[Music playing]

Speaker 1: Jamell and Shante Stagg welcomed baby Juju to their family in 2015, but just a few months later, their pediatrician expressed some concerns.

Shante Stagg:
We went for his pediatrician, his three-month checkup. 

[Text on screen:
Shante Stagg
Juju's Mother]

She didn't tell me personally that she felt like something was wrong, but she referred him to physical therapy because she said he didn't have head control at that time, and that's kind of how the whole journey began.

Speaker 1:
After testing, the family received a devastating diagnosis. Juju had a rare condition known as AADC deficiency.

[Text on screen:
Russell Lonser, MD
Chair, Department of Neurological Surgery
Ohio State Wexner Medical Center]

Russell Lonser, MD:
AADC deficiency is a genetic disorder affecting around 135 children worldwide. Children who are born with AADC deficiency are missing an enzyme that produces dopamine in the central nervous system. Without this enzyme, children lack muscle control and are unable to speak, feed themselves, or even hold up their head.

Speaker 1:
Children with AADC deficiency require round-the-clock care. They also suffer from seizure-like episodes called oculogyric crises that can last for hours.

Shante Stagg:
And it's painful. It's painful for them. They kind of just cry and cry and cry, and so that's what we literally would sit on the couch with him for however long he was in it, just trying to cuddle him and comfort him as much as we could.

Speaker 1:
Desperate for help, the Staggs, who live in Washington state, traveled across the country to The Ohio State University Wexner Medical Center, where a pioneer in human genetics, Dr. Krystof Bankiewicz, was conducting a clinical trial, testing a new type of gene therapy.

[Text on screen:
Krystof Bankiewicz, MD, PhD
Vice Chair of Research
The Ohio State University Wexner Medical Center]

Krystof Bankiewicz, MD, PhD:
In our work, we developed a method to reprogram the defective gene and to deliver correct one into the brains of children with AADC deficiency.

Speaker 1:
During the gene therapy procedure, the new genes are slowly infused into the patient's brain using devices Dr. Bankiewicz and his team helped develop. The team guides the new genes to a specific target within the brain and watch in real time through MRI imagery as the genes take place in the brain. From there, the brain cells begin to adopt the new gene and begin to perform the functions programmed in the new gene.

Krystof Bankiewicz, MD, PhD:
In the case of children with AADC, the cells begin the signaling process to tell the brain to produce dopamine, which children like Juju need to develop muscle control.

[Text on screen:
Jamell Stagg
Juju's Father]

Jamell Stagg:
The first night we actually saw him, we sat him down in front of the TV, but we put pillows around him and he was doing his little wobble thing and we're like, hold on, he's not falling back. And we both sat on the couch together and we looked, like we're actually sitting on the couch without him. This is crazy because we never had any alone time since he's been born. Now seeing him pick up toys, or if I can put something in my hand and he can just grab it out, it's really cool.

Speaker 1:
Today, Dr. Bankiewicz and Lonser are taking the methods they've developed in genetic conditions like AADC to apply them to broader neurologic conditions.

Russell Lonser, MD:
We are conducting clinical trials right now to investigate if these same methods can work to modify the genes of people experiencing neurologic conditions like Alzheimer's and Parkinson's disease.

[Text on screen:
Peter Mohler, PhD
Vice President of Research
The Ohio State University]

Peter Mohler, PhD:
These are the first trials of their kind to take place in humans, and it's happening right here at The Ohio State University at our Gene Therapy Institute. Using this game-changing technology, this interdisciplinary team has been able to change the lives of individuals with these rare and even potentially fatal genetic conditions. We believe that we can apply these same therapies to diseases that affect thousands and even millions of patients. Now, that is impact.

Speaker 1:
For the Stagg family, Dr. Bankiewicz and his gene therapy work has already been a game changer. Something that is not lost on Krystof Bankiewicz.

Krystof Bankiewicz, MD, PhD:
So helping patients like Juju has been the most profound moment of my scientific life.

[Text on screen:
The Ohio State University Wexner Medical Center
For more information, visit
wexnermedical.osu.edu]

Speaker 1:
Your support can fuel life-changing research at the Ohio State Gene Therapy Institute.

[Music fades]