Journal of Translational Medicine,
Год журнала:
2023,
Номер
21(1)
Опубликована: Июль 26, 2023
Abstract
Mitochondria
play
important
roles
in
maintaining
cellular
homeostasis
and
skeletal
muscle
health,
damage
to
mitochondria
can
lead
a
series
of
pathophysiological
changes.
Mitochondrial
dysfunction
atrophy,
its
molecular
mechanism
leading
atrophy
is
complex.
Understanding
the
pathogenesis
mitochondrial
useful
for
prevention
treatment
finding
drugs
methods
target
modulate
function
are
urgent
tasks
atrophy.
In
this
review,
we
first
discussed
normal
muscle.
Importantly,
described
effect
on
mechanisms
involved.
Furthermore,
regulatory
different
signaling
pathways
(AMPK-SIRT1-PGC-1α,
IGF-1-PI3K-Akt-mTOR,
FoxOs,
JAK-STAT3,
TGF-β-Smad2/3
NF-κB
pathways,
etc.)
factors
were
investigated
dysfunction.
Next,
analyzed
manifestations
caused
by
diseases.
Finally,
summarized
preventive
therapeutic
effects
targeted
regulation
including
drug
therapy,
exercise
diet,
gene
stem
cell
therapy
physical
therapy.
This
review
great
significance
holistic
understanding
role
muscle,
which
helpful
researchers
further
has
an
inspiring
development
strategies
targeting
future.
Cellular and Molecular Life Sciences,
Год журнала:
2024,
Номер
81(1)
Опубликована: Янв. 30, 2024
Abstract
Skeletal
muscle
is
a
highly
specialized
tissue
composed
of
myofibres
that
performs
crucial
functions
in
movement
and
metabolism.
In
response
to
external
stimuli
injuries,
range
stem/progenitor
cells,
with
stem
cells
or
satellite
(MuSCs)
being
the
predominant
cell
type,
are
rapidly
activated
repair
regenerate
skeletal
within
weeks.
Under
normal
conditions,
MuSCs
remain
quiescent
state,
but
become
proliferative
differentiate
into
new
injury.
addition
MuSCs,
some
interstitial
progenitor
(IPCs)
such
as
fibro-adipogenic
progenitors
(FAPs),
pericytes,
expressing
PW1
negative
for
Pax7
(PICs),
side
population
(SPCs),
CD133-positive
Twist2-positive
have
been
identified
playing
direct
indirect
roles
regenerating
tissue.
Here,
we
highlight
heterogeneity,
molecular
markers,
functional
properties
these
explore
role
homeostasis,
aging,
muscle-related
diseases.
This
review
provides
critical
insights
future
therapies
aimed
at
treating
Journal of Clinical Neurology,
Год журнала:
2025,
Номер
21(1), С. 40 - 40
Опубликована: Янв. 1, 2025
This
study
was
an
open-label,
dose-escalation,
phase
1
clinical
trial
to
determine
the
safety
and
dose
of
EN001
for
patients
with
Duchenne
muscular
dystrophy
(DMD).
EN001,
developed
by
ENCell,
are
allogeneic
early-passage
Wharton's
jelly-derived
mesenchymal
stem
cells
that
originate
at
umbilical
cord,
preclinical
studies
demonstrating
their
high
therapeutic
efficacy
DMD.
explored
tolerability
as
a
potential
treatment
option
Six
pediatric
participants
DMD
were
divided
into
two
subgroups
equal
size:
low-dose
(5.0×10⁵
cells/kg)
high-dose
(2.5×10⁶
cells/kg).
All
monitored
12
weeks
after
administration
assess
its
safety.
Dose-limiting
toxicity
(DLT)
evaluated
across
2
post
administration.
Exploratory
measuring
serum
creatine
kinase
levels,
functional
evaluations-including
spirometry,
myometry,
North
Star
Ambulatory
Assessment,
6-minute
walk
test-were
conducted
week
compared
baseline
values.
No
experienced
serious
adverse
events
related
injection
during
12-week
follow-up
period.
Mild
included
injection-related
local
erythema,
edema,
parosmia,
headache,
but
DLT
not
observed.
Functional
evaluations
revealed
no
significant
changes
from
baseline.
These
results
demonstrated
safe
well
tolerated
DMD,
did
cause
events.
The
could
be
confirmed
through
larger-scale
future
incorporate
repeated
dosing
have
randomized
controlled
design.
Journal of Veterinary Internal Medicine,
Год журнала:
2024,
Номер
38(1), С. 135 - 144
Опубликована: Янв. 1, 2024
Muscular
dystrophies
(MDs)
are
a
large,
heterogeneous
group
of
degenerative
muscle
diseases.
X-linked
dystrophin-deficient
MD
in
cats
is
the
first
genetically
characterized
cat
model
for
human
disease
and
few
novel
forms
have
been
identified.
Proteomes,
Год журнала:
2024,
Номер
12(1), С. 4 - 4
Опубликована: Янв. 16, 2024
This
perspective
article
is
concerned
with
the
question
of
how
proteomics,
which
a
core
technique
systems
biology
that
deeply
embedded
in
multi-omics
field
modern
bioresearch,
can
help
us
better
understand
molecular
pathogenesis
complex
diseases.
As
an
illustrative
example
monogenetic
disorder
primarily
affects
neuromuscular
system
but
characterized
by
plethora
multi-system
pathophysiological
alterations,
muscle-wasting
disease
Duchenne
muscular
dystrophy
was
examined.
Recent
achievements
dystrophinopathy
research
are
described
special
reference
to
proteome-wide
complexity
changes
and
body-wide
alterations/adaptations.
Based
on
description
current
applications
top-down
versus
bottom-up
proteomic
approaches
their
technical
challenges,
future
biological
outlined.
The
envisaged
holistic
integromic
bioanalysis
would
encompass
integration
diverse
omics-type
studies
including
inter-
intra-proteomics
as
disciplines
for
systematic
protein
evaluations,
sophisticated
biomolecular
analyses,
physiology,
biology,
biochemistry
histochemistry.
Integrated
findings
promise
be
instrumental
improving
our
detailed
knowledge
pathogenic
mechanisms
dysfunction,
widening
available
biomarker
signature
improved
diagnostic/prognostic
procedures,
advancing
identification
novel
therapeutic
targets
treat
dystrophy.
World Journal of Stem Cells,
Год журнала:
2025,
Номер
17(2)
Опубликована: Фев. 24, 2025
Skeletal
muscle
atrophy
results
from
disruptions
in
the
growth
and
metabolism
of
striated
muscle,
leading
to
a
reduction
or
loss
fibers.
This
condition
not
only
significantly
impacts
patients'
quality
life
but
also
imposes
substantial
socioeconomic
burdens.
The
complex
molecular
mechanisms
driving
skeletal
contribute
absence
effective
treatment
options.
Recent
advances
stem
cell
therapy
have
positioned
it
as
promising
approach
for
addressing
this
condition.
article
reviews
outlines
current
therapeutic
strategies,
focusing
on
mesenchymal
cells,
induced
pluripotent
their
derivatives.
Additionally,
challenges
these
cells
face
clinical
applications
are
discussed.
A
deeper
understanding
regenerative
potential
various
could
pave
way
breakthroughs
prevention
atrophy.
Duchenne
muscular
dystrophy
(DMD)
is
a
rare
and
severe
genetic
neuromuscular
disease,
characterized
by
rapid
progression
high
mortality,
highlighting
the
need
for
accurate
ambulatory
function
assessment
tools.
Ultrasound
imaging
methods
have
been
widely
used
quantitative
analysis.
Radiomics,
which
converts
medical
images
into
data,
combined
with
machine
learning
(ML),
offers
promising
solution.
This
study
aimed
at
utilizing
radiomics
to
analyze
different
stages
of
data
generated
during
B-mode
image
processing
evaluate
DMD
patients.
The
included
85
participants,
categorized
non-ambulatory
groups
based
on
their
functional
status.
scans
were
utilized
capture
backscattered
radiofrequency
then
processed
generate
envelope,
normalized,
images.
Radiomics
analysis
involved
manual
segmentation
grayscale
automatic
feature
extraction
using
specialized
software,
followed
selection
maximal
relevance
minimal
redundancy
method.
selected
features
input
five
ML
algorithms,
model
evaluation
conducted
via
area
under
receiver
operating
characteristic
curve
(AUROC).
To
ensure
robustness,
both
leave-one-out
cross-validation
repeated
splitting
employed.
Additionally,
multiple
models
constructed
tested
assess
performance.
intensity
values
across
all
types
increased
as
walking
ability
declined,
significant
differences
observed
between
(p
<
0.001).
These
exhibited
similar
diagnostic
performance
levels,
AUROC
below
0.8.
However,
(RF)
outperformed
other
when
was
applied,
notably
achieving
an
value
0.906.
combining
algorithms
yielded
higher
0.912
RF
input.
surpasses
conventional
ultrasound-derived
in
evaluating
DMD.
Moreover,
integrating
further
enhances
classification
proposed
method
this
framework
improving
accuracy
reliability
clinical
follow-up
evaluations,
supporting
more
effective
management
code
available
https://github.com/Goldenyan/radiomicsUS
.
