Military Medical Research,
Journal Year:
2023,
Volume and Issue:
10(1)
Published: March 10, 2023
Abstract
The
rapid
development
of
genome
editing
technology
has
brought
major
breakthroughs
in
the
fields
life
science
and
medicine.
In
recent
years,
clustered
regularly
interspaced
short
palindromic
repeats
(CRISPR)-based
toolbox
been
greatly
expanded,
not
only
with
emerging
CRISPR-associated
protein
(Cas)
nucleases,
but
also
novel
applications
through
combination
diverse
effectors.
Recently,
transposon-associated
programmable
RNA-guided
systems
have
uncovered,
adding
myriads
potential
new
tools
to
toolbox.
CRISPR-based
revolutionized
cardiovascular
research.
Here
we
first
summarize
advances
involving
newly
identified
Cas
orthologs,
engineered
variants
systems,
then
discuss
CRISPR-Cas
precise
editing,
such
as
base
prime
editing.
We
highlight
progress
research
using
technologies,
including
generation
genetically
modified
vitro
animal
models
diseases
(CVD)
well
treating
different
types
CVD.
Finally,
current
limitations
future
prospects
technologies
are
discussed.
Cell,
Journal Year:
2023,
Volume and Issue:
186(18), P. 3983 - 4002.e26
Published: Aug. 1, 2023
Prime
editing
enables
a
wide
variety
of
precise
genome
edits
in
living
cells.
Here
we
use
protein
evolution
and
engineering
to
generate
prime
editors
with
reduced
size
improved
efficiency.
Using
phage-assisted
evolution,
efficiencies
compact
reverse
transcriptases
by
up
22-fold
generated
that
are
516–810
base
pairs
smaller
than
the
current-generation
editor
PEmax.
We
discovered
different
specialize
types
used
this
insight
outperform
PEmax
PEmaxΔRNaseH,
truncated
dual-AAV
delivery
systems.
Finally,
Cas9
domains
improve
editing.
These
resulting
(PE6a-g)
enhance
therapeutically
relevant
patient-derived
fibroblasts
primary
human
T-cells.
PE6
variants
also
enable
longer
insertions
be
installed
vivo
following
delivery,
achieving
40%
loxP
insertion
cortex
murine
brain,
24-fold
improvement
compared
previous
state-of-the-art
editors.
Nature Communications,
Journal Year:
2023,
Volume and Issue:
14(1)
Published: Jan. 13, 2023
CRISPR-Cas
gene
editing
has
revolutionized
experimental
molecular
biology
over
the
past
decade
and
holds
great
promise
for
treatment
of
human
genetic
diseases.
Here
we
review
development
CRISPR-Cas9/Cas12/Cas13
nucleases,
DNA
base
editors,
prime
RNA
focusing
on
assessment
improvement
their
precision
safety,
pushing
limit
specificity
efficiency.
We
summarize
capabilities
limitations
each
CRISPR
tool
from
to
editing,
highlight
opportunities
future
improvements
applications
in
basic
research,
as
well
therapeutic
clinical
considerations
use
patients.
Trends in biotechnology,
Journal Year:
2023,
Volume and Issue:
41(8), P. 1000 - 1012
Published: March 30, 2023
Clustered
regularly
interspaced
short
palindromic
repeats-associated
protein
9
(CRISPR–Cas)-mediated
genome
editing
has
revolutionized
biomedical
research
and
will
likely
change
the
therapeutic
diagnostic
landscape.
However,
CRISPR–Cas9,
which
edits
DNA
by
activating
double-strand
break
(DSB)
repair
pathways,
is
not
always
sufficient
for
gene
therapy
applications
where
precise
mutation
required.
Prime
editing,
latest
revolution
in
genome-editing
technologies,
can
achieve
any
possible
base
substitution,
insertion,
or
deletion
without
requirement
DSBs.
prime
still
its
infancy,
further
development
needed
to
improve
efficiency
delivery
strategies
applications.
We
summarize
developments
optimization
of
editor
(PE)
variants
with
improved
precision.
Moreover,
we
highlight
some
potential
