Nature Communications,
Год журнала:
2021,
Номер
12(1)
Опубликована: Апрель 9, 2021
Abstract
Prime
editors
(PEs)
mediate
genome
modification
without
utilizing
double-stranded
DNA
breaks
or
exogenous
donor
as
a
template.
PEs
facilitate
nucleotide
substitutions
local
insertions
deletions
within
the
based
on
template
sequence
encoded
prime
editing
guide
RNA
(pegRNA).
However,
efficacy
of
in
adult
mice
has
not
been
established.
Here
we
report
an
NLS-optimized
SpCas9-based
editor
that
improves
efficiency
both
fluorescent
reporter
cells
and
at
endogenous
loci
cultured
cell
lines.
Using
this
system,
could
also
seed
tumor
formation
through
somatic
mouse.
Finally,
successfully
utilize
dual
adeno-associated
virus
(AAVs)
for
delivery
split-intein
demonstrate
system
enables
correction
pathogenic
mutation
mouse
liver.
Our
findings
further
establish
broad
potential
technology
directed
installation
modifications
vivo,
with
important
implications
disease
modeling
correction.
Cell Death and Disease,
Год журнала:
2022,
Номер
13(7)
Опубликована: Июль 23, 2022
Abstract
The
growing
understanding
of
RNA
functions
and
their
crucial
roles
in
diseases
promotes
the
application
various
RNAs
to
selectively
function
on
hitherto
“undruggable”
proteins,
transcripts
genes,
thus
potentially
broadening
therapeutic
targets.
Several
RNA-based
medications
have
been
approved
for
clinical
use,
while
others
are
still
under
investigation
or
preclinical
trials.
Various
techniques
explored
promote
intracellular
trafficking
metabolic
stability,
despite
significant
challenges
developing
therapeutics.
In
this
review,
mechanisms
action,
challenges,
solutions,
therapeutics
comprehensively
summarized.
Genes,
Год журнала:
2020,
Номер
11(8), С. 854 - 854
Опубликована: Июль 26, 2020
Being
the
most
common
musculoskeletal
progressive
condition,
osteoarthritis
is
an
interesting
target
for
research.
It
estimated
that
prevalence
of
knee
(OA)
among
adults
60
years
age
or
older
approximately
10%
in
men
and
13%
women,
making
OA
one
leading
causes
disability
elderly
population.
Today,
we
know
not
a
disease
characterized
by
loss
cartilage
due
to
mechanical
loading
only,
but
condition
affects
all
tissues
joint,
causing
detectable
changes
tissue
architecture,
its
metabolism
function.
All
these
are
mediated
complex
yet
fully
researched
interplay
proinflammatory
anti-inflammatory
cytokines,
chemokines,
growth
factors
adipokines,
which
can
be
measured
serum,
synovium
histological
samples,
potentially
serving
as
biomarkers
stage
progression.
Another
key
aspect
progression
epigenome
regulates
genetic
expression
through
DNA
methylation,
histone
modifications,
mRNA
interference.
A
lot
work
has
been
put
into
developing
non-surgical
treatment
options
slow
down
natural
course
postpone,
maybe
even
replace
extensive
surgeries
such
total
arthroplasty.
At
moment,
biological
treatments
platelet-rich
plasma,
bone
marrow
mesenchymal
stem
cells
autologous
microfragmented
adipose
containing
stromal
vascular
fraction
ordinarily
used.
Furthermore,
latter
two
mentioned
cell-based
seem
only
methods
so
far
increase
quality
patients.
Yet,
future,
gene
therapy
could
become
option
orthopedic
In
following
review,
summarized
latest
important
research
basic
sciences,
pathogenesis,
non-operative
treatment.
Nature Communications,
Год журнала:
2020,
Номер
11(1)
Опубликована: Ноя. 5, 2020
Abstract
Systemic
chemotherapy
remains
the
backbone
of
many
cancer
treatments.
Due
to
its
untargeted
nature
and
severe
side
effects
it
can
cause,
numerous
nanomedicine
approaches
have
been
developed
overcome
these
issues.
However,
targeted
delivery
therapeutics
challenging.
Engineering
microrobots
is
increasingly
receiving
attention
in
this
regard.
Their
functionalities,
particularly
their
motility,
allow
penetrate
tissues
reach
cancers
more
efficiently.
Here,
we
highlight
how
different
microrobots,
ranging
from
tailor-made
motile
bacteria
tiny
bubble-propelled
microengines
hybrid
spermbots,
be
engineered
integrate
sophisticated
features
optimised
for
precision-targeting
a
wide
range
cancers.
Towards
this,
importance
integrating
clinicians,
public
patients
early
on
development
novel
technologies.
Signal Transduction and Targeted Therapy,
Год журнала:
2023,
Номер
8(1)
Опубликована: Янв. 16, 2023
Abstract
Clustered
regularly
interspaced
short
palindromic
repeats
(CRISPR)/CRISPR-associated
protein
9
(Cas9)
gene-editing
technology
is
the
ideal
tool
of
future
for
treating
diseases
by
permanently
correcting
deleterious
base
mutations
or
disrupting
disease-causing
genes
with
great
precision
and
efficiency.
A
variety
efficient
Cas9
variants
derivatives
have
been
developed
to
cope
complex
genomic
changes
that
occur
during
diseases.
However,
strategies
effectively
deliver
CRISPR
system
diseased
cells
in
vivo
are
currently
lacking,
nonviral
vectors
target
recognition
functions
may
be
focus
research.
Pathological
physiological
resulting
from
disease
onset
expected
serve
as
identifying
factors
targeted
delivery
targets
gene
editing.
Diseases
both
varied
complex,
choice
appropriate
methods
different
important.
Meanwhile,
there
still
many
potential
challenges
identified
when
targeting
CRISPR/Cas9
treatment.
This
paper
reviews
current
developments
three
aspects,
namely,
type,
vector,
characteristics.
Additionally,
this
summarizes
successful
examples
clinical
trials
finally
describes
possible
problems
associated
applications.
