Exploration of Immunology,
Год журнала:
2024,
Номер
unknown, С. 577 - 615
Опубликована: Окт. 18, 2024
Complement
is
both
evolutionary
and
scientifically
old.
It
predates
the
adaptive
immunity
by
some
600
million
years
was
first
described
in
1905
Jules
Bordet
Paul
Ehrlich.
For
most
of
its,
existence
complement
system
has
been
ignored
scientists
clinicians
due
to
perception
it
being
complicated
its
relevance
for
pathogenesis
human
disease
unclear.
With
recent
US
Food
Drug
Administration
(FDA)
approvals
pegcetacoplan
paroxysmal
nocturnal
haemoglobinuria
(PNH)
geographic
atrophy
(GA),
avacincaptad
pegol
GA
iptacopan
danicopan
PNH,
we
are
at
a
crucial
juncture
complement-targeting
therapies.
A
number
companies
academic
institutions
developing
next-generation
therapies,
which
resulting
an
increasingly
competitive
landscape.
If
one
looks
serum
cascade,
all
3
pathways
now
have
biotechnology
or
pharmaceutical
industry
players
with
1
multiple
clinical-stage
inhibitors
that
expected
be
FDA
approved
within
next
few
years.
Furthermore,
limited
clinically
validated
targets
complement-mediated
disease,
competition
this
space
set
further
intensify
coming
In
review,
will
discuss
timeline
discoveries
led
development
current
crop
FDA-approved
therapeutics.
We
follow
discussion
crowded
therapy
scientific
advances
emerged
two
decades
underpinning
future
innovation,
including
our
understanding
biology,
such
as
local
intracellular
complement,
emerging
targets,
combinational
approaches
non-complement
therapeutics
unlock
new
indications
technologies
gene
therapy.
also
give
comprehensive
overview
landscape
how
can
utilized
target
dysregulation.
Plants,
Год журнала:
2023,
Номер
12(9), С. 1911 - 1911
Опубликована: Май 8, 2023
Targeted
genome
editing
technologies
are
becoming
the
most
important
and
widely
used
genetic
tools
in
studies
of
phytopathology.
The
"clustered
regularly
interspaced
short
palindromic
repeats
(CRISPR)"
its
accompanying
proteins
(Cas)
have
been
first
identified
as
a
natural
system
associated
with
adaptive
immunity
prokaryotes
that
successfully
various
genome-editing
techniques
because
flexibility,
simplicity,
high
efficiency
recent
years.
In
this
review,
we
provided
general
idea
about
different
CRISPR/Cas
systems
their
uses
This
review
focuses
on
benefits
knock-down
for
targeting
genes
involved
susceptibility
gaining
resistance
against
viral,
bacterial,
fungal
pathogens
by
negative
regulators
defense
pathways
hosts
crop
plants
via
systems.
Moreover,
possible
strategies
to
employ
improving
pathogen
studying
plant-pathogen
interactions
discussed.
Applied Microbiology and Biotechnology,
Год журнала:
2024,
Номер
108(1)
Опубликована: Фев. 14, 2024
Abstract
The
Cas9
endonuclease
of
the
CRISPR/Cas
type
IIA
system
from
Streptococcus
pyogenes
is
heart
genome
editing
technology
that
can
be
used
to
treat
human
genetic
and
viral
diseases.
Despite
its
large
size
other
drawbacks,
S.
remains
most
widely
editor.
A
vast
amount
research
aimed
at
improving
as
a
promising
therapy.
Strategies
include
directed
evolution
protein,
rational
design,
domain
swapping.
first
generation
editors
comes
directly
wild-type
protein.
next
obtained
by
combining
mutations
first-generation
variants,
adding
new
them,
or
refining
mutations.
This
review
summarizes
discusses
recent
advances
ways
in
creation
next-generation
genomic
derived
Cas9.
Key
points
•
Cas9-based
are
more
active
than
one.
PAM-relaxed
variants
improved
increased
specificity
activity.
Less
mutagenic
immunogenic
created.
World Journal of Gastroenterology,
Год журнала:
2024,
Номер
30(40), С. 4339 - 4353
Опубликована: Окт. 16, 2024
Diabetes
mellitus,
characterized
by
chronic
hyperglycemia
due
to
insulin
deficiency
or
resistance,
poses
a
significant
global
health
burden.
Central
its
pathogenesis
is
the
dysfunction
loss
of
pancreatic
beta
cells,
which
are
res-ponsible
for
production.
Recent
advances
in
beta-cell
regeneration
research
offer
promising
strategies
diabetes
treatment,
aiming
restore
endogenous
production
and
achieve
glycemic
control.
This
review
explores
physiological
basis
function,
recent
scientific
advan-cements,
challenges
translating
these
findings
into
clinical
applications.
It
highlights
key
developments
stem
cell
therapy,
gene
editing
technologies,
identification
novel
regenerative
molecules.
Despite
potential,
field
faces
hurdles
such
as
ensuring
safety
long-term
efficacy
regen-erative
therapies,
ethical
concerns
around
use,
complexity
differentiation
integration.
The
importance
interdisciplinary
collaboration,
increased
funding,
need
patient-centered
approaches
integration
new
treatments
comprehensive
care
overcome
challenges.
Through
continued
holds
potential
revolutionize
care,
turning
condition
manageable
even
curable
disease.
Journal of Clinical Medicine,
Год журнала:
2025,
Номер
14(6), С. 2040 - 2040
Опубликована: Март 17, 2025
Oncologists
increasingly
recognize
the
microbiome
as
an
important
facilitator
of
health
well
a
contributor
to
disease,
including,
specifically,
cancer.
Our
knowledge
etiologies,
mechanisms,
and
modulation
states
that
ameliorate
or
promote
cancer
continues
evolve.
The
progressive
refinement
adoption
“omic”
technologies
(genomics,
transcriptomics,
proteomics,
metabolomics)
utilization
advanced
computational
methods
accelerate
this
evolution.
academic
center
network,
with
its
immediate
access
extensive,
multidisciplinary
expertise
scientific
resources,
has
potential
catalyze
research.
Here,
we
review
our
current
understanding
role
gut
in
prevention,
predisposition,
response
therapy.
We
underscore
promise
operationalizing
network
uncover
structure
function
microbiome;
highlight
unique
microbiome-related
expert
resources
available
at
City
Hope
Comprehensive
Cancer
Center
example
team
science
achieve
novel
clinical
discovery.
Marfan
syndrome
(MFS)
is
an
inherited
autosomal
dominant
disorder
that
affects
connective
tissue
with
incidence
of
about
1
in
5,000
to
10,000
people.
90%
MFS
caused
by
mutations
the
fibrillin-1
(FBN1)
gene.
We
recruited
a
family
phenotype
South
China
and
identified
novel
variant.
This
study
investigated
whether
this
genetic
variant
pathogenic
potential
pathway
related
lipid
metabolism
MFS.
A
three-generation
consanguineous
was
for
study.
Whole
exome
sequencing
(WES)
utilized
on
members.
The
3D
structure
protein
predicted
using
AlphaFold.
CRISPR/Cas9
applied
generate
similar
fbn1
nonsense
mutation
(fbn1+/−)
zebrafish.
RNA-seq
analysis
zebrafish
performed
identify
pathways
pathogenesis.
Our
[NM_000138.5;
c.7764
C
>
G:
p.(Y2588*)]
FBN1
gene
from
same
site
among
proband
along
her
son
daughter.
Structural
modeling
showed
p.Y2588*
resulted
truncated
protein.
Compared
wild-type
zebrafish,
F2
generation
fbn1+/−
exhibited
phenotype.
indicated
many
genes
leptin
are
up-regulating,
which
could
affect
bone
development
adipose
homeostasis.
In
model,
we
found
functional
evidence
supporting
pathogenicity
detected
mutation.
research
proposes
possible
mechanism
underlying
relationship
between
These
findings
can
help
improve
clinical
diagnosis
treatment
