International Journal of Molecular Sciences,
Год журнала:
2020,
Номер
21(18), С. 6582 - 6582
Опубликована: Сен. 9, 2020
Messenger
ribonucleic
acid
(mRNA)-based
drugs,
notably
mRNA
vaccines,
have
been
widely
proven
as
a
promising
treatment
strategy
in
immune
therapeutics.
The
extraordinary
advantages
associated
with
including
their
high
efficacy,
relatively
low
severity
of
side
effects,
and
attainment
costs,
enabled
them
to
become
prevalent
pre-clinical
clinical
trials
against
various
infectious
diseases
cancers.
Recent
technological
advancements
alleviated
some
issues
that
hinder
vaccine
development,
such
efficiency
exist
both
gene
translation
vivo
deliveries.
immunogenicity
can
also
be
greatly
adjusted
result
upgraded
technologies.
In
this
review,
we
summarized
details
regarding
the
optimization
underlying
biological
mechanisms
form
vaccines.
Applications
vaccines
cancers
are
introduced.
It
includes
our
prospections
for
applications
caused
by
bacterial
pathogens,
tuberculosis.
At
same
time,
suggestions
future
development
about
storage
methods,
safety
concerns,
personalized
synthesis
found
context.
Molecular Therapy,
Год журнала:
2019,
Номер
27(4), С. 710 - 728
Опубликована: Фев. 19, 2019
mRNA
has
broad
potential
as
a
therapeutic.
Current
clinical
efforts
are
focused
on
vaccination,
protein
replacement
therapies,
and
treatment
of
genetic
diseases.
The
translation
therapeutics
been
made
possible
through
advances
in
the
design
manufacturing
intracellular
delivery
methods.
However,
application
is
still
limited
by
need
for
improved
systems.
In
this
review,
we
discuss
challenges
mRNA-based
therapeutics,
with
an
emphasis
recent
biomaterials
strategies,
present
overview
applications
therapy,
gene
editing,
vaccination.
Advanced Materials,
Год журнала:
2018,
Номер
30(29)
Опубликована: Май 7, 2018
Abstract
Advances
in
biomaterials
for
drug
delivery
are
enabling
significant
progress
biology
and
medicine.
Multidisciplinary
collaborations
between
physical
scientists,
engineers,
biologists,
clinicians
generate
innovative
strategies
materials
to
treat
a
range
of
diseases.
Specifically,
recent
advances
include
major
breakthroughs
cancer
immunotherapy,
autoimmune
diseases,
genome
editing.
Here,
the
design
implementation
reviewed.
A
brief
history
field
is
first
established,
then
commentary
on
RNA
delivery,
responsive
development,
immunomodulation
provided.
Current
challenges
associated
with
these
areas
as
well
opportunities
address
long‐standing
problems
medicine
discussed
throughout.
Proceedings of the National Academy of Sciences,
Год журнала:
2021,
Номер
118(52)
Опубликована: Дек. 21, 2021
Significance
Liver
accumulation
represents
a
significant
barrier
in
the
development
of
therapeutically
efficacious
nanoparticle
drug
delivery
systems.
Using
series
lipid
nanoparticles
with
distinct
organ-targeting
properties,
we
provide
evidence
for
plausible
mechanism
action
to
non-liver
tissues.
Following
intravenous
injection,
specific
proteins
blood
are
recruited
nanoparticle’s
surface
based
on
its
molecular
composition
and
they
endow
it
unique
biological
identity
that
governs
ultimate
fate
body.
An
innovative
paradigm
emerges
from
this
mechanistic
understanding
delivery—endogenous
targeting—wherein
is
rationally
engineered
interact
overcome
liver
target
organs.
Nature Communications,
Год журнала:
2020,
Номер
11(1)
Опубликована: Июнь 26, 2020
Abstract
CRISPR-Cas9
has
emerged
as
a
powerful
technology
that
relies
on
Cas9/sgRNA
ribonucleoprotein
complexes
(RNPs)
to
target
and
edit
DNA.
However,
many
therapeutic
targets
cannot
currently
be
accessed
due
the
lack
of
carriers
can
deliver
RNPs
systemically.
Here,
we
report
generalizable
methodology
allows
engineering
modified
lipid
nanoparticles
efficiently
into
cells
tissues
including
muscle,
brain,
liver,
lungs.
Intravenous
injection
facilitated
tissue-specific,
multiplexed
editing
six
genes
in
mouse
High
carrier
potency
was
leveraged
create
organ-specific
cancer
models
livers
lungs
mice
though
facile
knockout
multiple
genes.
The
developed
were
also
able
restore
dystrophin
expression
DMD
significantly
decrease
serum
PCSK9
level
C57BL/6
mice.
Application
this
strategy
will
facilitate
broad
nanoparticle
development
for
variety
disease
amenable
protein
delivery
precise
gene
correction
approaches.
