Cells,
Год журнала:
2024,
Номер
13(23), С. 1979 - 1979
Опубликована: Ноя. 29, 2024
Recently
approved
adeno-associated
viral
(AAV)
vectors
for
liver
monogenic
diseases
haemophilia
A
and
B
are
exemplifying
the
success
of
liver-directed
gene
therapy.
In
parallel,
additional
therapy
strategies
rapidly
emerging
to
overcome
some
inherent
AAV
limitations,
such
as
non-persistence
episomal
transgene
in
growing
immune
response.
Viral
integrating
vivo
lentiviral
non-viral
lipid
nanoparticles
encapsulating
mRNA
(LNP-mRNA)
being
developed,
currently
at
preclinical
clinical
stages,
respectively.
Macrophages
first
effector
cells
innate
response
triggered
by
vectors.
Macrophage
uptake
activation
following
administration
LNP
have
been
reported.
this
study,
we
assessed
biodistribution
AAV,
lentiviral,
LNP-mRNA
depletion
tissue
macrophages
clodronate
pre-treatment
neonatal
juvenile
mice.
Both
adult
clodronate-treated
mice
showed
a
significant
increase
lentiviral-transduced
hepatocytes.
contrast,
did
not
modify
hepatocyte
transduction
mediated
hepatotropic
AAV8
but
reduced
transfection
animals.
These
results
highlight
importance
age-specific
responses
will
translational
applications
programs.
Progress in Retinal and Eye Research,
Год журнала:
2025,
Номер
unknown, С. 101354 - 101354
Опубликована: Март 1, 2025
Retinal
gene
therapy
using
adeno-associated
viral
(AAV)
vectors
has
been
a
groundbreaking
step-change
in
the
treatment
of
inherited
retinal
diseases
(IRDs)
and
could
also
be
used
to
treat
more
common
such
as
age-related
macular
degeneration
diabetic
retinopathy.
The
delivery
expression
therapeutic
transgenes
eye
is
limited
by
innate
adaptive
immune
responses
against
components
vector
product,
which
termed
therapy-associated
uveitis
(GTAU).
This
clinically
important
intraocular
inflammation
lead
irreversible
loss
cells,
deterioration
visual
function
reduced
durability
effect
associated
with
costly
one-off
treatment.
For
achieve
an
improved
efficacy
safety
profile
for
treating
additional
IRDs
diseases,
risk
GTAU
must
minimised.
We
have
collated
insights
from
pre-clinical
research,
clinical
trials,
real-world
implementation
AAV-mediated
help
understand
factors
GTAU.
draw
attention
emerging
framework,
includes
patient
demographics,
construct,
dose,
route
administration,
choice
immunosuppression
regime.
Importantly,
we
consider
efforts
date
potential
future
strategies
mitigate
adverse
response
across
each
these
domains.
advocate
targeted
immunomodulatory
approaches
prevention
based
on
better
understanding
underlying
response.
Pharmaceutics,
Год журнала:
2024,
Номер
16(11), С. 1360 - 1360
Опубликована: Окт. 24, 2024
Over
80%
of
the
world's
deadliest
pandemics
are
caused
by
viral
infections,
and
vaccination
remains
most
effective
way
to
prevent
these
infections
from
spreading.
Since
discovery
first
vaccine
over
two
centuries
ago,
several
design
technologies
have
been
developed.
Next-generation
vaccines,
based
on
mRNA
vector
technologies,
recently
emerged
as
alternatives
traditional
vaccines.
Adenoviral
vector-based
vaccines
against
coronavirus
disease
2019
demonstrated
a
more
sustained
antibody
response
compared
However,
this
has
not
without
complications,
with
few
cases
severe
adverse
events
identified
in
vaccinated
individuals,
underlying
mechanism
is
subject
intense
investigation.
Adeno-associated
vectors
induce
weaker
cellular
immune
adenoviral
vectors,
it
mainly
for
reason
that
there
diminished
interest
exploring
them
platform
until
recently.
This
review
will
discuss
recent
developments
potential
adeno-associated
anti-viral
Viruses,
Год журнала:
2024,
Номер
16(10), С. 1507 - 1507
Опубликована: Сен. 24, 2024
While
adeno-associated
viral
(AAV)
vectors
are
successfully
used
in
a
variety
of
vivo
gene
therapy
applications,
they
continue
to
be
hampered
by
the
immune
system.
Here,
we
sought
identify
innate
and
cytokine
signaling
pathways
that
promote
CD8
Pharmaceuticals,
Год журнала:
2024,
Номер
17(9), С. 1213 - 1213
Опубликована: Сен. 14, 2024
Recombinant
AAV
(rAAV)
vectors
are
increasingly
favored
for
gene
therapy
due
to
their
useful
features
of
vectorology,
such
as
transfection
dividing
and
nondividing
cells,
the
presence
tissue-specific
serotypes,
biosafety
considerations.
This
study
investigates
impact
commonly
used
therapeutic
drugs—acetaminophen,
budesonide,
simvastatin—on
rAAV
transduction
efficiency
in
HEK-293
cells.
Cells
were
transduced
with
an
mosaic
vector
under
control
a
cytomegalovirus
(CMV)
promoter
encoding
green
fluorescent
protein
(GFP).
Transduction
was
assessed
by
qPCR
microscopy.
Analysis
functional
interactions
between
genes
potentially
involved
drug-exposed
cells
also
performed.
showed
clear
effect
drugs
on
transmission.
Notably,
acetaminophen
enhanced
9-fold,
while
budesonide
simvastatin
2-fold
3-fold
increases,
respectively.
The
analysis
illustrates
possible
involvement
related
cell
membranes
potentiation
induced
investigation.
Attention
should
be
paid
S100A8,
which
is
common
drug-modified
showing
anti-inflammatory
effects
(budesonide
simvastatin),
demonstrating
interaction
receptor
(HGFR).
underscores
significance
assessing
pharmacokinetics/pharmacodynamics
(PKs/PDs)
drug–gene
optimizing
protocols.
