So there are many unanswered questions really. One very fundamental question is, does this protein become toxic and does it cause disease? Or is the accumulation of the protein in aggregates just a bystander in the pathological process. We still do not have a definitive answer to this question. We have ideas that are backed up by data from many, many laboratories around the world. We think that there’s compelling evidence pointing in the direction that the protein may indeed become toxic, is associated with disease, both based on genetic evidence and as well as on studies in animals...
So there are many unanswered questions really. One very fundamental question is, does this protein become toxic and does it cause disease? Or is the accumulation of the protein in aggregates just a bystander in the pathological process. We still do not have a definitive answer to this question. We have ideas that are backed up by data from many, many laboratories around the world. We think that there’s compelling evidence pointing in the direction that the protein may indeed become toxic, is associated with disease, both based on genetic evidence and as well as on studies in animals. But we do not have this definitive answer. So this is a big unanswered question in the field. We also do not know what the normal function of the protein is. We know, we have some ideas of what it does in neurons, but we do not know what it does, for example, in red blood cells or in platelets or in other tissues besides the brain and we know the protein exists in other cells so we need to also understand the function so that we are then better at interpreting what happens when the protein becomes dysfunctional and accumulates.
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