Molecular Therapy — Methods & Clinical Development,
Journal Year:
2020,
Volume and Issue:
18, P. 532 - 557
Published: July 4, 2020
We
present
an
overview
of
clinical
trials
involving
gene
editing
using
clustered
interspaced
short
palindromic
repeats
(CRISPR)-CRISPR-associated
protein
9
(Cas9),
transcription
activator-like
effector
nucleases
(TALENs),
or
zinc
finger
(ZFNs)
and
discuss
the
underlying
mechanisms.
In
cancer
immunotherapy,
is
applied
ex
vivo
in
T
cells,
transgenic
cell
receptor
(tTCR)-T
chimeric
antigen
(CAR)-T
cells
to
improve
adoptive
therapy
for
multiple
types.
This
involves
knockouts
immune
checkpoint
regulators
such
as
PD-1,
components
endogenous
TCR
histocompatibility
leukocyte
(HLA)
complex
generate
universal
allogeneic
CAR-T
CD7
prevent
self-destruction
therapy.
cervix
carcinoma
caused
by
human
papillomavirus
(HPV),
E6
E7
genes
are
disrupted
topically
machinery.
HIV
infection,
CCR5
co-receptor
HIV-resistant
hematopoietic
stem
cells.
β-thalassemia
sickle
disease,
engineered
induce
production
fetal
hemoglobin.
AAV-mediated
exploit
liver
systemic
therapeutic
proteins
hemophilia
mucopolysaccharidoses,
eye
restore
splicing
CEP920
Leber's
congenital
amaurosis.
Close
consideration
safety
aspects
education
stakeholders
will
be
essential
a
successful
implementation
technology
clinic.
Computational and Structural Biotechnology Journal,
Journal Year:
2020,
Volume and Issue:
18, P. 2401 - 2415
Published: Jan. 1, 2020
Genome
editing
is
the
modification
of
genomic
DNA
at
a
specific
target
site
in
wide
variety
cell
types
and
organisms,
including
insertion,
deletion
replacement
DNA,
resulting
inactivation
genes,
acquisition
novel
genetic
traits
correction
pathogenic
gene
mutations.
Due
to
advantages
simple
design,
low
cost,
high
efficiency,
good
repeatability
short-cycle,
CRISPR-Cas
systems
have
become
most
widely
used
genome
technology
molecular
biology
laboratories
all
around
world.
In
this
review,
an
overview
will
be
introduced,
innovations,
applications
human
disease
research
therapy,
as
well
challenges
opportunities
that
faced
practical
application
systems.
BMC Plant Biology,
Journal Year:
2020,
Volume and Issue:
20(1)
Published: May 25, 2020
Abstract
Traditionally,
generation
of
new
plants
with
improved
or
desirable
features
has
relied
on
laborious
and
time-consuming
breeding
techniques.
Genome-editing
technologies
have
led
to
a
era
genome
engineering,
enabling
an
effective,
precise,
rapid
engineering
the
plant
genomes.
Clustered
regularly
interspaced
short
palindromic
repeats
(CRISPR)/CRISPR-associated
protein
9
(CRISPR/Cas9)
emerged
as
genome-editing
tool,
extensively
applied
in
various
organisms,
including
plants.
The
use
CRISPR/Cas9
allows
generating
transgene-free
genome-edited
(“null
segregants”)
period
time.
In
this
review,
we
provide
critical
overview
recent
advances
derived
for
inducing
mutations
at
target
sites
controlling
expression
genes.
We
highlight
major
breakthroughs
applying
challenges
toward
production
null
segregants.
also
update
efforts
Cas9
proteins,
newly
discovered
variants,
novel
CRISPR/Cas
systems
application
related
will
not
only
facilitate
molecular
crop
but
accelerate
progress
basic
research.
Plant Biotechnology Journal,
Journal Year:
2019,
Volume and Issue:
18(4), P. 895 - 915
Published: Dec. 7, 2019
Rice
sheath
blight
disease,
caused
by
the
basidiomycetous
necrotroph
Rhizoctonia
solani,
became
one
of
major
threats
to
rice
cultivation
worldwide,
especially
after
adoption
high-yielding
varieties.
The
pathogen
is
challenging
manage
because
its
extensively
broad
host
range
and
high
genetic
variability
also
due
inability
find
any
satisfactory
level
natural
resistance
from
available
germplasm.
It
time
remedies
combat
for
reducing
yield
losses
subsequently
minimize
threat
global
food
security.
development
alternative
means
avoid
use
hazardous
chemical
fungicides.
This
review
mainly
focuses
on
effort
better
understanding
host-pathogen
relationship,
finding
gene
loci/markers
imparting
response
modifying
genome
through
transgenic
development.
latest
trend
in
R.
solani-rice
pathosystem
research
with
gap
analysis
are
provided.
