Advanced Science,
Год журнала:
2024,
Номер
unknown
Опубликована: Ноя. 25, 2024
Abstract
Lipid
Nanoparticles
(LNPs)
recently
emerged
as
an
invaluable
RNA
delivery
platform.
With
many
LNP‐based
therapeutics
in
the
pre‐clinical
and
clinical
pipelines,
there
is
extensive
research
dedicated
to
improving
LNPs.
These
efforts
focus
mainly
on
tolerability
transfectability
of
new
ionizable
lipids
RNAs,
or
modulating
LNPs
biodistribution
with
active
targeting
strategies.
However,
most
formulations
follow
well‐established
lipid
proportions
used
clinically
approved
products.
Nevertheless,
investigating
effects
composition
their
can
expand
toolbox
for
particle
design,
leading
improved
Herein,
a
(30‐n‐LNPs)
formulation
increasing
amounts
phospholipids
investigated
possible
mRNA
system
treating
Inflammatory
Bowel
Diseases.
Compared
benchmark
(b‐LNPs),
n‐LNPs
containing
30%
distearoylphosphatidylcholine
(DSPC)
are
well
tolerated
following
intravenous
administration
display
natural
toward
inflamed
colon
dextran
sodium
sulfate
(DSS)‐colitis
bearing
mice,
while
de‐targeting
clearing
organs
such
liver
spleen.
Using
interleukin‐10‐encoding
therapeutic
cargo,
demonstrated
reduction
pathological
burden
colitis‐bearing
mice.
represent
starting
point
further
investigate
influence
systemic
biodistribution,
ultimately
opening
modalities
different
pathologies.
Signal Transduction and Targeted Therapy,
Год журнала:
2025,
Номер
10(1)
Опубликована: Апрель 29, 2025
Asbstract
Despite
aggressive
therapy,
glioblastoma
(GBM)
recurs
in
almost
all
patients
and
treatment
options
are
very
limited.
our
growing
understanding
of
how
cellular
transitions
associate
with
relapse
GBM,
critical
gaps
remain
ability
to
block
these
molecular
changes
treat
recurrent
disease.
In
this
study
we
combine
computational
biology,
forward-thinking
miRNA
biology
cutting-edge
nucleic
acid
delivery
vehicles
advance
targeted
therapeutics
for
GBM.
Computational
analysis
RNA
sequencing
from
clinical
GBM
specimens
identified
TGFβ
type
II
receptor
(TGFBR2)
as
a
key
player
the
mesenchymal
transition
associated
worse
outcome
Mechanistically,
show
that
elevated
levels
TGFBR2
is
conducive
reduced
temozolomide
(TMZ)
sensitivity.
This
effect
is,
at
least
partially,
induced
by
stem-cell
driving
events
coordinated
reprogramming
transcription
factors
Oct4
Sox2
lead
open
chromatin
states.
We
blocking
via
pharmacological
approaches
decreases
stem
cell
capacity
sensitivity
(rGBM)
isolates
TMZ
vitro.
Network
uncovered
miR-590-3p
tumor
suppressor
simultaneously
inhibits
multiple
oncogenic
nodes
downstream
TGFBR2.
also
developed
novel
biodegradable
lipophilic
poly(β-amino
ester)
nanoparticles
(LiPBAEs)
vivo
microRNA
(miRNAs)
delivery.
Following
direct
intra-tumoral
infusion,
nanomiRs
efficiently
distribute
through
tumors.
Importantly,
inhibited
growth
extended
survival
mice
bearing
orthotopic
human
rGBM
xenografts,
an
apparent
30%
cure
rate.
These
results
miRNA-based
provide
new
opportunities
bypass
resistance
standard
care
therapy.
Nanotechnology,
Год журнала:
2025,
Номер
36(22), С. 222003 - 222003
Опубликована: Май 16, 2025
Abstract
Chimeric
antigen
receptor
T
cells
(CAR-T)
immunotherapy
has
achieved
remarkable
progress
in
the
treatment
of
hematological
malignancies.
However,
it
encounters
challenges
including
complex
manufacturing
processes,
high
cost,
and
safety
issues.
Lipid
nanoparticle
(LNP)
technology,
as
an
advanced
gene
delivery
platform,
offers
significant
advancements
to
CAR-T
therapy
through
its
efficiency,
low
immunogenicity,
safety.
LNP
enable
vivo
production
cells,
thereby
improving
reducing
risks
immunogenicity
insertional
mutations,
simplifying
process
costs.
The
scalability
rapid
optimization
ability
position
them
promising
candidates
for
cell
production.
technology
is
expected
further
promote
development
provide
safer
more
economical
options.
Therefore,
this
paper
aims
a
comprehensive
systematic
review
application
therapy.
In
review,
we
initially
outline
fundamental
design,
process,
current
Subsequently,
present
characteristics
LNP,
their
advantages
vectors,
how
they
improve
efficacy
Finally,
summarize
research
landscape
applications
This
includes
enhancing
vitro
transfection
programming
situ
,
facilitating
T-cell
activation,
alleviating
side
effects
therapy,
combining
with
other
immunotherapies.
These
will
aid
design
mRNA
systems
based
on
promoting
Advanced Science,
Год журнала:
2024,
Номер
unknown
Опубликована: Ноя. 25, 2024
Abstract
Lipid
Nanoparticles
(LNPs)
recently
emerged
as
an
invaluable
RNA
delivery
platform.
With
many
LNP‐based
therapeutics
in
the
pre‐clinical
and
clinical
pipelines,
there
is
extensive
research
dedicated
to
improving
LNPs.
These
efforts
focus
mainly
on
tolerability
transfectability
of
new
ionizable
lipids
RNAs,
or
modulating
LNPs
biodistribution
with
active
targeting
strategies.
However,
most
formulations
follow
well‐established
lipid
proportions
used
clinically
approved
products.
Nevertheless,
investigating
effects
composition
their
can
expand
toolbox
for
particle
design,
leading
improved
Herein,
a
(30‐n‐LNPs)
formulation
increasing
amounts
phospholipids
investigated
possible
mRNA
system
treating
Inflammatory
Bowel
Diseases.
Compared
benchmark
(b‐LNPs),
n‐LNPs
containing
30%
distearoylphosphatidylcholine
(DSPC)
are
well
tolerated
following
intravenous
administration
display
natural
toward
inflamed
colon
dextran
sodium
sulfate
(DSS)‐colitis
bearing
mice,
while
de‐targeting
clearing
organs
such
liver
spleen.
Using
interleukin‐10‐encoding
therapeutic
cargo,
demonstrated
reduction
pathological
burden
colitis‐bearing
mice.
represent
starting
point
further
investigate
influence
systemic
biodistribution,
ultimately
opening
modalities
different
pathologies.
