Advanced Delivery Systems for Gene Editing: A Comprehensive Review from the GenE-HumDi COST Action Working group
Molecular Therapy — Nucleic Acids,
Journal Year:
2025,
Volume and Issue:
36(1), P. 102457 - 102457
Published: Jan. 18, 2025
Language: Английский
Basic Concepts and Indications of CAR T Cells
Hämostaseologie,
Journal Year:
2025,
Volume and Issue:
45(01), P. 014 - 023
Published: Feb. 1, 2025
Abstract
Chimeric
antigen
receptor
(CAR)
T
cell
therapy
has
revolutionized
cancer
immunotherapy,
particularly
for
hematological
malignancies.
This
personalized
approach
is
based
on
genetically
engineering
cells
derived
from
the
patient
to
target
antigens
expressed—among
others—on
malignant
cells.
Nowadays
they
offer
new
hope
where
conventional
therapies,
such
as
chemotherapy
and
radiation,
have
often
failed.
Since
first
FDA
approval
in
2017,
CAR
rapidly
expanded,
proving
highly
effective
against
previously
refractory
diseases
with
otherwise
a
dismal
outcome.
Despite
its
promise,
continues
face
significant
challenges,
including
complex
manufacturing,
management
of
toxicities,
resistance
mechanisms
that
impact
long-term
efficacy,
limited
access
well
high
costs,
which
continue
shape
ongoing
research
clinical
applications.
review
aims
provide
an
overview
therapy,
fundamental
concepts,
applications,
current
future
directions
Language: Английский
TCR-T cell therapy: current development approaches, preclinical evaluation, and perspectives on regulatory challenges
Journal of Translational Medicine,
Journal Year:
2024,
Volume and Issue:
22(1)
Published: Oct. 4, 2024
Language: Английский
Unlocking the potential of chimeric antigen receptor T cell engineering immunotherapy: Long road to achieve precise targeted therapy for hepatobiliary pancreatic cancers
Hongli Gao,
No information about this author
Lianyue Qu,
No information about this author
Mu Li
No information about this author
et al.
International Journal of Biological Macromolecules,
Journal Year:
2025,
Volume and Issue:
297, P. 139829 - 139829
Published: Jan. 13, 2025
Language: Английский
Autologous Paracrine Prostasin–Matriptase Serine Protease Interaction in Lymphoid Cancer Cells
Li-Mei Chen,
No information about this author
Karl X. Chai
No information about this author
Cells,
Journal Year:
2025,
Volume and Issue:
14(4), P. 247 - 247
Published: Feb. 10, 2025
The
serine
protease
prostasin
on
the
surface
of
exosomes
released
from
epithelial
cells
can
interact
with
ectopically
over-expressed
cell-surface
matriptase
in
cancerous
B
to
initiate
prostasin–matriptase
proteolytic
activation
cascade.
Matriptase
and
ensuing
self-activation
result
its
removal
cancer
cells,
reducing
cell
proliferation
migration.
In
this
study,
we
tested
hypothesis
that
lymphoid
could
be
removed
by
prostasin-initiated
using
genetically
engineered
autologous
carrying
prostasin.
co-cultures
prostasin-positive
prostasin-negative
vector-control
was
a
dose-dependent
manner,
as
determined
flow
cytometry.
This
paracrine
phenotype
requires
active
sites
both
proteases.
silico
analysis
RNA-seq
profiles
indicated
an
imbalanced
expression
high
low
prostasin,
their
cognate
inhibitors
B-cell
lymphoma
patient
specimens.
impact
exosomal
cluster
differentiation
molecules
activated
human
peripheral
blood
mononuclear
investigated
cytometry,
revealing
candidate
mechanisms
for
prostasin’s
role
regulating
cellular
adaptive
immunity.
interaction
exploited
method
targeting
diseases
such
lymphoma.
Language: Английский
Clinical Proof-of-Concept of a Non-Gene Editing Technology Using miRNA-Based shRNA to Engineer Allogeneic CAR T-Cells
Caroline Lonez,
No information about this author
Jennifer Bolsée,
No information about this author
Fanny Huberty
No information about this author
et al.
International Journal of Molecular Sciences,
Journal Year:
2025,
Volume and Issue:
26(4), P. 1658 - 1658
Published: Feb. 15, 2025
With
the
success
of
chimeric
antigen
receptor
(CAR)
T-cell
therapy
in
B-cell
malignancies,
efforts
are
being
made
to
extend
this
other
malignancies
and
broader
patient
populations.
However,
limitations
associated
with
time-consuming
highly
personalized
manufacturing
autologous
CAR
T-cells
remain.
Allogeneic
approaches
may
overcome
these
challenges
but
require
further
engineering
reduce
their
alloreactivity.
As
a
means
prevent
graft-versus-host
disease
(GvHD)
allogeneic
T-cells,
we
have
selected
micro
RNA
(miRNA)-based
short
hairpin
(shRNA)
targeting
CD3ζ
which
efficiently
downregulates
expression
(TCR)
below
detection
level.
We
generated
anti-B-cell
maturation
(CYAD-211)
that
co-express
an
anti-CD3ζ
miRNA-based
shRNA
within
construct
inhibited
TCR-mediated
signaling
vitro
GvHD
vivo.
CYAD-211
was
subsequently
evaluated
Phase-I
clinical
trial
(NCT04613557),
patients
relapsed
or
refractory
multiple
myeloma.
No
signs
were
observed
despite
evidence
engraftment,
demonstrating
efficient
downregulation
TCR.
Our
data
provide
proof
concept
non-gene-edited
technology
can
generate
fully
functional
without
any
GvHD.
is
needed
improve
persistence
long-term
activity.
Language: Английский
Nanotechnology in Advancing Chimeric Antigen Receptor T Cell Therapy for Cancer Treatment
Pharmaceutics,
Journal Year:
2024,
Volume and Issue:
16(9), P. 1228 - 1228
Published: Sept. 20, 2024
Chimeric
antigen
receptor
(CAR)
T
cell
therapy
has
emerged
as
a
groundbreaking
treatment
for
hematological
cancers,
yet
it
faces
significant
hurdles,
particularly
regarding
its
efficacy
in
solid
tumors
and
concerning
associated
adverse
effects.
This
review
provides
comprehensive
analysis
of
the
advancements
ongoing
challenges
CAR-T
therapy.
We
highlight
transformative
potential
nanotechnology
enhancing
by
improving
targeting
precision,
modulating
immune-suppressive
tumor
microenvironment,
overcoming
physical
barriers.
Nanotechnology
facilitates
efficient
CAR
gene
delivery
into
cells,
boosting
transfection
efficiency
potentially
reducing
costs.
Moreover,
offers
innovative
solutions
to
mitigate
cytokine
release
syndrome
(CRS)
immune
effector
cell-associated
neurotoxicity
(ICANS).
Cutting-edge
platforms
real-time
monitoring
activity
are
also
discussed.
By
integrating
these
advancements,
we
aim
provide
valuable
insights
pave
way
next
generation
therapies
overcome
current
limitations
enhance
therapeutic
outcomes.
Language: Английский
Egg Cooling After Oviposition Extends the Permissive Period for Microinjection-Mediated Genome Modification in Bombyx mori
International Journal of Molecular Sciences,
Journal Year:
2024,
Volume and Issue:
25(23), P. 12642 - 12642
Published: Nov. 25, 2024
In
general,
transgenesis
efficiency
is
largely
dependent
on
the
developmental
status
of
eggs
for
microinjection.
We
investigated
whether
relationship
between
and
cooling
in
silkworms,
Language: Английский
The potential of cellular homing behavior in tumor immunotherapy: from basic discoveries to clinical applications of immune, mesenchymal stem, and cancer cell homing
Dongtao Li,
No information about this author
Yixuan Yang,
No information about this author
Guangda Zheng
No information about this author
et al.
Frontiers in Immunology,
Journal Year:
2024,
Volume and Issue:
15
Published: Dec. 12, 2024
The
efficacy
of
immunotherapy,
a
pivotal
approach
in
the
arsenal
cancer
treatment
strategies,
is
contingent
on
capacity
effector
cells
to
localize
at
tumor
site.
navigational
these
intricately
linked
homing
behaviors
specific
cell
types.
Recent
studies
have
focused
leveraging
immune
and
mesenchymal
stem
(MSCs)
for
targeted
therapy
incorporating
properties
into
anti-tumor
strategies.
However,
research
development
immunotherapy
based
remain
their
preliminary
stages.
Enhancing
efficiency
essential;
therefore,
understanding
underlying
mechanisms
addressing
resistance
within
microenvironment
challenges
associated
with
vivo
therapeutic
agent
delivery
are
essential.
This
review
firstly
delineates
discovery
clinical
translation
three
principal
cell-homing
behaviors.
Secondly,
we
endeavor
conduct
an
in-depth
analysis
existing
therapy,
aim
identifying
common
applications,
potential
benefits,
barriers,
critical
success
factors
cellular
therapies.
Finally,
key
therapies,
provide
overview
outlook
enormous
harnessing
cells’
self-homing
treat
tumors.
Although
behavior
warrants
further
research,
it
remains
highly
competitive
modality
that
can
be
combined
classic
anti-cancer
In
general,
combining
optimize
effects
also
one
future
directions
field
transplantation.
Language: Английский