Our Mission is simple:  Cure Autism. 

 

We will use well designed, low cost and efficient studies directing research toward using gene therapy to correct the symptoms of autism.   Our studies will be faster and scientifically rigorous and will vector other research in the direction for a genetic cure.  Our agile approach will accelerate the race to a cure reducing the timeline that might otherwise take decades to achieve.  

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How you ask?

 

We start by curing autism in mice. We are going to use a new tool called CRISPR to modify the  expression of brain cells and reverse the symptoms.  From a  2007 study using mice  we have good reason to believe we can reverse symptoms by getting the DNA to express the right proteins to return the brain to normal functioning.  We also believe that this effect is not age dependent.  Children and adults may have symptoms reversed or greatly improved with the corrected genetic modification.  This can happen because disorders like autism are neurodevelopmental.  In neurodevelopmental disorders cells are not dying but are waiting for the right signals, or proteins, to function normally.  Brain cells, or neurons, in autistic individuals can appear immature or incapable of functioning like normal neurons because their DNA does not allow them to produce the right proteins to function normally.  Using CRISPR we will modify the DNA in brain cells of mice correcting the DNA expression and allowing the brain cells to mature and function normally. 

 

Learn more about CRISPR

 

Our study differs in important ways from the 2007 study cited above.  The 2007 study used mice that had both the good and bad gene right next to each other and could be turned on and off like a switch.  Rett Syndrome or other types of neurological disorders don’t work that way.  But the importance of the study was that Rett’s could be reversed even in young adult mice which is remarkable.

We will use our own mice genetically modified with a gene most associated with Rett Syndrome in humans.  This will be a much more realistic model to study.  We will then use CRISPR to correct the Rett gene to a normal gene expression the corrected protein.  With the gene correction we should see abnormal neurons revert to normal and correct the cognitive and neurological abnormalities.  We will compare our results with normal mice to show the degree of how well we did in reversing the symptoms. 

 

Steps to kicking off the study: 

 

Step 1:  Assembly an amazing team – done  Meet the Team

 

Step 2:  This is where you come in.  Help raise $250,000 US to fund the study.

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Step 3:  Launch the study and keep you informed every step of the way

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With your help we can make a difference in our children’s lifetime.

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Our goal is to raise the funds needed. If we do not meet our fundraising goal, we reserve the right to donate the funds to another USA registered non-profit in line with our mission.