From Wikipedia,
the free encyclopedia
Antisense therapy is a
theoretical form of treatment for
genetic disorders. When the
genetic sequence of a particular
gene is known to be causative
of a particular disease, it is
possible to synthesize a strand of
nucleic acid (DNA,
RNA or a chemical analogue)
that will bind to the
messenger RNA produced by that
gene, effectively turning that
gene "off".
This synthesized nucleic acid
is termed an "anti-sense" oligo
because its
base sequence is complementary
to the gene's messenger RNA
(mRNA), which is called the
"sense" sequence (so that a sense
segment of mRNA " 5'-AAGGUC-3' "
would be blocked by the anti-sense
mRNA segment " 3'-UUCCAG-5' ").
Most potential therapies have
not yet produced significant
clinical results, though one
antisense drug,
Formivirsen (marketed as
Vitravene), has been approved
by the US
FDA.
