Frontiers in Genome Editing,
Год журнала:
2024,
Номер
6
Опубликована: Авг. 23, 2024
The
liver
is
an
essential
organ
of
the
body
that
performs
several
vital
functions,
including
metabolism
biomolecules,
foreign
substances,
and
toxins,
production
plasma
proteins,
such
as
coagulation
factors.
There
are
hundreds
genetic
disorders
affecting
functions
and,
for
many
them,
only
curative
option
orthotopic
transplantation,
which
nevertheless
entails
risks
long-term
complications.
Some
peculiar
features
liver,
its
large
blood
flow
supply
tolerogenic
immune
environment,
make
it
attractive
target
in
vivo
gene
therapy
approaches.
In
recent
years,
genome-editing
tools
mainly
based
on
clustered
regularly
interspaced
short
palindromic
repeats
associated
protein
9
(CRISPR-Cas9)
system
have
been
successfully
exploited
context
liver-directed
preclinical
or
clinical
therapeutic
applications.
These
include
knock-out,
knock-in,
activation,
interference,
base
prime
editing
Despite
achievements,
important
challenges
still
need
to
be
addressed
broaden
applications,
optimization
delivery
methods,
improvement
efficiency,
risk
on-target
off-target
unwanted
effects
chromosomal
rearrangements.
this
review,
we
highlight
latest
progress
development
liver-targeted
genome
approaches
treatment
disorders.
We
describe
technological
advancements
currently
under
investigation,
overcome
applicability,
future
perspectives
technology.
MedComm – Biomaterials and Applications,
Год журнала:
2024,
Номер
3(1)
Опубликована: Янв. 14, 2024
Abstract
The
clustered
regularly
interspaced
short
palindromic
repeats
(CRISPR)/CRISPR
associated
protein
9
(CRISPR/Cas9)
systems
initiate
a
revolution
in
genome
editing,
which
have
significant
potential
for
treating
cancer.
A
amount
of
research
has
been
conducted
regarding
genetic
modification
using
CRISPR/Cas9
systems,
and
33
clinical
trials
ex
vivo
or
gene
editing
techniques
carried
out
to
treat
Despite
its
advantages,
the
main
obstacle
convert
technology
into
applications
is
safe
efficient
transport
material
owing
various
extra‐
intracellular
biological
hurdles.
We
outline
characteristics
three
forms
cargos,
plasmids,
mRNA/sgRNA,
ribonucleoprotein
(RNP)
complexes
this
review.
recent
nanotechnology‐based
delivery
these
categories
cancer
are
then
reviewed.
In
end,
we
prerequisites
effective
secure
contexts
discuss
challenges
with
current
nanocarriers.
This
review
offers
thorough
overview
nano‐delivery
system
treatment
cancer,
serving
as
resource
design
building
offering
fresh
perspectives
on
tumors.
Journal of Translational Medicine,
Год журнала:
2024,
Номер
22(1)
Опубликована: Авг. 8, 2024
The
Clustered
Regularly
Interspaced
Short
Palindromic
Repeat
(CRISPR)/Cas9
system,
a
groundbreaking
innovation
in
genetic
engineering,
has
revolutionized
our
approach
to
surmounting
complex
diseases,
culminating
CASGEVY™
approved
for
sickle
cell
anemia.
Derived
from
microbial
immune
defense
mechanism,
CRISPR/Cas9,
characterized
as
precision,
maneuverability
and
universality
gene
editing,
been
harnessed
versatile
tool
precisely
manipulating
DNA
mammals.
In
the
process
of
applying
it
practice,
consecutive
exploitation
novel
orthologs
variants
never
ceases.
It's
conducive
understanding
essentialities
particularly
cancer,
which
is
crucial
diagnosis,
prevention,
treatment.
CRISPR/Cas9
used
not
only
investigate
tumorous
genes
functioning
but
also
model
disparate
cancers,
providing
valuable
insights
into
tumor
biology,
resistance,
evasion.
Upon
cancer
therapy,
instrumental
developing
individual
precise
therapies
that
can
selectively
activate
or
deactivate
within
cells,
aiming
cripple
growth
invasion
sensitize
cells
treatments.
Furthermore,
facilitates
development
innovative
treatments,
enhancing
targeting
efficiency
reprogrammed
exemplified
by
advancements
CAR-T
regimen.
Beyond
potent
screening
susceptible
genes,
offering
possibility
intervening
before
initiative
progresses.
However,
despite
its
vast
potential,
application
research
therapy
accompanied
significant
efficacy,
efficiency,
technical,
safety
considerations.
Escalating
technology
innovations
are
warranted
address
these
issues.
system
revolutionizing
treatment,
opening
up
new
avenues
management
cancers.
integration
this
evolving
clinical
practice
promises
era
precision
oncology,
with
targeted,
personalized,
potentially
curative
patients.
Journal of Biomedical Science,
Год журнала:
2023,
Номер
30(1)
Опубликована: Июль 1, 2023
Abstract
Genome
editing
technologies
hold
great
promise
for
numerous
applications
including
the
understanding
of
cellular
and
disease
mechanisms
development
gene
therapies.
Achieving
high
frequencies
is
critical
to
these
research
areas
achieve
overall
goal
being
able
manipulate
any
target
with
desired
genetic
outcome.
However,
sometimes
suffer
from
low
efficiencies
due
several
challenges.
This
often
case
emerging
technologies,
which
require
assistance
translation
into
broader
applications.
Enrichment
strategies
can
support
this
by
selecting
edited
cells
non-edited
cells.
In
review,
we
elucidate
different
enrichment
strategies,
their
many
in
non-clinical
clinical
settings,
remaining
need
novel
further
improve
genome
therapy
studies.
Tuberculosis
is
an
infectious
disease
primarily
caused
by
the
bacterium
Mycobacterium
tuberculosis
.
This
infects
about
10
million
and
kills
1.6
people
annually.
M.
macrophages
manipulates
its
defense
functions
to
safely
replicate
inside
it.
Sequence
comparison
between
attenuated
bovis
bacille
Calmette‐Guérin
(BCG)
strain
identified
a
region
of
difference
1
(RD1)
which
encodes
highly
antigenic
proteins,
such
as
early
secretory
target‐6
(ESAT‐6)
10‐kDa
culture
filtrate
protein
(CFP‐10),
proteins
that
make
up
ATP‐dependent
apparatus.
Various
studies
suggest
ESAT‐6
counteracts
various
innate
adaptive
immune
host
involved
in
regulating
mycobacterial
virulence.
review
focuses
on
how
responses
are
critical
for
survival
virulence
We
also
discuss
possible
therapeutic
management
targeting
ESAT‐6.
International Journal of Molecular Sciences,
Год журнала:
2023,
Номер
24(24), С. 17320 - 17320
Опубликована: Дек. 10, 2023
Type
1
diabetes
mellitus
(T1D)
is
an
autoimmune
disease
caused
by
the
destruction
of
insulin-producing
β-cells
in
pancreas
cytotoxic
T-cells.
To
date,
there
are
no
drugs
that
can
prevent
development
T1D.
Insulin
replacement
therapy
standard
care
for
patients
with
This
treatment
life-saving,
but
expensive,
lead
to
acute
and
long-term
complications,
results
reduced
overall
life
expectancy.
has
stimulated
research
alternative
treatments
In
this
review,
we
consider
potential
therapies
T1D
using
cellular
regenerative
medicine
approaches
a
focus
on
CRISPR/Cas-engineered
products.
However,
CRISPR/Cas
as
genome
editing
tool
several
drawbacks
should
be
considered
safe
efficient
cell
engineering.
addition,
engineering
themselves
pose
hidden
threat.
The
purpose
review
critically
discuss
novel
strategies
technology.
A
well-designed
approach
β-cell
derivation
CRISPR/Cas-based
technology
will
significantly
reduce
risk
incorrectly
engineered
products
could
behave
"Trojan
horse".
Frontiers in Pharmacology,
Год журнала:
2024,
Номер
15
Опубликована: Март 18, 2024
Macrophages
contribute
to
adipose
tissue
homeostasis;
however,
they
are
also
thought
be
responsible
for
insulin
resistance
in
obesity.
Macrophages,
which
were
oversimplified
past
methodologies,
have
become
rather
difficult
understand
comprehensively
as
recent
developments
research
methodology
revealed
their
diversity.
This
review
highlights
studies
on
macrophages,
identifies
controversial
issues
that
need
resolved
and
proposes
a
scenario
further
development
of
macrophage
biology.
