The CRISPR Journal,
Journal Year:
2022,
Volume and Issue:
5(1), P. 109 - 122
Published: Feb. 1, 2022
Alzheimer's
disease
(AD)
is
the
result
of
abnormal
processing
amyloid
precursor
protein
(APP)
by
β-secretase
and
γ-secretase,
which
leads
to
formation
toxic
β-amyloid
peptides.
The
peptides
induce
neuron
death,
memory
problems,
AD
development.
Several
APP
mutations
increase
risk
developing
early-onset
AD.
However,
A673T
mutation
identified
in
Icelandic
population
prevents
development
reducing
cleavage
β-secretase.
In
this
study,
we
inserted
human
cells
using
CRISPR
prime
editing
(PE)
technique.
Repeated
PE
treatments
resulted
insertion
up
49.2%
genes
when
a
second
nick
was
induced
other
DNA
strand.
When
protospacer
adjacent
motif
used
for
also
mutated,
68.9%
contained
protective
mutation.
promising
approach
introduce
precisely
without
mutating
nearby
nucleotides.
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.
Biologics,
Journal Year:
2021,
Volume and Issue:
Volume 15, P. 353 - 361
Published: Aug. 1, 2021
Abstract:
Clustered
regularly
interspaced
short
palindromic
repeat
(CRISPR)
and
their
associated
protein
(Cas-9)
is
the
most
effective,
efficient,
accurate
method
of
genome
editing
tool
in
all
living
cells
utilized
many
applied
disciplines.
Guide
RNA
(gRNA)
CRISPR-associated
proteins
are
two
essential
components
CRISPR/Cas-9
system.
The
mechanism
contains
three
steps,
recognition,
cleavage,
repair.
designed
sgRNA
recognizes
target
sequence
gene
interest
through
a
complementary
base
pair.
While
Cas-9
nuclease
makes
double-stranded
breaks
at
site
3
pair
upstream
to
protospacer
adjacent
motif,
then
break
repaired
by
either
non-homologous
end
joining
or
homology-directed
repair
cellular
mechanisms.
genome-editing
has
wide
number
applications
areas
including
medicine,
agriculture,
biotechnology.
In
it
could
help
design
new
grains
improve
nutritional
value.
being
investigated
for
cancers,
HIV,
therapy
such
as
sickle
cell
disease,
cystic
fibrosis,
Duchenne
muscular
dystrophy.
technology
also
regulation
specific
genes
advanced
modification
protein.
However,
immunogenicity,
effective
delivery
systems,
off-target
effect,
ethical
issues
have
been
major
barriers
extend
clinical
applications.
Although
becomes
era
molecular
biology
countless
roles
ranging
from
basic
researches
applications,
there
still
challenges
rub
practical
various
improvements
needed
overcome
obstacles.
Keywords:
CRISPR,
Cas-9,
sgRNA,
gene-editing,
mechanism,
Journal of Controlled Release,
Journal Year:
2022,
Volume and Issue:
342, P. 345 - 361
Published: Jan. 10, 2022
The
discovery
of
clustered
regularly
interspaced
short
palindromic
repeats
(CRISPR)
genome
editing
technology
opened
the
door
to
provide
a
versatile
approach
for
treating
multiple
diseases.
Promising
results
have
been
shown
in
numerous
pre-clinical
studies
and
clinical
trials.
However,
safe
effective
method
deliver
genome-editing
components
is
still
key
challenge
vivo
therapy.
Adeno-associated
virus
(AAV)
one
most
commonly
used
vector
systems
date,
but
immunogenicity
against
capsid,
liver
toxicity
at
high
dose,
potential
genotoxicity
caused
by
off-target
mutagenesis
genomic
integration
remain
unsolved.
Recently
developed
transient
delivery
systems,
such
as
virus-like
particle
(VLP)
lipid
nanoparticle
(LNP),
may
solve
some
issues.
This
review
summarizes
existing
possible
solutions
overcome
their
limitations.
Also,
we
highlight
ongoing
trials
therapy
recently
tools
applications.
Nature Communications,
Journal Year:
2022,
Volume and Issue:
13(1)
Published: Feb. 9, 2022
Abstract
Prime
editing
(PE)
is
a
powerful
genome
engineering
approach
that
enables
the
introduction
of
base
substitutions,
insertions
and
deletions
into
any
given
genomic
locus.
However,
efficiency
PE
varies
widely
depends
not
only
on
region
targeted,
but
also
genetic
background
edited
cell.
Here,
to
determine
which
cellular
factors
affect
efficiency,
we
carry
out
focused
screen
targeting
32
DNA
repair
factors,
spanning
all
reported
pathways.
We
show
that,
depending
cell
line
type
edit,
ablation
mismatch
(MMR)
affords
2–17
fold
increase
in
across
several
human
lines,
types
edits
loci.
The
accumulation
key
MMR
MLH1
MSH2
at
sites
argues
for
direct
involvement
control.
Our
results
shed
new
light
mechanism
suggest
how
its
might
be
optimised.
Nature Biotechnology,
Journal Year:
2023,
Volume and Issue:
41(10), P. 1446 - 1456
Published: Feb. 16, 2023
Most
short
sequences
can
be
precisely
written
into
a
selected
genomic
target
using
prime
editing;
however,
it
remains
unclear
what
factors
govern
insertion.
We
design
library
of
3,604
various
lengths
and
measure
the
frequency
their
insertion
four
sites
in
three
human
cell
lines,
different
editor
systems
varying
DNA
repair
contexts.
find
that
length,
nucleotide
composition
secondary
structure
sequence
all
affect
rates.
also
discover
3'
flap
nucleases
TREX1
TREX2
suppress
longer
sequences.
Combining
features
machine
learning
model,
we
predict
relative
insertions
site
with
R
=
0.70.
Finally,
demonstrate
how
our
accurate
prediction
user-friendly
software
help
choose
codon
variants
common
fusion
tags
insert
at
high
efficiency,
provide
catalog
empirically
determined
rates
for
over
hundred
useful
Biologics,
Journal Year:
2024,
Volume and Issue:
Volume 18, P. 21 - 28
Published: Jan. 1, 2024
Abstract:
The
CRISPR-Cas
(Clustered
Regularly
Interspaced
Short
Palindromic
Repeat
(CRISPR))
and
the
associated
protein
(Cas9)
system,
a
young
but
well-studied
genome-editing
tool,
holds
plausible
solutions
to
wide
range
of
genetic
disorders.
single-guide
RNA
(sgRNA)
with
20-base
user-defined
spacer
sequence
Cas9
endonuclease
form
core
CRISPR-Cas9
system.
This
sgRNA
can
direct
nuclease
any
genomic
region
that
includes
protospacer
adjacent
motif
(PAM)
just
downstream
matches
sequence.
current
challenge
in
clinical
applications
technology
is
potential
off-target
effects
cause
DNA
cleavage
at
incorrect
sites.
Off-target
genome
editing
confuses
diminishes
therapeutic
addition
potentially
casting
doubt
on
scientific
findings
regarding
activities
genes.
In
this
review,
we
summarize
recent
technological
advancements
reducing
effect
editing.
Keywords:
CRISPR-Cas9,
editing,
effect,
advancements,
review
Plants,
Journal Year:
2022,
Volume and Issue:
11(19), P. 2625 - 2625
Published: Oct. 6, 2022
Climate
change
poses
a
serious
threat
to
global
agricultural
activity
and
food
production.
Plant
genome
editing
technologies
have
been
widely
used
develop
crop
varieties
with
superior
qualities
or
can
tolerate
adverse
environmental
conditions.
Unlike
conventional
breeding
techniques
(e.g.,
selective
mutation
breeding),
modern
tools
offer
more
targeted
specific
alterations
of
the
plant
could
significantly
speed
up
progress
developing
crops
desired
traits,
such
as
higher
yield
and/or
stronger
resilience
changing
environment.
In
this
review,
we
discuss
current
development
future
applications
in
mitigating
impacts
biotic
abiotic
stresses
on
agriculture.
We
focus
specifically
CRISPR/Cas
system,
which
has
center
attention
last
few
years
revolutionary
genome-editing
tool
various
species.
also
conducted
bibliographic
analysis
CRISPR-related
papers
published
from
2012
2021
(10
years)
identify
trends
potential
CRISPR/Cas-related
research.
addition,
review
article
outlines
shortcomings
challenges
employing
agriculture
notes
prospective.
believe
combining
innovative
would
be
key
optimizing
improvement
beyond
limitations
traditional
practices.
International Journal of Molecular Sciences,
Journal Year:
2023,
Volume and Issue:
24(22), P. 16077 - 16077
Published: Nov. 8, 2023
CRISPR
(clustered
regularly
interspaced
short
palindromic
repeats)/Cas9
is
a
unique
genome
editing
tool
that
can
be
easily
used
in
wide
range
of
applications,
including
functional
genomics,
transcriptomics,
epigenetics,
biotechnology,
plant
engineering,
livestock
breeding,
gene
therapy,
diagnostics,
and
so
on.
This
review
focused
on
the
current
CRISPR/Cas9
landscape,
e.g.,
Cas9
variants
with
improved
properties,
Cas9-derived
fusion
proteins,
delivery
methods,
pre-existing
immunity
against
anti-CRISPR
their
possible
roles
function
improvement.
Moreover,
this
presents
detailed
outline
CRISPR/Cas9-based
diagnostics
therapeutic
approaches.
Finally,
addresses
future
expansion
editors’
toolbox
orthologs
other
CRISPR/Cas
proteins.
Frontiers in Cell and Developmental Biology,
Journal Year:
2023,
Volume and Issue:
11
Published: March 3, 2023
The
use
of
next-generation
sequencing
(NGS)
technologies
has
been
instrumental
in
the
characterization
mutational
landscape
complex
human
diseases
like
cancer.
But
despite
enormous
rise
identification
disease
candidate
genetic
variants,
their
functionality
is
yet
to
be
fully
elucidated
order
have
a
clear
implication
patient
care.
Haploid
cell
models
become
tool
choice
for
functional
gene
studies,
since
they
only
contain
one
copy
genome
and
can
therefore
show
unmasked
phenotype
variants.
Over
past
few
years,
near-haploid
line
HAP1
widely
consolidated
as
favorite
studies.
Its
rapid
turnover
coupled
with
fact
that
allele
needs
modified
express
subsequent
desired
made
this
valuable
editing
by
CRISPR-Cas9
technologies.
This
review
examines
recent
uses
model
studies
high-throughput
screens
using
system.
It
covers
its
an
attempt
develop
new
relevant
further
elucidate
function,
create
ways
understand
basis
diseases.
We
will
cover
advantages
potential
technology
on
easily
efficiently
study
interpretation
function
single-nucleotide
variants
unknown
significance
identified
through
NGS
technologies,
implications
changes
clinical
practice
