Phase 1 Study of Intravenous Administration of Adeno-Associated Virus Gene Therapy for Individuals with Propionic Acidemia

Propionic acidemia (PA) is a severe metabolic disorder caused by mutations in the PCCA gene. Traditional treatments, including stringent life-long protein restrictions and liver transplants, have limited success and significant risks. This trial will evaluate the safety and preliminary efficacy of a groundbreaking gene therapy in young children, aiming to reduce metabolic crises, improve quality of life, and preserve cognitive function. The successful execution of this project will represent a significant advancement in the treatment of PA, offering hope to patients and their families.