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
BMC Bioinformatics,
Journal Year:
2021,
Volume and Issue:
22(1)
Published: March 2, 2021
Abstract
Background
The
rapid
expansion
of
the
CRISPR
toolbox
through
tagging
effector
domains
to
either
enzymatically
inactive
Cas9
(dCas9)
or
nickase
(nCas9)
has
led
several
promising
new
gene
editing
strategies.
Recent
additions
include
cytosine
adenine
base
editors
(CBEs
and
ABEs)
prime
(PEs),
in
which
a
deaminase
reverse
transcriptase
are
fused
nCas9,
respectively.
These
tools
hold
great
promise
model
correct
disease-causing
mutations
animal
plant
models.
But
so
far,
no
widely-available
exist
automate
design
both
BE
PE
reagents.
Results
We
developed
PnB
Designer,
web-based
application
for
pegRNAs
PEs
guide
RNAs
BEs.
Designer
makes
it
easy
targeting
single
multiple
targets
on
variant
reference
genome
from
organisms
spanning
kingdoms.
With
we
designed
all
known
disease
causing
available
ClinVar.
Additionally,
can
be
used
install
revert
SNV,
scanning
with
one
CBE
seven
different
ABE
PAM
variants
returning
best
use.
is
publicly
accessible
at
http://fgcz-shiny.uzh.ch/PnBDesigner/
Conclusion
created
user-friendly
tool
reagents,
should
simplify
choosing
strategy
avoiding
complications.
Nature Communications,
Journal Year:
2022,
Volume and Issue:
13(1)
Published: April 1, 2022
Deep
Learning
(DL)
has
recently
enabled
unprecedented
advances
in
one
of
the
grand
challenges
computational
biology:
half-century-old
problem
protein
structure
prediction.
In
this
paper
we
discuss
recent
advances,
limitations,
and
future
perspectives
DL
on
five
broad
areas:
prediction,
function
genome
engineering,
systems
biology
data
integration,
phylogenetic
inference.
We
each
application
area
cover
main
bottlenecks
approaches,
such
as
training
data,
scope,
ability
to
leverage
existing
architectures
new
contexts.
To
conclude,
provide
a
summary
subject-specific
general
for
across
biosciences.
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.
