When Sriansh was just three months old, his family grew concerned as he slept most of the day, arched his back, and could not control his eye movement. His parents brought him to Cincinnati Children’s, where he was diagnosed with AADC deficiency, a rare genetic disorder that disrupts dopamine and serotonin production in the brain. Sriansh’s family was told he might never walk, talk, or lift his head. But thanks to an experimental gene therapy clinical trial, his story took a groundbreaking turn. At just 16 months old, Sriansh became the youngest child in the world to receive a one-time gene therapy treatment, delivered directly into the brain through a minimally invasive surgical technique. His care team inserted an engineered virus carrying the missing gene, allowing his brain to begin producing the essential chemicals it lacked. Today, Sriansh is walking, running, and thriving because of this innovative clinical trial. This pioneering work helped lead to the first-ever FDA-approved gene therapy administered directly to the brain, offering hope for patients with rare genetic diseases once thought untreatable.