International Journal of Molecular Sciences,
Год журнала:
2023,
Номер
24(8), С. 7052 - 7052
Опубликована: Апрель 11, 2023
The
innovative
advances
in
transforming
clustered
regularly
interspaced
short
palindromic
repeats-associated
protein
9
(CRISPR/Cas9)
into
different
variants
have
taken
the
art
of
genome-editing
specificity
to
new
heights.
Allosteric
modulation
Cas9-targeting
by
sgRNA
sequence
alterations
and
protospacer
adjacent
motif
(PAM)
modifications
been
a
good
lesson
learn
about
activity
scores
Cas9
variants.
Some
high-fidelity
ranked
as
Sniper-Cas9,
eSpCas9
(1.1),
SpCas9-HF1,
HypaCas9,
xCas9,
evoCas9.
However,
selection
an
ideal
variant
for
given
target
remains
challenging
task.
A
safe
efficient
delivery
system
CRISPR/Cas9
complex
at
tumor
sites
faces
considerable
challenges,
nanotechnology-based
stimuli-responsive
approaches
significantly
contributed
cancer
management.
Recent
innovations
nanoformulation
design,
such
pH,
glutathione
(GSH),
photo,
thermal,
magnetic
responsive
systems,
modernized
approaches.
These
nanoformulations
possess
enhanced
cellular
internalization,
endosomal
membrane
disruption/bypass,
controlled
release.
In
this
review,
we
aim
elaborate
on
specific
endonuclease
system.
Furthermore,
critical
constraints
clinical
translations
towards
management
prospects
are
described.
Theranostics,
Год журнала:
2020,
Номер
10(10), С. 4374 - 4382
Опубликована: Янв. 1, 2020
CRISPR/Cas
genome
editing
is
a
simple,
cost
effective,
and
highly
specific
technique
for
introducing
genetic
variations.In
mammalian
cells,
can
facilitate
non-homologous
end
joining,
homologydirected
repair,
single-base
exchanges.Cas9/Cas12a
nuclease,
dCas9
transcriptional
regulators,
base
editors,
PRIME
editors
RNA
tools
are
widely
used
in
basic
research.Currently,
variety
of
CRISPR/Cas-based
therapeutics
being
investigated
clinical
trials.Among
many
new
findings
that
have
advanced
the
field,
we
highlight
few
recent
advances
relevant
to
gene
therapies
monogenic
human
diseases.
International Journal of Molecular Sciences,
Год журнала:
2019,
Номер
20(23), С. 6041 - 6041
Опубликована: Ноя. 30, 2019
The
gene
editing
tool
CRISPR-Cas
has
become
the
foundation
for
developing
numerous
molecular
systems
used
in
research
and,
increasingly,
medical
practice.
In
particular,
Cas
proteins
devoid
of
nucleolytic
activity
(dead
proteins;
dCas)
can
be
to
deliver
functional
cargo
programmed
sites
genome.
this
review,
we
describe
current
CRISPR
different
dCas-based
approaches
and
summarize
their
most
significant
applications.
We
conclude
with
comments
on
state-of-art
field
future
directions.
Journal of Experimental & Clinical Cancer Research,
Год журнала:
2020,
Номер
39(1)
Опубликована: Ноя. 4, 2020
Abstract
Long
noncoding
RNAs
(lncRNAs)
are
a
class
of
endogenous,
non-protein
coding
that
highly
linked
to
various
cellular
functions
and
pathological
process.
Emerging
evidence
indicates
lncRNAs
participate
in
crosstalk
between
tumor
stroma,
reprogramming
immune
microenvironment
(TIME).
TIME
possesses
distinct
populations
myeloid
cells
lymphocytes
influence
the
escape
cancer,
response
immunotherapy,
survival
patients.
However,
hitherto,
comprehensive
review
aiming
at
relationship
is
missing.
In
this
review,
we
focus
on
functional
roles
molecular
mechanisms
within
TIME.
Furthermore,
discussed
potential
immunotherapeutic
strategies
based
their
limitations.
npj Precision Oncology,
Год журнала:
2019,
Номер
3(1)
Опубликована: Март 18, 2019
Abstract
The
development
of
genetic
engineering
in
the
1970s
marked
a
new
frontier
genome-editing
technology.
Gene-editing
technologies
have
provided
plethora
benefits
to
life
sciences.
c
lustered
r
egularly
i
nterspaced
s
hort
p
alindromic
epeats/CRISPR
associated
protein
9
(CRISPR/
Cas9)
system
is
versatile
technology
that
provides
ability
add
or
remove
DNA
genome
sequence-specific
manner.
Serious
efforts
are
underway
improve
efficiency
CRISPR/Cas9
targeting
and
thus
reduce
off-target
effects.
Currently,
various
applications
used
cancer
biology
oncology
perform
robust
site-specific
gene
editing,
thereby
becoming
more
useful
for
biological
clinical
applications.
Many
variants
being
rapidly
developed.
Experimental
approaches
based
on
CRISPR
created
very
promising
tool
inexpensive
simple
developing
effective
therapeutics.
This
review
discusses
diverse
CRISPR-based
gene-editing
tools
potential
future
therapies.
Abstract
Intellectual
disability
(ID)
can
be
caused
by
non-genetic
and
genetic
factors,
the
latter
being
responsible
for
more
than
1700
ID-related
disorders.
The
broad
ID
phenotypic
heterogeneity,
as
well
difficulty
in
establishment
of
inheritance
pattern,
often
result
a
delay
diagnosis.
It
has
become
apparent
that
massive
parallel
sequencing
overcome
these
difficulties.
In
this
review
we
address:
(i)
aetiology,
(ii)
clinical/medical
settings
testing,
(iii)
sequencing,
(iv)
variant
filtering
prioritization,
(v)
classification
guidelines
functional
studies,
(vi)
diagnostic
yield.
Furthermore,
need
constant
update
methodologies
tests,
is
essential.
Thus,
international
collaborations,
to
gather
expertise,
data
resources
through
multidisciplinary
contributions,
are
fundamental
keep
track
fast
progress
gene
discovery.
