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Simon Frost, father to Annabel Frost, tells us how YOU can help shape the future for thousands and possibly millions og people by making this research happen.
Fund this AAV gene project and help save thousands and potentially millions of children all over the world.
AAV gene therapy is a new therapy that uses a virus to carry a string of healthy gene to the correct location where is it released and then it start to function normally and replaces the function of the broken gene.
This method if successful can be used in other disease where the cause is a mutated or a missing gene.