Novel Ectodysplasin-A Variants: Structural and Functional Basis of Hypohidrotic Ectodermal Dysplasia
Abstract
This
study
investigates
two
novel
variants
in
the
EDA ,
c.680G
>
A
(p.G227E)
and
c.649_666del
(Δ215–220),
identified
X-linked
ectodermal
dysplasia
syndromic
tooth
agenesis
cases.
These
were
through
Sanger
sequencing
mapped
to
highly
conserved
regions
of
EDA.
Bioinformatics
tools
consistently
classified
them
as
deleterious,
with
significant
disruptions
predicted
protein
stability,
hydrophobicity,
secondary
structure.
Structural
analysis
revealed
that
p.G227E
caused
a
glycine-to-glutamic
acid
substitution,
altering
hydrophobicity
structure,
while
Δ215–220
disrupted
hydrophobic
region,
leading
increased
instability
Functional
studies
reduced
expression
EDA
WNT4
proteins,
alongside
IκB
levels
decreased
NF-κB
mRNA
expression,
indicating
impaired
EDA-NF-κB
signaling.
Subcellular
localization
analyses
demonstrated
diminished
cytoplasmic
Variants
corroborated
by
silico
predictions.
Post-translational
modifications
(PTMs)
gene
ontology
(GO)
alterations
processes
critical
for
development,
including
macromolecule
biosynthesis,
nitrogen
metabolism,
receptor
Molecular
dynamics
simulations
highlighted
rigidity,
compact
flexibility
proteins
compared
Wild
Type
(WT).
Interestingly,
neither
variant
significantly
impacted
calcium
or
mitochondrial
potential
under
normal
experimental
conditions,
suggesting
their
pathogenic
effects
arise
primarily
from
interactions
signaling
pathways.
integrates
molecular,
bioinformatics,
functional
elucidate
pathogenicity
these
EDA
variants,
providing
insights
into
mechanisms
paving
way
future
therapeutic
strategies
targeting
variants.

Research Square (Research Square), Год журнала: 2025, Номер unknown
Опубликована: Янв. 3, 2025
Язык: Английский