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
Cell,
Journal Year:
2022,
Volume and Issue:
185(2), P. 250 - 265.e16
Published: Jan. 1, 2022
Methods
to
deliver
gene
editing
agents
in
vivo
as
ribonucleoproteins
could
offer
safety
advantages
over
nucleic
acid
delivery
approaches.
We
report
the
development
and
application
of
engineered
DNA-free
virus-like
particles
(eVLPs)
that
efficiently
package
base
editor
or
Cas9
ribonucleoproteins.
By
engineering
VLPs
overcome
cargo
packaging,
release,
localization
bottlenecks,
we
developed
fourth-generation
eVLPs
mediate
efficient
several
primary
mouse
human
cell
types.
Using
different
glycoproteins
alters
their
cellular
tropism.
Single
injections
into
mice
support
therapeutic
levels
multiple
tissues,
reducing
serum
Pcsk9
78%
following
63%
liver
editing,
partially
restoring
visual
function
a
model
genetic
blindness.
In
vitro
off-target
from
was
virtually
undetected,
an
improvement
AAV
plasmid
delivery.
These
results
establish
promising
vehicles
for
macromolecule
combine
key
both
viral
nonviral
Signal Transduction and Targeted Therapy,
Journal Year:
2023,
Volume and Issue:
8(1)
Published: Jan. 16, 2023
Abstract
Clustered
regularly
interspaced
short
palindromic
repeats
(CRISPR)/CRISPR-associated
protein
9
(Cas9)
gene-editing
technology
is
the
ideal
tool
of
future
for
treating
diseases
by
permanently
correcting
deleterious
base
mutations
or
disrupting
disease-causing
genes
with
great
precision
and
efficiency.
A
variety
efficient
Cas9
variants
derivatives
have
been
developed
to
cope
complex
genomic
changes
that
occur
during
diseases.
However,
strategies
effectively
deliver
CRISPR
system
diseased
cells
in
vivo
are
currently
lacking,
nonviral
vectors
target
recognition
functions
may
be
focus
research.
Pathological
physiological
resulting
from
disease
onset
expected
serve
as
identifying
factors
targeted
delivery
targets
gene
editing.
Diseases
both
varied
complex,
choice
appropriate
methods
different
important.
Meanwhile,
there
still
many
potential
challenges
identified
when
targeting
CRISPR/Cas9
treatment.
This
paper
reviews
current
developments
three
aspects,
namely,
type,
vector,
characteristics.
Additionally,
this
summarizes
successful
examples
clinical
trials
finally
describes
possible
problems
associated
applications.
Cell,
Journal Year:
2022,
Volume and Issue:
185(15), P. 2806 - 2827
Published: July 1, 2022
In
vivo
gene
editing
therapies
offer
the
potential
to
treat
root
causes
of
many
genetic
diseases.
Realizing
promise
therapeutic
in
requires
ability
safely
and
efficiently
deliver
agents
relevant
organs
tissues
vivo.
Here,
we
review
current
delivery
technologies
that
have
been
used
enable
editing,
including
viral
vectors,
lipid
nanoparticles,
virus-like
particles.
Since
no
single
modality
is
likely
be
appropriate
for
every
possible
application,
compare
benefits
drawbacks
each
method
highlight
opportunities
future
improvements.
Signal Transduction and Targeted Therapy,
Journal Year:
2024,
Volume and Issue:
9(1)
Published: July 17, 2024
Traditional
therapeutic
approaches
such
as
chemotherapy
and
radiation
therapy
have
burdened
cancer
patients
with
onerous
physical
psychological
challenges.
Encouragingly,
the
landscape
of
tumor
treatment
has
undergone
a
comprehensive
remarkable
transformation.
Emerging
fervently
pursued
modalities
are
small
molecule
targeted
agents,
antibody-drug
conjugates
(ADCs),
cell-based
therapies,
gene
therapy.
These
cutting-edge
not
only
afford
personalized
precise
targeting,
but
also
provide
enhanced
comfort
potential
to
impede
disease
progression.
Nonetheless,
it
is
acknowledged
that
these
strategies
still
harbour
untapped
for
further
advancement.
Gaining
understanding
merits
limitations
holds
promise
offering
novel
perspectives
clinical
practice
foundational
research
endeavours.
In
this
review,
we
discussed
different
modalities,
including
drugs,
peptide
antibody
cell
therapy,
It
will
detailed
explanation
each
method,
addressing
their
status
development,
challenges,
solutions.
The
aim
assist
clinicians
researchers
in
gaining
deeper
diverse
options,
enabling
them
carry
out
effective
advance
more
efficiently.
