Highly Branched Poly(β‐amino ester)s for Efficient mRNA Delivery and Nebulization Treatment of Silicosis
Advanced Materials,
Journal Year:
2025,
Volume and Issue:
unknown
Published: April 1, 2025
mRNA
therapeutics
hold
tremendous
promise
for
disease
prevention
and
treatment.
Development
of
high-performance
delivery
systems
with
enhanced
transfection
efficiency
a
safety
profile
will
further
fulfill
their
therapeutic
potential
expedite
translation.
The
synthesis
"four-in-one"
highly
branched
poly(β-amino
ester)s
(O-LhPAEs)
is
reported
by
integrating
the
essential
components
lipid
nanoparticles
(LNPs)
spleen-selective
enrichment
nebulization
treatment
silicosis.
60
O-LhPAEs
distinct
structure
chemical
composition,
including
tertiary/quaternary
amines,
cholesterol
moieties,
zwitterionic
species,
hydrophobic
alkyl
tails,
are
synthesized
using
sequential
Michael
addition,
ring-opening,
nucleophilic
substitution
reactions.
unique
topological
composition
collectively
O-LhPAEs/mRNA
polyplex
serum
resistance,
cellular
uptake,
endosomal
escape.
optimal
O-LhPAE,
20%b-3C-2P12,
exhibits
up
to
93.1%
across
11
different
cell
types,
epithelial
cells,
fibroblasts,
cancer
stem
neurological
astrocytes.
Biodistribution
study
reveals
that
20%b-3C-2P12/mRNA
polyplexes
mainly
enriched
in
spleen
following
systemic
administration.
Through
nebulization,
20%b-3C-2P12
mediated
high
Tbx2
expression
lungs
silicosis
mice,
effectively
restoring
lung
functions.
This
not
only
establishes
strategy
development
LNP-like
but
also
provides
promising
candidates
safe,
efficient,
Language: Английский
DNA polymer conjugates: Revolutionizing neurological disorder treatment through targeted drug delivery
Next Materials,
Journal Year:
2024,
Volume and Issue:
7, P. 100336 - 100336
Published: Aug. 3, 2024
Language: Английский
Synthetic-polymer-assisted antisense oligonucleotide delivery: targeted approaches for precision disease treatment
Anabel Álvarez,
No information about this author
Deborah Maguire,
No information about this author
Ruairí P. Brannigan
No information about this author
et al.
Beilstein Journal of Nanotechnology,
Journal Year:
2025,
Volume and Issue:
16, P. 435 - 463
Published: March 27, 2025
This
review
explores
the
recent
advancements
in
polymer-assisted
delivery
systems
for
antisense
oligonucleotides
(ASOs)
and
their
potential
precision
disease
treatment.
Synthetic
polymers
have
shown
significant
promise
enhancing
delivery,
stability,
therapeutic
efficacy
of
ASOs
by
addressing
key
challenges
such
as
cellular
uptake,
endosomal
escape,
reducing
cytotoxicity.
The
highlights
studies
from
past
decade
demonstrating
how
these
improve
gene
silencing
efficiencies,
particularly
cancer
neurodegenerative
models.
Despite
progress
achieved,
barriers
immunogenicity,
limitations,
scalability
still
need
to
be
overcome
broader
clinical
application.
Emerging
strategies,
including
stimuli-responsive
advanced
nanoparticle
systems,
offer
solutions
challenges.
underscores
transformative
polymer-enhanced
ASO
personalised
medicine,
emphasising
importance
continued
innovation
optimise
ASO-based
therapeutics
more
precise
effective
treatments.
Language: Английский
Nanotheranostics Revolutionizing Gene Therapy: Emerging Applications in Gene Delivery Enhancement
Journal of Nanotheranostics,
Journal Year:
2025,
Volume and Issue:
6(2), P. 10 - 10
Published: April 9, 2025
Nanotheranostics—where
nanoscale
materials
serve
both
diagnostic
and
therapeutic
functions—are
rapidly
transforming
gene
therapy
by
tackling
critical
delivery
challenges.
This
review
explores
the
design
engineering
of
various
nanoparticle
systems
(lipid-based,
polymeric,
inorganic,
hybrid)
to
enhance
stability,
targeting,
endosomal
escape
genetic
payloads.
We
discuss
how
real-time
imaging
capabilities
integrated
into
these
platforms
enable
precise
localization
controlled
release
genes,
improving
treatment
efficacy
while
reducing
off-target
effects.
Key
strategies
overcome
barriers
(such
as
proton
sponge
effect
photothermal
disruption)
achieve
nuclear
are
highlighted,
along
with
recent
advances
in
stimuli-responsive
that
facilitate
spatiotemporal
control
expression.
Clinical
trials
preclinical
studies
demonstrate
expanding
role
nanotheranostics
managing
cancer,
inherited
disorders,
cardiovascular
neurological
diseases.
further
address
regulatory
manufacturing
hurdles
must
be
for
widespread
clinical
adoption
nanoparticle-based
therapies.
By
synthesizing
progress
ongoing
challenges,
this
underscores
transformative
potential
effective,
targeted,
image-guided
delivery.
Language: Английский
The promising potential of gene therapy for diabetes mellitus
Nature Reviews Endocrinology,
Journal Year:
2024,
Volume and Issue:
unknown
Published: Aug. 29, 2024
Language: Английский
The Future of Nanomaterials Tackling the Challenge of Delivering Nucleic Acids to the Retina
Advanced Functional Materials,
Journal Year:
2024,
Volume and Issue:
unknown
Published: Dec. 4, 2024
Abstract
Ocular
gene
therapy
targets
eye
diseases
at
the
genetic
level.
Systemic
transport
of
nucleic
acids
to
ocular
tissues
poses
a
significant
challenge,
as
effectiveness
crossing
blood‐retina
barrier
limits
acid
penetration.
Therefore,
local
administration,
such
topical,
periocular,
or
intraocular,
can
improve
outcome
in
vivo
by
bypassing
first‐pass
effect
and
minimizing
systemic
toxic
effects.
The
is
an
immune‐privileged
organ
with
limited
immune
response,
making
it
ideal
candidate
for
therapy.
offers
promising
solution
treatment
wide
range
retinal
including
age‐related
macular
degeneration,
diabetic
retinopathy,
retinitis
pigmentosa,
Leber
congenital
amaurosis.
Gene
enables
replacement
mutated
genes
essential
visual
function,
delivery
expressing
neurotrophic
factors
anti‐apoptosis
anti‐angiogenic
proteins
neovascularization.
This
perspective
discusses
potential
nanoparticles
retina,
explores
challenges,
evaluates
different
methods,
non‐viral
agents
liposomes
polymers.
These
nonviral
present
advantages
over
traditional
viral
vectors,
showing
promise
overcoming
limitations
offering
viable
option
Language: Английский
Nano Delivery System for Atherosclerosis
Rong Zhang,
No information about this author
Xuan He,
No information about this author
T. Y. Fan
No information about this author
et al.
Journal of Functional Biomaterials,
Journal Year:
2024,
Volume and Issue:
16(1), P. 2 - 2
Published: Dec. 24, 2024
Atherosclerosis,
a
pathological
process
propelled
by
inflammatory
mediators
and
lipids,
is
principal
contributor
to
cardiovascular
disease
incidents.
Currently,
drug
therapy,
the
primary
therapeutic
strategy
for
atherosclerosis,
faces
challenges
such
as
poor
stability
significant
side
effects.
The
advent
of
nanomaterials
has
garnered
considerable
attention
from
scientific
researchers.
Nanoparticles,
liposomes
polymeric
nanoparticles,
have
been
developed
delivery
in
atherosclerosis
treatment.
This
review
will
focus
on
how
nanoparticles
effectively
improve
safety
efficacy,
well
continuous
development
optimization
same
material
further
explore
current
future
opportunities
this
field.
Language: Английский
Functionalized polyesters based on valerolactones and [12]aneN3 as effective non-viral gene vectors in HepG2 cells
Xi Zhang,
No information about this author
De‐Zhong Xu,
No information about this author
Zi-Han Zhang
No information about this author
et al.
Polymer Chemistry,
Journal Year:
2024,
Volume and Issue:
15(34), P. 3480 - 3491
Published: Jan. 1, 2024
Block
polyesters
with
BODIPY
and
[12]aneN
3
moieties
showed
efficient
cell-specific
nucleic
acid
expression
anti-tumor
gene
therapy
in
vitro
.
Language: Английский
Furan-rich, biobased transfection agents as potential oligomeric candidates for intracellular plasmid DNA delivery
RSC Advances,
Journal Year:
2024,
Volume and Issue:
14(44), P. 32637 - 32647
Published: Jan. 1, 2024
Derived
from
furan
diacrylate,
the
first
examples
of
biobased
gene
transfection
agents.
Language: Английский