Progress and prospects of mRNA-based drugs in pre-clinical and clinical applications
Yingying Shi,
No information about this author
Miaoyuan Shi,
No information about this author
Yì Wáng
No information about this author
et al.
Signal Transduction and Targeted Therapy,
Journal Year:
2024,
Volume and Issue:
9(1)
Published: Nov. 14, 2024
Abstract
In
the
last
decade,
messenger
ribonucleic
acid
(mRNA)-based
drugs
have
gained
great
interest
in
both
immunotherapy
and
non-immunogenic
applications.
This
surge
can
be
largely
attributed
to
demonstration
of
distinct
advantages
offered
by
various
mRNA
molecules,
alongside
rapid
advancements
nucleic
delivery
systems.
It
is
noteworthy
that
immunogenicity
presents
a
double-edged
sword.
context
immunotherapy,
extra
supplementation
adjuvant
generally
required
for
induction
robust
immune
responses.
Conversely,
non-immunotherapeutic
scenarios,
activation
unwanted
considering
host
tolerability
high
expression
demand
mRNA-encoded
functional
proteins.
Herein,
mainly
focused
on
linear
non-replicating
mRNA,
we
overview
preclinical
clinical
progress
prospects
medicines
encompassing
vaccines
other
therapeutics.
We
also
highlight
importance
focusing
host-specific
variations,
including
age,
gender,
pathological
condition,
concurrent
medication
individual
patient,
maximized
efficacy
safety
upon
administration.
Furthermore,
deliberate
potential
challenges
may
encounter
realm
disease
treatment,
current
endeavors
improvement,
as
well
application
future
advancements.
Overall,
this
review
aims
present
comprehensive
understanding
mRNA-based
therapies
while
illuminating
prospective
development
drugs.
Language: Английский
Why Do Lipid Nanoparticles Target the Liver? Understanding of Biodistribution and Liver-Specific Tropism
Molecular Therapy — Methods & Clinical Development,
Journal Year:
2025,
Volume and Issue:
33(1), P. 101436 - 101436
Published: Feb. 16, 2025
Lipid
nanoparticles
(LNPs)
are
now
highly
effective
transporters
of
nucleic
acids
to
the
liver.
This
liver-specificity
is
largely
due
their
association
with
certain
serum
proteins,
most
notably
apolipoprotein
E
(ApoE),
which
directs
them
liver
cells
by
binding
low-density
lipoprotein
(LDL)
receptors
on
hepatocytes.
The
liver's
distinct
anatomy,
its
various
specialized
cell
types,
also
influences
how
LNPs
taken
up
from
circulation,
cleared,
and
they
in
delivering
treatments.
In
this
review,
we
consider
factors
that
facilitate
LNP's
targeting
explore
latest
advances
liver-targeted
LNP
technologies.
Understanding
targeted
can
help
for
design
optimization
nanoparticle-based
therapies.
Comprehension
cellular
interaction
biodistribution
not
only
leads
better
treatments
diseases
but
delivers
insight
directing
other
tissues,
potentially
broadening
range
therapeutic
applications.
Language: Английский
Advances in Lipid Nanoparticle‐Based Disease Treatment
Yujie Zhou,
No information about this author
Qiqi Ge,
No information about this author
Xin Wang
No information about this author
et al.
ChemMedChem,
Journal Year:
2025,
Volume and Issue:
unknown
Published: Feb. 17, 2025
Abstract
Lipid
nanoparticles
(LNPs)
have
emerged
as
a
transformative
platform
for
the
targeted
delivery
of
therapeutic
agents,
revolutionizing
treatment
paradigms
across
spectrum
diseases.
Since
inception
liposomes
in
1960s,
lipid‐based
nanotechnology
has
evolved
to
address
limitations
such
poor
bioavailability,
off‐target
effects,
and
instability,
thereby
enhancing
efficacy
safety
drug
administration.
This
review
highlights
latest
advancements
LNPs
technology,
focusing
on
their
application
cancer
therapy,
gene
infectious
disease
management,
glaucoma,
other
clinical
areas.
Recent
studies
underscore
potential
deliver
messenger
RNA
(mRNA)
small
interfering
(siRNA)
precise
genetic
intervention,
exemplified
by
breakthroughs
interference
CRISPR‐Cas9
genome
editing.
Additionally,
been
successfully
employed
ameliorate
conditions,
demonstrating
versatility
addressing
both
acute
chronic
disorders.
However,
challenges
persist
concerning
large‐scale
manufacturing,
long‐term
stability,
comprehensive
evaluations.
Future
research
must
focus
optimizing
formulations,
exploring
synergistic
combinations
with
existing
therapies,
expanding
scope
treatable
The
integration
into
personalized
medicine
exploration
applications
diseases
represent
promising
avenues
further
investigation.
are
poised
play
an
increasingly
central
role
development
next‐generation
therapeutics.
Language: Английский
Comprehensive insights of etiological drivers of hepatocellular carcinoma: Fostering targeted nano delivery to anti-cancer regimes
Biochimica et Biophysica Acta (BBA) - Reviews on Cancer,
Journal Year:
2025,
Volume and Issue:
unknown, P. 189318 - 189318
Published: April 1, 2025
Language: Английский
In Vivo Engineered CAR-T Cell Therapy: Lessons Built from COVID-19 mRNA Vaccines
Sikun Meng,
No information about this author
Tomoaki Hara,
No information about this author
Yutaka Miura
No information about this author
et al.
International Journal of Molecular Sciences,
Journal Year:
2025,
Volume and Issue:
26(7), P. 3119 - 3119
Published: March 28, 2025
Chimeric
antigen
receptor
T
cell
(CAR-T)
therapy
has
revolutionized
cancer
immunotherapy
but
continues
to
face
significant
challenges
that
limit
its
broader
application,
such
as
targeting,
the
tumor
microenvironment,
and
persistence,
especially
in
solid
tumors.
Meanwhile,
global
implementation
of
mRNA
vaccines
during
COVID-19
pandemic
highlighted
transformative
potential
lipid
nanoparticle
(LNP)
technologies.
These
innovations,
characterized
by
their
swift
development
timelines,
precise
design,
efficient
delivery
mechanisms,
provide
a
promising
framework
address
some
limitations
CAR-T
therapy.
Recent
advancements,
including
mRNA-based
CAR
engineering
optimized
LNP
delivery,
have
demonstrated
capacity
enhance
efficacy,
particularly
context
This
review
explores
how
mRNA-LNP
technology
can
drive
vivo
engineered
therapies
current
discusses
future
directions,
advancements
optimization,
strategies
for
improving
functionality
safety.
By
bridging
these
technological
insights,
may
evolve
into
versatile
accessible
treatment
paradigm
across
diverse
oncological
landscapes.
Language: Английский
Targeted lipid nanoparticles distributed in hydrogel treat osteoarthritis by modulating cholesterol metabolism and promoting endogenous cartilage regeneration
Zhibo Zhao,
No information about this author
Peng Wang,
No information about this author
Ziyang Li
No information about this author
et al.
Journal of Nanobiotechnology,
Journal Year:
2024,
Volume and Issue:
22(1)
Published: Dec. 20, 2024
Osteoarthritis
(OA)
is
the
most
common
disease
in
aging
joints
and
has
characteristics
of
cartilage
destruction
inflammation.
It
currently
considered
a
metabolic
disease,
CH25H-CYP7B1-RORα
axis
cholesterol
metabolism
chondrocytes
plays
crucial
catabolic
regulatory
role
its
pathogenesis.
Targeting
this
may
provide
therapeutic
approach
for
OA
treatment.
Here,
study,
we
propose
to
use
combination
stem
cell-recruiting
hydrogels
lipid
nanoparticles
(LNPs)
that
modulate
jointly
promote
regenerative
microenvironment.
Specifically,
first
developed
an
injectable,
bioactive
hydrogel
composed
self-assembling
peptide
nanofibers
recruits
endogenous
synovial
cells
(SMSCs)
promotes
their
chondrogenic
differentiation.
At
same
time,
LNPs
regulate
are
incorporated
into
slowly
released,
thereby
improving
inflammatory
environment
OA.
Enhancements
were
noted
conditions
associated
with
OA,
alongside
successful
attraction
mesenchymal
(MSCs)
from
membrane.
These
then
observed
differentiate
chondrocytes,
contributing
effective
restoration
chondrocyte
regeneration,
offering
promising
In
summary,
provides
feasible
siRNA-based
option,
potential
nonsurgical
solution
treatment
Language: Английский