For Dr. Alysson Muotri, unlocking autism’s biology is personal. His son has autism, and Muotri hopes to help him and millions of others.
A Mission to Understand Autism
He launched the Tooth Fairy Project, which collected more than 3,000 donated baby teeth from children with and without autism. Then he turned cells from those teeth into neurons, the brain cells affected by autism. Using these neurons, he studies how autistic and neurotypical brain cells differ.
“We use these cells to identify gene alterations and test whether we can reverse them,” he explained. “Autism has a strong genetic component, so genome sequencing can reveal affected genes. Some genes are promising candidates for gene therapy. When we correct a mutated gene in the lab, the neural networks behave more normally. We see tremendous potential to help people.”
Brain Organoids Offer New Research Possibilities
Muotri is also creating brain organoids, which are three‑dimensional structures grown from stem cells. These organoids mimic some brain functions and may predict treatment risks more accurately than animal models.
“We can test drugs in the brain organoids of the person and see if it works, see if there’s any toxicity before you actually give the drug to a person, and it will save us time and money and will increase our knowledge about the human brain.”
He says he still gets excited seeing how these cells work. “It’s amazing, it’s a miracle. Every time I see it, it’s like seeing dolphins in the sea because it’s so beautiful.”
Dr. Muotri is also a big proponent of diversity, equity and inclusion in scientific research. He says in the past it was very much a top-down model with scientists deciding what was important. He says we need to change that and give patients and communities a bigger role in shaping the direction of research.
“I think this is something we scientists have to learn, how to incorporate patients in our research. These communities are the ones we are studying, and we need to know what they want and not assume that what we want is what they want. They should be consulted on our grants, and they should participate in the design of our experiments. That is the future.”
Our 2021-22 Annual Report is now online. It’s filled with information about the work we have done over the last year (we are on a fiscal calendar year from July 1 – June 30), the people who have helped us do that work, and some of the people who have benefited from that work. One of those is Dr. Alysson Muotri, a professor in the Departments of Pediatrics and Cellular & Molecular Medicine at the University of California, San Diego.