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Fluorous iminoalditols: a new family of glycosidase inhibitors and pharmacological chaperones.
[gm1 gangliosidosis]
A
collection
of
new
reversible
glycosidase
inhibitors
of
the
iminoalditol
type
featuring
N-
substituents
containing
perfluorinated
regions
has
been
prepared
for
evaluation
of
physicochemical
,
biochemical
and
diagnostic
properties
.
The
vast
variety
of
feasible
oligofluoro
moieties
allows
for
modular
approaches
to
customised
structures
according
to
the
intended
applications
,
which
are
influenced
by
the
fluorine
content
as
well
as
the
distance
of
the
fluorous
moiety
from
the
ring
nitrogen
.
The
first
examples
,
in
particular
in
the
D-
galacto
series
,
exhibited
excellent
inhibitory
activities
.
A
preliminary
screen
with
two
human
cell
lines
showed
that
,
at
subinhibitory
concentrations
,
they
are
powerful
pharmacological
chaperones
enhancing
the
activities
of
the
catalytically
handicapped
lysosomal
D-
galactosidase
mutants
associated
with
GM
1
gangliosidosis
and
Morquio
B
disease
.