bioRxiv (Cold Spring Harbor Laboratory),
Journal Year:
2024,
Volume and Issue:
unknown
Published: Sept. 14, 2024
Diabetic
retinopathy
(DR),
a
leading
cause
of
vision
impairment
and
blindness,
is
characterized
by
abnormal
retinal
vascular
changes
due
to
chronic
hyperglycemia.
The
Tie-1
signaling
pathway,
essential
for
growth
remodeling,
has
emerged
as
key
therapeutic
target,
though
its
molecular
mechanisms
interactome
remain
largely
unclear.
Through
protein-centric
approach,
we
identified
novel
lncRNA
named
it
Tie1-associated
angiogenic
(TAAL).
TAAL
regulates
endothelial
cell
migration,
proliferation,
tube
formation,
permeability
modulating
ER-calcium
homeostasis
cytoskeleton
dynamics.
In
zebrafish,
taal
modulation
led
defects,
which
were
rescued
human
orthologue.
Our
studies
further
revealed
that
negatively
Tie1
protein
via
ubiquitin-mediated
degradation.
Notably,
expression
upregulated
in
the
blood
DR
patients
downregulated
models.
Overexpression
restored
VE-cadherin
surface
expression.
These
findings
establish
regulator
turnover,
with
potential
implications
diabetic
retinopathy.
Nucleosides Nucleotides & Nucleic Acids,
Journal Year:
2025,
Volume and Issue:
unknown, P. 1 - 25
Published: Jan. 13, 2025
The
field
of
biomedical
science
has
witnessed
another
milestone
with
the
advent
RNA-based
therapeutics.
This
review
explores
three
major
RNA
molecules,
namely:
messenger
(mRNA),
interference
technology
(RNAi),
and
Antisense
Oligonucleotide
(ASO),
analyses
U.S.
Food
Drug
Administration
drugs
from
14
pharmaceutical
companies
in
terms
targeted
genes,
diseases
types,
clinical
trials
status,
mode
delivery,
year
production.
Many
such
are
clinically
approved
or
pending
approval
by
(FDA)
alongside
other
leading
agencies.
Regarding
diseases,
this
article
emphasizes
cancer
therapy,
genetic
viral
infections,
two
categories
drug
delivery
systems
include
vectors
nanoparticles.
Despite
tremendous
progress
made,
key
issues
associated
these
stability,
off-target
activities
payloads,
efficiency
cellular
uptake,
innovative
need
for
engineering
techniques
modifications.
transformative
potential
therapeutics
role
technologies
addressing
needs,
paving
way
a
new
era
precision
medicine.
Oncology Research Featuring Preclinical and Clinical Cancer Therapeutics,
Journal Year:
2024,
Volume and Issue:
32(10), P. 1543 - 1564
Published: Jan. 1, 2024
The
advent
of
RNA
therapy,
particularly
through
the
development
mRNA
cancer
vaccines,
has
ushered
in
a
new
era
field
oncology.
This
article
provides
concise
overview
key
principles,
recent
advancements,
and
potential
implications
vaccines
as
groundbreaking
modality
treatment.
represent
revolutionary
approach
to
combatting
by
leveraging
body's
innate
immune
system.
These
are
designed
deliver
specific
sequences
encoding
cancer-associated
antigens,
prompting
system
recognize
mount
targeted
response
against
malignant
cells.
personalized
adaptive
nature
holds
immense
for
addressing
heterogeneity
tailoring
treatments
individual
patients.
Recent
breakthroughs
exemplified
success
COVID-19
have
accelerated
their
application
platform's
versatility
allows
rapid
adaptation
vaccine
candidates
various
types,
presenting
an
agile
promising
avenue
therapeutic
intervention.
Clinical
trials
demonstrated
encouraging
results
terms
safety,
immunogenicity,
efficacy.
Pioneering
candidates,
such
BioNTech's
BNT111
Moderna's
mRNA-4157,
exhibited
outcomes
targeting
melanoma
solid
tumors,
respectively.
successes
underscore
elicit
robust
durable
anti-cancer
responses.
While
great
promise,
challenges
manufacturing
complexities
cost
considerations
need
be
addressed
widespread
adoption.
scalable
cost-effective
processes,
along
with
ongoing
clinical
research,
will
pivotal
realizing
full
vaccines.
Overall,
cutting-edge
that
promise
transforming
As
research
progresses,
refining
processes
crucial
advancing
these
from
mainstream
oncology
practice,
offering
hope
patients
fight
cancer.
Molecular Therapy — Nucleic Acids,
Journal Year:
2024,
Volume and Issue:
35(3), P. 102313 - 102313
Published: Aug. 19, 2024
The
use
of
mRNA
and
ribonucleoproteins
(RNPs)
as
therapeutic
agents
is
a
promising
strategy
for
treating
diseases
such
cancer
infectious
diseases.
This
review
provides
recent
advancements
challenges
in
mRNA-
RNP-based
therapies,
focusing
on
delivery
systems
lipid
nanoparticles
(LNPs),
which
ensure
efficient
to
target
cells.
Strategies
microfluidic
devices
are
employed
prepare
LNPs
loaded
with
RNPs,
demonstrating
effective
genome
editing
protein
expression
vitro
vivo.
These
applications
extend
treatment
disease
management,
results
therapy
using
encapsulating
Cas9
single-guide
RNA.
In
addition,
tissue-specific
targeting
strategies
offer
potential
improved
outcomes
reduced
off-target
effects.
Despite
progress,
optimizing
efficiency
remain.
Future
research
should
enhance
efficiency,
explore
targeting,
investigate
combination
advance
clinical
translation.
conclusion,
therapies
avenue
various
have
the
revolutionize
medicine,
providing
new
hope
patients
worldwide.
Advanced Science,
Journal Year:
2025,
Volume and Issue:
unknown
Published: April 7, 2025
Abstract
The
hyperactivation
of
autoreactive
B
cells
and
plasma
leads
to
the
development
systemic
lupus
erythematosus
(SLE)
rheumatoid
arthritis
(RA),
therefore,
targeting
abnormal
might
hold
promise
for
treatment
these
refractory
relapsing
diseases.
This
study
developed
l
ipid
n
ano
p
article‐encapsulated
mRN
A‐encoding
a
nti
b
odies
(mRNab‐LNPs)
CD19,
evaluated
their
therapeutic
efficacy
in
RA
mice.
mRNab‐LNPs
enabled
robust
production
anti‐CD19
antibodies
multiple
cell
lines
vitro.
Interestingly,
intramuscular
injection
resulted
high
sustained
In
particular,
numbers
CD19+
circulating
tissue‐resident
are
significantly
reduced
by
As
result,
histopathological
changes
tissue
injuries
both
Collectively,
findings
demonstrate
translational
potential
SLE
RA.
