Why There Is Hope For a Cure...

  • We know what is wrong - the function of one gene, UBE3A, is missing
  • We know that loss of UBE3A doesn't affect neuronal development; only neuronal function
  • We have excellent Angelman Syndrome animal models where research indicates it can be cured
  • We have strong ideas and approaches for restoring UBE3A function in neurons

*the hyperlinks below redirect to the US site in a new browser window

Why is it an advantage that Angelman Syndrome is caused by defects in one gene?

Loss of UBE3A does not appear to affect neuronal development. Why is this important?

Is there any evidence that restoring UBE3A function would allow neurons to function better?

Are there real possibilities of restoring UBE3A in the neurons of individuals with Angelman Syndrome?

Everyone uses the word "cure". What makes this situation different?

If it is so easy, why hasn't it been accomplished by now?

How long will this take?

How much money will it take?

Will this benefit anyone else?

Why we need money for a cure

   


 


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