Trends in Pharmacological Sciences,
Journal Year:
2024,
Volume and Issue:
45(5), P. 406 - 418
Published: April 12, 2024
T
cells
modified
to
express
intelligently
designed
chimeric
antigen
receptors
(CARs)
are
exceptionally
powerful
therapeutic
agents
for
relapsed
and
refractory
blood
cancers
have
the
potential
revolutionize
therapy
many
other
diseases.
To
circumvent
complexity
cost
associated
with
broad-scale
implementation
of
ex
vivo
manufactured
adoptive
cell
products,
alternative
strategies
generate
CAR
in
by
direct
infusion
nanoparticle-formulated
nucleic
acids
or
engineered
viral
vectors
under
development
received
a
great
deal
attention
past
few
years.
Here,
we
outline
manufacturing
process
as
motivating
framework
discuss
emerging
data
from
preclinical
models
highlight
potency
approach,
applicability
new
disease
indications,
remaining
challenges
clinical
readiness,
including
delivery
specificity,
long
term
efficacy,
safety.
Science,
Journal Year:
2022,
Volume and Issue:
375(6576), P. 91 - 96
Published: Jan. 6, 2022
Fibrosis
affects
millions
of
people
with
cardiac
disease.
We
developed
a
therapeutic
approach
to
generate
transient
antifibrotic
chimeric
antigen
receptor
(CAR)
T
cells
in
vivo
by
delivering
modified
messenger
RNA
(mRNA)
cell–targeted
lipid
nanoparticles
(LNPs).
The
efficacy
these
vivo–reprogrammed
CAR
was
evaluated
injecting
CD5-targeted
LNPs
into
mouse
model
heart
failure.
Efficient
delivery
mRNA
encoding
the
lymphocytes
observed,
which
produced
transient,
effective
vivo.
Antifibrotic
exhibited
trogocytosis
and
retained
target
as
they
accumulated
spleen.
Treatment
mRNA-targeted
reduced
fibrosis
restored
function
after
injury.
In
generation
may
hold
promise
platform
treat
various
diseases.
Advanced Materials,
Journal Year:
2023,
Volume and Issue:
35(51)
Published: May 17, 2023
Abstract
Messenger
RNA
(mRNA)
has
received
great
attention
in
the
prevention
and
treatment
of
various
diseases
due
to
success
coronavirus
disease
2019
(COVID‐19)
mRNA
vaccines
(Comirnaty
Spikevax).
To
meet
therapeutic
purpose,
it
is
required
that
must
enter
target
cells
express
sufficient
proteins.
Therefore,
development
effective
delivery
systems
necessary
crucial.
Lipid
nanoparticle
(LNP)
represents
a
remarkable
vehicle
indeed
accelerated
applications
humans,
as
several
mRNA‐based
therapies
have
already
been
approved
or
are
clinical
trials.
In
this
review,
focus
on
mRNA‐LNP‐mediated
anticancer
therapy.
It
summarizes
main
strategies
mRNA‐LNP
formulations,
discusses
representative
approaches
cancer,
points
out
current
challenges
possible
future
directions
research
field.
hoped
these
delivered
messages
can
help
further
improve
application
technology
cancer
Science,
Journal Year:
2023,
Volume and Issue:
381(6656), P. 436 - 443
Published: July 27, 2023
Hematopoietic
stem
cells
(HSCs)
are
the
source
of
all
blood
over
an
individual's
lifetime.
Diseased
HSCs
can
be
replaced
with
gene-engineered
or
healthy
through
HSC
transplantation
(HSCT).
However,
current
protocols
carry
major
side
effects
and
have
limited
access.
We
developed
CD117/LNP-messenger
RNA
(mRNA),
a
lipid
nanoparticle
(LNP)
that
encapsulates
mRNA
is
targeted
to
cell
factor
receptor
(CD117)
on
HSCs.
Delivery
anti-human
CD117/LNP-based
editing
system
yielded
near-complete
correction
hematopoietic
sickle
cells.
Furthermore,
in
vivo
delivery
pro-apoptotic
PUMA
(p53
up-regulated
modulator
apoptosis)
CD117/LNP
affected
function
permitted
nongenotoxic
conditioning
for
HSCT.
The
ability
target
offers
regimen
HSCT,
this
platform
could
basis
genome
cure
genetic
disorders,
which
would
abrogate
need
