Landscape of small nucleic acid therapeutics: moving from the bench to the clinic as next-generation medicines
Signal Transduction and Targeted Therapy,
Journal Year:
2025,
Volume and Issue:
10(1)
Published: March 10, 2025
Abstract
The
ability
of
small
nucleic
acids
to
modulate
gene
expression
via
a
range
processes
has
been
widely
explored.
Compared
with
conventional
treatments,
acid
therapeutics
have
the
potential
achieve
long-lasting
or
even
curative
effects
editing.
As
result
recent
technological
advances,
efficient
delivery
for
therapeutic
and
biomedical
applications
achieved,
accelerating
their
clinical
translation.
Here,
we
review
increasing
number
classes
most
common
chemical
modifications
platforms.
We
also
discuss
key
advances
in
design,
development
application
each
platform.
Furthermore,
this
presents
comprehensive
profiles
currently
approved
drugs,
including
11
antisense
oligonucleotides
(ASOs),
2
aptamers
6
siRNA
summarizing
modifications,
disease-specific
mechanisms
action
strategies.
Other
candidates
whose
trial
status
recorded
updated
are
discussed.
consider
strategic
issues
such
as
important
safety
considerations,
novel
vectors
hurdles
translating
academic
breakthroughs
clinic.
Small
produced
favorable
results
trials
address
previously
“undruggable”
targets,
suggesting
that
they
could
be
useful
guiding
additional
candidates.
Language: Английский
Metal-phenolic networks specifically eliminate hypoxic tumors by instigating oxidative and proteotoxic stresses
Jia Liu,
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Zuoyu Chen,
No information about this author
Lixue Deng
No information about this author
et al.
Bioactive Materials,
Journal Year:
2025,
Volume and Issue:
47, P. 361 - 377
Published: Feb. 12, 2025
Language: Английский
In situ carrier-free nanovaccines reversing the immunosuppressive microenvironment for boosting tumor immunotherapy
Yi‐Lei Zhao,
No information about this author
Guoxin Zhu,
No information about this author
Xuechun Wang
No information about this author
et al.
Chinese Chemical Letters,
Journal Year:
2025,
Volume and Issue:
unknown, P. 111031 - 111031
Published: March 1, 2025
Language: Английский
A carrier-free DNA nanoplatform for efficient three-in-one tumor therapy in vivo
Hong Wang,
No information about this author
Xuehe Lu,
No information about this author
Jing Fan
No information about this author
et al.
Nano Today,
Journal Year:
2025,
Volume and Issue:
62, P. 102734 - 102734
Published: March 27, 2025
Language: Английский
tert-Butoxycarbonyl-Modification Driven Disturbance of Molecular Ordering Enables High-Efficiency Dual Drugs Co-Assembly for Synergistic Tumor Inhibition
Xuequan Zhang,
No information about this author
Xiaoxian Zhang,
No information about this author
Jiahui Zhu
No information about this author
et al.
ACS Nano,
Journal Year:
2025,
Volume and Issue:
unknown
Published: April 1, 2025
The
development
of
carrier-free
drug
delivery
systems
(CDDS)
for
tailored
combinations
posed
a
significant
challenge,
particularly
in
achieving
efficient
co-assembly
while
maintaining
therapeutic
efficacy.
Herein,
we
proposed
strategy
based
on
molecular
engineering.
Paclitaxel
(PTX)
and
7-ethyl-10-hydroxycamptothecin
(SN38)
were
chemically
modified
with
tert-butoxycarbonyl
(BOC)
groups.
successful
incorporation
the
BOC
groups
was
confirmed
by
proton
nuclear
magnetic
resonance
mass
spectrometry
analyses.
Further
characterization
using
polarized
light
microscopy
X-ray
diffraction
revealed
that
this
modification
significantly
reduced
crystallinity
both
drugs,
simultaneously
disrupting
their
original
ordered
stacking
structure.
Molecular
dynamics
simulations
indicated
increased
spacing,
density,
expanded
volume,
resulting
looser
packing
arrangement.
This
structural
alteration
enabled
molecules
to
efficiently
coassemble
α-tocopherol
succinate
(α-TOS)
into
spherical
nanoparticles
at
nearly
predefined
ratio.
exhibited
high
loading
capacity
52.66%
remained
stable
4
°C
over
50
days.
Notably,
these
displayed
controllable
release
characteristics
pH
5.0.
Both
vitro
vivo
studies
demonstrated
BOC-modified
drugs
retained
bioactivity.
When
co-assembled
α-TOS,
synergistic
antitumor
effect
suppressed
tumor
metastasis
through
downregulation
matrix
metalloproteinase-9
(MMP-9)
expression.
study
provided
solid
theoretical
foundation
innovative
approach
CDDS,
utilizing
molecular-scale
regulation
co-assembly.
Language: Английский
Tactics to Improve the Photodynamic Therapeutic Efficacy Based on Nanomaterials
Yinghao Cao,
No information about this author
Xiaoping Qian,
No information about this author
Mengqi Shi
No information about this author
et al.
Chinese Journal of Chemistry,
Journal Year:
2025,
Volume and Issue:
unknown
Published: April 19, 2025
Comprehensive
Summary
The
development
of
photodynamic
therapy
(PDT),
from
its
initial
discovery
effects
to
current
use
in
various
medical
conditions,
is
a
testament
therapeutic
potential.
Recent
breakthroughs
nanotechnology
have
significantly
enhanced
the
effectiveness
PDT.
Typical
nanomaterials
(NMs),
including
metal‐organic
frameworks
(MOFs),
covalent
organic
(COFs),
and
nanozymes
been
introduced
enhance
efficacy
because
they
can
delivery
PSs,
effectively
overcome
insufficient
targeting
specificity,
limited
tissue
penetration
depth,
hypoxic
microenvironments,
thereby
amplifying
efficacy.
However,
clinical
application
these
NMs
PDT
faces
several
challenges,
concerns
regarding
biocompatibility,
long‐term
biosafety,
economic
feasibility.
To
further
advance
PDT,
researchers
should
focus
on
designing
improve
outcomes,
exploring
combination
therapies
with
conducting
translational
trials
validate
safety
novel
approaches.
This
review
summarizes
recent
progress
based
NMs,
especially
MOFs,
COFs
their
disease
treatment.
We
aim
provide
guidance
for
future
research
practice
advancing
NMs‐enhanced
paving
way
more
effective
strategies.
Key
Scientists
Language: Английский
Nanometer preparation of natural bioactive compounds for treatment of rheumatoid arthritis
Junping Zhu,
No information about this author
Xiang Qin,
No information about this author
Qi Li
No information about this author
et al.
Journal of Pharmaceutical Analysis,
Journal Year:
2025,
Volume and Issue:
unknown, P. 101341 - 101341
Published: May 1, 2025
Language: Английский
Multifunctional nanomaterials composed entirely of active pharmaceutical ingredients for synergistically enhanced antitumor and antibacterial effects
Qi An,
No information about this author
Dongmei Wang,
No information about this author
Liang Huang
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et al.
Frontiers in Pharmacology,
Journal Year:
2024,
Volume and Issue:
15
Published: Oct. 21, 2024
Introduction
Multifunctional
nanomaterials
are
emerging
as
promising
tools
for
treating
both
cancer
and
bacterial
infections.
However,
integrating
dual
therapeutic
capabilities
into
a
single
system
remains
challenging.
This
study
presents
multifunctional
nanoparticles
(ECI-NPs)
based
on
Epigallocatechin
gallate
(EGCG)
oligomers,
Curcumin
(CUR),
Indocyanine
Green
(ICG)
combined
treatment.
Methods
ECI-NPs
were
synthesized
via
oxidative
coupling
of
EGCG,
CUR,
ICG.
The
characterized
stability,
size,
drug
loading,
release
profiles.
Cellular
uptake,
phototoxicity
in
melanoma
cells,
antibacterial
activity
against
Escherichia
coli
Staphylococcus
aureus
also
evaluated.
Results
demonstrated
optimal
high
controlled
release.
studies
showed
increased
uptake
greater
cells
compared
to
free
drugs.
exhibited
enhanced
anticancer
effects
strong
activity,
outperforming
the
individual
components.
Discussion
polyphenol-based
offer
synergistic
effects,
overcoming
limitations
drugs
terms
solubility
efficacy.
dual-function
platform
shows
potential
broader
biomedical
applications,
addressing
challenges
Further
research
will
focus
vivo
clinical
translation.
Language: Английский
ROS Regulation in CNS Disorder Therapy: Unveiling the Dual Roles of Nanomedicine
Zhengyang Quan,
No information about this author
Sa Wang,
No information about this author
Huanhuan Xie
No information about this author
et al.
Small,
Journal Year:
2024,
Volume and Issue:
unknown
Published: Dec. 15, 2024
Abstract
The
treatment
of
brain
diseases
has
always
been
the
focus
attention.
Due
to
presence
blood‐brain
barrier
(BBB),
most
small
molecule
drugs
are
difficult
reach
brain,
leading
undesirable
therapeutic
outcomes.
Recently,
nanomedicines
that
can
cross
BBB
and
precisely
target
lesion
sites
have
emerged
as
thrilling
tools
enhance
early
diagnosis
treat
various
intractable
disorders.
Extensive
research
shown
reactive
oxygen
species
(ROS)
play
a
crucial
role
in
occurrence
progression
diseases,
including
tumors
neurodegenerative
(NDDs)
such
Alzheimer's
disease,
Parkinson's
stroke,
or
traumatic
injury,
making
ROS
potential
target.
In
this
review,
on
structure
function
well
mechanisms
first
elaborated
through
which
nanomedicine
traverses
it.
Then,
recent
studies
production
summarized
photodynamic
therapy
(PDT),
chemodynamic
(CDT),
sonodynamic
(SDT)
for
treating
tumors,
depletion
NDDs.
This
provides
valuable
guidance
future
design
ROS‐targeted
disease
treatment.
ongoing
challenges
perspectives
developing
nanomedicine‐based
management
also
discussed
outlined.
Language: Английский