Signal Transduction and Targeted Therapy,
Journal Year:
2023,
Volume and Issue:
8(1)
Published: Nov. 24, 2023
Abstract
Mesoporous
silica
nanoparticles
(MSNs)
are
recognized
as
a
prime
example
of
nanotechnology
applied
in
the
biomedical
field,
due
to
their
easily
tunable
structure
and
composition,
diverse
surface
functionalization
properties,
excellent
biocompatibility.
Over
past
two
decades,
researchers
have
developed
wide
variety
MSNs-based
nanoplatforms
through
careful
design
controlled
preparation
techniques,
demonstrating
adaptability
various
application
scenarios.
With
continuous
breakthroughs
MSNs
fields
biosensing,
disease
diagnosis
treatment,
tissue
engineering,
etc.,
gradually
moving
from
basic
research
clinical
trials.
In
this
review,
we
provide
detailed
summary
beginning
with
comprehensive
overview
development
history.
We
then
discuss
types
nanostructured
architectures,
well
classification
nanocomposites
according
elements
existed
inorganic
functional
components.
Subsequently,
summarize
primary
purposes
surface-functionalized
modifications
MSNs.
following,
applications
MSNs,
highlight
targeted
therapeutic
modalities
currently
developed.
Given
importance
translation,
also
progress
Finally,
take
perspective
on
future
direction
remaining
challenges
field.
Molecular Therapy,
Journal Year:
2020,
Volume and Issue:
29(2), P. 464 - 488
Published: Dec. 10, 2020
Hereditary
diseases
are
caused
by
mutations
in
genes,
and
more
than
7,000
rare
affect
over
30
million
Americans.
For
years,
hundreds
of
researchers
have
maintained
that
genetic
modifications
would
provide
effective
treatments
for
many
inherited
human
diseases,
offering
durable
possibly
curative
clinical
benefit
with
a
single
treatment.
This
review
is
limited
to
gene
therapy
using
adeno-associated
virus
(AAV)
because
the
delivered
this
vector
does
not
integrate
into
patient
genome
has
low
immunogenicity.
There
now
five
approved
commercialization
currently
available,
i.e.,
Luxturna,
Zolgensma,
two
chimeric
antigen
receptor
T
cell
(CAR-T)
therapies
(Yescarta
Kymriah),
Strimvelis
(the
gammaretrovirus
adenosine
deaminase-severe
combined
immunodeficiency
[ADA-SCID]
Europe).
Dozens
other
under
trials.
The
article
presents
broad
overview
field
vivo
transfer.
We
neuromuscular
disorders
(spinal
muscular
atrophy
[SMA];
Duchenne
dystrophy
[DMD];
X-linked
myotubular
myopathy
[XLMTM];
central
nervous
system,
including
Alzheimer's
disease,
Parkinson's
Canavan
aromatic
l-amino
acid
decarboxylase
[AADC]
deficiency,
giant
axonal
neuropathy),
ocular
(Leber
congenital
amaurosis,
age-related
macular
degeneration
[AMD],
choroideremia,
achromatopsia,
retinitis
pigmentosa,
retinoschisis),
bleeding
disorder
hemophilia,
lysosomal
storage
disorders.
Cell,
Journal Year:
2021,
Volume and Issue:
184(19), P. 4919 - 4938.e22
Published: Sept. 1, 2021
Replacing
or
editing
disease-causing
mutations
holds
great
promise
for
treating
many
human
diseases.
Yet,
delivering
therapeutic
genetic
modifiers
to
specific
cells
in
vivo
has
been
challenging,
particularly
large,
anatomically
distributed
tissues
such
as
skeletal
muscle.
Here,
we
establish
an
strategy
evolve
and
stringently
select
capsid
variants
of
adeno-associated
viruses
(AAVs)
that
enable
potent
delivery
desired
tissues.
Using
this
method,
identify
a
class
RGD
motif-containing
capsids
transduces
muscle
with
superior
efficiency
selectivity
after
intravenous
injection
mice
non-human
primates.
We
demonstrate
substantially
enhanced
potency
efficacy
these
engineered
vectors
compared
naturally
occurring
AAV
two
mouse
models
disease.
The
top
from
our
selection
approach
show
conserved
across
variety
inbred
strains,
cynomolgus
macaques
primary
myotubes,
transduction
dependent
on
target
cell
expressed
integrin
heterodimers.
International Journal of Molecular Sciences,
Journal Year:
2020,
Volume and Issue:
21(17), P. 6240 - 6240
Published: Aug. 28, 2020
Many
genetic
diseases
and
undesirable
traits
are
due
to
base-pair
alterations
in
genomic
DNA.
Base-editing,
the
newest
evolution
of
clustered
regularly
interspaced
short
palindromic
repeats
(CRISPR)-Cas-based
technologies,
can
directly
install
point-mutations
cellular
DNA
without
inducing
a
double-strand
break
(DSB).
Two
classes
base-editors
have
been
described
thus
far,
cytosine
(CBEs)
adenine
(ABEs).
Recently,
prime-editing
(PE)
has
further
expanded
CRISPR-base-edit
toolkit
all
twelve
possible
transition
transversion
mutations,
as
well
small
insertion
or
deletion
mutations.
Safe
efficient
delivery
editing
systems
target
cells
is
one
most
paramount
challenging
components
for
therapeutic
success
BEs.
Due
its
broad
tropism,
well-studied
serotypes,
reduced
immunogenicity,
adeno-associated
vector
(AAV)
emerged
leading
platform
viral
genome
agents,
including
DNA-base-editors.
In
this
review,
we
describe
development
various
base-editors,
assess
their
technical
advantages
limitations,
discuss
potential
treat
debilitating
human
diseases.
Chemical Reviews,
Journal Year:
2021,
Volume and Issue:
121(18), P. 11527 - 11652
Published: May 3, 2021
The
advent
of
genome
editing
has
transformed
the
therapeutic
landscape
for
several
debilitating
diseases,
and
clinical
outlook
gene
therapeutics
never
been
more
promising.
potential
nucleic
acids
limited
by
a
reliance
on
engineered
viral
vectors
delivery.
Chemically
defined
polymers
can
remediate
technological,
regulatory,
challenges
associated
with
modes
Because
their
scalability,
versatility,
exquisite
tunability,
are
ideal
biomaterial
platforms
delivering
acid
payloads
efficiently
while
minimizing
immune
response
cellular
toxicity.
While
polymeric
delivery
progressed
significantly
in
past
four
decades,
translation
vehicles
faces
formidable
challenges.
aim
our
Account
is
to
illustrate
diverse
concepts
designing
towards
meeting
goals
vivo
ex
therapy.
Here,
we
highlight
classes
employed
summarize
recent
work
understanding
contributions
chemical
architectural
design
parameters.
We
touch
upon
characterization
methods
used
visualize
understand
events
transpiring
at
interfaces
between
polymer,
acids,
physiological
environment.
conclude
that
interdisciplinary
approaches
methodologies
motivated
fundamental
questions
key
high-performing
Signal Transduction and Targeted Therapy,
Journal Year:
2021,
Volume and Issue:
6(1)
Published: June 7, 2021
Abstract
Over
the
past
decades,
great
interest
has
been
given
to
biomimetic
nanoparticles
(BNPs)
since
rise
of
targeted
drug
delivery
systems
and
nanotechnology.
Biological
vectors
including
cell
membranes,
extracellular
vesicles
(EVs),
viruses
are
considered
promising
candidates
for
owing
their
biocompatibility
biodegradability.
BNPs,
integration
biological
functional
agents,
anticipated
load
cargos
or
camouflage
synthetic
achieve
delivery.
Despite
excellent
intrinsic
properties,
natural
deliberately
modified
endow
multiple
functions
such
as
good
permeability,
improved
loading
capability,
high
specificity.
Through
structural
modification
transformation
vectors,
they
pervasively
utilized
more
effective
vehicles
that
can
deliver
contrast
chemotherapy
drugs,
nucleic
acids,
genes
target
sites
refractory
disease
therapy.
This
review
summarizes
recent
advances
in
based
on
EVs,
viruses,
highlighting
potential
applications
BNPs
fields
biomedical
imaging
therapy
industry,
well
discussing
possibility
clinical
translation
exploitation
trend
these
BNPs.
Chemical Society Reviews,
Journal Year:
2021,
Volume and Issue:
50(5), P. 3355 - 3423
Published: Jan. 1, 2021
Nanomaterials
offer
unique
physical,
chemical
and
biological
properties
of
interest
for
medical
imaging
therapy.
Over
the
last
two
decades,
there
has
been
an
increasing
effort
to
translate
nanomaterial-based
medicinal
products
(so-called
nanomedicines)
into
clinical
practice
and,
although
multiple
nanoparticle-based
formulations
are
clinically
available,
is
still
a
disparity
between
number
pre-clinical
those
that
reach
approval.
To
facilitate
efficient
translation
nanomedicinal-drugs,
it
important
study
their
whole-body
biodistribution
pharmacokinetics
from
early
stages
development.
Integrating
this
knowledge
with
therapeutic
profile
and/or
toxicity
should
provide
powerful
combination
efficiently
inform
nanomedicine
trials
allow
selection
most
promising
candidates.
In
context,
radiolabelling
nanomaterials
allows
non-invasive
in
vivo
tracking
by
sensitive
techniques
positron
emission
tomography
(PET),
single
photon
computed
(SPECT).
Furthermore,
certain
radionuclides
specific
nuclear
emissions
can
elicit
effects
themselves,
leading
radionuclide-based
ensure
robust
information
during
development
PET/SPECT
radionuclide
therapy,
appropriate
method
its
limitations
critical.
Different
strategies
available
depending
on
type
material,
final
application.
review
we
describe
different
currently
critical
vision
over
advantages
disadvantages.
The
aim
relevant
up-to-date
field,
support
future
nanomedicinal
