Stoke Therapeutics (STOK): Change the Channel

You need to know a little something about how gene products work

Stoke Therapeutics (STOK) is targeting diseases that are autosomal dominant. This means that it takes only one abnormal gene copy to produce the disease; you often see this in diseases in which there is a structural protein, as opposed to an enzymatic protein (something that catalyzes an action) – because one defect in the structure often ruins the whole structure: if one pillar on the entrance to a Neo-Classical building is perfectly strong, but the pillar on the opposite side has cracks throughout its marble, the whole portico collapses.

It’s different when an enzyme is a gene’s protein product, at least usually, which is why these types of diseases are usually recessive in their inheritance patterns: you