Frontiers in Molecular Neuroscience,
Journal Year:
2023,
Volume and Issue:
15
Published: Jan. 9, 2023
Inherited
retinal
diseases
(IRDs)
are
associated
with
mutations
in
over
250
genes
and
represent
a
major
cause
of
irreversible
blindness
worldwide.
While
gene
augmentation
or
editing
therapies
could
address
the
underlying
genetic
small
subset
patients,
their
utility
remains
limited
by
great
heterogeneity
IRDs
costs
developing
individualised
therapies.
Gene-agnostic
therapeutic
approaches
target
common
pathogenic
pathways
that
drive
degeneration
provide
functional
rescue
vision
independent
cause,
thus
offering
potential
clinical
benefits
to
all
IRD
patients.
Here,
we
review
key
gene-agnostic
approaches,
including
cell
reprogramming
replacement,
neurotrophic
support,
immune
modulation
optogenetics.
The
relative
limitations
these
strategies
timing
interventions
discussed.
Progress in Retinal and Eye Research,
Journal Year:
2021,
Volume and Issue:
86, P. 100975 - 100975
Published: May 29, 2021
Inherited
and
age-related
retinal
degeneration
is
the
hallmark
of
a
large
group
heterogeneous
diseases
main
cause
untreatable
blindness
today.
Genetic
factors
play
major
pathogenic
role
in
degenerations
for
both
monogenic
(such
as
retinitis
pigmentosa)
complex
with
established
genetic
risk
macular
degeneration).
Progress
genotyping
techniques
back
eye
imaging
are
completing
our
understanding
these
their
manifestations
patient
populations
suffering
from
degenerations.
It
clear
that
whatever
cause,
majority
vision
loss
results
photoreceptor
function.
The
timing
circumstances
surrounding
function
determine
adequate
therapeutic
approach
to
use
each
patient.
Among
such
approaches,
gene
therapy
rapidly
becoming
reality
applicable
clinic.
This
massive
move
laboratory
work
towards
clinical
application
has
been
propelled
by
advances
disease
genetics
mechanisms,
delivery
vectors,
editing
systems,
compensatory
strategies
Here,
we
provide
an
overview
existing
modalities
relevance
based
on
needs
inherited
