
research
advances
that
have
fueled
development
treatments
for
patients
with
IRD
AMD,
including
first
approved
gene-augmentation
treatment
RPE65-related
complement
inhibition
therapies
to
slow
progression
geographic
atrophy
(GA)
AMD.
outlines
opportunities
address
gaps
unmet
needs
should
lead
additional
toward
IRDs
non-neovascular
AMD
future.
bioRxiv (Cold Spring Harbor Laboratory),
Journal Year:
2024,
Volume and Issue:
unknown
Published: June 9, 2024
Abstract
Purpose
CLN3
Batten
disease
(also
known
as
Juvenile
Neuronal
Ceroid
Lipofuscinosis;
JNCL)
is
a
lysosomal
storage
disorder
that
typically
initiates
with
retinal
degeneration
but
followed
by
seizure
onset,
motor
decline
and
premature
death.
Patient-derived
iPSC-RPE
cells
show
defective
phagocytosis
of
photoreceptor
outer
segments
(POSs).
Because
modifier
genes
are
implicated
in
disease,
our
goal
here
was
to
investigate
direct
link
between
mutation
POS
defect.
Methods
Isogenic
control
mutant
stem
cell
lines
were
generated
CRISPR-Cas9mediated
biallelic
deletion
exons
7
8.
A
transgenic
Δ
7-8/
7-8
(
)
Yucatan
miniswine
also
used
study
the
impact
on
phagocytosis.
cultured
RPE
analyzed
Western
blotting
immunohistochemistry.
Electroretinogram,
optical
coherence
tomography
histological
analysis
7/8
wild-type
eyes
carried
out
at
6-,
36-,
or
48-month
age.
Results
RPE)
displayed
reduced
binding
consequently
decreased
uptake
compared
isogenic
cells.
Furthermore,
phagocytosed
less
efficiently
than
POS.
Consistent
phagocytosis,
lipofuscin/autofluorescence
36
months-of-age
almost
complete
loss
photoreceptors
48
months
Conclusions
(that
affects
up
85%
patients)
both
POSs
leads
disease.
primary
dysfunction
independently
contribute
impaired
