Biomedical Journal,
Journal Year:
2023,
Volume and Issue:
46(1), P. 70 - 80
Published: Jan. 13, 2023
Since
the
COVID-19
pandemic
was
declared,
vaccines
against
SARS-CoV-2
have
been
urgently
developed
around
world.
On
basis
of
mRNA
vaccine
technology
previously,
were
promptly
tested
in
animals,
advanced
to
clinical
trials,
and
then
authorized
for
emergency
use
humans.
The
administration
has
successfully
reduced
hospitalization
mortality
caused
by
viral
infection,
although
virus
continuously
evolves
with
its
transmission.
Therefore,
development
technology,
including
RNA
modification
delivery
systems,
is
well
recognized
contribution
moderating
harms
pandemic.
scientists
who
these
technologies,
Katalin
Karikó,
Drew
Weissman,
Pieter
Cullis,
awarded
2022
Tang
Prize
Biopharmaceutical
Science.
In
this
review,
we
summarize
principles,
safety
efficacy
as
immune
response
vaccines.
approaches
could
be
practical
prevention
infectious
diseases,
also
briefly
describe
other
human
pathogens
trials.
ACS Nano,
Journal Year:
2023,
Volume and Issue:
17(7), P. 6150 - 6164
Published: March 21, 2023
The
selective
removal
of
misfolded,
aggregated,
or
aberrantly
overexpressed
protein
plays
an
essential
role
in
maintaining
protein-dominated
biological
processes.
In
parallel,
the
precise
knockout
abnormal
proteins
is
inseparable
from
accurate
identification
within
complex
environments.
Guided
by
these
precepts,
small
molecules,
antibodies,
are
commonly
used
as
recognition
tools
for
developing
targeted
degradation
(TPD)
technology.
Indeed,
TPD
has
shown
tremendous
prospects
chronic
diseases,
rare
cancer
research,
and
other
fields.
Meanwhile,
aptamers
short
RNA
DNA
oligonucleotides
that
can
bind
to
target
with
high
specificity
strong
affinity.
Accordingly,
actively
designing
constructing
this
perspective,
we
provide
a
brief
introduction
technology
its
current
progress,
summarize
application
challenges.
Recent
advances
aptamer-based
reviewed,
together
corresponding
challenges
outlooks.
Nano Letters,
Journal Year:
2024,
Volume and Issue:
24(5), P. 1477 - 1486
Published: Jan. 23, 2024
Lipid
nanoparticle
(LNP)-mediated
nucleic
acid
therapies,
including
mRNA
protein
replacement
and
gene
editing
hold
great
potential
in
treating
neurological
disorders
neurodegeneration,
brain
cancer,
stroke.
However,
delivering
LNPs
across
the
blood-brain
barrier
(BBB)
after
systemic
administration
remains
underexplored.
In
this
work,
we
engineered
a
high-throughput
screening
transwell
platform
for
BBB
(HTS-BBB),
specifically
optimized
LNPs.
Unlike
most
assays,
which
only
assess
transport
an
endothelial
monolayer,
HTS-BBB
simultaneously
measures
LNP
transfection
of
cells
themselves.
We
then
use
to
screen
library
14
made
with
structurally
diverse
ionizable
lipids
demonstrate
it
is
predictive
npj Genomic Medicine,
Journal Year:
2024,
Volume and Issue:
9(1)
Published: Jan. 9, 2024
Abstract
In
the
past
decade,
RNA
therapeutics
have
gone
from
being
a
promising
concept
to
one
of
most
exciting
frontiers
in
healthcare
and
pharmaceuticals.
The
field
is
now
entering
what
many
call
renaissance
or
“RNAissance”
which
fueled
by
advances
genetic
engineering
delivery
systems
take
on
more
ambitious
development
efforts.
However,
this
occurring
at
an
unprecedented
pace,
will
require
different
way
thinking
if
live
up
its
full
potential.
Recognizing
need,
article
provide
forward-looking
perspective
medical
products
potential
long-term
innovations
policy
shifts
enabled
revolutionary
game-changing
technological
platform.
Acta Pharmaceutica Sinica B,
Journal Year:
2024,
Volume and Issue:
14(9), P. 3802 - 3817
Published: May 13, 2024
Small
nucleic
acid
drugs,
composed
of
nucleotides,
represent
a
novel
class
pharmaceuticals
that
differ
significantly
from
conventional
small
molecule
and
antibody-based
therapeutics.
These
agents
function
by
selectively
targeting
specific
genes
or
their
corresponding
messenger
RNAs
(mRNAs),
further
modulating
gene
expression
regulating
translation-related
processes.
Prominent
examples
within
this
category
include
antisense
oligonucleotides
(ASO),
interfering
(siRNAs),
microRNAs
(miRNAs),
aptamers.
The
emergence
drugs
as
focal
point
in
contemporary
biopharmaceutical
research
is
attributed
to
remarkable
specificity,
facile
design,
abbreviated
development
cycles,
expansive
target
spectrum,
prolonged
activity.
Overcoming
challenges
such
poor
stability,
immunogenicity,
permeability
issues
have
been
addressed
through
the
integration
chemical
modifications
drug
delivery
systems.
This
review
provides
an
overview
current
status
prospective
trends
development.
Commencing
with
historical
context,
we
introduce
primary
classifications
mechanisms
drugs.
Subsequently,
delve
into
advantages
U.S.
Food
Drug
Administration
(FDA)
approved
mainly
discuss
encountered
during
Apart
researching
modification
system
efficiently
deliver
enrich
tissues,
promoting
endosomal
escape
critical
scientific
question
important
direction
siRNA
Future
directions
field
will
prioritize
addressing
these
facilitate
clinical
transformation
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.
ACS Nano,
Journal Year:
2023,
Volume and Issue:
17(16), P. 15231 - 15253
Published: Aug. 3, 2023
During
the
COVID-19
pandemic,
mRNA
(mRNA)
vaccines
emerged
as
leading
vaccine
candidates
in
a
record
time.
Nonreplicating
(NRM)
and
self-amplifying
(SAM)
technologies
have
been
developed
into
high-performing
clinically
viable
against
range
of
infectious
agents,
notably
SARS-CoV-2.
demonstrate
efficient
vivo
delivery,
long-lasting
stability,
nonexistent
risk
infection.
The
stability
translational
efficiency
vitro
transcription
(IVT)-mRNA
can
be
further
increased
by
modulating
its
structural
elements.
In
this
review,
we
present
comprehensive
overview
recent
advances,
key
applications,
future
challenges
field
mRNA-based
vaccinology.
Theranostics,
Journal Year:
2023,
Volume and Issue:
14(1), P. 1 - 16
Published: Oct. 30, 2023
Lipid
nanoparticles
(LNPs)
have
emerged
as
a
viable,
clinically-validated
platform
for
the
delivery
of
mRNA
therapeutics.
LNPs
been
utilized
systems
applications
including
vaccines,
gene
therapy,
and
cancer
immunotherapy.
However,
LNPs,
which
are
typically
composed
ionizable
lipids,
cholesterol,
helper
lipid-anchored
polyethylene
glycol,
often
traffic
to
liver
limits
therapeutic
potential
platform.
Several
approaches
proposed
resolve
this
tropism
such
post-synthesis
surface
modification
or
addition
synthetic
cationic
lipids.
BME Frontiers,
Journal Year:
2023,
Volume and Issue:
5
Published: Dec. 18, 2023
Protein
biologics
are
powerful
therapeutic
agents
with
diverse
inhibitory
and
enzymatic
functions.
However,
their
clinical
use
has
been
limited
to
extracellular
applications
due
inability
cross
plasma
membranes.
Overcoming
this
physiological
barrier
would
unlock
the
potential
of
protein
drugs
for
treatment
many
intractable
diseases.
In
review,
we
highlight
progress
made
toward
achieving
cytosolic
delivery
recombinant
proteins.
We
start
by
first
considering
intracellular
as
a
drug
modality
compared
existing
Food
Drug
Administration-approved
modalities.
Then,
summarize
strategies
that
have
reported
achieve
internalization.
These
techniques
can
be
broadly
classified
into
3
categories:
physical
methods,
direct
engineering,
nanocarrier-mediated
delivery.
Finally,
challenges
offer
an
outlook
future
advances.
