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Question: What will be the impact of your research on autism treatment?

Michael Wigler: Yes, well there are two ways in which our work could inform clinical treatment.  In the area of early diagnosis.  If there’s a child and it’s developing—it’s giving off developmental clues there might be something wrong, if we had a list of the kind of genetic lesions we could screen for, we might be able to determine early on that this child is going to develop a form of autism.  And if it’s correct—most disorders are correctable to the extent that they are correctable, are more correctable early than late, when we know how to correct or treat, we’ll be able to start that sooner.  So, early diagnosis is going to be important for any disorder.  That’s one way. 

Another way is children with a particular genetic abnormality, that is, those children who share genetic abnormality, may have one particular way of treating them that’s different than children who have a different abnormality.  We will only learn about that once we can separate these children according to their genetic abnormalities.  That’s going to take many, many years. 

The third way is that in some cases, we will be identifying genes, who by their very nature, tell us this is a correctable, treatable, syndrome.  For example, we find a gene that’s involved in metabolism.  This child is perhaps got really a storage disorder of some type, but altering the diet in those cases might be able to treat the child.  But unfortunately, we don’t yet know the identities of the autism genes.  We have regions and there’s a huge effort underway.  I would say, in particular by doing very exhaustive sequence comparisons of children to their parents, we will identify the actual culprit genes.  And that will take us two to four years.  And there may be, unfortunately, I’m estimating around 400 such genes that each one of which can cause autism.  But when we have those genes, we see what they do; we can see what pathways they are interacting with, some of those will suggest immediately treatments that can be tested.  We will be able to make animal models and test drugs in animals to correct these things. 

So, in general, the way to understand a disorder is to understand its causes and then address those causes.  In the case of autism, most people would agree, I think most scientists would agree the causes are genetic, and we have a pathway to discover the genes.  So it will be easier to diagnose, classify by diagnosis into behavioral and even drug treatments, and discover new drug treatments.

Recorded April 12, 2010

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