Journal of Inherited Metabolic Disease,
Journal Year:
2024,
Volume and Issue:
47(1), P. 176 - 191
Published: Jan. 1, 2024
Abstract
Inborn
errors
of
neurotransmitter
(NT)
metabolism
are
a
group
rare,
heterogenous
diseases
with
predominant
neurological
features,
such
as
movement
disorders,
autonomic
dysfunction,
and
developmental
delay.
Clinical
overlap
other
disorders
has
led
to
delayed
diagnosis
treatment,
some
conditions
refractory
oral
pharmacotherapies.
Gene
therapies
have
been
developed
translated
clinics
for
paediatric
inborn
metabolism,
38
interventional
clinical
trials
ongoing
date.
Furthermore,
efforts
in
restoring
dopamine
synthesis
neurotransmission
through
viral
gene
therapy
Parkinson's
disease.
Along
the
recent
European
Medicines
Agency
(EMA)
Healthcare
Products
Regulatory
(MHRA)
approval
an
AAV2
supplementation
AADC
deficiency,
promising
efficacy
safety
profiles
can
be
achieved
this
diseases.
In
review,
we
present
preclinical
advances
address
NT‐related
diseases,
summarise
potential
challenges
that
require
careful
considerations
NT
studies.
ACS Synthetic Biology,
Journal Year:
2023,
Volume and Issue:
12(1), P. 17 - 26
Published: Jan. 10, 2023
Gene
therapy
has
demonstrated
enormous
potential
for
changing
how
we
combat
disease.
By
directly
engineering
the
genetic
composition
of
cells,
it
provides
a
broad
range
options
improving
human
health.
Adeno-associated
viruses
(AAVs)
represent
leading
gene
vector
and
are
expected
to
address
wide
conditions
in
coming
decade.
Three
AAV
therapies
have
already
been
approved
by
FDA
treat
Leber's
congenital
amaurosis,
spinal
muscular
atrophy,
hemophilia
B.
Yet
these
cost
around
$850,000,
$2,100,000,
$3,500,000,
respectively.
Such
prices
limit
applicability
make
inaccessible
most
patients.
Much
this
problem
arises
from
high
manufacturing
costs
AAVs.
At
same
time,
field
synthetic
biology
grown
rapidly
displayed
special
aptitude
addressing
biomanufacturing
problems.
Here,
discuss
emerging
efforts
apply
design
decrease
price
production,
propose
that
such
could
play
major
role
making
much
more
widely
accessible.
Advanced Materials,
Journal Year:
2024,
Volume and Issue:
36(31)
Published: June 6, 2024
Abstract
The
recent
success
of
gene
therapy
during
the
COVID‐19
pandemic
has
underscored
importance
effective
and
safe
delivery
systems.
Complementing
lipid‐based
systems,
polymers
present
a
promising
alternative
for
delivery.
Significant
advances
have
been
made
in
past,
with
multiple
clinical
trials
progressing
beyond
phase
I
several
companies
actively
working
on
polymeric
systems
which
provides
assurance
that
carriers
can
soon
achieve
translation.
massive
advantage
structural
tunability
vast
chemical
space
is
being
leveraged
to
mitigate
shortcomings
traditional
polycationic
improve
translatability
Tailored
approaches
diverse
nucleic
acids
specific
subcellular
targets
are
now
designed
therapeutic
efficacy.
This
review
describes
polymer
design
improved
by
polyplexes
covalent
polymer‐nucleic
acid
conjugates.
also
offers
brief
note
novel
computational
techniques
design.
concludes
an
overview
current
state
therapies
clinic
as
well
future
directions
their
translation
clinic.
The Journal of Gene Medicine,
Journal Year:
2024,
Volume and Issue:
26(8)
Published: Aug. 1, 2024
Abstract
To
date,
3,900
gene
therapy
clinical
trials
have
been
completed,
are
ongoing
or
approved
worldwide.
Our
database
brings
together
global
information
on
activity
from
trial
databases,
official
agency
sources,
published
literature,
conference
presentations
and
posters
kindly
provided
to
us
by
individual
investigators
sponsors.
This
review
presents
our
analysis
of
that,
the
best
knowledge,
being
performed
As
March
2023
update,
we
entries
undertaken
in
46
countries.
We
analyzed
geographical
distribution
trials,
disease
indications
(or
other
reasons)
for
proportions
which
different
vector
types
used,
genes
transferred.
Details
analyses
presented,
searchable
The
Journal
Gene
Medicine
Therapy
Clinical
Trials
Worldwide
website
at
https://a873679.fmphost.com/fmi/webd/GTCT
.
also
provide
an
overview
progress
made
around
world,
discuss
key
trends
since
previous
review,
namely
unprecedented
increase
activity,
including
implementation
genome
editing
technology
with
potential
transform
field
moving
forward.
Journal of Inherited Metabolic Disease,
Journal Year:
2024,
Volume and Issue:
47(1), P. 176 - 191
Published: Jan. 1, 2024
Abstract
Inborn
errors
of
neurotransmitter
(NT)
metabolism
are
a
group
rare,
heterogenous
diseases
with
predominant
neurological
features,
such
as
movement
disorders,
autonomic
dysfunction,
and
developmental
delay.
Clinical
overlap
other
disorders
has
led
to
delayed
diagnosis
treatment,
some
conditions
refractory
oral
pharmacotherapies.
Gene
therapies
have
been
developed
translated
clinics
for
paediatric
inborn
metabolism,
38
interventional
clinical
trials
ongoing
date.
Furthermore,
efforts
in
restoring
dopamine
synthesis
neurotransmission
through
viral
gene
therapy
Parkinson's
disease.
Along
the
recent
European
Medicines
Agency
(EMA)
Healthcare
Products
Regulatory
(MHRA)
approval
an
AAV2
supplementation
AADC
deficiency,
promising
efficacy
safety
profiles
can
be
achieved
this
diseases.
In
review,
we
present
preclinical
advances
address
NT‐related
diseases,
summarise
potential
challenges
that
require
careful
considerations
NT
studies.
