International Journal of Molecular Sciences,
Journal Year:
2023,
Volume and Issue:
24(8), P. 7052 - 7052
Published: April 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.
Science,
Journal Year:
2018,
Volume and Issue:
361(6405), P. 866 - 869
Published: Aug. 30, 2018
The
diversity,
modularity,
and
efficacy
of
CRISPR-Cas
systems
are
driving
a
biotechnological
revolution.
RNA-guided
Cas
enzymes
have
been
adopted
as
tools
to
manipulate
the
genomes
cultured
cells,
animals,
plants,
accelerating
pace
fundamental
research
enabling
clinical
agricultural
breakthroughs.
We
describe
basic
mechanisms
that
set
toolkit
apart
from
other
programmable
gene-editing
technologies,
highlighting
diverse
naturally
evolved
now
functionalized
biotechnologies.
discuss
rapidly
evolving
landscape
applications,
gene
editing
transcriptional
regulation,
imaging,
diagnostics.
Continuing
functional
dissection
an
expanding
applications
position
at
cutting
edge
nucleic
acid
manipulation
is
rewriting
biology.
Science,
Journal Year:
2023,
Volume and Issue:
379(6629)
Published: Jan. 19, 2023
The
advent
of
clustered
regularly
interspaced
short
palindromic
repeat
(CRISPR)
genome
editing,
coupled
with
advances
in
computing
and
imaging
capabilities,
has
initiated
a
new
era
which
genetic
diseases
individual
disease
susceptibilities
are
both
predictable
actionable.
Likewise,
genes
responsible
for
plant
traits
can
be
identified
altered
quickly,
transforming
the
pace
agricultural
research
breeding.
In
this
Review,
we
discuss
current
state
CRISPR-mediated
manipulation
human
cells,
animals,
plants
along
relevant
successes
challenges
present
roadmap
future
technology.
Neuron,
Journal Year:
2019,
Volume and Issue:
104(2), P. 239 - 255.e12
Published: Aug. 15, 2019
Highlights•A
CRISPR
interference
platform
for
genetic
screens
in
human
iPSC-derived
neurons•Survival
uncover
genes
essential
neurons,
but
not
iPSCs
or
cancer
cells•Single-cell
RNA-seq
reveal
distinct
neuronal
roles
ubiquitous
genes•Arrayed
high-content
controlling
morphologySummaryCRISPR/Cas9-based
functional
genomics
have
transformed
our
ability
to
elucidate
mammalian
cell
biology.
However,
most
previous
CRISPR-based
were
conducted
lines
rather
than
healthy,
differentiated
cells.
Here,
we
describe
a
(CRISPRi)-based
neurons
derived
from
induced
pluripotent
stem
cells
(iPSCs).
We
demonstrate
robust
and
durable
knockdown
of
endogenous
such
present
results
three
complementary
screens.
First,
survival-based
screen
revealed
neuron-specific
that
improved
survival
upon
knockdown.
Second,
with
single-cell
transcriptomic
readout
uncovered
several
examples
whose
had
strikingly
cell-type-specific
consequences.
Third,
longitudinal
imaging
detected
consequences
gene
on
morphology.
Our
highlight
the
power
unbiased
types
provide
systematic
interrogation
normal
disease
states
neurons.Video
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video
(mp4,
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abstract
Seminars in Cancer Biology,
Journal Year:
2018,
Volume and Issue:
55, P. 106 - 119
Published: April 16, 2018
CRISPR/Cas9
has
become
a
powerful
method
for
making
changes
to
the
genome
of
many
organisms.
First
discovered
in
bacteria
as
part
an
adaptive
immune
system,
and
modified
versions
have
found
widespread
use
engineer
genomes
activate
or
repress
expression
genes.
As
such,
promises
accelerate
cancer
research
by
providing
efficient
technology
dissect
mechanisms
tumorigenesis,
identify
targets
drug
development,
possibly
arm
cells
cell-based
therapies.
Here,
we
review
current
applications
therapy.
We
describe
novel
Cas9
variants
how
they
are
used
functional
genomics
discover
cancer-specific
vulnerabilities.
Furthermore,
highlight
impact
generating
organoid
mouse
models
cancer.
Finally,
provide
overview
first
clinical
trials
that
apply
therapeutic
approach
against
Frontiers in Medicine,
Journal Year:
2021,
Volume and Issue:
8
Published: March 3, 2021
Cancer
is
one
of
the
most
leading
causes
mortalities
worldwide.
It
caused
by
accumulation
genetic
and
epigenetic
alterations
in
2
types
genes:
tumor
suppressor
genes
(TSGs)
proto-oncogenes.
In
recent
years,
development
clustered
regularly
interspaced
short
palindromic
repeats
(CRISPR)
technology
has
revolutionized
genome
engineering
for
different
cancer
research
ranging
from
fundamental
science
to
translational
medicine
precise
treatment.
The
CRISPR/CRISPR
associated
proteins
(CRISPR/Cas)
are
prokaryote-derived
editing
systems
that
have
enabled
researchers
detect,
image,
manipulate
annotate
specific
DNA
RNA
sequences
various
living
cells.
CRISPR/Cas
significant
contributions
discovery
proto-oncogenes
TSGs,
cell
epigenome
normalization,
targeted
delivery,
identification
drug
resistance
mechanisms,
high-throughput
screening,
models
establishment,
immunotherapy
gene
therapy
clinics.
Robust
technical
improvements
shown
a
considerable
degree
efficacy,
specificity,
flexibility
target
locus
desired
applications.
Recent
developments
CRISPRs
offers
hope
medical
cure
against
other
deadly
diseases.
Despite
this
field,
several
challenges
need
be
addressed,
such
as
off-target
activity,
insufficient
indel
or
low
homology-directed
repair
(HDR)
efficiency,
vivo
delivery
Cas
system
components,
immune
responses.
This
study
aims
overview
technological
advancements,
preclinical
perspectives
on
clinical
applications
CRISPR
along
with
their
advantages
limitations.
Moreover,
potential
research,
genetic,
treatments
discussed.
