Every 90 minutes,
someone is diagnosed with and passes from Amyotrophic Lateral Sclerosis, or ALS.
Once diagnosed,
ALS patients face a life expectancy of 2–4 years.
48 months remaining
(~4.0 years)
Under cellular stress in ALS,
Stress Granules (SGs) become dysfunctional and form additional "organelles".
Excessive SG formation and dysfunction is linked to pathological TDP-43 protein aggregation in neurons.
We have developed
novel antisense oligonucleotides
to knock down proteins implicated in stress granule formation.
Furthermore, we used computational modeling and in-vitro testing to create a
RNA aptamer
that prevents the formation of TDP-43 aggregates.
Our solution in action!
