Nanotheranostics Revolutionizing Gene Therapy: Emerging Applications in Gene Delivery Enhancement
Journal of Nanotheranostics,
Год журнала:
2025,
Номер
6(2), С. 10 - 10
Опубликована: Апрель 9, 2025
Nanotheranostics—where
nanoscale
materials
serve
both
diagnostic
and
therapeutic
functions—are
rapidly
transforming
gene
therapy
by
tackling
critical
delivery
challenges.
This
review
explores
the
design
engineering
of
various
nanoparticle
systems
(lipid-based,
polymeric,
inorganic,
hybrid)
to
enhance
stability,
targeting,
endosomal
escape
genetic
payloads.
We
discuss
how
real-time
imaging
capabilities
integrated
into
these
platforms
enable
precise
localization
controlled
release
genes,
improving
treatment
efficacy
while
reducing
off-target
effects.
Key
strategies
overcome
barriers
(such
as
proton
sponge
effect
photothermal
disruption)
achieve
nuclear
are
highlighted,
along
with
recent
advances
in
stimuli-responsive
that
facilitate
spatiotemporal
control
expression.
Clinical
trials
preclinical
studies
demonstrate
expanding
role
nanotheranostics
managing
cancer,
inherited
disorders,
cardiovascular
neurological
diseases.
further
address
regulatory
manufacturing
hurdles
must
be
for
widespread
clinical
adoption
nanoparticle-based
therapies.
By
synthesizing
progress
ongoing
challenges,
this
underscores
transformative
potential
effective,
targeted,
image-guided
delivery.
Язык: Английский
Towards a Cure for Diamond–Blackfan Anemia: Views on Gene Therapy
Cells,
Год журнала:
2024,
Номер
13(11), С. 920 - 920
Опубликована: Май 27, 2024
Diamond–Blackfan
anemia
(DBA)
is
a
rare
genetic
disorder
affecting
the
bone
marrow’s
ability
to
produce
red
blood
cells,
leading
severe
and
various
physical
abnormalities.
Approximately
75%
of
DBA
cases
involve
heterozygous
mutations
in
ribosomal
protein
(RP)
genes,
classifying
it
as
ribosomopathy,
with
RPS19
being
most
frequently
mutated
gene.
Non-RP
mutations,
such
GATA1,
have
also
been
identified.
Current
treatments
include
glucocorticosteroids,
transfusions,
hematopoietic
stem
cell
transplantation
(HSCT),
HSCT
only
curative
option,
albeit
challenges
like
donor
availability
immunological
complications.
Gene
therapy,
particularly
using
lentiviral
vectors
CRISPR/Cas9
technology,
emerges
promising
alternative.
This
review
explores
potential
gene
focusing
on
technology
combination
non-integrating
vectors,
solution
for
DBA.
It
highlights
transformative
advancements
treatment
landscape
DBA,
offering
hope
individuals
affected
by
this
condition.
Язык: Английский
The Hard Work of Developing New Therapies for Pediatric Populations
Clinical Therapeutics,
Год журнала:
2024,
Номер
unknown
Опубликована: Окт. 1, 2024
Язык: Английский
Nucleases: From Primitive Immune Defenders to Modern Biotechnology Tools
Immunology,
Год журнала:
2024,
Номер
unknown
Опубликована: Дек. 16, 2024
ABSTRACT
The
story
of
nucleases
begins
on
the
ancient
battlefields
early
Earth,
where
simple
bacteria
fought
to
survive
against
viral
invaders.
Nucleases
are
enzymes
that
degrade
nucleic
acids,
with
restriction
endonucleases
emerging
as
some
earliest
defenders,
cutting
foreign
DNA
protect
their
hosts.
However,
sought
more
than
just
defence.
They
evolved
CRISPR‐Cas
system,
an
adaptive
immune
mechanism
capable
remembering
past
now‐famous
Cas9
nuclease,
a
key
player
in
this
has
been
harnessed
for
genome
editing,
revolutionising
biotechnology.
Over
time,
from
basic
defence
tools
into
complex
regulators
function
higher
organisms.
In
humans,
DNases
and
RNases
maintain
balance
by
clearing
cellular
debris,
preventing
autoimmunity,
defending
pathogens.
These
have
transformed
bacterial
defenders
critical
players
both
human
immunity
This
review
explores
evolutionary
history
vital
roles
protectors
life's
mechanisms.
Язык: Английский