Chemistry, manufacturing and controls strategies for using novel excipients in lipid nanoparticles
Nature Nanotechnology,
Год журнала:
2025,
Номер
unknown
Опубликована: Янв. 16, 2025
Язык: Английский
In Vivo Endothelial Cell Gene Silencing by siRNA‐LNPs Tuned with Lipoamino Bundle Chemical and Ligand Targeting
Small,
Год журнала:
2024,
Номер
20(42)
Опубликована: Июнь 25, 2024
Although
small-interfering
RNAs
(siRNAs)
are
specific
silencers
for
numerous
disease-related
genes,
their
clinical
applications
still
require
safe
and
effective
means
of
delivery
into
target
cells.
Highly
efficient
lipid
nanoparticles
(LNPs)
developed
siRNA
delivery,
showcasing
the
advantages
novel
pH-responsive
lipoamino
xenopeptide
(XP)
carriers.
These
sequence-defined
XPs
assembled
by
branched
lysine
linkages
between
cationizable
polar
succinoyl
tetraethylene
pentamine
(Stp)
units
apolar
fatty
acids
(LAFs)
at
various
ratios
bundle
or
U-shape
topologies.
Formulation
siRNA-LNPs
using
LAF
Язык: Английский
Lipo-Xenopeptide Polyplexes for CRISPR/Cas9 based Gene editing at ultra-low dose
Janin Germer,
Anna-Lina Lessl,
Jana Pöhmerer
и другие.
Journal of Controlled Release,
Год журнала:
2024,
Номер
370, С. 239 - 255
Опубликована: Апрель 27, 2024
Double
pH-responsive
xenopeptide
carriers
containing
succinoyl
tetraethylene
pentamine
(Stp)
and
lipo
amino
fatty
acids
(LAFs)
were
evaluated
for
CRISPR/Cas9
based
genome
editing.
Different
carrier
topologies,
variation
of
LAF/Stp
ratios
LAF
types
as
Cas9
mRNA/sgRNA
polyplexes
screened
in
three
different
reporter
cell
lines
using
genomic
targets
(Pcsk9,
eGFP,
mdx
exon
23).
One
U-shaped
bundle
(B2)-shaped
lipo-xenopeptides
exhibiting
remarkable
efficiencies
identified.
Genome
editing
potency
top
observed
at
sub-nanomolar
EC
Язык: Английский
Nanoparticle Targeting Strategies for Lipid and Polymer‐Based Gene Delivery to Immune Cells In Vivo
Small Science,
Год журнала:
2024,
Номер
unknown
Опубликована: Июль 30, 2024
Lipid
nanoparticles
and
polymeric
are
promising
biomaterial
platforms
for
robust
intracellular
DNA
mRNA
delivery,
highlighted
by
the
widespread
use
of
nanoparticle‐
(NP)
based
vaccines
to
help
end
COVID‐19
pandemic.
Recent
research
has
sought
adapt
this
nanotechnology
transfect
engineer
immune
cells
in
vivo.
The
system
is
an
especially
appealing
target
due
its
involvement
many
different
diseases,
ex
vivo‐engineered
cell
therapies
like
chimeric
antigen
receptor
(CAR)
T
therapy
have
already
demonstrated
remarkable
clinical
success
certain
blood
cancers.
Although
gene
delivery
can
potentially
address
some
cost
manufacturing
concerns
associated
with
current
autologous
therapies,
transfecting
vivo
challenging.
Not
only
extrahepatic
NP
lymphoid
organs
difficult,
but
particular
resistance
transfection.
Despite
these
challenges,
modular
nature
NPs
allows
researchers
examine
critical
structure–function
relationships
between
a
particle's
properties
ability
specifically
Herein,
several
nanomaterial
components
outlined,
including
targeting
ligands,
nucleic
acid
cargo,
chemical
properties,
physical
route
administration
optimal
Язык: Английский
MicroRNAs in the Mitochondria–Telomere Axis: Novel Insights into Cancer Development and Potential Therapeutic Targets
Genes,
Год журнала:
2025,
Номер
16(3), С. 268 - 268
Опубликована: Фев. 25, 2025
The
mitochondria–telomere
axis
is
recognized
as
an
important
factor
in
the
processes
of
metabolism,
aging
and
oncogenesis.
MicroRNAs
(miRNAs)
play
essential
function
this
complex
interaction,
having
impact
on
aspects
such
cellular
homeostasis,
oxidative
responses
apoptosis.
In
recent
years,
miRNAs
have
been
found
to
be
crucial
for
telomeric
stability,
well
mitochondrial
behavior,
factors
that
influence
cell
proliferation
viability.
Furthermore,
(mitomiRs)
are
associated
with
gene
expression
activity
cGAS/STING
pathway
activity,
linking
DNA
recognition
immune
system
responses.
Hence,
maintain
a
link
biogenesis,
metabolic
changes
cancer
organelles.
This
review
focuses
roles
variety
progression
their
potential
application
biomarkers
or
therapeutic
agents.
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