DR. ALESSANDRO PRIGIONE
Member of the Board of Directors, Senses Cultural, Inc.
Team member, Senses Cultural research project on:
Gene Engineering Approaches to the Treatment of Fragile X-Associated Neurodevelopment and Neurodegenerative Disorders Including Autism
Professor of general pediatrics at Heinrich-Heine-University and expert on various forms of intellectual disability
After months of major professional and advisory consultations with our team of scientists, we are thrilled to share the news of the launch of our latest research on autism and neurodevelopmental disorders!
Senses Cultural is proud to launch a new groundbreaking research project, the Gene Engineering Approaches to the Treatment of Fragile X-Associated Neurodevelopmental and Neurodegenerative Disorders Including Autism. This project will engage top world scientists from the University of California, Davis, School of Medicine and the Heinrich-Heine-University in Germany.
Our team will study the fragile X gene responsible for the leading inherited form of intellectual disability (Fragile X Syndrome) and for autism, and a leading form of adult-onset neurodegeneration, the fragile X-associated tremor ataxia syndrome (FXTAS). Our scientists will take an alternative approach, one that will involve the use of gene engineering to target the expression of the fragile X gene itself. A second form of gene engineering that involves a special form of the CRISPR machinery will be studied.
CRISPR is a fast and affordable breakthrough in the technology of genetic therapy. It has changed the way researchers think about approaching the treatment of genetic diseases. As the technology is studied further and methods of safely delivering the therapy to humans are established, CRISPR therapy will provide an affordable approach to gene therapy that makes it accessible to more people.
Advancements in CRISPR have offered hope to autistic individuals and their families, especially after 2020 when research on the topic received recognition by the Nobel Prize Committee. It is widely accepted that there is a strong genetic component to autism. As autism researchers are able to identify more specific autism-related genes, CRISPR researchers can develop specific interventions to target those genes, repair them, and reduce symptoms of autism.
Our research project will use a form of CRISPR that has been modified to target the gene of interest but without actually cutting it. Our scientists will only use the targeting aspect of CRISPR, and then modify the CRISPR protein further to use it to shut off the gene. The advantage of this type of approach over regular CRISPR is that it is “reversible” so that if there is a problem, then it can be reversed. For traditional CRISPR, if there is a problem with cutting, it cannot be reversed.
We anticipate that our new research will provide a path toward clinical studies to offer new treatments for autism and neurodevelopmental disorders. You can help us with this project, by helping fund two post-doctoral positions to assist our team of scientists at the University of California, Davis and the Heinrich-Heine University, or by funding the project in whole (sponsorship funder(s)) or in part. We accept your tax-deductible gifts and donations here.
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