CRISPR-Cas9 Gene Therapy: Non-Viral Delivery and Stimuli-Responsive Nanoformulations
Molecules,
Год журнала:
2025,
Номер
30(3), С. 542 - 542
Опубликована: Янв. 24, 2025
The
CRISPR-Cas9
technology,
one
of
the
groundbreaking
genome
editing
methods
for
addressing
genetic
disorders,
has
emerged
as
a
powerful,
precise,
and
efficient
tool.
However,
its
clinical
translation
remains
hindered
by
challenges
in
delivery
efficiency
targeting
specificity.
This
review
provides
comprehensive
analysis
structural
features,
advantages,
potential
applications
various
non-viral
stimuli-responsive
systems,
examining
recent
progress
to
emphasize
address
these
limitations
advance
therapeutics.
We
describe
how
reports
that
nonviral
vectors,
including
lipid-based
nanoparticles,
extracellular
vesicles,
polymeric
gold
mesoporous
silica
can
offer
diverse
advantages
enhance
stability,
cellular
uptake,
biocompatibility,
based
on
their
structures
physio-chemical
stability.
also
summarize
nanoformulations,
type
vector,
introduce
precision
control
delivery.
Stimuli-responsive
nanoformulations
are
designed
respond
pH,
redox
states,
external
triggers,
facilitate
controlled
targeted
delivery,
minimize
off-target
effects.
insights
our
suggest
future
gene
therapy
technologies
highlight
systems
CRISPR-Cas9’s
efficacy,
positioning
them
pivotal
tools
gene-editing
therapies.
Язык: Английский
Efficient intracellular delivery of CRISPR-Cas9 ribonucleoproteins using dendrimer nanoparticles for robust genomic editing
Nano Today,
Год журнала:
2025,
Номер
61, С. 102654 - 102654
Опубликована: Янв. 31, 2025
Язык: Английский
Dendritic Cell-Based Glioblastoma Vaccines: Advances and Challenges
IntechOpen eBooks,
Год журнала:
2025,
Номер
unknown
Опубликована: Фев. 14, 2025
Glioblastoma
(GBM)
is
the
most
aggressive
malignancy
of
central
nervous
system.
Despite
advances
in
standard
treatments
such
as
surgery,
radiotherapy,
and
chemotherapy,
patients
have
a
very
poor
prognosis.
Tumor
vaccines
based
on
dendritic
cells
(DCs)
provide
promising
new
approach
for
GBM
treatment.
DCs,
effective
antigen-presenting
cells,
initiate
adaptive
immune
responses
by
activating
tumor-specific
T
cells.
However,
immunosuppressive
microenvironment
(characterized
regulatory
myeloid
suppressor
factors)
physical
barrier
blood-brain
(BBB)
greatly
limit
efficacy
DC
vaccines.
This
chapter
explores
biological
basis,
preparation
process,
clinical
progress,
challenges,
future
directions
DC-based
Key
aspects
antigen
selection,
vitro
culture
activation,
loading,
delivery
strategies
are
analyzed
detail.
Early
trials
demonstrated
safety
potential
vaccines,
while
combination
therapies
reprogramming
being
used
to
overcome
existing
obstacles.
precision
personalization
highlight
their
focus
immunotherapy
research.
We
believe
that
with
continuous
advancement
technology
interdisciplinary
collaboration,
can
significantly
improve
survival
rate
quality
life
patients.
Язык: Английский
Engineered Metal–Organic Frameworks for Targeted CRISPR/Cas9 Gene Editing
ACS Pharmacology & Translational Science,
Год журнала:
2025,
Номер
8(4), С. 1028 - 1049
Опубликована: Март 12, 2025
The
development
of
precise
and
efficient
delivery
systems
is
pivotal
for
advancing
CRISPR/Cas9
gene-editing
technologies,
particularly
therapeutic
applications.
Engineered
metal-organic
frameworks
(MOFs)
have
emerged
as
a
promising
class
inorganic
nonviral
vectors,
offering
unique
advantages
such
tunable
porosity,
high
cargo-loading
capacity,
biocompatibility.
This
review
explores
the
design
application
MOF-based
nanoplatforms
tailored
targeted
components,
aiming
to
enhance
precision
efficiency.
By
incorporating
stimuli-responsive
linkers
bioactive
ligands,
these
MOFs
enable
controlled
release
payloads
at
target
site.
Comparative
discussions
demonstrate
superior
performance
over
conventional
in
terms
stability,
transfection
efficiency,
reduced
off-target
effects.
Additionally,
intracellular
trafficking
mechanisms
potential
platforms
preclinical
models
are
discussed.
These
findings
highlight
transformative
overcoming
challenges
associated
with
immunogenicity
cytotoxicity,
paving
way
their
medicine.
provides
blueprint
integration
nanotechnology
genome
editing,
frontier
systems.
Язык: Английский
Evidence-Based Nutraceuticals Derived from Antrodia cinnamomea
Foods,
Год журнала:
2025,
Номер
14(7), С. 1212 - 1212
Опубликована: Март 30, 2025
Antrodia
cinnamomea
(A.
cinnamomea),
a
medicinal
and
edible
mushroom
endemic
to
Taiwan,
has
been
traditionally
valued
as
health
tonic.
Recent
studies
have
highlighted
the
diverse
specialized
metabolites
bioactive
potential
of
this
substance,
primarily
attributed
key
secondary
such
benzenoids,
maleic
succinic
acids,
ubiquinone,
triterpenoids,
primary
metabolite
polysaccharides.
These
compounds
exhibit
broad
spectrum
pharmacological
properties,
including
those
related
antibacterial,
antitumor,
anti-inflammation,
hepatoprotection,
hypoglycaemia,
antioxidant
activities,
immunomodulation
gut
microbiota
regulation.
findings
highlight
therapeutic
A.
its
applications
in
supplements
functional
foods.
This
review
evaluated
recent
advancements
cultivation,
extraction,
characterization
from
cinnamomea,
with
particular
focus
on
submerged
solid-state
fermentation
methods.
We
hope
provide
comprehensive
framework
for
promoting
efficient
scientific
evidence
based
utilization
novel
strategies
health-related
innovations.
Язык: Английский
Efficient DNA- and virus-free engineering of cellular transcriptomic states using dCas9 ribonucleoprotein (dRNP) complexes
Nucleic Acids Research,
Год журнала:
2025,
Номер
53(6)
Опубликована: Март 20, 2025
Abstract
For
genome
editing,
the
use
of
CRISPR
ribonucleoprotein
(RNP)
complexes
is
well
established
and
often
superior
choice
over
plasmid-based
or
viral
strategies.
RNPs
containing
dCas9
fusion
proteins,
which
enable
targeted
manipulation
transcriptomes
epigenomes,
remain
significantly
less
accessible.
Here,
we
describe
production,
delivery,
optimization
second
generation
CRISPRa
(dRNPs).
We
characterize
transcriptional
cellular
consequences
dRNP
treatments
in
a
variety
human
target
cells
show
that
uptake
very
efficient.
The
activation
genes
demonstrates
remarkable
potency,
even
for
are
strongly
silenced,
such
as
developmental
master
transcription
factors.
In
contrast
to
DNA-based
strategies,
gene
immediate
characterized
by
sharp
temporal
precision.
also
dRNPs
allow
high-target
multiplexing,
enabling
undiminished
multiple
simultaneously.
Applying
these
insights,
find
intensive
multiplexing
at
single
promoters
synergistically
elevates
transcription.
Finally,
demonstrate
stem
differentiated
preferable
features
instruct
convert
cell
fates
efficiently
without
need
DNA
delivery
vectors.
Язык: Английский
Interallelic gene conversion of leukemia-associated single nucleotide variants
Gene,
Год журнала:
2025,
Номер
958, С. 149493 - 149493
Опубликована: Апрель 11, 2025
Язык: Английский
Current Non-Viral-Based Strategies to Manufacture CAR-T Cells
International Journal of Molecular Sciences,
Год журнала:
2024,
Номер
25(24), С. 13685 - 13685
Опубликована: Дек. 21, 2024
The
successful
application
of
CAR-T
cells
in
the
treatment
hematologic
malignancies
has
fundamentally
changed
cancer
therapy.
With
increasing
numbers
registered
cell
clinical
trials,
efforts
are
being
made
to
streamline
and
reduce
costs
manufacturing
while
improving
their
safety.
To
date,
all
approved
products
have
relied
on
viral-based
gene
delivery
genomic
integration
methods.
While
viral
vectors
offer
high
transfection
efficiencies,
concerns
regarding
potential
malignant
transformation
coupled
with
costly
time-consuming
vector
constant
drivers
search
for
cheaper,
easier-to-use,
safer,
more
efficient
alternatives.
In
this
review,
we
examine
different
non-viral
transfer
methods
as
alternatives
production,
advantages
disadvantages,
examples
applications.
Transposon-based
lead
stable
but
non-targeted
integration,
easy
handle,
achieve
rates.
Programmable
endonucleases
allow
targeted
reducing
risk
integration-mediated
cells.
Non-integrating
CAR-encoding
avoid
completely
only
transient
CAR
expression.
these
promising
alternative
techniques
transfer,
avenues
open
fully
exploiting
next-generation
therapy
applying
it
a
wide
range
Язык: Английский