An essential question for further research on the Angelman Syndrome is to understand, if the Angelman Syndrome and its effects are actually reversible.

Although the brain structure of an Angelman Syndrome patient seems to be developed correctly, the question remains if they were to function correctly, if the perfect treatment could be found.

In addition this is important, as with a positive answer the pharmaceutical industry can be contacted to interest them in creating a drug for therapy.

The approach will be to genetically modify a mouse to have at first the Angelman Syndrome. In contrary to the earlier mouse models of Angelman this will be not a deletion of the Ube3a gene, but there will be a block on this part of the DNA. This will be a new mouse model for Angelman Syndrome. Then this block will be removed by injection with a specific chemical. This chemical is then in effect the perfect treatment as it removes the block.

The financial support for this research comes from French (www.angelman-afsa.org ) and Italian (www.sindromediangelman.org ) Angelman organisations. The Nina Foundation played a key role in the organisation of the funding. The funding provided is by far not enough and only for the initial start up. In order to  continue a lot of additional funding is needed. The next step is to create this new mouse model. This is a timely and therefore cost intensive process. Please support us to make this a high priority project and to get the answer that is so important to all of us, as soon as possible.