There is mounting evidence to suggest that a treatment for Angelman syndrome is not just
possible, but probable. The lack of known molecular targets associated with AS has hampered
the development of specific therapeutics. However, a recent surge of potential therapeutics
for other disorders associated with cognitive disruption has begun to be used in human
clinical trials. The molecular modes of action for many of these new therapeutic agents have
correlates to counter the molecular defects observed in AS. One such agent is minocycline
(MC), a drug traditionally used as an antibiotic. This compound administered to a mouse model
of AS showed a significant decrease in motor deficit and an increase in long term
potentiation. The investigators believe a similar result will be observed when minocycline is
administered to the AS patient and may lead to the development of an effective AS
therapeutic.