The
discovery
of
RNA
interference
(RNAi)
has
revolutionized
molecular
biology
and
therapeutic
development,
offering
a
novel
approach
to
gene
silencing.
Despite
significant
progress,
critical
gap
remains
in
fully
harnessing
the
potential
RNAi-based
therapies
for
broader
range
diseases,
particularly
beyond
hepatic
targets.
This
review
focuses
on
strategies
overcome
unique
barriers
posed
by
skin
examines
recent
advances
nanoparticle-based
delivery
systems
RNAi
targeting
diseases.
Nanoparticles
(NPs)—including
lipid-based,
inorganic,
polymer-based
carriers—have
demonstrated
improved
drug
delivery,
sustained
release
precise
effects.
Although
several
drugs
have
already
been
approved,
further
research
is
necessary
expand
their
application
Furthermore,
innovative
are
crucial
enhancing
efficacy,
overcoming
barrier.
findings
indicate
that
despite
considerable
innovation
NP
design
mechanisms
essential
exploit
dermatology.
Nucleic
acid
therapeutics
represent
a
highly
promising
treatment
approach
in
modern
medicine,
treating
diseases
at
the
genetic
level.
However,
these
face
numerous
challenges
practical
applications,
particularly
regarding
their
stability,
effectiveness,
cellular
uptake
efficiency,
and
limitations
delivering
them
specifically
to
target
tissues.
To
overcome
obstacles,
researchers
have
developed
various
innovative
delivery
systems,
including
viral
vectors,
lipid
nanoparticles,
polymer
inorganic
protein
carriers,
exosomes,
antibody
oligonucleotide
conjugates,
DNA
nanostructure-based
systems.
These
systems
enhance
therapeutic
efficacy
of
nucleic
drugs
by
improving
targeting
specificity,
half-life
vivo.
In
this
review,
we
systematically
discuss
different
types
drugs,
analyze
major
barriers
encountered
delivery,
summarize
current
research
progress
emerging
We
also
highlight
latest
advancements
application
for
diseases,
infectious
cancer,
brain
wound
healing.
This
review
aims
provide
comprehensive
overview
drug
systems'
status
future
directions
integrating
nanotechnology,
biomaterials
science,
gene
editing
technologies,
emphasizing
transformative
potential
precision
medicine.
Bioconjugate Chemistry,
Год журнала:
2025,
Номер
unknown
Опубликована: Фев. 12, 2025
Brain
tumors,
particularly
glioblastomas,
represent
the
most
complicated
cancers
to
treat
and
manage
due
their
highly
invasive
nature
protective
barriers
of
brain,
including
blood-brain
barrier
(BBB).
The
efficacy
currently
available
treatments,
viz.,
radiotherapy,
chemotherapy,
immunotherapy,
are
frequently
limited
by
major
side
effects,
drug
resistance,
restricted
penetration
into
brain.
Lipid
nanoparticles
(LNPs)
have
emerged
as
a
promising
targeted
delivery
system
for
brain
tumors.
nanocarriers
gained
tremendous
attention
tumor
therapeutics
multiple
encapsulation
abilities,
controlled
release,
better
biocompatibility,
ability
cross
BBB.
Herein,
detailed
analysis
design,
mechanisms,
therapeutic
benefits
LNPs
in
treatment
is
discussed.
Moreover,
we
also
discuss
safety
issues
clinical
developments
current
future
challenges.
Further,
focused
on
transformation
therapy
eliminating
effects
engineering
overcome
related
biological
barriers,
which
provide
personalized,
affordable,
low-risk
options.
Abstract
Small
molecule
drugs
are
increasingly
emerging
as
innovative
and
effective
treatments
for
various
diseases,
with
mRNA
therapeutics
being
a
notable
representative.
The
success
of
COVID‐19
vaccines
has
underscored
the
transformative
potential
in
RNA
therapeutics.
Within
family,
there
is
another
unique
type
known
circRNA.
This
single‐stranded
closed‐loop
offers
advantages
over
mRNA,
including
enhanced
stability
prolonged
protein
expression,
which
may
significantly
impact
therapeutic
strategies.
Furthermore,
circRNA
plays
pivotal
role
pathogenesis
such
cancers,
autoimmune
disorders,
cardiovascular
making
it
promising
clinical
intervention
target.
Despite
these
benefits,
application
settings
remains
underexplored.
review
provides
comprehensive
overview
current
state
synthetic
therapeutics,
focusing
on
its
synthesis,
optimization,
delivery,
diverse
applications.
It
also
addresses
challenges
impeding
advancement
from
bench
to
bedside.
By
summarizing
aspects,
aims
equip
researchers
insights
into
ongoing
developments
future
directions
Highlighting
both
progress
existing
gaps
research,
this
valuable
perspectives
advancing
field
guiding
investigations.
Pharmaceutics,
Год журнала:
2025,
Номер
17(2), С. 157 - 157
Опубликована: Янв. 24, 2025
Background/Objectives:
Transforming
Growth
Factor-beta
(TGFβ1)
plays
a
core
role
in
the
process
of
pulmonary
fibrosis
(PF).
The
progression
can
be
alleviated
by
siRNA-based
inhibiting
TGF-β1.
However,
limitations
naked
siRNA
lead
to
failure
achieving
therapeutic
effect.
This
study
aimed
design
lipid
nanoparticles
(LNPs)
that
deliver
siTGF-β1
lungs
for
purposes.
Methods:
cytotoxicity
and
transfection
assay
vitro
were
used
screen
ionizable
lipids
(ILs).
Design
Experiments
(DOE)
was
obtain
novel
LNPs
enhance
resistance
atomization
shear
forces.
Meanwhile,
impact
encapsulating
(siTGFβ1-LNPs)
on
PF
investigated.
Results:
When
DLin-DMA-MC3
(MC3)
as
ILs,
phase
ratio
MC3:DSPC:DMG-PEG2000:cholesterol
=
50:10:3:37,
N/P
3.25;
siTGFβ1-LNPs
could
stably
delivered
via
converting
solution
into
an
aerosol
(atomization).
In
experiments
have
confirmed
high
safety,
encapsulation,
promote
cellular
uptake
endosomal
escape.
addition,
significantly
reduced
inflammatory
infiltration
attenuated
deposition
extracellular
matrix
(ECM)
protected
lung
tissue
from
toxicity
bleomycin
(BLM)
without
causing
systemic
toxicity.
Conclusions:
effectively
lungs,
resulting
silencing
TGF-β1
mRNA
inhibition
epithelial–mesenchymal
transition
pathway,
thereby
delaying
PF,
which
provides
new
method
treatment
intervention
PF.
Gene
and
RNA-based
therapeutics
represent
a
promising
frontier
in
oncology,
enabling
targeted
modulation
of
tumor-associated
genes
proteins.
This
review
explores
the
latest
advances
payload
vectorization
delivery
systems
developed
for
vivo
cancer
treatments.
We
discuss
viral
non-viral
organic
particles,
including
lipid
based
nanoparticles
polymeric
structures,
effective
transport
plasmids,
siRNA,
self-amplifying
RNA
therapeutics.
Their
physicochemical
properties,
strategies
to
overcome
intracellular
barriers,
innovations
cell-based
carriers
engineered
extracellular
vesicles
are
highlighted.
Moreover,
we
consider
oncolytic
viruses,
novel
capsid
modifications,
approaches
that
refine
tumor
targeting
immunomodulation.
Ongoing
clinical
trials
regulatory
frameworks
guide
future
directions
emphasize
need
safe,
scalable
production.
The
potential
convergence
these
with
combination
therapies
paves
way
toward
personalized
medicine.
Advanced Materials,
Год журнала:
2025,
Номер
unknown
Опубликована: Фев. 9, 2025
Lipid
nanoparticles
(LNPs)
with
highly
efficient
and
specific
extrahepatic
targeting
abilities
are
promising
in
gene
delivery,
the
lipopeptides
(LPs)
excellent
designability
functionality
expected
to
empower
construction
of
functional
LNPs.
This
study
aims
develop
ionizable
components
that
accurately
match
different
lipid
systems
through
modular
design
LPs.
Based
on
this,
a
lipopeptide-based
organ-specific
(POST)
LNP
screening
strategy
is
constructed,
which
lysine-histidine-based
(KH-LPs)
designed
as
components.
The
optimal
KH-LP
screened
vitro
shows
siRNA/mRNA
transfecting
ability
various
hard-to-transfect
cell
lines.
Compared
classic
LNPs,
POST
LNPs
vivo
achieve
even
higher
(or
at
least
comparable)
efficiency
specificity
delivering
mRNA
siRNA
lung,
liver,
spleen,
respectively.
structure-activity
relationship
(SAR)
proves
regulation
LP
structures
can
provide
for
systems,
demonstrating
potential
this
developing
selective
open
up
more
possibilities
therapy.
Frontiers in Pharmacology,
Год журнала:
2025,
Номер
15
Опубликована: Янв. 8, 2025
The
application
of
nanomedicine
in
inflammatory
bowel
disease
(IBD)
has
gained
significant
attention
the
recent
years.
As
field
rapidly
evolves,
analyzing
research
trends
and
identifying
hotpots
are
essential
for
guiding
future
advancements,
a
comprehensive
bibliometric
can
provide
valuable
insights.
current
focused
on
publications
from
2001
to
2024,
was
sourced
Web
Science
Core
Collection
(WoSCC).
CiteSpace
VOSviewer
were
employed
visualize
authors,
institutions,
countries,
co-cited
references,
keywords,
thereby
mapping
intellectual
structure
emerging
field.
analysis
covered
1,518
literature
across
447
journals,
authored
by
9,334
researchers
5,459
institutions
287
countries/regions.
global
publication
numbers
exhibited
an
upward
trend,
particularly
last
decade,
with
China
leading
as
top
publishing
country
Chinese
Academy
Sciences
foremost
institution.
Dr.
Xiao
Bo
is
prominent
figure
advanced
drug
delivery
systems.
This
interdisciplinary
field,
which
spans
materials
science,
pharmacy,
medicine,
seen
influential
mainly
concentrated
targeted
nanoparticles
treatment
IBD.
Keyword
revealed
that
hotspots
include
delivery,
immune
cell
regulation,
antioxidant
damage,
intestinal
microbiota
homeostasis,
nanovesicles.
study
offers
overview
landscape,
emphasizing
rapid
growth
increasing
complexity
this
It
identifies
key
trends,
including
efforts
enhance
precision,
efficacy,
safety
applications.
Emerging
directions
highlighted
crucial
further
progress
evolving
area.
